A Phase 2 study of the safety, efficacy and pharmacokinetics of PRAX-562 in paediatric patients
Research type
Research Study
Full title
A Phase 2, Double-Blind, Randomized Clinical Trial to Explore the Safety, Tolerability, Efficacy, and Pharmacokinetics of PRAX-562 in Pediatric Participants with Developmental and Epileptic Encephalopathies Followed by an Open-Label Extension
IRAS ID
1010967
Contact name
William Motel
Contact email
Sponsor organisation
Praxis Precision Medicines
Clinicaltrials.gov Identifier
Research summary
The aim of this clinical study is to investigate the safety and tolerability, as well as the effectiveness and the levels in the body, of a potential new medicine called PRAX-562, which is being developed as a treatment for rare, debilitating epilepsy and developmental disorders in children. Patients with these disorders have mutations in certain genes which are involved in the production of proteins called voltage-gated sodium channels. PRAX-562 is expected to work by closing (blocking) these sodium channels to reduce unwanted activity, and maintain normal electrical activity in the brain. This is expected to reduce epileptic seizures and improve other symptoms of the disease. This study is open to male and female patients aged at least 2 years old and no more than 18 years old, with a confirmed diagnosis of these disorders. There are two parts to this study, Part A and Part B, and there will be four groups (cohorts) of patients. Part A is randomized and double-blind, which means a patient will be allocated at random to receive, once daily, either PRAX-562 for 16 weeks, or PRAX-562 for 12 weeks and a matching dummy medicine (placebo) for 4 weeks. The timing of when a patient receives placebo will be unknown (blinded) for both them and the investigator. The first dose will be administered either during a clinic visit, or at the patient's home under the supervision of study staff. Subsequent doses will be administered at the patient's home. The total length of Part A will be 26 weeks, which includes a screening period of up to 6 weeks, and a 4 week follow up period if a patient does not wish to enter Part B. Part B will be an open-label extension, which means all patients are invited to continue treatment with PRAX-562, for a period of 144 weeks, after which there is a 4 week safety follow up period. Patients will undergo health checks to assess their general health, including the collection of blood and urine samples. The study will involve two sites in the UK.
REC name
London - Hampstead Research Ethics Committee
REC reference
24/LO/0848
Date of REC Opinion
31 Jan 2025
REC opinion
Further Information Favourable Opinion