RecruitingPhase 2NCT06924970
A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)
A Phase 2, Double-blind, Randomized, Placebo-Controlled, Multicenter, Dose- Finding, Efficacy, and Safety Study of Tebapivat in Participants With Sickle Cell Disease
Sponsor
Agios Pharmaceuticals, Inc.
Enrollment
56 participants
Start Date
May 1, 2025
Study Type
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Min Age: 16 Years
Inclusion Criteria3
- Documented diagnosis of SCD (HbSS, HbSC \[combined heterozygosity for hemoglobins S and C\], sickle hemoglobin \[HbS\]/β0-thalassemia, HbS/β+-thalassemia, or other sickle cell syndrome variants).
- Hemoglobin ≥5.5 and ≤10.5 grams per decilitre (g/dL). Hemoglobin concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the screening period.
- If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before randomization. Discontinuation of hydroxyurea requires a 90-day washout before providing informed consent.
Exclusion Criteria8
- Receiving regularly scheduled red blood cell (RBC) transfusion therapy (also termed chronic, prophylactic, or preventative transfusion); episodic transfusion in response to worsened anemia or vaso-occlusive crisis (VOC) is permitted. Additionally, a participant who requires episodic transfusion(s) may not have received a transfusion(s) within 60 days before providing informed consent or during the screening period.
- \>10 sickle cell pain crisis (SCPCs) in the 12 months before providing informed consent.
- Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed; the testosterone dose and preparation must be stable for ≥10 weeks before randomization.
- Hospitalized for an SCPC and/or other vaso-occlusive event within 14 days before providing informed consent or within 14 days before randomization. If an SCPC occurs during the screening period, the screening period may be extended with Medical Monitor approval.
- Receiving treatment with voxelotor, crizanlizumab, or L-glutamine within 90 days before randomization.
- Platelet count \<lower limit of normal (LLN) for the local laboratory or \<150×109/liter (L) (whichever is lower) during screening. Platelet transfusions received within 28 days before consent or during screening.
- Receiving treatment with hematopoietic stimulating agents within 90 days before randomization.
- Prior exposure to gene therapy or prior bone marrow or stem cell transplantation, including any conditioning regimen.
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
DRUGTebapivat
Oral tablets.
DRUGTebapivat Matched Placebo
Oral tablets.
Locations(32)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06924970
Related Trials
The Efficacy and Safety of Rilzabrutinib in Patients Aged 10 to 65 Years With Sickle-cell Disease
NCT0697586553 locations
Sickle Cell, Pain and Mediterranean Diet
NCT068864771 location
Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
NCT039378171 location
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
NCT071879733 locations
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)
NCT0643908230 locations