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139 studies
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- Migraine / chronic headache
BOTOX® vs. XEOMIN® for Chronic Migraine
Comparing two injections to treat chronic migraine
📍 Naval Medical Center Camp Lejeune · Jacksonville, NC · Get directions →
Chronic migraine (CM) is a disabling disorder that sidelines active duty personnel and diminishes their quality of life. It affects 1.3% to 2.4% of the general population.
- 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.
- Migraine / chronic headache
A Study of Eptinezumab in Pediatric Participants With Episodic Migraine
Testing whether eptinezumab lowers the number of days with episodic migraine in children
📍 Ki Health Partners LLC DBA New England Institute for Clinical Research · Stamford, CT · +14 more US sites · Get directions →
The main goal of this trial is to learn whether eptinezumab helps reduce the number of days with episodic migraine in pediatric participants.
- 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)
A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 2a Study With an Open-Label Extension Evaluating the Efficacy and Safety of VENT-03 in Adult Participants With Active Cutaneous Lupus Erythematosus With or Without Systemic Lupus Erythematosus
Testing a treatment called VENT-03 for skin lupus, with or without body lupus
📍 Investigative Site · Beverly Hills, CA · +9 more US sites · Get directions →
The goal of this clinical trial is to learn if VENT-03 works to treat patients with cutaneous lupus erythematosus (CLE) who may or may not have systemic lupus erythematosus (SLE). Another goal is to learn about the safety of VENT-03 and how it is processed by the body.
- 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.
- Migraine / chronic headache
Efficacy and Safety Study of Rimegepant for the Preventative Treatment of Migraine in Pediatric Subjects
Testing rimegepant versus placebo as a migraine prevention in kids
📍 The Belinga Clinic · Fort Smith, AR · +39 more US sites · Get directions →
The purpose of this study is to compare the efficacy and safety of rimegepant to placebo as a preventative treatment for migraine in children and adolescents ≥ 6 to \<18 years with episodic migraine.
- Lupus (SLE)
CD19-Directed Chimeric Antigen Receptor Autologous T Cells (CART19) for Lupus
Testing a cell therapy made from a person’s own immune cells for lupus
📍 Children's Hospital of Philadelphia · Philadelphia, PA · Get directions →
This is a single-center, single-arm, open-label phase 1/2 study of CART19 in children and young adults with refractory Systemic lupus erythematosus (SLE), including both patients diagnosed with lupus nephritis (LN) and patients with non-renal Systemic lupus erythematosus (SLE). Phase 1 will evaluate the safety of CART19 in 6-12 patients with Systemic lupus erythematosus (SLE).
- Lupus (SLE)
US Zamto-cel Autoimmune Diseases
Testing the safety of an experimental cell therapy for lupus
📍 Froedtert Hospital and the Medical College of Wisconsin · Milwaukee, WI · Get directions →
AID is a phase I multi-cohort study to assess the safety and tolerability of zamtocabtagene autoleucel (zamto-cel) in patients with refractory autoimmune diseases (SLE-Non renal, SLE-LN, SSc/dcSSc) after receiving standard therapy.
- 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.
- Lupus (SLE)
A Study of Enpatoran in Participants With Cutaneous Manifestations of Lupus With or Without Systemic Disease (ELOWEN-2)
Testing enpatoran over 24 weeks for skin lupus symptoms, with or without whole-body disease
📍 Inland Rheumatology Clinical Trials, Inc. · Upland, CA · +7 more US sites · Get directions →
The purpose of this global, multicenter, Phase 3 study is to evaluate the efficacy and safety of enpatoran over 24 weeks in participants with active cutaneous manifestations of lupus erythematosus with or without systemic disease. Study details include: Study Duration: Up to 35 weeks.
- Sickle cell disease
Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults
Testing whether a low dose of ketamine helps treat sickle cell pain in children and young adults
📍 Harbor UCLA Medical Center · Torrance, CA · Get directions →
The purpose of this research is to see if ketamine is effective and safe in treating children and young adults with sickle cell disease experiencing sickle cell related pain. In this study, we will compare the outcomes (such as pain scores) in persons who receive standard of care pain medicine (an opioid such as morphine) plus a low dose (amount) of ketamine to those who receive only standard of care pain medicine.
- Lupus (SLE)
A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease
Testing the safety and early effects of a new treatment for lupus in adults
📍 Research Site 4 · Redwood City, CA · +6 more US sites · Get directions →
This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult subjects with refractory autoimmune diseases, including active systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM).
- 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).
- Lupus (SLE)
A Phase 1b Study of Budoprutug in Systemic Lupus Erythematosus (SLE)
Testing the safety and tolerance of budoprutug in adults with lupus
📍 Climb Bio Investigative Site #100104 · Allen, TX · +1 more US site · Get directions →
The main objective is to assess the safety and tolerability of budoprutug in adults with SLE. Pharmacokinetics, pharmacodynamics, and preliminary clinical efficacy will also be assessed.
- Migraine / chronic headache
A Study of Oral Atogepant Tablets to Assess Adverse Events and Change in Disease Activity To Prevent Migraine in Participants Aged 12 to 17 Years
Testing an oral medicine to track side effects and migraine changes in teens aged 12 to 17
📍 Rehabilitation and Neurological Services /ID# 270782 · Huntsville, AL · +30 more US sites · Get directions →
Migraine is a disease that most often causes moderate to severe headache on one side of the head. A migraine attack is a headache that may be accompanied by throbbing, nausea, vomiting, sensitivity to light and sound, or other symptoms.
- Lupus (SLE)
A Study to Test Whether Different Doses of BI 3000202 Help People With Systemic Lupus Erythematosus (SLE)
Testing whether different doses of BI 3000202 help adults with lupus
📍 Wallace & Lee Center · Beverly Hills, CA · +29 more US sites · Get directions →
This study is open to adults with systemic lupus erythematosus (SLE). The purpose of this study is to find out whether a medicine called BI 3000202 helps people with SLE.
- Lupus (SLE)Rheumatoid arthritis
A Study of CC-97540, CD-19-Targeted Nex-T CAR T Cells, in Participants With Severe, Refractory Autoimmune Diseases (Breakfree-1)
Testing a CD19-targeted CAR T cell treatment in people with severe lupus or rheumatoid arthritis
📍 University of Colorado Anschutz Medical Campus · Aurora, CO · +30 more US sites · Get directions →
The purpose of this study is to establish the tolerability, preliminary efficacy, and pharmacokinetics of CC-97540 in participants with severe, refractory autoimmune diseases (Breakfree-1).
- 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.
- Migraine / chronic headache
A Study to Learn About the Study Medicine Called Rimegepant in Adolescents With Frequent Migraine
Testing rimegepant tablets in adolescents with frequent migraine
📍 Xenoscience Inc · Phoenix, AZ · +26 more US sites · Get directions →
The purpose of this study is to learn about the effect of a study medicine called rimegepant in adolescents who have frequent migraine attacks. Rimegepant is a tablet that dissolves when you put it on or under your tongue.
- Sickle cell disease
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
Testing a once-daily mouth medicine in teens with sickle cell disease
📍 The Hospital for Sick Children · Toronto, Ontario · Get directions →
This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat.
- Lupus (SLE)
Study to Evaluate the Efficacy of Ruxolitinib 1.5% Cream in Adult Subjects with Discoid Lupus Erythematosus
Testing a 1.5% ruxolitinib cream for discoid lupus in adults
📍 INNO-6051 Site 03 · Fredericton, New Brunswick · Get directions →
This study is a single-blind, intraindividual study to evaluate the efficacy of ruxolitinib 1.5 % cream in adult subjects with discoid lupus erythematosus.
- Lupus (SLE)
The Pediatric Lupus Nephritis Mycophenolate Mofetil (PLUMM) Study
Testing two ways of giving mycophenolate to children with lupus kidney disease
📍 University of California, San Francisco · San Francisco, CA · +18 more US sites · Get directions →
The study is a 1-year 2-part double-blinded placebo controlled 2-arm clinical trial. Treatment arms are (1) MMF dosed as per body-surface area (MMFBSA; 600mg/m2 body surface area per dose about every 12 hours) and (2) pharmacokinetically-guided precision-dosing of MMF (MMFPK; MMF dosed twice daily to achieve an area under the concentration-time curve (AUC0-12h) of MPA \>60-70 mg\*h/L.
- Lupus (SLE)
A Study of GSK5926371 in Participants With B-cell Driven Autoimmune Rheumatic Diseases (ARD)
Testing different doses of an experimental medicine for lupus
📍 GSK Investigational Site · Buenos Aires, · Get directions →
This is a 2-part study of GSK5926371 in participants with autoimmune rheumatic diseases (ARD). In part 1, participants will receive different doses of GSK5926371 to find a suitable priming dose.
- Lupus (SLE)
A Phase 1 Study of FT819 in B-cell Mediated Autoimmune Disease
Testing an immune treatment for safety and effects on B-cells in lupus
📍 Wallace Rheumatic Center · Beverly Hills, CA · +15 more US sites · Get directions →
This is a phase 1 study designed to evaluate the safety, pharmacokinetics (PK), and anti-B-cell activity of FT819 following treatment with or without auxiliary medicinal product (AMP) in participants with moderate-to-severe active systemic lupus erythematosus (SLE) with or without nephritis, antineutrophilic cytoplasmic antibody (ANCA)-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). The study will consist of a dose-escalation stage, followed by an expansion stage to further evaluate the safety and activity of FT819.
- Lupus (SLE)
RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus
Testing the safety and effects of CABA-201 in people with active lupus
📍 University of California Irvine · Orange, CA · +20 more US sites · Get directions →
RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus
- Lupus (SLE)
Phase 3 Extension Study to Evaluate Long-term Safety of Ianalumab in Participants With Systemic Lupus Erythematosus (SIRIUS-SLE Extension).
Testing long-term safety of ianalumab for people with lupus
📍 Pinnacle Research Group Llc · Anniston, AL · +20 more US sites · Get directions →
The purpose of this study is to evaluate long-term safety and tolerability of ianalumab in participants with systemic lupus erythematosus who have previously completed the treatment period in one of the two SIRIUS-SLE core studies (CVAY736F12301 or CVAY736F12302).
- Lupus (SLE)
BAFF CAR-T Cells (LMY-920) for Systemic Lupus Erythematosus
Testing safety and best dose for CAR-T cell treatment in adults with lupus
📍 Nationwide Children's · Columbus, OH · Get directions →
This phase 1 study seeks to examine the safety and recommended phase 2 dose (RP2D) of BAFF-ligand CAR-T cells (LMY-920) in adult patients with refractory systemic lupus erythematosus (SLE). It is hypothesized that BAFF CAR-T cells will be safe and will improve SLE disease activity scores.
- Migraine / chronic headache
A Study to Evaluate the Efficacy and Safety of Elismetrep (K-304) in the Acute Treatment of Migraine
Testing whether elismetrep works and is safe for treating migraine attacks
📍 Central Research Associates, LLC (CRA) dba Flourish Research · Birmingham, AL · +39 more US sites · Get directions →
This is a double-blind, randomized, multicenter, outpatient evaluation of the efficacy, safety, and tolerability of elismetrep, as compared with placebo, in the acute treatment of migraine.
- Migraine / chronic headache
A Study to Investigate the Safety and Efficacy of MEDI0618 Compared to Placebo in Adult Participants With Episodic Migraine
Testing a medicine shot to treat episodic migraine vs placebo
📍 Flourish - Birmingham · Birmingham, AL · +15 more US sites · Get directions →
The purpose of this Phase 2 study is to evaluate the safety and efficacy of SC MEDI0618 compared to placebo in participants with episodic migraine.
- Migraine / chronic headache
Study of EVO756 in Adults With Migraine
Testing a medicine called EVO756 compared with placebo in adults with migraine
📍 CA Medical Clinic for Headache · Los Angeles, CA · +12 more US sites · Get directions →
This study will evaluate the efficacy and safety of EVO756 against placebo in adults with migraine.
- Migraine / chronic headache
Study to Assess Adverse Events and Disease Activity of Oral Ubrogepant Tablets for the Acute Treatment of Migraine in Children and Adolescents (Ages 6-17)
Testing side effects and migraine symptom activity of a tablet for kids
📍 Rehabilitation & Neurological Services /ID# 229969 · Huntsville, AL · +39 more US sites · Get directions →
Migraine is a common neurological disorder typically characterized by attacks of throbbing, moderate to severe headache, often associated with nausea, vomiting, and sensitivity to light and sound. Migraine is extremely common and disabling in children.
- 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.
- Lupus (SLE)
Effects of Stopping Hydroxychloroquine in Elderly Lupus Disease
Testing what happens to older people with lupus if they stop their hydroxychloroquine
📍 University of California, Los Angeles · Los Angeles, CA · +9 more US sites · Get directions →
Hydroxychloroquine (HCQ) is a systemic lupus erythematosus (SLE) medication that has been very effective in reducing lupus disease activity and keeping patients stable with reduced symptoms. Despite a track record of safety with regard to infection compared to traditional immunosuppressive agents, the risk of HCQ retinal toxicity escalates with continued use.
- Lupus (SLE)
A Study to Investigate the Safety, Tolerability, and Efficacy of AZD0120 in Adults With Refractory SLE
Testing a CAR T cell treatment for adults with hard-to-treat lupus
📍 Research Site · San Francisco, CA · +14 more US sites · Get directions →
This is a Phase 1b/2, single-arm, open-label, multi-center, clinical study of AZD0120, a CD19/BCMA dual CAR T cell therapy, to evaluate the safety, tolerability, and efficacy in adult participants with refractory Systemic Lupus Erythematosus.
- Lupus (SLE)
A Study to Assess Adverse Events, How the Drug Moves Through the Body and Effectiveness of Intravenous Infusions of ABBV-319 in Adult Participants With Systemic Lupus Erythematosus (SLE) and Sjogren's Disease (SjD)
Testing side effects and how an intravenous drug works in lupus and Sjogren’s
📍 Life Clinical Trials - Colonial Drive - Margate /ID# 276050 · Margate, FL · +3 more US sites · Get directions →
Systemic lupus erythematosus (SLE) is a chronic, systemic autoimmune disease characterized by B cell hyperactivity and Sjorgren's disease (SjD) is a chronic, multisystem autoimmune disease characterized by lacrimal and salivary gland inflammation, with resultant dryness of the eyes and mouth and occasional glandular enlargement. ABBV-319 exhibits potential B cell depletion in SLE and SjD which are characterized by B cell hyperactivity.
- 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).
- Lupus (SLE)💵 May compensate
A Research Trial to Assess if Cenerimod is Efficacious and Safe to Treat Active Lupus Nephritis on Top of Regular Treatment
Testing whether cenerimod added to usual treatment helps adults with active lupus nephritis
📍 Alloy Clinical Research LLC · Kissimmee, FL · +10 more US sites · Get directions →
The goal of this clinical trial is to learn if cenerimod, on top of regular treatment, works to treat active lupus nephritis in adults with systemic lupus erythematosus and active lupus nephritis. It will also learn about the safety of cenerimod.
- Migraine / chronic headache
Effectiveness of Magnesium in Addition to Prochlorperazine for the Treatment of Migraines
Testing whether adding magnesium to prochlorperazine reduces migraine pain
📍 Advocate Christ Medical Center Emergency Department · Oak Lawn, IL · Get directions →
The purpose of this research study is to find out if using magnesium in addition to prochlorperazine will help reduce your migraine pain.
- Lupus (SLE)
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Obinutuzumab in Adolescents With Active Class III or IV Lupus Nephritis and the Safety and PK of Obinutuzumab in Pediatric Participants
Testing a medicine in children and teens with active lupus kidney disease
📍 Loma Linda University health · Loma Linda, CA · +10 more US sites · Get directions →
This phase II, randomized, double-blind, placebo-controlled study is designed to evaluate the safety, efficacy and pharmacokinetics (PK) of obinutuzumab in adolescent participants (AP) aged 12 to less than 18 with biopsy-confirmed proliferative lupus nephritis (LN). It will also evaluate open label safety and PK of obinutuzumab in pediatric participants (PP), aged 5 to \<12 with LN.
- Lupus (SLE)
REACT-01: Reversing Autoimmunity Through Cell Therapy
Testing a modified immune cell therapy for children and young adults with lupus
📍 Seattle Children's Hospital · Seattle, WA · Get directions →
This is a phase 1, open-label, non-randomized study enrolling pediatric and young adult research participants with treatment-refractory Systemic Lupus Erythematosus (SLE), to examine the safety, feasibility, and efficacy of administering T cell products derived from peripheral blood mononuclear cells (PBMC) that have been genetically modified to express CD19 specific chimeric antigen receptor (CAR) A child or young adult meeting all eligibility criteria and meeting none of the exclusion criteria will have their T cells collected. The T cells will then be bioengineered into a CAR T cell that targets circulating and tissue residing B cells.
- 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.
- Lupus (SLE)
Alnuctamab for Refractory SLE (LATTE Study)
Testing alnuctamab in people with moderate to severe lupus not helped
📍 Icahn School of Medicine at Mount Sinai · New York, NY · Get directions →
This study will assess the safety and preliminary efficacy of the bi-specific TCE, alnuctamab (known as BMS-986349, CC-93269, EM901), targeting BCMA in patients with moderate to severe SLE, refractory to standard-of-care treatments.
- Lupus (SLE)
Phase I/II Study of AD-PluReceptor Plus Tafasitamab-cxix and Lymphodepleting Chemotherapy in Patients With Autoimmune Disorders
Testing the safety and best dose of an immune cell treatment with lupus drugs and chemotherapy
📍 MD Anderson Cancer Center · Houston, TX · +1 more US site · Get directions →
The goal of Safety Lead-In is to confirm the safety of tafasitamab when given to patients with SSc, SLE, and LN. The goal of Phase 1 is to find the recommended dose of AD-PluReceptor-NK cells in combination with tafasitamab and lymphodepleting chemotherapy that can be given to patients with the disease.
- Sickle cell disease
Early Screening and Treatment of Heart Complication in Sickle Cell Disease
Testing early heart screening and iron overload treatment in people with sickle cell disease
📍 Inova Schar Cancer · Fairfax, VA · +1 more US site · Get directions →
This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.
- Sickle cell disease
Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial
Testing two daily doses of zinc in people with sickle cell disease to see changes in bone-related
📍 UCSF Benioff Children's Hospital Oakland · Oakland, CA · +5 more US sites · Get directions →
The goal of this short term prospective Phase II study is to compare the effects of two alternate daily doses of zinc (25 and 40 mg/day) in 34 randomly assigned homozygous Sickle Cell Disease (SCD-SS) patients aged 15-40 years old. The main question it aims to answer is: Which biomarkers are most responsive to zinc supplementation, and what is the maximum tolerated zinc dose that induces the desired changes in biomarkers of bone turnover?
- 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
Allo HSCT for High Risk Hemoglobinopathies
Testing donor stem cell transplant for people with high-risk sickle cell
📍 Masonic Cancer Center · Minneapolis, MN · Get directions →
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.
- 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.
- Heart / cardiovascular diseaseLupus (SLE)
The Role of Anifrolumab in Improving Markers of Vascular Risk in Patients With Systemic Lupus Erythematosus (SLE) - IFN-CVD
Testing whether anifrolumab improves blood vessel risk markers in lupus
📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →
Background: People with systemic lupus erythematosus (SLE) are at risk of developing complications in their blood vessels. This can increase the risk of heart attacks or stroke.
- Lupus (SLE)
A Study of GSK4527363 in Healthy Participants, Systemic Lupus Erythematosus (SLE) Participants, Healthy Chinese, and Japanese Participants and CTD-ILD Participants
Testing the safety and immune effects of a drug in people with lupus and related lung disease
📍 GSK Investigational Site · Scottsdale, AZ · +5 more US sites · Get directions →
This study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of GSK4527363 in healthy participants (Part A), participants with active SLE (Part B), healthy participants of Chinese and Japanese descent (Part C), and participants with interstitial lung disease associated with connective tissue disease (Part D)
- Lupus (SLE)
A Study of Rapcabtagene Autoleucel in Active, Refractory Systemic Lupus Erythematosus (SLE) or Lupus Nephritis (LN) Patients (AUTOGRAPH - SLE/LN)
Testing a one-time cell treatment plus chemotherapy to treat severe lupus
📍 University Of Alabama · Birmingham, AL · +18 more US sites · Get directions →
The purpose of this study is to evaluate the efficacy and safety of rapcabtagene autoleucel (administered once following lymphodepletion) in patients with active, refractory systemic lupus erythematosus (SLE) or active, refractory lupus nephritis (LN).
- Lupus (SLE)
A Study of Enpatoran in Participants With Cutaneous Manifestations of Lupus With or Without Systemic Disease
Testing a treatment called enpatoran for skin lupus symptoms over 24 weeks
📍 Dermatology Research Associates - 300204025 · Los Angeles, CA · +10 more US sites · Get directions →
The purpose of this global, multicenter, Phase 3 study is to evaluate the efficacy and safety of enpatoran over 24 weeks in participants with active cutaneous manifestations of lupus erythematosus with or without systemic disease. Study details include: Study Duration: Up to 35 weeks.
- Lupus (SLE)
Obe-cel in Severe, Refractory Systemic Lupus Erythematosus (SLE) With Active Lupus Nephritis (LN)
Testing a one-time cell therapy approach for severe lupus with kidney involvement
📍 State University of New York Upstate Medical Center (SUNY) · Syracuse, NY · +1 more US site · Get directions →
The purpose of this trial is to evaluate the efficacy and safety of obecabtagene autoleucel (obe-cel) administered once following lymphodepletion in participants with severe, refractory systemic lupus erythematosus (SLE) and active lupus nephritis (LN).
- Lupus (SLE)Rheumatoid arthritisCrohn's / ulcerative colitis (IBD)
COVID-19 Booster and IIV Schedule in Immunocompromised Hosts
Testing COVID-19 booster and flu vaccine schedules in people with lupus, rheumatoid arthritis, or
📍 Maisonneuve -Rosemont Installation · Montreal, Quebec · Get directions →
The goal of this pragmatic embedded open-label, 2 x 2 factorial phase II randomized controlled trial is to evaluate strategies to improve COVID-19 booster and influenza vaccine immunogenicity in people living with immunocompromising conditions (PLIC). The main questions it aims to answer are: 1.
- Sickle cell disease
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Testing immune-suppressing treatment before stem cell transplant for people with sickle cell disease
📍 Memorial Sloan Kettering at Basking Ridge (Consent only) · Basking Ridge, NJ · +5 more US sites · Get directions →
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD.
- 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.
- 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.
- Lupus (SLE)
A Study to Investigate the Safety and Preliminary Efficacy of ALLO-329, an Allogeneic CAR T-cell Therapy, in Adults With Autoimmune Disease
Testing safety and early effects of a CAR T-cell treatment in adults with lupus
📍 Mayo Clinic · Phoenix, AZ · +12 more US sites · Get directions →
This is a first-in-human, single-arm, open-label study evaluating the safety, tolerability, and preliminary efficacy of ALLO-329 in adults with autoimmune diseases: systemic lupus erythematosus (SLE) with and without renal involvement, idiopathic inflammatory myopathy (IIM), and systemic sclerosis (SSc).The purpose of this trial is to evaluate the safety and tolerability of ALLO-329, an allogeneic anti-CD19, anti-CD70 dual chimeric antigen receptor (CAR) T cell therapy, in adults with autoimmune disorders, provide initial evidence of biological activity and clinical response to the treatment and determine the recommended Phase 2 regimen (RP2R).
- Lupus (SLE)
Descartes-08 for Children, Adolescents, and Young Adults With Autoimmune Disorders
Testing the safety and effects of Descartes-08 in young people with lupus
📍 H01- Children's National Hospital · Washington D.C., DC · +1 more US site · Get directions →
Safety, tolerability and efficacy of Descarte-08 in children, adolescents and young adults with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis
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