Clinical trials: Sickle Cell Anemia
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
Bone Marrow Transplant (BMT)
HPC Offered for PRESERVE Expansion
Ossium recognizes that not all patients who could benefit from Ossium's HPC, Marrow can access the PRESERVE trial for logistical or eligibility reasons.
- Trial name
- HOPE (NCT07145411)
- Testing
- Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors)
- Where
- 4 sites in United States
- Volunteers
- 10 people
- Sponsor
- Ossium Health, Inc.
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
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Healthy Volunteers Sickle Cell Disease, Thalassemia
A Research Study of the Effect of Food on Etavopivat in Healthy Participants
The purpose of this study is to evaluate the effect of food on the amount of etavopivat in the bloodstream of healthy participants. Participants will take a single oral dose of etavopivat following a high-fat meal (i.e.
- Trial name
- NCT06433661
- Testing
- Etavopivat
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Sickle Cell Disease
Codeine in Sickle Cell Disease
The objective of this study is to determine if a subject's genetic make-up would affect the treatment response to codeine in subjects with sickle cell disease.
- Trial name
- NCT00174538
- Testing
- Codeine (30 mg)
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- PriCara, Unit of Ortho-McNeil, Inc.
Sickle Cell Disease
Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease
This clinical trial is a study to evaluate the pharmacokinetics of the tablet formulation Pociredir in fasted and fed state participants with Sickle Cell Disease (SCD).
- Trial name
- NCT07431398
- Testing
- Pociredir
- Where
- 6 sites in United States
- Volunteers
- 13 people
- Sponsor
- Fulcrum Therapeutics
Renal Impairment
Study of GBT021601 in Participants With Renal Impairment
Renal Impairment study of GBT021601.
- Trial name
- NCT05878704
- Testing
- GBT021601
- Where
- 4 sites in United States
- Volunteers
- 8 people
- Sponsor
- Pfizer
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
Plasmodium Falciparum Malaria, Malaria
Effect of Plasmodium Falciparum Exposure and Sickle Cell Trait on Infection Rates and Kinetics After IV Administration of PfSPZ Challenge
The study is designed to establish infectivity of Plasmodium falciparum sporozoites (PfSPZ) via intravenous (IV) administration in three groups with different malaria immunity-status: 1.
- Trial name
- NCT02237586
- Testing
- PfSPZ Challenge
- Where
- Gabon
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Sanaria Inc.
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
The primary objective of this multicenter observational registry is to characterize the clinical, epidemiological, and biological profiles of individuals of Middle Eastern, North African, and Levantine ancestry, including…
- Conditions
- Antiphospholipid (aPL)-Positive, Rheumatic Arthritis, Celiac Disease, Focal Segmental Glomerulosclerosis (FSGS)
- Trial name
- MERIT (NCT07892651)
- Where
- Jordan
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Axiom Bioregistry
Sickle Cell Disease
An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001
This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).
- Trial name
- NCT03041909
- Testing
- GBT440
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- Global Blood Therapeutics
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
Anemia, Sickle Cell, Sickle Cell Anemia
Safety Study of MP4CO in Adult Sickle Cell Patients
Sickle Cell Anemia is caused by an inherited hemoglobin disorder. Healthy red blood cells are discoid and can deform and move through small blood vessels to carry oxygen to all parts of the body.
- Conditions
- Anemia, Sickle Cell, Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell Disorders
- Trial name
- NCT01356485
- Testing
- MP4CO, Sodium chloride solution
- Where
- 5 sites in France, Jamaica and 2 more
- Volunteers
- 32 people
- Sponsor
- Sangart
Sickle Cell Disease
Safety and Efficacy of Orally Administered NUV001 Nutraceutical Supplement in Sickle Cell Disease Patients
This multicenter, randomized, double-blind, parallel-group, placebo-controlled pilot study evaluated the safety, tolerability, and exploratory efficacy of orally administered NUV001 in adult participants with sickle cell disease…
- Trial name
- NCT05791591
- Testing
- NUV001 - IR, NUV001 - GR
- Where
- 9 sites in India
- Volunteers
- 168 people
- Sponsor
- LGD
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease
The purpose of this study is to determine the correct prasugrel dosage to be given to children with sickle cell disease (SCD).
- Trial name
- Crescent (NCT01476696)
- Testing
- Prasugrel
- Where
- 9 sites in United States
- Volunteers
- 33 people
- Sponsor
- Eli Lilly and Company
Cancer, Breakthrough Pain
Efficacy/Safety Study of ACTIQ® for Opioid-Tolerant Children and Adolescents With Breakthrough Pain (BTP)
The primary objective of the study is to evaluate the efficacy of ACTIQ treatment for the management of breakthrough pain (BTP) compared to placebo treatment in children with cancer and non-cancer pain who are receiving…
- Trial name
- NCT00236041
- Testing
- ACTIQ®
- Where
- 31 sites in United States, Puerto Rico
- Volunteers
- 38 people
- Sponsor
- Cephalon
Sickle Cell Disease
A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)
The main purpose of this study is to compare the effect of tebapivat versus placebo on anemia and to detect a dose-response for hemoglobin (Hb) response in participants with SCD.
- Trial name
- NCT06924970
- Testing
- Tebapivat
- Where
- 20 sites in United States, Belgium and 5 more
- Volunteers
- 59 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Disease
Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing Vaso-Occlusive Crises Rate in Pediatric Patients With Sickle Cell Disease.
The purpose of this study is to compare the effect of ticagrelor vs placebo for the reduction of Vaso-Occlusive crises in paediatric patients with Sickle Cell Disease
- Trial name
- HESTIA5 (NCT04293172)
- Testing
- Brilinta
- Sponsor
- AstraZeneca