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75 studies

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  • Crohn's / ulcerative colitis (IBD)

    A Long-term Extension (LTE) Study of Guselkumab in Pediatric Participants

    Testing the long-term safety of an under-the-skin injection in children with ulcerative colitis,

    📍 Emory University · Atlanta, GA · +4 more US sites · Get directions →

    The purpose of this study is to evaluate long-term safety of subcutaneous guselkumab in pediatric participants with moderately to severely active ulcerative colitis, or moderately to severely active Crohn's disease, or juvenile psoriatic arthritis (jPsA).

    ✓ Study care freePhase 3Ages 3+Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Study of Guselkumab in Pediatric Participants With Moderately to Severely Active Crohn's Disease

    Testing guselkumab in children with Crohn's disease at week 52

    📍 Cedars Sinai Medical Center · Los Angeles, CA · +13 more US sites · Get directions →

    The purpose of this study is to evaluate the clinical and endoscopic efficacy of guselkumab in pediatric participants with Crohn's Disease (CD) at the end of maintenance therapy (Week 52) among participants who were in clinical response to guselkumab at Week 12.

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Maintenance Study to Investigate the Efficacy and Safety of Duvakitug in Participants With Moderately to Severely Active Crohn's Disease

    Testing a drug given during maintenance to help people with Crohn's disease stay stable

    📍 Peak Gastroenterology Associates - Colorado Springs-Site Number: 8400039 · Colorado Springs, CO · +21 more US sites · Get directions →

    This is a multicenter, randomized, double-blind, placebo-controlled, maintenance, Phase 3 study to evaluate the efficacy and safety of duvakitug in participants with moderately to severely active Crohn's Disease (CD). Study details include: The study duration may be up to 286 weeks including: * 40-week Pivotal Maintenance Sub-Study * 240-week Open-Label Extension (OLE) Sub-Study * 45-day Follow-Up visit Note: For the participants who do not enroll into OLE Sub-Study, the duration will be up to 46 weeks, including the 40-week maintenance period and a 45-day follow-up visit.

    ✓ Study care freePhase 3Ages 16–80Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Maintenance Study to Investigate the Efficacy and Safety of Duvakitug in Participants With Moderately to Severely Active Ulcerative Colitis

    Testing a treatment during maintenance for moderately to severely active ulcerative colitis

    📍 Del Sol Research Management, LLC - Site Number: 8400012 · Tucson, AZ · +25 more US sites · Get directions →

    This is a multicenter, randomized, double-blind, placebo-controlled Phase 3 maintenance study to evaluate the efficacy and safety of duvakitug in participants with moderately to severely active Ulcerative Colitis (UC). Study details include: The study duration may be up to 286 weeks including: * 40-week Pivotal Maintenance Sub-Study * 240-week Open-Label Extension (OLE) Sub-Study * 45-day Follow-up Visit Note: For the participants who do not enroll into OLE Sub-Study, the duration will be up to 46 weeks, including the 40-week maintenance period and a 45-day follow-up visit.

    ✓ Study care freePhase 3Ages 16–80Learn more →
  • Sickle cell disease

    A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)

    Testing different doses of DISC-3405 to check safety in sickle cell disease

    📍 University of Alabama at Birmingham · Birmingham, AL · +6 more US sites · Get directions →

    This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Type 2 diabetesHigh cholesterol

    📍 Clinical Research Institute of Arizona · Sun City West, AZ · +19 more US sites · Get directions →

    This study will be a placebo-controlled, double-blind, randomized, Phase 3 study to evaluate the efficacy, safety, and tolerability of obicetrapib 10 mg, both as a fixed-dose combination (FDC) with ezetimibe 10 mg and as monotherapy, on top of guideline-recommended lipid-lowering therapy in patients with metabolic syndrome and/or Type 2 Diabetes Mellitus.

    ✓ Study care freePhase 3Ages 18+Learn more →
  • Sickle cell disease

    Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease

    Testing higher doses of VAS-101 in people with stable sickle cell disease

    💵 May compensate

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen.

    ✓ Study care freePhase 1Ages 18–90Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Study of Vedolizumab in Children and Teenagers With Ulcerative Colitis or Crohn's Disease

    Testing how a medicine given under the skin works in children with IBD

    📍 Phoenix Children's Hospital · Phoenix, AZ · +13 more US sites · Get directions →

    The main aim of this study is to learn how the body of a child or teenager with moderately to severely active ulcerative colitis (UC) or Crohn's disease (CD) processes vedolizumab (pharmacokinetics) given just under the skin subcutaneously (SC). The participants will be treated with vedolizumab for up to 34 weeks.

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • Sickle cell disease

    A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)

    Testing the safety and how an oral carbon monoxide liquid works in sickle cell disease

    📍 Cevaxin - The Panama Clinic · Panama City, · Get directions →

    This is a multi-center, open label Phase 2a clinical trial in subjects with sickle cell disease to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with doses daily for 14 days.

    ✓ Study care freePhase 1Ages 18–55Learn more →
  • Crohn's / ulcerative colitis (IBD)

    📍 Erick H. Alayo Medical Corporation - Gastro SB Clinic · Chula Vista, CA · +1 more US site · Get directions →

    The purpose of this study is to evaluate the disease-drug-drug interaction (DDDI) potential of afimkibart (also known as RO7790121). This will be assessed by the characterization of the pharmacokinetics (PK) of cytochrome P450 (CYP) enzyme substrates alone and after administration of afimkibart in participants with moderately to severely active ulcerative colitis (UC).

    ✓ Study care freePhase 1Ages 18–60Learn more →
  • Sickle cell disease

    The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease

    Testing rilzabrutinib for safety and effectiveness in sickle-cell disease

    📍 University of Alabama at Birmingham- Site Number : 8400003 · Birmingham, AL · +15 more US sites · Get directions →

    This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study details include: * Study duration: a 52-week double-blind period (Part A), followed by an open-label LTE period (Part B).

    ✓ Study care freePhase 3Ages 10–65Learn more →
  • Crohn's / ulcerative colitis (IBD)

    Autologous Stem Cell Transplantation for Crohn's Disease

    Testing stem cell treatment for severe Crohn's disease in teens and adults

    📍 UPMC Prebyterian- Adult Gastroenterology · Pittsburgh, PA · +1 more US site · Get directions →

    The objective of this study is to evaluate the safety and effectiveness of administering high-dose chemotherapy followed by infusion of autologous CD34-selected peripheral blood stem cells (PBSC) in pediatric and adult patients with severe Crohn's disease.

    ✓ Study care freePhase 1Ages 10–60Learn more →
  • Sickle cell disease

    Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease

    Testing whether extra arginine lowers pain in children with sickle cell

    📍 Children's Healthcare fo Atlanta at Hughes Spalding · Atlanta, GA · +1 more US site · Get directions →

    The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful events (VOE) will decrease pain scores, decrease need for pain medications or decrease length of hospital stay or emergency department visit.

    ✓ Study care freePhase 1Ages 7–21Learn more →
  • Crohn's / ulcerative colitis (IBD)

    An Open-Label, Proof of Consent Study of Vorinostat for the Treatment of Mdoerate-to-Severe Crohn s Disease and Maintenance Therapy With Ustekinumab

    Testing vorinostat in people with moderate to severe Crohn’s disease and using ustekinumab to

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    Background: Crohn s disease (CD) is an inflammatory bowel disease. It causes inflammation of the gut.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Study of Vedolizumab With and Without Upadacitinib in Adults With Crohn's Disease

    Testing a combination of two medicines to treat Crohn’s bowel inflammation vs one medicine

    📍 UCSD Medical Center · La Jolla, CA · +18 more US sites · Get directions →

    The main aim of this study is to learn whether vedolizumab and upadacitinib given together (also called dual targeted therapy or DTT) reduces bowel inflammation and ulcers in the bowel compared to vedolizumab only (also called monotherapy) in adults with moderately or severely active Crohn's Disease (CD) after 12 weeks of treatment. Other aims are to learn how safe and effective DTT is compared to monotherapy for these participants.

    ✓ Study care freePhase 3Ages 18–65Learn more →
  • Crohn's / ulcerative colitis (IBD)

    Testing the Role of Anti-fungal Therapy in Improving the Response to Therapies for Crohn's Disease

    Testing whether fluconazole improves responses to IL-23 therapy in Crohn’s disease

    📍 Weill Cornell Medicine · New York, NY · Get directions →

    The goal of this clinical trial is to learn about the effects of fluconazole in patients who plan to start or are currently undergoing standard of care treatment and plan to dose-escalate an IL-23 therapy for their Crohn's disease. The main question it aims to assess is whether or not patient response to IL-23 therapies improve when simultaneously treated with fluconazole.

    ✓ Study care freePhase 3Ages 18+Learn more →
  • Crohn's / ulcerative colitis (IBD)

    📍 Phoenix Children's Hospital /ID# 255766 · Phoenix, AZ · +10 more US sites · Get directions →

    Crohn's Disease (CD) is a gastrointestinal disease that can cause chronic diarrhea with or without gross bleeding, abdominal pain, weight loss, and fever. This study will assess the pharmacokinetics, efficacy, and safety of risankizumab in pediatric participants with moderately to severely active CD aged 2 to \< 18 years old who have had intolerance or inadequate response to other therapies.

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • Crohn's / ulcerative colitis (IBD)

    Treatment of Moderate to Severe Refractory Crohn's Disease

    Testing different doses of a new treatment for Crohn’s disease

    📍 Mayo Clinic Arizona · Scottsdale, AZ · +12 more US sites · Get directions →

    This research study is testing an investigational research product called TRX103 as a possible treatment for individuals suffering from Crohn's Disease (CD). The primary purpose of this study is to learn how safe and effective different doses of TRX103 are when administered to individuals with CD.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Study to Learn More About How Risankizumab Works in Young Participants With Ulcerative Colitis

    Studying how risankizumab works and its safety for youth with ulcerative colitis

    📍 Phoenix Children's Hospital /ID# 273015 · Phoenix, AZ · +10 more US sites · Get directions →

    Ulcerative colitis (UC) is a type of inflammatory bowel disease that causes inflammation and bleeding from the lining of the rectum and colon (large intestine). This study will assess how Risankizumab moves through the body as well as how safe and effective it is in treating pediatric participants with moderate to severely active UC.

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • Sickle cell disease

    RH Genotype Matched RBC Transfusions

    Testing whether matching donor red blood cells by RH type helps people with sickle cell who need

    📍 Children's Hospital of Philadelphia · Philadelphia, PA · Get directions →

    To determine the feasibility and efficacy of matching donor red cells by RH genotype for a cohort of chronically transfused patients with SCD.

    ✓ Study care freePhase 1Ages 1+Learn more →
  • Crohn's / ulcerative colitis (IBD)

    An Induction Study to Investigate the Efficacy and Safety of Duvakitug in Participants With Moderately to Severely Active Ulcerative Colitis

    Testing a medicine called duvakitug for safety and benefit in ulcerative colitis

    📍 Onyx Clinical Research - Site Number: 8400021 · Peoria, AZ · +39 more US sites · Get directions →

    This is a multinational, multicenter, randomized, double-blind, placebo-controlled, Phase 3 induction study to evaluate the efficacy and safety of duvakitug in participants with moderately to severely active Ulcerative Colitis (UC). Study details include: The study duration may be up to 35 weeks with: * Screening period * 12-week Sub-Study 1 (Single-Arm Open-Label Feeder Induction) or Sub-Study 2 (Pivotal Induction) * 12-week Sub-Study 3 (Extended Induction for non-responders) * 45 days follow-up visit for participants who do not enroll into the maintenance study (EFC18359) The treatment duration will be up to 12 weeks in each sub-study.

    ✓ Study care freePhase 3Ages 16–80Learn more →
  • Sickle cell disease

    Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

    Testing a one-time blood stem cell transplant using gene editing for severe sickle cell disease

    📍 University of California, Los Angeles · Los Angeles, CA · +1 more US site · Get directions →

    This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR\_SCD001 Drug Product).

    ✓ Study care freePhase 1Ages 12–35Learn more →
  • Crohn's / ulcerative colitis (IBD)

    📍 Children's Healthcare of Atlanta · Atlanta, GA · +1 more US site · Get directions →

    This Phase III, randomized, double-blind, multicenter, induction and maintenance study will evaluate the safety and efficacy of Afimkibart (RO7790121) in pediatric participants with moderate to severe active ulcerative colitis (UC).

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Study of Guselkumab Versus Risankizumab in Participants With Moderately to Severely Active Crohn's Disease

    Comparing two medicines for Crohn’s disease with moderate to severe activity

    📍 Digestive Health Specialists of the Southeast · Dothan, AL · +24 more US sites · Get directions →

    The purpose of this study is to assess how well guselkumab works when compared to risankizumab in participants with moderately to severely active Crohn's Disease (CD; a long-term condition causing severe inflammation of the intestinal tract).

    ✓ Study care freePhase 3Ages 18+Learn more →
  • Sickle cell disease

    Cannabidiol in Sickle Cell Disease

    Testing twice-daily cannabidiol doses for people with sickle cell disease

    📍 Icahn School of Medicine at Mount Sinai · Manhattan, NY · Get directions →

    Randomized, placebo-controlled, double masked, dose finding study of twice daily cannabidiol given at 3 dose levels, 200mg, 400mg, and 600mg, compared to placebo for 4 weeks.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Obesity / overweightCrohn's / ulcerative colitis (IBD)

    Mirikizumab Administered at the Same Time as Tirzepatide in Adult Participants With Moderately to Severely Active Ulcerative Colitis and Obesity or Overweight: Phase 3b Study

    Testing two medicines together to reduce ulcerative colitis symptoms and weight in adults with

    📍 Digestive Health Specialists · Dothan, AL · +36 more US sites · Get directions →

    The main purpose of this study is to show whether in these individuals, treatment with both mirikizumab and tirzepatide, compared with treatment with mirikizumab and placebo, leads to decrease or disappearance of UC symptoms, and loss of at least one-tenth of the overall body weight. Participation in this study will last up to 61 weeks, including 52 weeks of treatment.

    ✓ Study care freePhase 3Ages 18–70Learn more →
  • Obesity / overweightCrohn's / ulcerative colitis (IBD)

    Mirikizumab and Tirzepatide Administered in Adult Participants With Moderately to Severely Active Crohn's Disease and Obesity or Overweight

    Testing treatments for adults with Crohn’s disease and excess weight

    📍 Digestive Health Specialists · Dothan, AL · +34 more US sites · Get directions →

    The main purpose of this study is to evaluate the efficacy and safety of mirikizumab and placebo compared with mirikizumab and concomitantly administered tirzepatide in adult participants with moderately to severely active CD and obesity, or overweight. The maximum duration of this study is up to 61 weeks.

    ✓ Study care freePhase 3Ages 18–70Learn more →
  • Sickle cell disease

    Gene Editing For Sickle Cell Disease

    Testing whether gene editing is safe for people with sickle cell disease

    📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →

    This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.

    ✓ Study care freePhase 1Ages 18–24Learn more →
  • Sickle cell disease

    Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease

    Testing a single pneumococcal vaccine dose in children with sickle cell disease

    📍 Site # 8400006 · Atlanta, GA · +4 more US sites · Get directions →

    The purpose of this study is to measure whether PCV21 vaccine (investigational pneumococcal vaccine) is safe and can help the body to develop germ-fighting agents called "antibodies" (immunogenicity) compared with 20vPCV (licensed pneumococcal vaccine) when given as a single dose to children aged 2 to 17 years with sickle cell disease who had received or not a previous vaccination with pneumococcal conjugate or pneumococcal polysaccharide vaccine.

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Study to Evaluate the Efficacy and Safety of Tulisokibart (MK-7240) in Participants With Moderate to Severe Crohn's Disease (MK-7240-008)

    Testing whether tulisokibart works and is safe for people with Crohn’s disease

    📍 Digestive Health Specialists ( Site 5064) · Dothan, AL · +39 more US sites · Get directions →

    The purpose of this protocol is to evaluate the efficacy and safety of tulisokibart in participants with moderately to severely active Crohn's disease. Study 1's primary hypotheses are that at least 1 tulisokibart dose level is superior to placebo in the proportion of participants achieving clinical remission per Crohn's Disease Activity Index score (\<150, US/FDA) or per stool frequency and abdominal pain score (EU/EMA) and in the proportion of participants achieving endoscopic response at Week 52 (US/FDA and EU/EMA), and that at least 1 tulisokibart dose level is superior to placebo in the proportion of participants achieving clinical remission per Crohn's Disease Activity Index score (\<150, US/FDA) or per stool frequency and abdominal pain score (EU/EMA) and in the proportion of participants achieving endoscopic response at Week 12 (US/FDA and EU/EMA).

    ✓ Study care freePhase 3Ages 16–80Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Study of Mirikizumab (LY3074828) in Pediatric Participants With Crohn's Disease

    Testing mirikizumab in kids with Crohn’s disease for safety and how it works

    📍 Cedars Sinai Medical Center · Los Angeles, CA · +14 more US sites · Get directions →

    Study participants will be screened during the platform study and randomly assigned to receive mirikizumab or another intervention. The purpose of the mirikizumab study is to evaluate efficacy, safety, tolerability, and how well mirikizumab absorbs into the body of pediatric participants with Crohn's disease.

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • Crohn's / ulcerative colitis (IBD)

    R-3750 in Patients With Mild to Moderate Ulcerative Colitis

    Testing the safety of an oral probiotic in people with mild to moderate ulcerative colitis

    📍 Amicis Research Center · Valencia, CA · +4 more US sites · Get directions →

    The goal of this study is to determine the safety and tolerability of orally taken probiotic (R-3750) in patients with mild to moderate ulcerative colitis. Patients will take an oral dosage of probiotic (R-3750) and provide patient-reported and physician scored measures of their colitis.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Sickle cell disease

    A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

    Testing long-term treatment with a new medicine for sickle cell or thalassaemia

    📍 Univ of Alabama Birmingham · Birmingham, AL · +38 more US sites · Get directions →

    Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.

    ✓ Study care freePhase 3Ages 2+Learn more →
  • Sickle cell disease

    A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease

    Testing how BMS-986470 affects the body and safety in sickle cell disease

    📍 University of Alabama at Birmingham · Birmingham, AL · +18 more US sites · Get directions →

    The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Study of JNJ-78934804 in Participants With Moderately to Severely Active Crohn's Disease

    Testing a Crohn’s disease medicine against guselkumab for safety and effects

    📍 Clinnova Research · Anaheim, CA · +5 more US sites · Get directions →

    The purpose of this study is to assess how well JNJ-78934804 works (efficacy) and how safe it is (safety) as compared to guselkumab at Week 48 in participants with moderately to severely active Crohn's disease (a long-term, progressive \[worsens with time\] and life-threatening disease of the intestine).

    ✓ Study care freePhase 3Ages 18+Learn more →
  • Crohn's / ulcerative colitis (IBD)

    Fecal Microbiota Transplantation in an Expanded Ulcerative Colitis Population

    Testing fecal transplant for ulcerative colitis in a feasibility study

    📍 University of Calgary · Calgary, Alberta · Get directions →

    This is a multi-centre, randomised controlled trial comparing fecal microbiota transplantation to placebo in an expanded ulcerative colitis population: a feasibility study (FRONTIER-UC) to determine whether a full-scale randomized controlled trial (RCT) to investigate fecal microbiota transplantation (FMT) in ulcerative colitis (UC) is feasible.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Sickle cell disease

    Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

    Testing a partial stem cell transplant from matched donors for severe sickle cell disease or

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens.

    ✓ Study care freePhase 1Ages 4–65Learn more →
  • Sickle cell disease

    Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

    Testing how safe escalating doses of fostamatinib are in people with stable sickle cell disease

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Sickle cell disease

    Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

    Testing gene editing in stem cells for severe sickle cell disease

    📍 Boston Children's Hospital · Boston, MA · Get directions →

    A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.

    ✓ Study care freePhase 1Ages 13–40Learn more →
  • Crohn's / ulcerative colitis (IBD)

    Crohn's Disease: Efficacy, Safety, and Pharmacokinetics of Upadacitinib in Pediatric Subjects With Moderately to Severely Active Crohn's Disease

    Testing how well and how safely upadacitinib works in children with Crohn’s disease

    📍 UCSF Benioff Children's Hospital - Oakland /ID# 262217 · Oakland, CA · +11 more US sites · Get directions →

    Crohn's disease (CD) is a long-lasting disease that causes severe inflammation (redness, swelling), in the digestive tract, most often affecting the bowels. It can cause many different symptoms including abdominal pain, diarrhea, tiredness, and weight loss.

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • Crohn's / ulcerative colitis (IBD)

    Beta-Hydroxybutyrate Feasibility Treating IBD

    Testing whether taking a ketone supplement for 4 weeks is doable in people with Crohn’s disease

    📍 University of Texas at Austin · Austin, TX · Get directions →

    This clinical trial aims to understand the feasibility of patients taking ketone body supplement beta-hydroxybutyrate (BHB) for 4 weeks with a confirmed diagnosis of Crohn's disease and starting new therapy for active disease. The main questions it aims to answer are: * BHB supplementation will be feasible and acceptable to patients.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Crohn's / ulcerative colitis (IBD)

    Study of XmAb942 in Healthy Participants and Participants With Ulcerative Colitis

    Testing the safety and effects of a drug called XmAb942 in ulcerative colitis

    📍 Xencor Investigative Site · Scottsdale, AZ · +23 more US sites · Get directions →

    Brief summary The Phase 1 study described herein will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of XmAb942 in healthy volunteers (Parts A and B). Part C of this study will be a Phase 2 study to evaluate XmAb942 in participants with ulcerative colitis (UC).

    ✓ Study care freePhase 1Ages 18–75Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Long-Term Extension Study of Vedolizumab in Children and Teenagers With Ulcerative Colitis (UC) or Crohn's Disease (CD)

    Testing long-term skin shots of vedolizumab in children with ulcerative colitis or Crohn’s and

    📍 Phoenix Childrens Hospital · Phoenix, AZ · +13 more US sites · Get directions →

    The main aim of this study is to learn about medical problems (adverse events) if vedolizumab subcutaneously (SC) is given to a child or teenager with UC or CD for a long time. Other aims are to understand if the long time use of vedolizumab SC has an impact on the time period until hospital visits because of bowel swelling (inflammation) are needed and has an impact on the quality of life of children and teenagers who received vedolizumab SC.

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • Sickle cell disease

    SCD Stem Cell Mobilization and Apheresis Using Motixafortide

    Testing whether motixafortide is safe and can raise stem cells in people with sickle cell disease

    📍 University of Alabama at Birmingham · Birmingham, AL · +2 more US sites · Get directions →

    This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Sickle cell disease

    📍 University Of Alabama · Birmingham, AL · +13 more US sites · Get directions →

    A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.

    ✓ Study care freePhase 3Ages 12–100Learn more →
  • Crohn's / ulcerative colitis (IBD)

    An Induction Study to Investigate the Efficacy and Safety of Duvakitug in Participants With Moderately to Severely Active Crohn's Disease

    Testing the safety and effectiveness of duvakitug for active Crohn’s disease

    📍 Onyx Clinical Research - Site Number: 8400021 · Peoria, AZ · +39 more US sites · Get directions →

    This is a multinational, multicenter, randomized, double-blind, placebo-controlled, Phase 3 induction study, comprised of 3 sub-studies, to evaluate the efficacy and safety of duvakitug in participants with moderately to severely active CD. Study details include: The study duration may be up to 35 weeks with: * Up to 5-week Screening Period.

    ✓ Study care freePhase 3Ages 16–80Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Study of Icotrokinra in Participants With Moderately to Severely Active Crohn's Disease

    Testing how well and how safely icotrokinra works for Crohn's disease

    📍 AZ Gastro Care · Chandler, AZ · +39 more US sites · Get directions →

    The purpose of this study is to evaluate how-well icotrokinra works (clinical efficacy) and how safe it is (safety) in participants with moderately to severely active Crohn's disease (CD; a long-term condition causing severe inflammation of the intestinal tract).

    ✓ Study care freePhase 2Ages 18+Learn more →
  • Sickle cell disease

    Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease

    Testing the safety and effects of panobinostat in adults with sickle cell disease

    📍 Augusta University · Augusta, GA · Get directions →

    The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Sickle cell disease

    A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

    Testing how a new medicine works in healthy people and sickle cell patients

    📍 University of Alabama Birmingham · Birmingham, AL · +5 more US sites · Get directions →

    The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).

    ✓ Study care freePhase 1Ages 12–55Learn more →
  • Sickle cell disease

    Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

    Testing a bone marrow transplant with lower-intensity treatment for kids with sickle cell and

    📍 Yale School of Medicine · New Haven, CT · +3 more US sites · Get directions →

    This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.

    ✓ Study care freePhase 1Learn more →
  • Crohn's / ulcerative colitis (IBD)

    📍 Digestive Health Specialists of the Southeast (Gastroenterology Associates of Dothan) - Dothan · Dothan, AL · +39 more US sites · Get directions →

    This Phase III, multicenter, double-blind, placebo-controlled study will evaluate the efficacy and safety of induction therapy with Afimkibart (RO7790121) compared with placebo in participants with moderately to severely active ulcerative colitis (UC).

    ✓ Study care freePhase 3Ages 16–80Learn more →
  • Sickle cell disease

    A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

    Testing how well etavopivat lowers painful sickle cell crises and organ damage

    📍 Uni of Alabama at Birmingham · Birmingham, AL · +39 more US sites · Get directions →

    This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo.

    ✓ Study care freePhase 3Ages 12+Learn more →
  • Crohn's / ulcerative colitis (IBD)

    Low Dose IL-2 for the Treatment of Crohn's Disease

    Testing low-dose interleukin-2 to find a safe dose for Crohn's disease

    📍 Boston Children's Hospital · Boston, MA · +2 more US sites · Get directions →

    The purpose of this study is to determine the safety and maximum effective dose (MED) of Interleukin-2 in subjects with moderate-to-severe crohn's disease.

    ✓ Study care freePhase 1Ages 12–80Learn more →
  • Crohn's / ulcerative colitis (IBD)

    Extension Study of Long-term Safety and Efficacy of Tulisokibart in Participants With Crohn's Disease or Ulcerative Colitis (MK-7240-011)

    Studying long-term safety and effectiveness of tulisokibart in Crohn’s and ulcerative colitis

    📍 Connecticut Clinical Research Institute ( Site 0297) · Bristol, CT · +11 more US sites · Get directions →

    Researchers want to learn more about tulisokibart (also known as MK-7240) in an extension study. Tulisokibart is a medicine designed to treat active, moderate to severe Crohn's disease (CD) and ulcerative colitis (UC).

    ✓ Study care freePhase 3Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Study of Vedolizumab in Children and Teenagers With Moderate to Severe Crohn's Disease (CD)

    Testing a medicine (vedolizumab) for children with moderate to severe Crohn’s disease

    📍 Phoenix Childrens Hospital · Phoenix, AZ · +21 more US sites · Get directions →

    Vedolizumab is a medicine that helps to reduce inflammation and pain in the digestive system. In this study, children and teenagers with moderate to severe Crohn's disease will be treated with vedolizumab.

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • Sickle cell disease

    Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease

    Testing whether children with sickle cell disease can take daily ADHD medicine and monitoring its

    📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →

    The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork problems and if they have any side effects. The study will assess any thinking or attention problems participants may have both before taking this drug and after.

    ✓ Study care freePhase 1Ages 8–17Learn more →
  • Crohn's / ulcerative colitis (IBD)

    A Study of Vedolizumab in Children With Ulcerative Colitis (UC) or Crohn's Disease (CD)

    Testing vedolizumab in children with ulcerative colitis or Crohn’s disease

    📍 University of South Alabama (USA) Physicians Group · Mobile, AL · +21 more US sites · Get directions →

    The study is an extension of two parent studies (MLN0002-3024 \[NCT04779307\] and MLN0002-3025 \[NCT04779320\]). Participants must have participated in one of the previous studies.

    ✓ Study care freePhase 3Ages 2+Learn more →
  • Sickle cell disease

    Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease

    Testing if adding ruxolitinib lowers graft failure after transplant in children with sickle cell

    📍 Children's Hospital of Colorado · Aurora, CO · +3 more US sites · Get directions →

    This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD). This study will enroll and treat up to 24 participants.

    ✓ Study care freePhase 1Ages 12–45Learn more →
  • Crohn's / ulcerative colitis (IBD)

    Study to Evaluate the Fecal Microbiota Transplantation (FMT) in the Treatment of Ulcerative Colitis

    Testing frozen stool transplant methods for people with active ulcerative colitis

    📍 The University of Texas Health Science Center at Houston · Houston, TX · Get directions →

    The study is to evaluate the safety, feasibility, and preliminary efficacy of frozen FMT delivery via retention enema compared to lyophilized powder given in oral capsules as induction FMT in subjects with active UC. This study will also determine changes in microbiome (diversity and genera) and proportion of antibody-coated microbiota from baseline to after completion of FMT.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Sickle cell disease

    Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease

    Testing a new transplant plan using immune-suppressing treatment before a blood stem cell transplant

    📍 City of Hope Medical Center · Duarte, CA · Get directions →

    This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.

    ✓ Study care freePhase 1Ages 1–30Learn more →
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