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
Sex
ALL
Min age
16 Years
Max age
—
Healthy volunteers
No
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.
Primary outcome measure(s)
Percentage of Participants With Hb Response — Baseline, Week 10 through Week 12
Trial sites (20)
Facility
City
Region
Status
UConn Health
Farmington
Connecticut
MedStar Washington Hospital Center
Washington D.C.
District of Columbia
Emory-Children's Center/ Children's Healthcare of Atlanta: Arthur M. Blank Hospital
Atlanta
Georgia
Henry Ford Health System
Detroit
Michigan
Children's Hospital of Michigan
Detroit
Michigan
Icahn School of Medicine at Mt. Sinai
New York
New York
University of Pittsburgh Medical Center
Pittsburgh
Pennsylvania
Prisma Health Cancer Institute - Farris Road
Greenville
South Carolina
University of Texas Health Science Center of Houston
Houston
Texas
Fred Hutchinson Cancer Center, University of Washington
Seattle
Washington
CHR de la Citadelle
Liège
Wallonne
CHU Montreal
Montreal
Quebec
Hôpital Edouard Herriot, CHU de Lyon
Lyon
Auvergne-Rhône-Alpes
Institut Universitaire du Cancer de Toulouse - Oncopole
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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