PIONEER
Research type
Research Study
Full title
Multi-center, Randomized, Double-blind, Parallel-group, Double-dummy, Active-controlled, Comparative Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Ponesimod Versus Fingolimod During 108 Weeks of Treatment in Pediatric Participants, 10 to <18 Years Old, with Relapsing-remitting Multiple Sclerosis
IRAS ID
1007040
Contact name
David Wright
Contact email
Sponsor organisation
Janssen-Cilag International NV
Eudract number
2020-004431-24
Research summary
Multiple Sclerosis (MS) is a disease, in which the body’s own immune cells (e.g. lymphocytes) attack the covering on the nerves called myelin and damage nerve function in the brain and spinal cord. Relapsing-remitting MS (RRMS) is the most common type of MS. It is marked by periods of new or worsening of symptoms (relapses) followed by periods of improvement or disappearance of symptoms (remission).
Ponesimod is a drug that blocks a receptor* called sphingosine-1-phosphate receptor 1 (S1P1), which prevents migration of lymphocytes into the brain and spinal cord, and thereby reduces the damage to the nerves in the brain and spinal cord.
*Protein that binds to specific molecule.
Researchers want to know if ponesimod is not less effective than fingolimod in reducing relapses in children and adolescents with RRMS. This will be measured by time from start of the treatment to the first relapse experienced by the participant.This study will include male and female participants 10 to less than 18 years old.
This study will include two participant groups, one with body weights above 40 kg, and the other with body weights 40 kg or below. In the first cohort (>40 kg) participants will be randomly divided into 2 groups as below:
Arm A (Ponesimod): Participants will receive up to 20 mg ponesimod once daily for about 2 years.
Arm B (Fingolimod): Participants will receive fingolimod 0.5 mg orally once daily for about 2 years.
Participants will also receive matching placebo (a pill which contains no medicine) for about 2 years.
A parallel second cohort will be a sub-study to assess if a dose below 20 mg ponesimod results in drug concentrations and effects comparable to those with 20 mg in adults. It will be conducted in participants with body weight 40 kg or below.
During the study, some tests will be performed such as blood and urine tests, physical and neurological exams, vital signs, MRI, ECG, and questionnaires. All side effects will be recorded until study ends.REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
23/NW/0006
Date of REC Opinion
31 May 2023
REC opinion
Further Information Favourable Opinion