Clinical trials: Sickle Cell
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.
Sickle Cell Disease
Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease
This is a phase 1, first-in-human, multi-center, open-label, single dose cohort study to evaluate the safety and tolerability, pharmacokinetics (PK), exploratory pharmacodynamics (PD), and biomarkers of target engagement of…
- Trial name
- NCT04285827
- Testing
- CSL889
- Where
- 15 sites in United States, Netherlands and 1 more
- Volunteers
- 28 people
- Sponsor
- CSL Behring
Healthy Volunteers Sickle Cell Disease, Thalassemia
A Research Study of the Effect of Etavopivat on Other Drugs in Healthy Participants
The study aims to test if a new medicine called etavopivat potentially affects other medicines in healthy participants. The purpose of the study is to investigate whether the use of etavopivat affects the breakdown and metabolism…
- Trial name
- NCT06813924
- Testing
- Etavopivat, Digoxin, Pitavastatin
- Where
- United States
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Sickle Cell Disease
A Single Dose Study of the Safety, Blood Levels and Biological Effects of Aes-103 Compared to Placebo in Subjects With Stable Sickle Cell Disease
The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of Aes-103 (active ingredient 5-hydroxymethyl-2-furfural [5-HMF]) compared with placebo in subjects with stable sickle…
- Trial name
- NCT01597401
- Testing
- Aes-103
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Baxalta now part of Shire
Healthy Volunteers, Sickle Cell Disease
A SAD/MAD to Assess the Safety, PK/PD of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients
FT-4202 is an oral small-molecule agonist of pyruvate kinase red blood cell isozyme (PKR) being developed for the treatment of hemolytic anemias.
- Trial name
- NCT03815695
- Where
- 18 sites in United States
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- Forma Therapeutics, Inc.
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
Healthy Volunteers, Sickle Cell Disease
A Study on the Effect of Etavopivat on Heart Rhythm in Healthy Participants
Novo Nordisk is developing a new study medicine, Etavopivat, to treat individuals with sickle cell disease (SCD). The purpose of the study is to determine the effect of Etavopivat on the electrical activity of the heart in…
- Conditions
- Healthy Volunteers, Sickle Cell Disease, Thalassemia
- Trial name
- NCT07023029
- Testing
- Etavopivat, Moxifloxacin
- Where
- United States
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Acute Vaso Occlusive Crisis (VOC)
L-citrulline Injection in Patients Aged 6-21 Years Old with Sickle Cell Disease Presenting with Vaso-Occlusive Crisis (VOC)
The purpose of this study is to determine if intravenous L-citrulline can abrogate an active vaso-occlusive crisis in sickle cell disease, resulting in decreased pain, reduction or elimination of opiate usage, and reduction or…
- Trial name
- NCT04852172
- Testing
- L-citrulline, D5 1/2NS
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Asklepion Pharmaceuticals, LLC
Sickle Cell Anemia
Sickle Cell Disease: A Retrospective Chart Review
This study is a retrospective chart review of sickle cell patients and will include patients whom have received blood transfusions and those whom have not.
- Trial name
- NCT01441375
- Where
- 2 sites in United States
- Volunteers
- 261 people
- Sponsor
- Novartis Pharmaceuticals
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.
Sickle Cell Anemia
Phase III Randomized Study of Poloxamer 188 for Vaso-Occlusive Crisis of Sickle Cell Disease
OBJECTIVES: I. Assess the efficacy of poloxamer 188 in reducing the duration of painful vaso-occlusive crisis in patients with sickle cell disease. II.
- Trial name
- NCT00004408
- Testing
- poloxamer 188
- Volunteers
- 300 people
- Sponsor
- Mast Therapeutics, Inc.
Sickle Cell Disease, Hemoglobinopathies
A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).
- Trial name
- NCT04853576
- Testing
- EDIT-301
- Where
- 24 sites in United States, Canada
- Volunteers
- 45 people
- Sponsor
- Editas Medicine, Inc.
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
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD).
- Trial name
- HIBISCUS KIDS (NCT06198712)
- Testing
- Etavopivat
- Where
- 18 sites in Canada, France and 5 more
- Volunteers
- 95 people
- Sponsor
- Forma Therapeutics, Inc.
Sickle Cell Disease, Pharmacokinetics
Pharmacokinetics and Safety of Endari (L-glutamine) in Sickle Cell Disease Patients
L-glutamine has been approved in the US to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients 5 years of age and older.
- Trial name
- NCT04684381
- Testing
- L-glutamine
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Emmaus Medical, Inc.
Sickle Cell Disease
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.
- Trial name
- NCT07656415
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 159 people
- Sponsor
- Agios Pharmaceuticals, 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
Pyruvate Kinase Deficiency, Anemia, Hemolytic
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.
- Conditions
- Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, Thalassemia
- Trial name
- NCT05777993
- Testing
- Mitapivat
- Where
- 23 sites in Belgium, Brazil and 6 more
- Volunteers
- 55 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Disease, Sickle-Beta Zero Thalassemia
Dose-Escalation Study of SCD-101 in Sickle Cell Disease
The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.
- Trial name
- NCT02380079
- Testing
- SCD-101
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Invenux, LLC
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