A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease

Sponsor
Forma Therapeutics, Inc.
Study ID
NCT06198712
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
6 Months - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • Etavopivat — DRUG
    Participants will receive oral tablets of etavopivat once daily.

Study Details

This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat. Eligible participants who enter the study will start a 96-week treatment period. At the end of the 96 weeks, participants will have an end of study visit that occurs 4 weeks later. The participants will receive etavopivat every day throughout the treatment period.

Key Dates

Start date
Jan 12, 2023
Status verified
Apr 2026
Primary completion
Feb 23, 2028
Completion
Aug 8, 2029

Study Design

Enrollment
95 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Etavopivat
    Participants will receive Etavopivat once daily (QD) orally.

Primary Outcome Measure

Single-dose: maximum plasma concentration (Cmax) [ Time Frame: During the 24-week primary treatment period ]

Central Contacts

Related Studies