Clinical trials: Anemia, Sickle Cell
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
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.
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
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
Sickle Cell Disease
Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects
The purpose of this study to evaluate the effect of concomitant administration of GBT440 on caffeine (a CYP1A2 probe substrate), S warfarin (a CYP2C9 probe substrate), omeprazole (a CYP2C19 probe substrate), and midazolam (a…
- Trial name
- NCT02567682
- Testing
- GBT440
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Global Blood Therapeutics
Sickle Cell Disease, Sickle Cell Anemia
A Low-Interventional Study of an Electronic Sickle Cell Disease Patient Reported Outcomes in Sickle Cell Participants
The purpose of this study is to learn more about painful crisis in people with Sickle Cell Disease. For this reason, Pfizer is conducting a study to understand how people with Sickle Cell Disease feel on a daily basis, treat…
- Trial name
- NCT06503458
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Sanguine Biosciences
Vaso-occlusive Crisis, Sickle Cell Disease
Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)
The purpose of this study is to evaluate whether MST-188 can reduce the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease.
- Trial name
- EPIC (NCT01737814)
- Testing
- Saline, MST-188
- Where
- 72 sites in United States, Belgium and 9 more
- Volunteers
- 388 people
- Sponsor
- Mast Therapeutics, Inc.
Sickle Cell Disease
A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease
This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell…
- Trial name
- NCT03653247
- Testing
- Plerixafor, Busulfan, BIVV003
- Where
- 5 sites in United States
- Volunteers
- 7 people
- Sponsor
- Sangamo Therapeutics
Sickle Cell Disease
Clinical Study of BRL-101 in the Treatment of Sickle Cell Disease
This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem…
- Trial name
- NCT06287099
- Testing
- BRL-101
- Volunteers
- 5 people
- Sponsor
- Bioray Laboratories
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
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, 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
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 Stratified Sickle Event Randomized Trial (ASSERT)
The purpose of this study is to compare the effects of ICA-17043 to placebo with or without hydroxyurea (an oral drug used for treatment of sickle cell disease) in patients with sickle cell disease who have had 2 or more acute…
- Trial name
- ASSERT (NCT00102791)
- Testing
- ICA-17043
- Where
- 49 sites in United States, Brazil and 4 more
- Volunteers
- 297 people
- Sponsor
- Icagen
Transfusional Iron Overload, Beta-Thalassemia
Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload
This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of SSP-004184AQ. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of SSP-004184AQ; and the…
- Trial name
- NCT01363908
- Testing
- SPD602
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 30 people
- Sponsor
- Shire
Sickle Cell Disease
An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia
This is an open-label extension study of IMR-687 in adult patients who completed Imara's blinded Phase 2a study (IMR-SCD-102). The open-label extension study will evaluate long-term safety and tolerability.
- Trial name
- NCT04053803
- Testing
- IMR-687
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 30 people
- Sponsor
- Imara, 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
Iron Overload, Sickle Cell Disease
Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias
This research is being done so that we can look at the safety and efficacy of deferiprone in people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron from the body.
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Trial name
- FIRST (NCT02041299)
- Testing
- Deferiprone, Deferoxamine
- Where
- 33 sites in United States, Brazil and 6 more
- Volunteers
- 230 people
- Sponsor
- ApoPharma
Anemia, Sickle Cell
Safety of ICL670 vs. Deferoxamine in Sickle Cell Disease Patients With Iron Overload Due to Blood Transfusions
The purpose of this study is to determine if the new orally active iron chelator, ICL670, is as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.
- Trial name
- NCT00067080
- Testing
- ICL670, deferoxamine
- Where
- 34 sites in United States
- Volunteers
- 195 people
- Sponsor
- Novartis Pharmaceuticals
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