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- Obesity / overweightCrohn's / ulcerative colitis (IBD)
📍 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.
- Obesity / overweightCrohn's / ulcerative colitis (IBD)
📍 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.
- Sickle cell disease
📍 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.
- Crohn's / ulcerative colitis (IBD)
📍 Digestive Health Specialsits · Dothan, AL · +38 more US sites · Get directions →
The main aim of this study is to learn about the effect of treatment with vedolizumab IV (vedolizumab) together with tofacitinib in adults with moderate and severe ulcerative colitis (UC). Another aim is to learn about treatment with Vedolizumab alone after the double treatment.
- Sickle cell disease
📍 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.
- Crohn's / ulcerative colitis (IBD)
📍 Digestive Health Specialsits · Dothan, AL · +37 more US sites · Get directions →
The main aim of this study is to learn about the effect of treatment with vedolizumab IV (vedolizumab) together with adalimumab or vedolizumab (VDZ) together with ustekinumab (UST) in adults with moderate to severe Crohn's Disease, and the effect of treatment with vedolizumab alone, after the dual targeted treatment. The study is conducted in two parts.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- Crohn's / ulcerative colitis (IBD)
📍 Sun City Clinical Research · Glendale, AZ · +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 (also known as RO7790121) in participants with moderately to severely active Crohn's disease (CD).
- Crohn's / ulcerative colitis (IBD)
📍 Phoenix Children's Hospital · Phoenix, AZ · +21 more US sites · Get directions →
Crohn's disease (CD) is a condition that causes inflammation (swelling, redness) of the lining and wall of the small intestine, large intestine, or both. CD may be associated with abdominal cramps/pain, diarrhea, blood in the stool, weight loss, or delayed growth in children.
- Crohn's / ulcerative colitis (IBD)
📍 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).
- 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).
- 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).
- Lupus (SLE)Rheumatoid arthritisMultiple sclerosis (MS)
📍 University of Alabama at Birmingham Cancer Center · Birmingham, AL · +39 more US sites · Get directions →
This phase Ib trial studies the side effects of nivolumab and to see how well it works alone and in combination with other treatments, such as ipilimumab, cabozantinib, platinum containing therapy, and fluoropyrimidine, in treating patients with autoimmune disorders and cancer that has spread from where it first started (primary site) to nearby tissue, lymph nodes, or distant parts of the body (advanced), to other places in the body (metastatic) or cannot removed by surgery (unresectable). Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
- Crohn's / ulcerative colitis (IBD)
📍 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).
- 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.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- Crohn's / ulcerative colitis (IBD)
📍 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).
- 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.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- Crohn's / ulcerative colitis (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.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- 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.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- Crohn's / ulcerative colitis (IBD)
A Study of Vedolizumab in Adults With Ulcerative Colitis or Crohn's Disease in the Community Setting
📍 Gastro Health Research- St. Vincents East · Birmingham, AL · +39 more US sites · Get directions →
Ulcerative Colitis (UC) and Crohn's Disease (CD) are long-term conditions in the gut that can cause diarrhea, swelling (inflammation), bleeding from the anus, and belly pain. The main aim of this study is to check for how many participants with UC and CD signs and symptoms disappear after 3.5 months (14 weeks) of treatment with Vedolizumab (this is called remission).
- Crohn's / ulcerative colitis (IBD)
📍 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.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- Sickle cell disease
📍 Smilow Cancer Hospital · New Haven, CT · +23 more US sites · Get directions →
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- Crohn's / ulcerative colitis (IBD)
📍 UCSF Medical Center at Mission Bay · San Francisco, CA · +10 more US sites · Get directions →
The main purpose of this study is to evaluate the long-term efficacy of mirikizumab in pediatric participants with ulcerative colitis (UC) or Crohn's disease (CD). The study will last about 172 weeks and may include up to 44 visits.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- 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 treat-through study will evaluate the efficacy and safety of induction and maintenance therapy with Afimkibart (also known as RO7790121) in participants with moderately to severely active Crohn's disease (CD).
- Crohn's / ulcerative colitis (IBD)
📍 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).
- Crohn's / ulcerative colitis (IBD)
📍 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).
- Crohn's / ulcerative colitis (IBD)
📍 Children's Hospital of Los Angeles · Los Angeles, CA · +10 more US sites · Get directions →
Approximately 3 million people in the United States are living with inflammatory bowel disease, which includes Crohn's Disease (CD). There are limited treatment options approved for use in children and adults with Crohn's disease.
- Sickle cell disease
📍 University Of Alabama · Birmingham, AL · +6 more US sites · Get directions →
This is a multi-center multi-national rollover study to allow continued access to crizanlizumab for patients with sickle cell disease (SCD) who are on crizanlizumab treatment in a Novartis-sponsored study (parent study) and are benefiting from the treatment as judged by the investigator.
- 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.
- Sickle cell disease
📍 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).
- Crohn's / ulcerative colitis (IBD)
📍 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).
- Crohn's / ulcerative colitis (IBD)
📍 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).
- 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.
- Sickle cell disease
📍 Children's Hospital Los Angeles · Los Angeles, CA · +5 more US sites · Get directions →
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
- Sickle cell disease
📍 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.
- Sickle cell disease
📍 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.
- Crohn's / ulcerative colitis (IBD)
📍 Bausch Health Site 008 · Garden Grove, CA · +3 more US sites · Get directions →
This is a randomized, double-blind, placebo-controlled, multicenter, study to evaluate the efficacy, safety, and pharmacokinetics (PK) of budesonide extended-release tablets for the induction of remission in pediatric subjects, with active, mild to moderate ulcerative colitis (UC). Subjects will be permitted to continue taking background oral or rectal 5-aminosalicylate (5-ASA) products.
- Crohn's / ulcerative colitis (IBD)
📍 TLC Clinical Research Inc - Los Angeles · Los Angeles, CA · +2 more US sites · Get directions →
Transmural healing (TMH) is recognized as a potentially important measure of Crohn's disease (CD) activity but not a formal target. Observational studies suggest that TMH may be associated with better long-term outcomes.
- Sickle cell disease
📍 New York Presbyterian Hospital - Morgan Stanley Children's Hospital · New York, NY · +2 more US sites · Get directions →
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.
- Crohn's / ulcerative colitis (IBD)
📍 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.
- 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.
- 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).
- 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).
- Crohn's / ulcerative colitis (IBD)
📍 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.
- Crohn's / ulcerative colitis (IBD)
📍 Connecticut Children's Medical Center · Hartford, CT · +2 more US sites · Get directions →
Randomized, placebo-controlled dose-ranging study of 2'-FL in IBD, Crohn's Disease (CD) and ulcerative colitis (UC). The overarching hypothesis is that 2'-FL supplementation in IBD will be safe and well tolerated, while increasing fecal Bifidobacterium abundance and butyrate in a dose dependent manner.
- Sickle cell disease
📍 Emory University · Atlanta, GA · +1 more US site · Get directions →
This research is being conducted to see if using an injectable contraception, Depot Medroxyprogesterone Acetate (Depo-Provera), can reduce the pain experienced by women with sickle cell disease. Participants in this study will be adult women with sickle cell disease who regularly experience sickle cell pain.
- Crohn's / ulcerative colitis (IBD)
Open-label Single-arm Study to Assess the Efficacy of Mirikizumab in Patients With Inflammatory Strictures Due to CD
💵 May compensate📍 Digestive & Liver Center of Florida · Orlando, FL · +1 more US site · Get directions →
This is an open-label, single-arm, phase 4 study to assess the safety and efficacy of mirikizumab in approximately 60 participants with stricturing CD.
- Sickle cell disease
📍 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.
- 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).
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