Phase 3 study to evaluate mitapivat in pediatrics with PKD who are regularly transfused
Research type
Research Study
Full title
A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period
IRAS ID
1004452
Contact name
Christina Baladi
Contact email
Sponsor organisation
Agios Pharmaceuticals, Inc.
Eudract number
2021-003265-36
Clinicaltrials.gov Identifier
Research summary
Mitapivat is being studied as a possible treatment for PK deficiency, a rare genetic blood disease. This study aims to assess if mitapivat helps reduce the number of blood transfusions in paediatric patients with PK deficiency who have regular transfusions. PK Deficiency is a defect in a specific protein called “pyruvate kinase R”, PKR, needed to maintain healthy red blood cells. Red blood cells are essential for carrying oxygen to the tissues throughout the body and when PKR protein is abnormal, red blood cells can’t make enough energy, resulting in cells breaking down sooner than normal. Those with PK deficiency have fewer healthy red blood cells resulting in anaemia which causes fatigue, jaundice, and sometimes difficulty breathing. Patients may require regular blood transfusions. There is no known cure for PK deficiency, Mitapivat attaches to the PKR protein and helps it function more normally, allowing red blood cells to make more energy and perform their normal functions. In studies in adult patients with PK deficiency who received regular blood transfusions, 37% of patients who received mitapivat required fewer blood transfusions. Mitapivat is a tablet or granule taken orally, twice daily. It is estimated that 45 patients will take part in this study globally, participants will be randomly assigned in a 2:1 ratio to receive study drug mitapivat or placebo, respectively. Randomisation will be stratified by age 1 to <6 years, 6 to <12 years and 12 to <18 years. Randomisation will be generated and implemented by an interactive voice/web response system (IXRS) provider. Mitapivat and matched placebo will be packaged and labelled identically to maintain the study blind. Patients will receive either mitapivat or placebo for 32 weeks, Double-Blind period. After this time, all patients will be given the option to receive mitapivat for an additional 5 years in an Open-Label Extension Period. The total length of your study participation is approximately 5.9 years.
REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
22/NE/0016
Date of REC Opinion
2 Mar 2022
REC opinion
Further Information Favourable Opinion