Phase 3 study to evaluate mitapivat in pediatrics with PKD who are not 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 Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period
IRAS ID
1004456
Contact name
María Trolliet
Contact email
Sponsor organisation
Agios Pharmaceuticals, Inc.
Eudract number
2021-003333-11
Clinicaltrials.gov Identifier
Research summary
Phase 3 study to evaluate the efficacy and safety of mitapivat versus placebo, assessed by an increase in haemoglobin concentrations in paediatric patients with pyruvate kinase (PK) deficiency who do not regularly receive blood transfusions. PK Deficiency is a genetic condition resulting in the defect of a specific protein called “pyruvate kinase R”, PKR, is needed to maintain healthy red blood cells. Red blood cells are essential for carrying oxygen throughout the body, but when PKR protein is abnormal, red blood cells can’t make enough energy, causing red blood cells to breakdown sooner than normal. PK deficiency patients have fewer healthy red blood cells resulting in anaemia which causes fatigue, jaundice, and sometimes difficulty breathing. Current support treatments do not target the underlying causes of the disease, Mitapivat is a first-in-class drug which works to improve PK Deficiency symptoms by attaching to the PKR protein helping it function more normally, allowing cells to make more energy and perform their normal functions, therefore reducing complications of chronic haemolytic anaemia in patients with PK deficiency, preventing/reducing the need for transfusions, decreasing disease burden, and improve quality of life. Paediatric patients between 1 and 18 years of age will be recruited from a hospital setting. Patients will receive either mitapivat or placebo for 32 weeks, Double-Blind period, after which, 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. Patients will receive age/weight-based doses of mitapivat or matched placebo for oral administration and will be regularly reviewed by the study team, patients undergo blood testing and scans to assess disease progress and safety. Patients will keep an e-diary and regularly answer questionnaires to access disease burden and quality of life.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
22/NW/0109
Date of REC Opinion
13 May 2022
REC opinion
Further Information Favourable Opinion