Clinical trials: Sickle Cell Disorders
Sickle Cell Disease
Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial
The trial is designed to test intravenous (IV) arginine therapy in children with sickle cell disease (SCD) and vaso-occlusive painful episodes (VOE) to further knowledge on efficacy and safety of this orphan drug.
- Trial name
- STArT (NCT04839354)
- Testing
- L-Arginine Hydrochloride, Normal saline
- Where
- 10 sites in United States
- Volunteers
- 271 people
- Sponsor
- Claudia R. Morris
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)
Sickle Cell Disease
Inhaled Nitric Oxide for Pediatric Painful Sickle Crisis
Randomized, double blind placebo controlled clinical trial to evaluate effectiveness and safety of inhaled nitric oxide for the treatment of sickle cell painful crisis in pediatric patients with sickle cell disease.
- Trial name
- NCT00142051
- Testing
- nitric oxide, oxygen
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Boston Children's Hospital
Sickle Cell Anemia
Hydroxyurea for the Treatment of Patients With Sickle Cell Anemia
A total of fifty severely affected patients with homozygous sickle cell disease or other sickling disorders (e.g. B negative or B positive Thalassemia/Sickle) who are greater than 18 years of age will be eligible for treatment.
- Trial name
- NCT00001197
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Laboratory-based Hypnosis Intervention on Pain Responsivity in Adolescents With Sickle Cell Disease
The purpose of this study is to test the effects of a laboratory-based hypnosis session compared to an attention control condition on peripheral blood flow, autonomic stress responses, and acute pain responses in adolescents…
- Trial name
- NCT02987725
- Testing
- Hypnosis, Attention Control
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of California, Los Angeles
Sickle Cell Disease, Sickle B+ Thalassemia
The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.
The primary objective of this study is to better understand factors contributing to variations in hydroxyurea (HU) adherence behavior in adolescents and young adults (AYA) with sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
- Trial name
- NCT04691323
- Testing
- HU-Go app
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Sickle Cell Disease
Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease
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…
- Trial name
- R34 pK/PD (NCT02447874)
- Testing
- Arginine, Arginine (Loading), Arginine (Continuous)
- Where
- 2 sites in United States
- Volunteers
- 21 people
- Sponsor
- Emory University
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
Sickle Cell Disease
Advancing Feasibility and Acceptability of Digital Cognitive Rehabilitation in Sickle Cell Disease
This is a single site nonrandomized pilot clinical trial of the feasibility, acceptability, and efficacy of a combination treatment involving digital Cogmed working memory skills training, social support components, and memory…
- Trial name
- ALL IN (NCT06770010)
- Testing
- Cogmed+
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Children's National Research Institute
Sickle Cell Disease
Collect of Cord Blood From Subjects at Risk for Sickle Cell Disease, for the Purpose of Laboratory Research
The study consists in collecting umbilical cord blood cells from newborns at risk of sickle cell disease, to perform laboratory experiments aiming to characterize the cells with HbS/HbS mutation, to develop methods to prepare, to…
- Trial name
- DREPACORD (NCT03876821)
- Where
- France
- Volunteers
- 44 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Sickle Cell Disease
Angiotensin-converting Enzyme Inhibitors and Early Sickle Cell Renal Disease in Children
Patients with sickle cell anaemia may develop renal disease. In fact, renal disease occurred in 40% of adults patients (macroalbuminuria) with evolution to end-stage renal disease for half of them.
- Trial name
- MADREPIEC (NCT01096121)
- Testing
- Enalapril
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Dose-Finding Study of SC411 in Children With Sickle Cell Disease
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD).
- Trial name
- SCOT (NCT02973360)
- Testing
- Docosahexaenoic Acid
- Where
- 11 sites in United States
- Volunteers
- 68 people
- Sponsor
- Micelle BioPharma Inc
Chronic Pain, Sickle Cell Disease
Focused Ultrasound Neuromodulation for Chronic Pain
This study will evaluate the feasibility, tolerability, and preliminary analgesic effects of individualized Precision Confidence Mapping-guided low-intensity focused ultrasound neuromodulation in adults with chronic pain.
- Conditions
- Chronic Pain, Sickle Cell Disease, Chronic Pelvic Pain Syndrome
- Trial name
- NCT07910396
- Testing
- Focused PCM-Guided CMN LIFU, Unfocused Control LIFU
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- University of Minnesota
Sickle Cell Disease (SCD)
Pharmacokinetics and Pharmacodynamics Study of SEG101 (Crizanlizumab) in Sickle Cell Disease (SCD) Patients With Vaso- Occlusive Crisis (VOC)
The purpose of the CSEG101A2202 study was to characterize the Pharmacokinetic (PK) and Pharmacodynamic (PD) of SEG101/crizanlizumab and to evaluate the safety and efficacy of SEG101/crizanlizumab in sickle cell disease (SCD)…
- Trial name
- NCT03264989
- Testing
- crizanlizumab
- Where
- 12 sites in United States
- Volunteers
- 57 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Leg Ulcer
Test of the Safety, Effectiveness, & Acceptability of An Improvised Dressing for Sickle Cell Leg Ulcers in the Tropics
One in 300 Jamaicans have HbSS sickle cell disease, and of these, up to 70% will suffer from sickle cell leg ulcers (SCLUs). Of these, 24% will have a chronic SCLU (one lasting longer than 6 months).
- Trial name
- SCLUJamaica (NCT04479618)
- Testing
- Usual Practice, Improvised dressing, Advanced dressing
- Where
- Jamaica
- Volunteers
- 48 people
- Sponsor
- Benskin, Linda, PhD, RN, SRN (Ghana), CWCN, CWS, DAPWCA
Sickle Cell Disease
ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study
As safety information pertaining to the long-term use of HU remains incomplete in spite of the first safety study (ESCORT-HU), an extension of the latter is proposed.
- Trial name
- NCT04707235
- Testing
- Hydroxycarbamide
- Where
- 87 sites in France, French Guiana and 6 more
- Volunteers
- 2,093 people
- Sponsor
- Theravia
Hemoglobinopathies, Sickle Cell Disease
Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies
The purpose of this study is to determine if treatment with reduced-dose busulfex, fludarabine and alemtuzumab (CAMPATH) followed by sten cell infusion will allow for donor stem cells to grow in patients with hemoglobinopathies…
- Conditions
- Hemoglobinopathies, Sickle Cell Disease, Thalassemia
- Trial name
- NCT00153985
- Testing
- Busulfex, Fludarabine, Alemtuzumab
- Where
- 6 sites in United States
- Volunteers
- 2 people
- Sponsor
- Dana-Farber Cancer Institute
Sickle Cell Disease
A Sickle CEll Disease ComplicatioN Trial
The objective of this study is to assess the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects receiving SC411 compared to those subjects receiving placebo.
- Trial name
- ASCENT (NCT02604368)
- Testing
- SC411
- Volunteers
- 210 people
- Sponsor
- Micelle BioPharma Inc
Sickle Cell Disease
Hemolysis Related Complications in SCD. A Phase II Study With Voxelotor
Intro: Sickle cell disease is a genetic disorder caused by a mutation of the β hemoglobin called HbS, which causes red blood cell (RBC) abnormalities responsible for hemolysis, mainly intravascular, leading to chronic anemia.
- Trial name
- HEMOPROVE (NCT05199766)
- Testing
- Voxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell disease
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease, Pregnancy Related
Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease
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…
- Trial name
- ProTIP (NCT06979492)
- Testing
- Prophylactic Transfusion Intervention group: Transfusion, Control group
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Emory University
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