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151 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).
- Eczema / atopic dermatitis
Molecular Signatures of Cutaneous Dupilumab Response
Studying how dupilumab affects immune cells in eczema skin
📍 University of California, San Francisco · San Francisco, CA · Get directions →
This study examines the effect of IL4RA blockade with dupilumab on the immune cells of atopic dermatitis skin lesions.
- 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.
- 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.
- 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.
- 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.
- Crohn's / ulcerative colitis (IBD)
Clinical, Imaging, and Endoscopic Outcomes of Children Newly Diagnosed With Crohn's Disease
Looking at tests and scans in children newly diagnosed with Crohn’s disease
📍 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.
- Multiple sclerosis (MS)
Efficacy and Safety of Remibrutinib After Switching From Ocrelizumab in Participants Living With Relapsing Multiple Sclerosis.
Testing remibrutinib in people with relapsing multiple sclerosis who switch from ocrelizumab versus
📍 Perseverance Research Center · Scottsdale, AZ · +39 more US sites · Get directions →
The purpose of this Phase 3b study is to assess the efficacy, safety and tolerability of remibrutinib after switching from ocrelizumab and compared to continuous ocrelizumab treatment, in patients living with relapsing multiple sclerosis (plwRMS).
- Multiple sclerosis (MS)
Open-label, Multi-center, Phase I/II Study to Assess Safety, Disease Progression and Cellular Kinetics Following YTB323 Administration in Participants With Non-active Progressive Multiple Sclerosis (PMS)
Testing the safety and effects on disease and immune cells of one dose of YTB323 in people with
📍 Novartis Investigative Site · Darlinghurst, New South Wales · Get directions →
This is an open-label, multi-center, non-confirmatory study to assess the safety, disease progression, and cellular kinetics following YTB323 administration to 28 participants with non-active Progressive Multiple Sclerosis (PMS). The study design utilizes an ascending single dose design consisting of 3 sentinel cohorts followed by an expansion cohort.
- Multiple sclerosis (MS)
Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of a Modified Regimen of Ublituximab
Testing a modified ublituximab treatment for relapsing multiple sclerosis
📍 TG Therapeutics Investigational Trial Site · Birmingham, AL · +34 more US sites · Get directions →
The primary purpose of this phase 3b study is to assess the efficacy of a modified regimen of ublituximab in participants with relapsing multiple sclerosis (RMS) as measured by T1 Gadolinium (Gd)-enhancing lesions in Part A; PK in Part B along with efficacy of ublituximab as measured by T1 Gd-enhancing lesions in participants who had a suboptimal experience on prior anti-CD20 therapy in Part C. The study consists of 3 parts: Part A is single-armed and open-label, Part B is randomized, double-blind, placebo-controlled, and Part C is single-armed and open-label.
- Multiple sclerosis (MS)
An Open-label Study of AZD0120 in Adults With Multiple Sclerosis
Testing safety of a new immune cell therapy in adults with multiple sclerosis
📍 Research Site · Tucson, AZ · +8 more US sites · Get directions →
This trial is a Phase 1b, open-label, multi-center, clinical study of AZD0120, a BCMA/CD19 dual targeting CAR+ T-cell therapy, to evaluate the safety and tolerability in adult participants with Multiple Sclerosis.
- Multiple sclerosis (MS)
Ublituximab (Briumvi) for Early Forms of Relapsing Multiple Sclerosis
Testing safety of a twice-yearly medicine for early relapsing MS
📍 Northwestern Memorial Hospital · Chicago, IL · Get directions →
In this prospective, open-label, single-arm, single-institution trial, the investigators will accomplish the following two aims: 1. study the safety and tolerability of Ublituximab (Briumvi) twice annually in participants with early MS over a treatment observation period of \~12 months.
- Sickle cell disease💵 May compensate
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
📍 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.
- Lupus (SLE)Eczema / atopic dermatitis
Evaluating the Safety and Tolerability of Baricitinib in Patients With Job Syndrome With Lupus-Like Disease and/or Atopic Dermatitis
Testing whether baricitinib is safe in people with Job syndrome and lupus or eczema
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: Autosomal dominant hyper-IgE syndrome (HIES), also called Job syndrome, is a genetic disorder that affects the immune system. It can cause skin and lung infections and problems with blood vessels, connective tissues, and bones.
- Crohn's / ulcerative colitis (IBD)
A Study of Vedolizumab With Tofacitinib in Adults With Ulcerative Colitis (UC)
Testing a medicine combination and then a single medicine for ulcerative colitis in adults
📍 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.
- 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.
- Multiple sclerosis (MS)
A Study Evaluating the Safety and Efficacy of KITE-363 in Relapsed/Refractory Autoimmune Neurologic Diseases
Testing the safety and effects of KITE-363 in people with multiple sclerosis
📍 Stanford Neuroscience Health Center · Palo Alto, CA · +3 more US sites · Get directions →
This study will have two Phases: Phase 1a and Phase 1b. The goals of this clinical study are to learn more about the study drug KITE-363, by evaluating its safety, tolerability and efficacy in participants with relapsed/refractory autoimmune neurologic diseases.
- Multiple sclerosis (MS)
Dalfampridine Combined With Physical Therapy for Mobility Impairment in Multiple Sclerosis
Testing whether combining a walking medication with physical therapy improves mobility in people
📍 MGH Institute of Health Professions · Boston, MA · Get directions →
The goal of this clinical trial is to evaluate if combining a medication that can help improve walking in people with multiple sclerosis (MS) with a physical therapy program is better for improving walking than either treatment alone. The main questions this study will answer are: * Does combining dalfampridine with physical therapy improve mobility more than physical therapy without concurrent dalfampridine?
- Multiple sclerosis (MS)
A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell ( CAR-T) Therapy in Subjects With Non-relapsing and Progressive Forms of Multiple Sclerosis
Testing a CD19 CAR-T cell treatment for progressive multiple sclerosis
📍 Stanford Multiple Sclerosis Center · Palo Alto, CA · Get directions →
A Study of Anti-CD19 Chimeric Antigen Receptor T Cell Therapy in Subjects with Non-relapsing and Progressive Forms of Multiple Sclerosis
- 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.
- Crohn's / ulcerative colitis (IBD)
An Early-Stage Study in Multiple Clinics of How Afimkibart May Affect the Body's Processing of Medicines That Rely on Cytochrome P450 Enzymes in Participants With Ulcerative Colitis
Testing how afimkibart in people with ulcerative colitis affects drug processing in the liver
📍 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).
- 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).
- Eczema / atopic dermatitis
A Study of the Medicine Called Abrocitinib in Children 6 to Less Than 12 Years of Age With Moderate-to-Severe Eczema
Testing whether a medicine called abrocitinib helps children with eczema and is safe
📍 Cahaba Dermatology & Skin Health Center, LLC · Birmingham, AL · +9 more US sites · Get directions →
This research study is being conducted to find out if the test medicine, abrocitinib, improves eczema and is safe for children 6 to \<12 years of age who have moderate-to-severe eczema. Research study participants who meet the study criteria will be assigned by chance (like the flip of a coin) to receive either abrocitinib test medicine or placebo (pretend medicine that looks just like the test medicine) for 16 weeks.
- Eczema / atopic dermatitis
A Study of BBT001 in Healthy Volunteers (HVs) and in Adult Patients With Atopic Dermatitis (AD)
Testing BBT001 in healthy adults and adults with eczema
📍 First OC Dermatology · Irvine, CA · +2 more US sites · Get directions →
This is a Phase 1, randomized, blinded, placebo controlled, single-ascending dose (SAD) and multiple-ascending dose (MAD) study of BBT001 in healthy volunteers (HVs) and adult patients with moderate to severe Atopic Dermatitis (AD).
- Multiple sclerosis (MS)
A Study of Orelabrutinib in Patients With Primary Progressive Multiple Sclerosis
Testing a drug called orelabrutinib versus placebo in people with PPMS
📍 Arizona Neuroscience Research, LLC · Pheonix, AZ · +12 more US sites · Get directions →
Orelabrutinib is a CNS-penetrable BTK inhibitor. This is a phase 3, randomized, double-blind, parallel-group, multicenter study to evaluate the efficacy and safety of orelabrutinib compared with placebo in patients with PPMS.
- Eczema / atopic dermatitis
Dupilumab De-escalation in Pediatric Atopic Dermatitis
Testing whether stopping or lowering eczema treatment works for kids
📍 Johns Hopkins Univerisity · Baltimore, MD · Get directions →
This is a pilot investigator-blinded, randomized clinical trial to assess the feasibility of dupilumab treatment discontinuation or dose-reduction in children aged 1-17 years who have achieved sustained atopic dermatitis (AD) control on dupilumab.
- 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.
- 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.
- Multiple sclerosis (MS)
Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS)
Comparing stem cell transplant vs best available treatment for relapsing multiple sclerosis
📍 Stanford Multiple Sclerosis Center · Palo Alto, CA · +21 more US sites · Get directions →
This is a multi-center prospective rater-masked (blinded) randomized controlled trial of 156 participants, comparing the treatment strategy of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) to the treatment strategy of Best Available Therapy (BAT) for treatment-resistant relapsing multiple sclerosis (MS). Participants will be randomized at a 1 to 1 (1:1) ratio.
- 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.
- 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.
- 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.
- Crohn's / ulcerative colitis (IBD)
A Study to Assess Adverse Events, Change in Disease Activity, and How Intravenous and Subcutaneous Risankizumab Moves Through the Body of Pediatric Participants With Moderately to Severely Active Crohn's Disease
Testing how a medicine given by IV or shot affects Crohn’s disease in children
📍 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)
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.
- Eczema / atopic dermatitis
Ph 1a/1b Single Ascending Dose and Multiple Ascending Dose Study of ARQ-234
Testing a new drug dose in people with eczema and healthy volunteers
📍 Clinical Site 101 · Fair Lawn, NJ · Get directions →
This is a first-in-human, Phase 1, double-blind, randomized, placebo-controlled, dose-escalation study evaluating ARQ-234. The study is designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ARQ-234 in two populations: healthy volunteers and participants with moderate to severe atopic dermatitis (AD).
- 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.
- Multiple sclerosis (MS)
A Study of Orelabrutinib in Patients With Secondary Progressive Multiple Sclerosis
Testing orelabrutinib versus placebo for people with non-active secondary progressive multiple
📍 Arizona Neuroscience Research, LLC · Phoenix, AZ · +12 more US sites · Get directions →
Orelabrutinib is a CNS-penetrable BTK inhibitor. This is a phase 3, randomized, double-blind, parallel-group, multicenter study to evaluate the efficacy and safety of orelabrutinib compared with placebo in patients with non-active Secondary Progress MS.
- 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.
- Multiple sclerosis (MS)
A Study to Investigate the Safety, Tolerability, and Processing by the Body of Intravenous and Subcutaneous RO7121932 Administration in Participants With Multiple Sclerosis
Testing how a new medicine given through a vein or under the skin is handled and tolerated in people
📍 Stanford University Medical Center · Stanford, CA · +4 more US sites · Get directions →
The primary purpose of the study is to evaluate the safety and tolerability of a single-ascending intravenous (IV) dose (Part 1), a single-ascending subcutaneous (SC) dose (Part 2), and multiple ascending SC doses (Part 3) of RO7121932 in participants with multiple sclerosis (MS).
- Multiple sclerosis (MS)
Home Based Infusions for Ocrelizumab
Testing the safety and effectiveness of home infusions of ocrelizumab for multiple sclerosis
📍 Amerita · Centennial, CO · Get directions →
The goal is to assess the safety and effectiveness of home ocrelizumab infusion.
- Multiple sclerosis (MS)
Study to Evaluate the Effectiveness and Safety of Ozanimod Compared to Fingolimod in Children and Adolescents With Relapsing Remitting Multiple Sclerosis
Testing ozanimod versus fingolimod in children with multiple sclerosis
📍 Local Institution - 0114 · Loma Linda, CA · +11 more US sites · Get directions →
The purpose of this study is to evaluate the effectiveness, safety, tolerability, drug levels and drug effects of ozanimod compared to fingolimod in children and adolescents with relapsing remitting multiple sclerosis (RRMS).
- Multiple sclerosis (MS)
Treatment of Participants With Primary or Secondary Progressive Multiple Sclerosis
Testing whether TRX319 is safe for people with progressive multiple sclerosis
📍 University of Kansas Medical Center · Kansas City, KS · +1 more US site · Get directions →
The goal of this clinical trial is to treat male and female participants with two types of Multiple Sclerosis (MS) called primary progressive or secondary progressive MS. The main questions the trial aims to answer are the following: * Is TRX319 safe when administered to patients with progressive forms of MS?
- 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.
- 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).
- Crohn's / ulcerative colitis (IBD)
A Study to Evaluate the Pharmacokinetics, Safety and Efficacy of Afimkibart (RO7790121) in Children With Moderately to Severely Active Ulcerative Colitis
Testing a medicine in children with moderate to severe ulcerative colitis
📍 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).
- Multiple sclerosis (MS)
A Study to Evaluate the Safety, Tolerability, Efficacy, and Drug Levels of CC-97540 in Participants With Relapsing Forms of Multiple Sclerosis, Progressive Forms of Multiple Sclerosis or Refractory Myasthenia Gravis (MG) (Breakfree-2)
Testing a new drug’s safety and effects in people with multiple sclerosis
📍 Local Institution - 0011 · Birmingham, AL · +16 more US sites · Get directions →
The purpose of this study is to evaluate the safety, tolerability, efficacy, and drug levels of CC-97540 in participants with Relapsing Forms of Multiple Sclerosis (RMS), Progressive Forms of Multiple Sclerosis (PMS) or Refractory Myasthenia Gravis (MG).
- Eczema / atopic dermatitis
Lebrikizumab in Moderate-to-severe Atopic Dermatitis
Testing a medicine called lebrikizumab for long-term improvement in eczema
📍 Physioseq USA - CA · Folsom, CA · +1 more US site · Get directions →
This research is studying a drug already approved for the treatment of atopic dermatitis (AD). This research collects health-related information and blood and skin samples to understand if the study drug, lebrikizumab, leads to long-term improvement in AD skin.
- 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).
- Dementia / Alzheimer'sMultiple sclerosis (MS)
Exploring the Utility of [18F]3F4AP for Demyelination Imaging
Testing how an imaging tracer works in people with memory loss and multiple sclerosis
📍 Yale University PET Center · New Haven, CT · Get directions →
The overall objective is to obtain an assessment of the pharmacokinetics of \[18F\]3F4AP in healthy volunteers and subjects with demyelinating diseases such as mild cognitive impairment (MCI), Alzheimer's Disease (AD), Multiple Sclerosis (MS), Spinal Cord Injury (SCI) and Spinal radiculopathy (SR).
- Eczema / atopic dermatitis
A Study to Assess Adverse Events and Change in Disease Activity Comparing Oral Upadacitinib to Subcutaneous Dupilumab in Children From 2 to Less Than 12 Years of Age With Moderate to Severe Atopic Dermatitis
Comparing side effects and disease activity between two eczema treatments in children
📍 Applied Research Center Of Arkansas /ID# 268547 · Little Rock, AR · +29 more US sites · Get directions →
Atopic dermatitis (AD) is a skin condition that may cause a rash and itching due to inflammation of the skin. Topical therapies applied over the skin may not be enough to control the AD in trial participants who require systemic anti-inflammatory treatment.
- 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.
- 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.
- 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.
- Multiple sclerosis (MS)
A Study to Evaluate the Efficacy and Safety of Remibrutinib in Secondary Progressive Multiple Sclerosis
Testing how well and how safely remibrutinib works in secondary progressive multiple sclerosis
📍 Alabama Neurology Associates PC · Birmingham, AL · +32 more US sites · Get directions →
The purpose of this study is to provide efficacy and safety data for remibrutinib in patients with secondary progressive multiple sclerosis (SPMS)
- Sickle cell disease
Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease
Testing whether routine red blood cell transfusions in pregnancy reduce sickle cell flare-ups or
📍 Grady Health System · Atlanta, GA · Get directions →
The goal of this study is to determine if there is a positive effect of prophylactic red blood cell (RBC) transfusion of leukoreduced, ABO, Rh (D/Cc/Ee) and Kell matched blood compared to standard of care on the number of episodes of acute sickle cell disease (SCD) manifestations or pregnancy-related complications requiring acute health care encounters (acute care/ER/Hospital visits) or resulting in death over the entirety of pregnancy until 2 months post-partum in women with SCD. RBC transfusion is the only disease-modifying therapy for pregnant women with SCD, and it is considered a standard treatment option however, there exists no consensus on the role of transfusion therapy in preventing SCD-related pregnancy complications.
- Eczema / atopic dermatitis
The Impact of Botox on Neuroimmune Interactions in Atopic Dermatitis
Testing how Botox changes skin immune signals in people with eczema
📍 University of Pittsburgh Medical Center · Pittsburgh, PA · +1 more US site · Get directions →
The purpose of this study is to understand cellular and molecular interactions in the skin of participants with mild-to-moderate AD, and how botulinum toxin alters these interactions.
- Eczema / atopic dermatitis
Ethnic Differences in Mechanisms of Action of Dupilumab
Studying how a common eczema treatment works differently by race
📍 University of Michigan · Ann Arbor, MI · Get directions →
Previous research has shown that Asian and African Americans are more likely to develop atopic dermatitis (AD) than their Caucasian counterparts. However, limited information is known about AD in Asian and African American populations because most molecular studies have focused on Caucasians with AD.
- Crohn's / ulcerative colitis (IBD)
VECTORS - A Study to Evaluate Transmural Healing as a Treatment Target in Crohn's Disease
Testing whether healing through the whole bowel wall is a good treatment goal in Crohn’s disease
📍 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
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.
- 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.
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