Clinical trials: Sickle Cell Disorders
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, Sickle-Cell; Hb-SC
A Study of IMR-687 in Healthy Adult Volunteers
The purpose of this Phase 1a, first in human, randomized, double-blind, placebo-controlled study is to evaluate the safety, tolerability, PK and PD profile of the orally administered IMR-687 in healthy adult subjects.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hb-SC, Sickle Beta 0 Thalassemia
- Trial name
- NCT02998450
- Testing
- IMR-687
- Where
- United States
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- Cardurion Pharmaceuticals, Inc.
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 Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease
The purpose of this study is to determine whether ticagrelor is effective in reducing the number of days of pain, intensity of pain, and reducing the use of analgesics due to sickle cell disease
- Trial name
- Hestia2 (NCT02482298)
- Testing
- Ticagrelor
- Where
- 20 sites in United States, Egypt and 6 more
- Volunteers
- 87 people
- Sponsor
- AstraZeneca
Sickle Cell Disease
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies.
- Trial name
- NCT04628585
- Testing
- Safety and efficacy assessments
- Where
- 16 sites in United States, France
- Volunteers
- 85 people
- Sponsor
- Genetix Biotherapeutics Inc.
Priapism
A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism
The goal of the study was to evaluate the efficacy and safety of crizanlizumab in sickle cell disease (SCD) patients with priapism.
- Trial name
- SPARTAN (NCT03938454)
- Testing
- Crizanlizumab
- Where
- 13 sites in United States
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
PK and PD Responses to Oral L-Citrulline in Patients With Sickle Cell Disease
To evaluate PK and PD responses to L-citrulline given orally for four weeks to patients with sickle cell disease who are otherwise healthy.
- Trial name
- NCT00343954
- Testing
- L-citrulline tablets, 1000 mg
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Angiogenix
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
Sickle Cell Disease
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512.
- Trial name
- NCT06546670
- Testing
- ITU512
- Where
- 14 sites in United States, Brazil and 1 more
- Volunteers
- 161 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease
The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) in subjects with sickle cell disease.
- Trial name
- NCT00842088
- Testing
- HQK-1001
- Where
- 9 sites in United States, Jamaica
- Volunteers
- 24 people
- Sponsor
- HemaQuest Pharmaceuticals Inc.
Sickle Cell Disease
A Study of Intravenous L-Citrulline in Patients Aged 6-21 Years Old With Sickle Cell Disease Presenting to Emergency Departments in Acute Vaso-Occlusive Crisis
This study is being done to learn more about a possible new treatment for pain episodes (called vaso-occlusive crises or VOCs) in children, teens, and young adults with sickle cell disease (SCD).
- Trial name
- NCT06989567
- Testing
- L-citrulline
- Where
- 6 sites in United States
- Volunteers
- 120 people
- Sponsor
- Asklepion Pharmaceuticals, LLC
Sickle Cell Disease, Sickle Cell Anemia
Actigraphy Improvement With Voxelotor (ActIVe) Study
This is a study to evaluate the effect of voxelotor on daily physical activity and sleep quality, as measured by a wrist-worn device in participants with sickle cell disease (SCD) and chronic moderate anemia.
- Trial name
- ActIVe (NCT04400487)
- Testing
- Voxelotor
- Where
- 10 sites in United States
- Volunteers
- 25 people
- Sponsor
- Pfizer
Pneumococcal Infections
Immunogenicity and Safety Study of Pneumococcal 7-Valent Conjugate Vaccine in Sickle Cell Disease Infants.
The primary objectives of this study were to assess the immunogenicity and the tolerance of the heptavalent pneumococcal conjugate vaccine (Prevenar) in young infants (2 months of age) with sickle cell disease when administred at…
- Trial name
- NCT00368186
- Testing
- Pneumo 23
- Where
- France
- Volunteers
- 51 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
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
PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD).
- Trial name
- PRECISE (NCT06923111)
- Testing
- Xromi
- Where
- 12 sites in Germany, United Kingdom
- Volunteers
- 180 people
- Sponsor
- Nova Laboratories Limited
Sickle Cell Disease
A Study of IMR-687 in Subjects With Sickle Cell Disease
A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease
- Trial name
- NCT04474314
- Testing
- IMR-687
- Where
- 49 sites in United States, Ghana and 11 more
- Volunteers
- 115 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.
Sickle Cell Disease
A Sickle CEll Disease ComplicatioN Trial
The objective of this study is to assess the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects receiving SC411 compared to those subjects receiving placebo.
- Trial name
- ASCENT (NCT02604368)
- Testing
- SC411
- Volunteers
- 210 people
- Sponsor
- Micelle BioPharma Inc
Sickle Cell Disease Vaso-occlusive Crisis
Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis
This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with…
- Trial name
- NCT06699849
- Testing
- CSL889
- Where
- 30 sites in United States, Turkey (Türkiye) and 1 more
- Volunteers
- 70 people
- Sponsor
- CSL Behring
Beta-Thalassemia, Thalassemia
A Long-term Follow-up Study in Participants Who Received CTX001
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161…
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases
- Trial name
- NCT04208529
- Testing
- CTX001
- Where
- 20 sites in United States, Belgium and 4 more
- Volunteers
- 160 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Blood and Lymphatic Diseases
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: Long-term efficacy of the…
- Trial name
- NCT05145062
- Testing
- BIVV003, ST-400
- Where
- 7 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sangamo Therapeutics