Clinical trials: Sickle Cell Disorder
Sickle Cell Disease
Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD
Study participants will undergo up to four periods of voxelotor administered orally at progressively higher dose levels from 1500 mg until either a maximum tolerated dose (MTD) or 3000 mg/day dose is reached, whichever occurs…
- Trial name
- NCT04247594
- Testing
- Voxelotor
- Where
- 6 sites in United Kingdom
- Volunteers
- 6 people
- Sponsor
- Pfizer
Sickle Cell Disease
A Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab as Adjunct Treatment in Prevention of Vaso-Occlusive Episodes (VOE) in Sickle Cell Disease (SCD)
This study is designed to evaluate the efficacy, safety and pharmacokinetics of crovalimab compared with placebo as adjunct therapy in the prevention of VOEs in participants with SCD.
- Trial name
- CROSSWALK-c (NCT05075824)
- Testing
- Crovalimab
- Where
- 27 sites in United States, Brazil and 9 more
- Volunteers
- 95 people
- Sponsor
- Hoffmann-La Roche
Sickle Cell Disease
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
- Trial name
- GBT_HOPE (NCT03036813)
- Testing
- voxelotor
- Where
- 62 sites in United States, Canada and 10 more
- Volunteers
- 449 people
- Sponsor
- Pfizer
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
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
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
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
Research Study Investigating How Well NDec Works in People With Sickle Cell Disease
This study examines how well a new, potential medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine).
- Trial name
- ASCENT1 (NCT05405114)
- Testing
- NDec - oral decitabine-tetrahydrouridine, HU - Hydroxyurea
- Where
- 118 sites in United States, Canada and 12 more
- Volunteers
- 96 people
- Sponsor
- Novo Nordisk A/S
Sickle Cell Disease
Evaluation of Repeat Administration of Purified Poloxamer 188
The purpose of this study is to evaluate the safety of repeat administration of MST-188 during vaso-occlusive crisis of sickle cell disease.
- Trial name
- EPIC-E (NCT02449616)
- Testing
- MST-188
- Where
- 9 sites in United States
- Volunteers
- 16 people
- Sponsor
- Mast Therapeutics, Inc.
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, 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 in Pediatrics With Sickle Cell Disease
This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years.
- Trial name
- HOPE-KIDS (NCT02850406)
- Testing
- Voxelotor
- Where
- 20 sites in United States, Lebanon and 1 more
- Volunteers
- 147 people
- Sponsor
- Pfizer
Anemia, Sickle Cell
Nitric Oxide Inhalation to Treat Sickle Cell Pain Crises
This study will examine whether nitric oxide (NO) gas can reduce the time it takes for pain to go away in patients who are in sickle cell crisis. NO is important in regulating blood vessel dilation, and consequently, blood flow.
- Trial name
- NCT00094887
- Testing
- Nitric Oxide
- Where
- 11 sites in United States
- Volunteers
- 150 people
- Sponsor
- Mallinckrodt
Sickle Cell Disease
Characteristics of Patients With Sickle Cell Disease
This was a retrospective descriptive analysis of health care claims data using the IQVIA open source medical and pharmacy claims databases.
- Trial name
- NCT05494541
- Testing
- Crizanlizumab
- Where
- United States
- Volunteers
- 540 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).
- Trial name
- NCT04055818
- Testing
- Nicotinamide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- EpiDestiny, Inc.
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.
Sickle Cell Disease
Voxelotor CYP and Transporter Cocktail Interaction Study
This research study is examining multiple doses of voxelotor (a study drug intended for treatment of sickle cell disease) and how it interacts with additional substrates (substrates are drugs or other substances that are…
- Trial name
- NCT05981365
- Testing
- Voxelotor, Bupropion, Repaglinide
- Where
- United States
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Pfizer
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
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
Patient Characteristics and Treatment Patterns From Early Crizanlizumab Use in Real-world Setting: Preliminary Analysis From Select Sickle Cell Centers
This was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with at least five patients who initiated crizanlizumab.
- Trial name
- NCT05833022
- Where
- 11 sites in United States
- Volunteers
- 297 people
- Sponsor
- Novartis Pharmaceuticals