A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)

Launched by AGIOS PHARMACEUTICALS, INC. · Apr 10, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a medication called tebapivat to see how well it works in treating anemia in people with Sickle Cell Disease (SCD). Anemia is a condition where you don’t have enough healthy red blood cells to carry oxygen in your body, which can make you feel weak and tired. The trial aims to find out the right dose of tebapivat and to compare its effects with a placebo, which is a treatment that doesn’t have any active medication in it.

To be eligible for this trial, participants need to have a confirmed diagnosis of Sickle Cell Disease and a specific range of hemoglobin levels (5.5 to 10.5 grams per deciliter). They should not have received regular blood transfusions in the past, and certain other medications must be stable or stopped before joining. Participants can expect to be monitored closely throughout the study to assess their health and response to the treatment. This trial is important because it could help improve treatment options for people living with Sickle Cell Disease.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• • 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.
• Key Exclusion Criteria:
• • 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.