Clinical trials: Sickle Cell Disorders
Pulmonary Hypertension
Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients With Pulmonary Arterial Hypertension (PAH)
The study will assess the effect of bosentan on pulmonary vascular resistance and exercise capacity in sickle cell disease (SCD) patients diagnosed with pulmonary arterial hypertension.
- Trial name
- NCT00310830
- Testing
- Bosentan
- Volunteers
- 14 people
- Sponsor
- Actelion
Sickle Cell Disease
Open-Label Extension of Voxelotor
Open-label extension study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials.
- Trial name
- NCT04188509
- Testing
- Voxelotor
- Where
- 20 sites in United States, Egypt and 3 more
- Volunteers
- 162 people
- Sponsor
- Pfizer
Healthy Volunteers, Sickle Cell Disease
A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese Participants
The study tests a new medicine called etavopivat in healthy Chinese participants. During the study, the participant will be given 1 dose of the study medicine. A dose is 2 tablets which are swallowed together with a cup of water.
- Conditions
- Healthy Volunteers, Sickle Cell Disease, Thalassemia
- Trial name
- NCT06581627
- Testing
- Etavopivat
- Where
- China
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
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
Sickle Cell Disease
Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype
The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).
- Trial name
- NCT05951205
- Testing
- Exa-cel
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease
Plasma DNA and Vascular Remodelling in Patients With Sickle Cell Disease
The purpose of this study is to evaluate the relationship between plasma DNA levels and micro- and macro-circulatory vascular remodelling in patients with sickle cell disease
- Trial name
- PADRE (NCT02721472)
- Testing
- micro- and macro-circulatory vascular remodelling measures not practice in routine care, Biological measures not practice in routine care
- Where
- France
- Volunteers
- 44 people
- Sponsor
- Theravia
Sickle Cell Disease
Evaluation of Spectra Optia Red Blood Cell Exchange in Sickle Cell Patients
The purpose of this study is to evaluate the performance of the Spectra Optia system red blood cell exchange (RBCx) protocols (exchange and depletion/exchange) in study participants with sickle cell disease.
- Trial name
- ESSENTIAL (NCT01736657)
- Testing
- Red blood cell exchange in sickle cell
- Where
- 5 sites in United States
- Volunteers
- 73 people
- Sponsor
- Terumo BCT
Sickle Cell Disease
Study to Assess Efficacy and Safety of VIT-2763 (Vamifeport) in Subjects With Sickle Cell Disease
The purpose of this study is to investigate the effect of VIT-2763 on markers of hemolysis (breakdown in red blood cells) in sickle cell disease (SCD).
- Trial name
- ViSionSerenity (NCT04817670)
- Testing
- VIT-2763 120 mg, VIT-2763 360 mg, VIT-2763 240 mg
- Where
- 22 sites in United States, France and 3 more
- Volunteers
- 25 people
- Sponsor
- Vifor (International) Inc.
Sickle Cell Disease, Sickle Cell Anemia
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pociredir
This is a study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of Pociredir in participants with sickle cell disease.
- Trial name
- NCT05169580
- Testing
- Pociredir oral capsule(s)
- Where
- 18 sites in United States, Nigeria and 1 more
- Volunteers
- 45 people
- Sponsor
- Fulcrum Therapeutics
Sickle Cell Disease
Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease
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…
- Trial name
- NCT07247188
- Testing
- PCV21, 20vPCV
- Where
- 7 sites in United States, Dominican Republic
- Volunteers
- 100 people
- Sponsor
- Sanofi
Sickle Cell Disease
A GBT021601 ADME Microtracer Study in Healthy Volunteers
An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601.
- Trial name
- NCT05718687
- Testing
- GBT021601
- Where
- Netherlands
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Pfizer
Sickle Cell Disease
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease
- Trial name
- NCT05632354
- Testing
- Osivelotor
- Where
- 9 sites in United States, Nigeria
- Volunteers
- 47 people
- Sponsor
- Pfizer
Anemia, Sickle Cell, Healthy Volunteers
A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease
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…
- Trial name
- NCT06481306
- Testing
- BMS-986470, Famotidine, Pantoprazole
- Where
- 32 sites in United States, Canada and 3 more
- Volunteers
- 224 people, healthy volunteers welcome
- Sponsor
- Bristol-Myers Squibb
Transfusion-dependent Iron Overload
A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload
This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload.
- Trial name
- NCT00171821
- Testing
- Deferasirox
- Where
- 97 sites in Australia, Austria and 21 more
- Volunteers
- 1,784 people
- Sponsor
- Novartis Pharmaceuticals
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, Transfusion-dependent Beta-Thalassemia
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
- Conditions
- Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
- Trial name
- NCT06363760
- Testing
- Safety and efficacy assessments
- Where
- 18 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Editas Medicine, Inc.
Sickle Cell Disease, Nephropathy
A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy
The primary purpose of this study is to evaluate the effect of mitapivat on albumin creatinine ratio (ACR) response in participants with sickle cell disease (SCD) and nephropathy.
- Trial name
- NCT06286046
- Testing
- Mitapivat
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Disease
A Study of SHP655 (rADAMTS13) in Sickle Cell Disease
TAK-755 (previously known as SHP655) is a medicine used to treat sickle cell disease (SCD). The main aim of the study is to measure the safety and tolerability of TAK-755 in SCD participants.
- Trial name
- RAISE (NCT03997760)
- Testing
- TAK-755
- Where
- 14 sites in United States
- Volunteers
- 19 people
- Sponsor
- Shire
Sickle Cell Disease, Vaso-occlusive Crisis
IMPACTS Trial: Investigation of the Modulation of Phospholipase in Acute Chest Syndrome
The study will be conducted at 15-20 US centers in a randomized, placebo-controlled, double-blind fashion. Enrollees will be hospitalized sickle cell disease (SCD) patients at-risk for acute chest syndrome (ACS) based on the…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Acute Chest Syndrome
- Trial name
- NCT00434473
- Testing
- A-001
- Where
- 6 sites in United States
- Volunteers
- 30 people
- Sponsor
- Anthera Pharmaceuticals
Sickle Cell Disease
A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disease (SCD).
The purpose of this study is to evaluate crovalimab for the treatment of a sickle cell pain crisis (also known as a VOE) that requires hospitalisation in adult and adolescent participants with SCD.
- Trial name
- CROSSWALK-a (NCT04912869)
- Testing
- Crovalimab
- Where
- 16 sites in United States, Brazil and 8 more
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche