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

Purpose

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.

Condition

Sickle Cell Disease

Eligibility

Eligible Ages: Over 16 Years
Eligible Sex: All
Accepts Healthy Volunteers: No

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.

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.

Study Design

Phase: Phase 2
Study Type: Interventional
Allocation: Randomized
Intervention Model: Parallel Assignment
Primary Purpose: Treatment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

Arm	Description	Assigned Intervention
Experimental Tebapivat 2.5 milligrams (mg)	Participants will receive 2.5 mg tebapivat orally, once daily (QD) for 12-weeks in the double-blind (DB) period. Participants who complete the DB Period will be eligible to receive the same dose in the Open-Label Extension (OLE) period for up to 52 weeks.	Drug: Tebapivat Oral tablets. Other names: AG-946
Experimental Tebapivat 5.0 mg	Participants will receive 5.0 mg tebapivat orally, QD, for 12-weeks in the DB period. Participants who complete the DB Period will be eligible to receive the same dose in the OLE period for up to 52 weeks.	Drug: Tebapivat Oral tablets. Other names: AG-946
Experimental Tebapivat 7.5 mg	Participants will receive 7.5 mg tebapivat orally, QD, for 12-weeks in the DB period. Participants who complete the DB Period will be eligible to receive the same dose in the OLE period for up to 52 weeks.	Drug: Tebapivat Oral tablets. Other names: AG-946
Placebo Comparator Tebapivat Matched Placebo	Participants will receive a matched placebo, orally, QD, for 12-weeks in the DB period. Participants who complete the DB Period will be randomized in 1:1:1 to receive tebapivat 2.5 mg QD, tebapivat 5.0 mg QD, or tebapivat 7.5 mg QD in the OLE period for up to 52 weeks	Drug: Tebapivat Oral tablets. Other names: AG-946 Drug: Tebapivat Matched Placebo Oral tablets.

Recruiting Locations

UCHealth at University of Colorado Anschutz Medical Campus
Aurora 5412347, Colorado 5417618 80045

UConn Health
Farmington 4834272, Connecticut 4831725 06030-0001

MedStar Washington Hospital Center
Washington D.C. 4140963, District of Columbia 4138106 20010

Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago 4887398, Illinois 4896861 60611

Boston Medical Center & Boston University School of Medicine
Boston 4930956, Massachusetts 6254926 02118

Children's Hospital of Michigan
Detroit 4990729, Michigan 5001836 48304

Icahn School of Medicine at Mt. Sinai
New York 5128581, New York 5128638 10029

Montefiore Medical Center
The Bronx 5110266, New York 5128638 10460

University of Pittsburgh Medical Center
Pittsburgh 5206379, Pennsylvania 6254927 15232

Lifespan at Rhode Island Hospital
Providence 5224151, Rhode Island 5224323 02903-4923

Prisma Health Cancer Institute - Farris Road
Greenville 4580543, South Carolina 4597040 29605

University of Texas Health Science Center of Houston
Houston 4699066, Texas 4736286 77030

More Details

NCT ID: NCT06924970
Status: Recruiting
Sponsor: Agios Pharmaceuticals, Inc.

Study Contact

Agios Medical Affairs
833-228-8474
medinfo@agios.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.