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RECRUITINGPhase 2INTERVENTIONAL

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Key Who May Qualify: - 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). - blood count (hemoglobin) at least 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 willing to sign a consent form. Key Who Should NOT Join This Trial: - 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 willing to sign a consent form or during the screening period. - \>10 sickle cell pain crisis (SCPCs) in the 12 months before providing willing to sign a consent form. - 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 willing to sign a consent form 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

DRUG

Tebapivat

Oral tablets.

DRUG

Tebapivat Matched Placebo

Oral tablets.

Locations (20)

UCHealth at University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States
UConn Health
Farmington, Connecticut, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
MedStar Washington Hospital Center
Washington D.C., District of Columbia, United States
Emory-Children's Center/ Children's Healthcare of Atlanta: Arthur M. Blank Hospital
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Boston Medical Center & Boston University School of Medicine
Boston, Massachusetts, United States
Henry Ford Health System
Detroit, Michigan, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Icahn School of Medicine at Mt. Sinai
New York, New York, United States
Montefiore Medical Center
The Bronx, New York, United States
Thomas Jefferson University Hospital
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Lifespan at Rhode Island Hospital
Providence, Rhode Island, United States
Prisma Health Cancer Institute - Farris Road
Greenville, South Carolina, United States
University of Texas Health Science Center of Houston
Houston, Texas, United States
Fred Hutchinson Cancer Center, University of Washington
Seattle, Washington, United States
CHR de la Citadelle
Liège, Wallonne, Belgium
Clinique CHC MontLégia
Liège, Wallonne, Belgium
Scarborough Health Network - Centenary Hospital
Scarborough Village, Ontario, Canada