Clinical trials: Hemoglobinopathy
Transfusion-dependent Hemachromatosis, Thalassemia Major
Pilot Study for Patients With Poor Response to Deferasirox
This purpose of this study is to understand the differences between people who have a good response to deferasirox (exjade) compared to people who have a poor response to this medication when used for transfusion-dependent iron…
- Conditions
- Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
- Trial name
- NCT00749515
- Testing
- Deferoxamine, Deferasirox, HIDA
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Boston Children's Hospital
Sickle Cell Disease, Sickle Cell Anemia
Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited blood disorder. It results from a single genetic change (mutation) in red blood cells (RBCs). RBCs are the cells that carry oxygen to the body.
- Trial name
- NCT04476277
- Testing
- Biotin label
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, COVID-19
COVID-19 Vaccine Response in Sickle Cell Disease
The purpose of this study is to assess the antibody response to COVID-19 vaccination in a cohort of patients with sickle cell disease (SCD) and to assess vaccine and SCD related complications around the time of vaccination.
- Trial name
- NCT05139992
- Testing
- COVID-19 Vaccine
- Where
- 8 sites in United States
- Volunteers
- 59 people
- Sponsor
- ASH Research Collaborative
Sickle Cell Disease
Voxelotor CYP and Transporter Cocktail Interaction Study
This research study is examining multiple doses of voxelotor (a study drug intended for treatment of sickle cell disease) and how it interacts with additional substrates (substrates are drugs or other substances that are…
- Trial name
- NCT05981365
- Testing
- Voxelotor, Bupropion, Repaglinide
- Where
- United States
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Pfizer
Sickle Cell Anemia, Sickle Cell-Beta0-Thalassemia
The Effect of Rivaroxaban in Sickle Cell Disease
The primary study hypothesis is that inhibition of factor Xa with rivaroxaban will reduce inflammation, coagulation and endothelial cell activation, and improve microvascular blood flow in patients with sickle cell disease (SCD)…
- Trial name
- NCT02072668
- Testing
- rivaroxaban
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of North Carolina, Chapel Hill
Hemoglobin SC Disease
Treatment of Hemoglobin SC Disease With Hydroxyurea
Sickle cell disease (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle cell disease with typically higher hemoglobin and milder or later disease complications.
- Trial name
- SCYTHE (NCT02336373)
- Testing
- hydroxyurea
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Baylor College of Medicine
Transfusion Dependent Beta-thalassemia
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age.
- Trial name
- NCT03432364
- Testing
- ST-400 Investigational product
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Sangamo Therapeutics
Sickle Cell Disease
Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) who have high risk features.
- Trial name
- STRIDE (NCT01565616)
- Testing
- Conditioning Regimen with Bone Marrow Transplant
- Where
- 8 sites in United States
- Volunteers
- 22 people
- Sponsor
- Emory University
Anemia, Iron Deficiency
Is Iron Deficiency the Cause of Anemia Among Women in Cambodia?
Globally, the most common cause of anemia is thought to be iron deficiency anemia (IDA). This was assumed to be the major cause of anemia in Cambodia, because Cambodian diets, which consist mainly of rice, lack iron-rich animal…
- Conditions
- Anemia, Iron Deficiency, Hemoglobin Disorder, Infection
- Trial name
- NCT02481375
- Testing
- Multiple micronutrients, Iron
- Where
- Cambodia
- Volunteers
- 809 people, healthy volunteers welcome
- Sponsor
- University of British Columbia
Sickle Cell Disease
Inhaled Mometasone to Reduce Painful Episodes in Patients With Sickle Cell Disease
The proposed research is designed to test the global hypothesis that inhaled corticosteroids (ICS), a therapy developed to treat asthma, will prevent vasoocclusive painful episodes in adults with Sickle Cell Disease (SCD) who…
- Trial name
- IMPROVE (NCT02061202)
- Testing
- Mometasone Furoate
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- Jeffrey Glassberg
Sickle Cell Disease, Thalassemia
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
Background: Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs.
- Conditions
- Sickle Cell Disease, Thalassemia, Stem Cell Transplantation, Graft vs Host Disease
- Trial name
- NCT02105766
- Testing
- Alemtuzumab, Sirolimus, Cyclophosphamide
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Beta-thalassemia Major, Hemosiderosis
Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
To allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available.
- Conditions
- Beta-thalassemia Major, Hemosiderosis, Iron Overload, Rare Anemia
- Trial name
- ESCALATOR (NCT00171301)
- Testing
- Deferasirox
- Where
- 5 sites in Egypt, Lebanon and 3 more
- Volunteers
- 233 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Hematological Diseases
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants with severe sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Trial name
- NCT03745287
- Testing
- Exa-cel
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease
Building Adaptive Coping and Knowledge to Improve Daily Life
The purpose of this study is to find out how teenagers with chronic pain and sickle cell disease respond to a new training program called Back2Life and get their feedback about how to modify the program to best fit their needs.
- Trial name
- Back2Life (NCT04602728)
- Testing
- Back2Life
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Emory University
Pain, Sickle Cell Disease
Choosing Opioid Management for Pain and Analyzing Acute Chest Syndrome (ACS) Rates Equally
The pathophysiology of sickle cell disease (SCD) manifestations, are complex with interactions of intracellular hemoglobin, membrane and endothelial activation but the hallmark remains recurrent and painful vaso-occlusive…
- Trial name
- COMPARE (NCT01380197)
- Testing
- Morphine, Nubain
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Children's Healthcare of Atlanta
Sickle Cell Disease
Music Therapy in Sickle Cell Pain Mixed Methods Study
The purpose of this research study is to: 1. Investigate the effects of a single 20-minute music therapy intervention with a music therapist on the pain intensity, pain relief, and mood of adult patients with sickle cell disease…
- Trial name
- NCT02270060
- Testing
- Music Therapy, Music Listening
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- University Hospitals Cleveland Medical Center
Beta Thalassemia Major, Beta Thalassemia Intermedia
Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.
Dose finding study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with Beta (β)-Thalassemia
- Trial name
- NCT01571635
- Testing
- SOTATERCEPT (ACE-011)
- Where
- 18 sites in France, Greece and 2 more
- Volunteers
- 46 people
- Sponsor
- Celgene
Pulmonary Hypertension
Anticoagulation For Pulmonary Hypertension in Sickle Cell Disease
Sickle cell disease (SCD) is often referred to as a hypercoagulable state. However, the contribution of coagulation activation to the pathogenesis of SCD remains uncertain.
- Trial name
- NCT01036802
- Testing
- Warfarin
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of North Carolina, Chapel Hill
Hepatitis C Virus Infection
Ledipasvir/Sofosbuvir Fixed-Dose Combination for 12 or 24 Weeks in Genotype 1 or 4 HCV Infected Adults With Sickle Cell Disease
The primary objectives of this study are to evaluate the antiviral efficacy, safety, and tolerability of ledipasvir/sofosbuvir (LDV/SOF) fixed-dose combination (FDC) administered for 12 or 24 weeks in adults with genotype 1 or 4…
- Trial name
- NCT02301936
- Testing
- LDV/SOF
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Gilead Sciences
Sickle Cell Disease, Renal Function Disorder
Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH)
The use of hydroxyurea in sickle cell disease patients with glomerular hyperfiltration and renal failure requires a specific monitoring and dose adjustment in order to remain within the therapeutic interval while limiting the…
- Trial name
- NCT02522104
- Testing
- Siklos
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Theravia