Clinical trials: Hemoglobinopathy (Disorder)
Sickle Cell Anemia
Prasugrel Versus Placebo in Adult Sickle Cell Disease
The purpose of this trial is to assess the safety of Prasugrel in adult patients with sickle cell disease (SCD) by monitoring the rate and severity of hemorrhagic events requiring medical intervention compared to placebo for 30…
- Trial name
- NCT01167023
- Testing
- Prasugrel
- Where
- 14 sites in United States
- Volunteers
- 62 people
- Sponsor
- Eli Lilly and Company
Sickle Cell Disease, Sickle Cell Anemia
A Study of HQK-1001 in Patients With Sickle Cell Disease
The purpose of this study is to evaluate the safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease
- Trial name
- NCT01322269
- Testing
- HQK-1001
- Where
- 16 sites in United States, Canada and 3 more
- Volunteers
- 52 people
- Sponsor
- HemaQuest Pharmaceuticals Inc.
Beta-Thalassemia
CS-101 in Patients With β-thalassemia
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.
- Trial name
- NCT06328764
- Testing
- CS-101
- Where
- China
- Volunteers
- 10 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Sickle Cell Disease, Thalassemia
A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease
This clinical trial is a Phase 2 study that will evaluate the safety and clinical activity of etavopivat in patients with thalassemia or sickle cell disease and test how well etavopivat works to lower the number of red blood cell…
- Trial name
- GLADIOLUS (NCT04987489)
- Testing
- Etavopivat tablets
- Where
- 32 sites in United States, Canada and 4 more
- Volunteers
- 53 people
- Sponsor
- Forma Therapeutics, Inc.
Sickle Cell Disease, Vaso-occlusive Crisis
A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
- Trial name
- NCT04927247
- Testing
- Inclacumab
- Where
- 59 sites in United States, Brazil and 12 more
- Volunteers
- 72 people
- Sponsor
- Pfizer
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease.
- Trial name
- HOPE Kids 2 (NCT04218084)
- Testing
- Voxelotor
- Where
- 35 sites in United States, Egypt and 7 more
- Volunteers
- 236 people
- Sponsor
- Pfizer
Sickle Cell Disease (SCD)
Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease
The primary objective of this study is to assess the safety and tolerability of ALXN1820 SC (subcutaneous) in participants with SCD (Sickle Cell Disease).
- Trial name
- PHOENIX (NCT05565092)
- Testing
- ALXN1820
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Beta-Thalassemia
A Long-term Follow-up Study in Participants Who Received CS-101
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-06 (NCT06024876),No investigational drug product will be administered in the study.
- Trial name
- NCT06685536
- Testing
- Safety and efficacy assessments
- Where
- China
- Volunteers
- 5 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Vaso-occlusive Crisis, Sickle Cell Disease
Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)
The purpose of this study is to evaluate whether MST-188 can reduce the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease.
- Trial name
- EPIC (NCT01737814)
- Testing
- Saline, MST-188
- Where
- 72 sites in United States, Belgium and 9 more
- Volunteers
- 388 people
- Sponsor
- Mast Therapeutics, Inc.
Beta-Thalassemia, Thalassemia
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT03655678
- Testing
- CTX001
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 59 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease
A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age)…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis
- Trial name
- NCT04935879
- Testing
- Inclacumab
- Where
- 58 sites in United States, Brazil and 10 more
- Volunteers
- 241 people
- Sponsor
- Pfizer
Sickle Cell Disease
PK Study of Ticagrelor in Children Aged Less Than 24 Months, With Sickle Cell Disease (HESTIA4)
The purpose of this Phase I study is to investigate the pharmacokinetic properties of ticagrelor in pediatric patients from 0 to less than 24 months with sickle cell disease.
- Trial name
- HESTIA4 (NCT03492931)
- Testing
- Ticagrelor
- Where
- 8 sites in Belgium, Italy and 4 more
- Volunteers
- 21 people
- Sponsor
- AstraZeneca
Sickle Cell Disease, Vaso-occlusive Crisis
Study of GMI-1070 for the Treatment of Sickle Cell Pain Crisis
GMI-1070 is a new drug that may reduce the stickiness of cells in the blood. The purpose of this study is to evaluate whether GMI-1070 can reduce the time it takes for pain to go away in patients with vaso-occlusive crisis (also…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Pain Crisis
- Trial name
- NCT01119833
- Testing
- GMI-1070
- Where
- 22 sites in United States, Canada
- Volunteers
- 81 people
- Sponsor
- GlycoMimetics Incorporated
Iron Overload, Sickle Cell Disease
Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias
This research is being done so that we can look at the safety and efficacy of deferiprone in people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron from the body.
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Trial name
- FIRST (NCT02041299)
- Testing
- Deferiprone, Deferoxamine
- Where
- 33 sites in United States, Brazil and 6 more
- Volunteers
- 230 people
- Sponsor
- ApoPharma
Sickle Cell Disease
A Relative Bioavailability Study of a Prasugrel Orally Disintegrating Tablet
This study compares the clinical tablet formulation of prasugrel taken orally with an orally disintegrating tablet (ODT) taken orally.
- Trial name
- NCT01430091
- Testing
- Prasugrel (clinical formulation), Prasugrel (Orally Disintegrating Tablet [ODT])
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Eli Lilly and Company
Chronic Cutaneous Ulcer, Sickle Cell Disease
Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment
Approximately 60 subjects will be enrolled into this double-blind, placebo-controlled study for the Deferoxamine Intradermal Delivery Patch (DIDP).
- Trial name
- D-SCOUT (NCT04058197)
- Testing
- Deferoxamine Product
- Where
- 4 sites in United States
- Sponsor
- TauTona Group
Sickle Cell Disease, Umbilical Cord Blood
US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease
The application of experimental hematopoietic cell transplantation (HCT) therapy in sickle-cell disease (SCD) must strike a balance between the underlying disease severity and the possibility of a direct benefit of the treatment…
- Conditions
- Sickle Cell Disease, Umbilical Cord Blood, Hematopoietic Cell Proliferation
- Trial name
- NCT04594031
- Testing
- ECT-001-CB (UM171-Expanded Cord Blood Transplant)
- Where
- 2 sites in United States
- Sponsor
- ExCellThera inc.
Transfusion Dependent Thalassemia
Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE)
This is a prospective, multi-center, randomized, crossover trial to evaluate the clinical effectiveness of red blood cells (RBCs) derived from Mirasol-treated whole blood (WB) versus conventional RBCs in transfusion dependent…
- Trial name
- PRAISE (NCT03329404)
- Testing
- Mirasol Red Blood Cells (MIR RBCs), Reference Red Blood Cells (REF RBCs)
- Where
- 9 sites in United States, Israel and 2 more
- Volunteers
- 9 people
- Sponsor
- Terumo BCTbio
Sickle Cell Disease
Retrospective Real World Oxbryta® Data Collection and Analysis Study
The aim of this study is to collect and analyze retrospective data on Oxbryta in a real-world setting. This is a multicenter, retrospective data collection and analysis study to characterize health outcomes in approximately 300…
- Trial name
- RETRO (NCT04930328)
- Testing
- Oxbryta® (voxelotor) 500-mg Tablets
- Where
- 12 sites in United States
- Volunteers
- 216 people
- Sponsor
- Pfizer
Beta Thalassemia Major Anemia, Iron Overload
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on…
- Trial name
- START (NCT03591575)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in Egypt, Indonesia
- Volunteers
- 64 people
- Sponsor
- Chiesi Canada Corp