Sickle Cell Disease Clinical Trial
Official title:
A Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of Etavopivat in Patients With Thalassemia or Sickle Cell Disease
This clinical trial is a Phase 2 study that will evaluate the safety and clinical activity of etavopivat in patients with thalassemia or sickle cell disease and test how well etavopivat works to lower the number of red blood cell transfusions required and increase hemoglobin.
Etavopivat is a potent, selective, orally bioavailable, small-molecule activator of pyruvate kinase red blood cell (PKR) being developed by Forma Therapeutics, Inc and is intended for use as a treatment for patients with sickle cell disease (SCD) or other inherited hemoglobinopathies or refractory anemias. This study is a multicenter, Phase 2, open-label, multiple-cohort study examining the safety and efficacy of etavopivat for the treatment of patients, age 12 to 65 years, with SCD or thalassemia. Three treatment cohorts based on the patients hemoglobinopathy (SCD or thalassemia) and transfusion requirements will be evaluated. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT02227472 -
Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease
|
||
| Recruiting |
NCT06301893 -
Uganda Sickle Surveillance Study (US-3)
|
||
| Recruiting |
NCT04398628 -
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
|
||
| Completed |
NCT02522104 -
Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH)
|
Phase 4 | |
| Recruiting |
NCT04688411 -
An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease
|
N/A | |
| Terminated |
NCT03615924 -
Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease
|
Phase 3 | |
| Not yet recruiting |
NCT06300723 -
Clinical Study of BRL-101 in Severe SCD
|
N/A | |
| Recruiting |
NCT03937817 -
Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
|
||
| Completed |
NCT04917783 -
Health Literacy - Neurocognitive Screening in Pediatric SCD
|
N/A | |
| Completed |
NCT04134299 -
To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease
|
N/A | |
| Completed |
NCT02580565 -
Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease
|
||
| Recruiting |
NCT04754711 -
Interest of Nutritional Care of Children With Sickle Cell Disease on Bone Mineral Density and Body Composition
|
N/A | |
| Completed |
NCT04388241 -
Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD
|
N/A | |
| Recruiting |
NCT05431088 -
A Phase 2/3 Study in Adult and Pediatric Participants With SCD
|
Phase 2/Phase 3 | |
| Completed |
NCT01158794 -
Genes Influencing Iron Overload State
|
||
| Recruiting |
NCT03027258 -
Point-of-Delivery Prenatal Test Results Through mHealth to Improve Birth Outcome
|
N/A | |
| Withdrawn |
NCT02960503 -
Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease
|
Phase 1/Phase 2 | |
| Not yet recruiting |
NCT02525107 -
Prevention of Vaso-occlusive Painful Crisis by Using Omega-3 Fatty Acid Supplements
|
Phase 3 | |
| Completed |
NCT02567682 -
Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects
|
Phase 1 | |
| Completed |
NCT02565082 -
Evaluation of the Hemostatic Potential in Sickle Cell Disease Patients
|
N/A |