Evaluating the effects of tebapivat on anemia in sickle cell disease
A Phase 2, Double-blind, Randomized, Placebo-Controlled, Multicenter, Dose- Finding, Efficacy, and Safety Study of Tebapivat in Participants With Sickle Cell Disease
This study is testing if a new drug called tebapivat can help improve anemia in people with sickle cell disease compared to a placebo.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 56 (estimated) |
| Ages | 16 Years and up |
| Sex | All |
| Sponsor | Agios Pharmaceuticals, Inc. Industry-sponsored |
| Drugs / interventions | crizanlizumab |
| Locations | 1 site (New York, New York) |
| Trial ID | NCT06924970 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the efficacy and safety of tebapivat in participants diagnosed with sickle cell disease (SCD). It will compare the effects of tebapivat against a placebo, focusing on the dose-response relationship for hemoglobin levels. Participants will be monitored for changes in anemia and overall health outcomes related to SCD. The study will include individuals with specific hemoglobin levels and stable hydroxyurea treatment, if applicable.
Who should consider this trial
Good fit: Ideal candidates are individuals with a documented diagnosis of sickle cell disease and hemoglobin levels between 5.5 and 10.5 g/dL.
Not a fit: Patients who are receiving regular red blood cell transfusions or have not stabilized their hydroxyurea treatment may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option that improves hemoglobin levels and reduces anemia in patients with sickle cell disease.
How similar studies have performed: Other studies have explored treatments for sickle cell disease, but the specific approach of using tebapivat is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
New York, New York
- Icahn School of Medicine at Mt. Sinai — New York, New York, United States (Recruiting)
Study contacts
- Study coordinator: Agios Medical Affairs
- Email: medinfo@agios.com
- Phone: 833-228-8474
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.