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
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Conditions

Eligibility Criteria

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

Interventions

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

Study Details

The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD). Participants will receive etavopivat and will be enrolled in a staggered manner, starting with the oldest age group and followed sequentially by younger cohorts after review of pharmacokinetic and safety data from the preceding cohort. All participants will undergo a 24-week primary treatment period followed by a 72-week extension treatment period to further evaluate long-term safety and pharmacokinetics of etavopivat. The total duration of the study will be approximately 96 weeks.

Key Dates

First listed
Jan 10, 2024
Start date
Jan 12, 2023
Status verified
Aug 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: Cohort 1: Etavopivat (12 to less than [<] 18 years)
    Participants aged 12 to less than 18 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in this age group.
  • Experimental: Cohort 2: Etavopivat (6 to <12 years)
    Participants aged 6 to less than 12 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in this age group.
  • Experimental: Cohort 3: Etavopivat (2 to <6 years)
    Participants aged 2 to less than 6 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in younger children.
  • Experimental: Cohort 4: Etavopivat (6 months to <2 years)
    Participants aged 6 months to less than 2 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in infants.

Primary Outcome Measure

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

Central Contacts

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