Clinical trials: Sickle Cells Disease
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.
Anemia, Sickle Cell
Study of SANGUINATE™ In the Treatment of Sickle Cell Disease Patients With Vaso-Occlusive Crisis
Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis
- Trial name
- NCT02411708
- Testing
- SANGUINATE
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Prolong Pharmaceuticals
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with Hemoglobinopathies to halt disease progression.
- Trial name
- NCT00777231
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Talaris Therapeutics Inc.
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
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, Vaso-occlusive Crisis
A Study of Varespladib Infusion in Subjects With Sickle Cell Disease.
The purpose of this study is to determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).
- Trial name
- IMPACTS-2 (NCT01522196)
- Testing
- Varespladib
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Anthera Pharmaceuticals
Sickle Cell Disease
Dose-Finding Study of SC411 in Children With Sickle Cell Disease
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD).
- Trial name
- SCOT (NCT02973360)
- Testing
- Docosahexaenoic Acid
- Where
- 11 sites in United States
- Volunteers
- 68 people
- Sponsor
- Micelle BioPharma Inc
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
Sickle Cell Disease
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to…
- Trial name
- NCT05031780
- Testing
- Mitapivat
- Where
- 91 sites in United States, Belgium and 14 more
- Volunteers
- 286 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Healthy Subjects, Sickle Cell Disease
A Study of the Safety, Blood Levels and Biological Effects of GBT440 in Healthy Subjects and Subjects With Sickle Cell Disease
The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of GBT440 compared with placebo in healthy subjects and subjects with sickle cell disease (SCD).
- Trial name
- NCT02285088
- Testing
- GBT440
- Where
- United Kingdom
- Volunteers
- 133 people, healthy volunteers welcome
- Sponsor
- Global Blood Therapeutics
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
Pneumococcal Conjugate Vaccine
Study Evaluating 13-valent Pneumococcal Conjugate Vaccine (13vPnC) in Children With Sickle Cell Disease
This study is to evaluate the safety, tolerability and immunogenicity of 13-valent pneumococcal Conjugate Vaccine in children with Sickle Cell Disease who have already been vaccinated with 23-valent polysaccharide vaccine.
- Trial name
- NCT00918580
- Testing
- 13-valent Pneumoccocal Conjugate Vaccine
- Where
- 19 sites in United States, Egypt and 5 more
- Volunteers
- 158 people
- Sponsor
- Pfizer
Sickle Cell Disease
A Study to Compare if the Uptake of Ticagrelor in the Body Differs When Different Tablets Are Administered
To evaluate the relative bioavailability of ticagrelor for the different formulations. A randomized cross-over design has been chosen to minimize the effects of between-subject variability and any period effects on the overall…
- Trial name
- NCT03126695
- Testing
- Ticagrelor granule, Ticagrelor pediatric tablets, Ticagrelor pediatric tablets suspended in water
- Where
- Germany
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Anemia, Sickle Cell
CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease
This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor…
- Trial name
- NCT04091737
- Testing
- Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- CSL Behring
Sickle Cell Disease
TRF-1101 Assessment in Sickle Cell Disease
This study is designed to assess the safety, tolerability, and activity of TRF-1101 on microvascular blood flow, vascular endothelial injury, and vasoocclusive pain associated with sickle cell disease.
- Trial name
- NCT00773890
- Testing
- TRF-1101
- Where
- 6 sites in United States
- Volunteers
- 80 people
- Sponsor
- TRF Pharma, Inc
Sickle Cell Disease, Sickle Cell Anemia
A Study Evaluating the Long-Term Safety of ICA-17043 in Sickle Cell Disease Patients With or Without Hydroxyurea Therapy
This trial is a follow-up companion study to Protocol ICA-17043-10, a Phase III, multi-center, efficacy and safety study of ICA-17043.
- Trial name
- NCT00294541
- Testing
- ICA-17043
- Where
- 31 sites in United States
- Volunteers
- 51 people
- Sponsor
- Icagen
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
Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease
The purpose of the study is to Evaluate the Effect of Ticagrelor versus Placebo in Reducing the Rate of Vaso-Occlusive Crises in Paediatric Patients with Sickle Cell Disease
- Trial name
- HESTIA3 (NCT03615924)
- Testing
- Ticagrelor
- Where
- 55 sites in United States, Belgium and 14 more
- Volunteers
- 193 people
- Sponsor
- AstraZeneca
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.