HINALEA 1
Research type
Research Study
Full title
A PHASE IV OPEN-LABEL STUDY EVALUATING THE EFFECTIVENESS AND SAFETY OF RISDIPLAM ADMINISTERED AS AN EARLY INTERVENTION IN PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY AFTER GENE THERAPY
IRAS ID
1008990
Contact name
Head, EU/International Pharmaceuticals Division, PDR
Contact email
Sponsor organisation
F. Hoffmann-La Roche Ltd
Eudract number
2023-504508-26
Clinicaltrials.gov Identifier
Research summary
Spinal Muscular Atrophy (SMA) is genetic condition that begins in infancy and effects the nerves that control muscle movement such as moving, walking and breathing , but does not effect mental development. It is the leading genetic cause of mortality in infants and young children, with an incidence of 1 in approximately 11,000 live births and a carrier frequency estimated at 1 in 50-70 individuals.
The objectives are to evaluate the effectiveness, safety and tolerability of risdiplam (Evrysdi®) when administered to patients under 2 years of who have been diagnosed with SMA and who have previously received gene therapy. The dose will be adapted for weight and age. Risdiplam will be home administered and recorded in a daily patient diary by a caregiver. The doses and dosing schedule of risdiplam used in this study match those in the approved prescribing information.
Participants will receive risdiplam (taken by mouth or feeding tube) once daily for 72 weeks. This study has four parts:
1. Screening (to see if your child/ward is eligible for the study)
2. Treatment Period (72 weeks [approximately 18 months])
3. Treatment Extension (48 weeks [approximately 11.5 months])
4. Study Completion Visit (Week 120 [approximately 30 months]; to check on your child/ward at his/her last dose after treatment is finished)
OR
Early Discontinuation Visit (within 28 days after his/her last dose if he/she discontinues treatment early)
During this study, there will be visits approximately every 12 weeks during the Treatment Period. There will be one visit (Week 96) during the Treatment Extension Period. Visits may last 2-4 hours. The total time in the study will be about 120 weeks (approximately 30 months).The study will take place in approximately 15 centres in North America, and Europe (Germany, Poland and UK) with about 28 children (aged 2 months to 2 years) with SMA.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
24/SC/0077
Date of REC Opinion
22 Apr 2024
REC opinion
Further Information Favourable Opinion