A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period

Conditions

• Pediatric Hemolytic Anemia
• Pediatric Pyruvate Kinase Deficiency

Eligibility Criteria

Sex

ALL

Age

1 Year - 17 Years

Healthy Volunteers

Not accepted

Interventions

• Mitapivat — DRUG
  Tablets or granules
• Mitapivat-matching placebo — DRUG
  Tablets or granules

Study Details

Study ACTIVATE-Kids (AG348-C-023) will evaluate the efficacy and safety of orally administered mitapivat as compared with placebo in pediatric participants with pyruvate kinase deficiency (PKD) who are not regularly receiving blood transfusions. Participants will be randomized 2:1 to receive either mitapivat or matching placebo. Randomization will be stratified by age (1 to \< 6 years, 6 to \< 12 years, 12 to \< 18 years). Participants will be dosed by age and weight during a double-blind period consisting of an 8-week dose titration period followed by a 12-week fixed-dose period. Participants who complete the double-blind period will be eligible to receive mitapivat for up to 5 years in the open-label extension (OLE) period.

Key Dates

Start date

Jun 6, 2022

Status verified

May 2026

Primary completion

Dec 13, 2024

Completion

Jan 31, 2030

Study Design

Enrollment

30 participants (actual)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Mitapivat
  Double-Blind Period: Participants will receive mitapivat orally, at doses based on age and weight, for 8 weeks in the dose titration period and for 12 weeks in the fixed-dose period.
• Placebo Comparator: Placebo
  Double-Blind Period: Participants will receive mitapivat-matching placebo orally for 8 weeks in the dose titration period and for 12 weeks in the fixed-dose period.
• Experimental: Mitapivat (OLE period)
  Participants who have completed the double-blind period will be eligible to receive mitapivat for up to 5 years in the OLE period. Participants entering the OLE period will first receive blinded mitapivat and placebo for 8 weeks to maintain the double-blind treatment assignment before being transitioned to only receive active, open-label drug (mitapivat).

Primary Outcome Measure

Percentage of Participants Achieving a Hemoglobin (Hb) Response [ Time Frame: Baseline up to Week 20 ]

Locations (11)

Related coverage on Hipa.ai

• Mitapivat Phase 3 Trial for Pediatric PKD Completes Primary Study
  Mitapivat · Dec 13, 2024 · ClinicalTrials.gov

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