This is a multicentric, randomized, double-blind study versus placebo, with two parallel groups treated to evaluate the efficacy and the tolerance of Riluzole in children and young adults (6 to 20 years of age) with SMA. (Type II and Type III).
ASIRI study should allow to evaluate the efficacy and the tolerance of Riluzole in children and young adults (6 to 20 years of age) with SMA. This is a multicentric, randomized, double-blind study versus placebo, with two parallel groups treated and followed during 2 years. It has been preceded by a 7 days pharmacokinetic phase, concerning 14 patients, aiming to provide information on the kinetic profile of Riluzole in children. The drug could stabilize patients condition, and especially interrupt paralysis progression; those are the desired effects. There is an open-label study of the long term safety of riluzole therapy in spinal muscular atrophies types II and III, with patients previously enrolled in ASIRI double-blind study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
141
50 mg per day during 24 months
Hopital Raymond Poincare
Garches, France
The drug could stabilize patients condition during 24 months, and especially interrupt paralysis progression: Motor function (MFM scale)
Time frame: 6, 12, 18 and 24 months
Forced vital capacity (spirometry)
Time frame: 6, 12, 18 and 24 months
Quality of life (OKado questionnaire)
Time frame: 12 and 24 months
Measure of functional independence (MFI)
Time frame: 6,12,18 and 24 months
Tolerance evaluation: Somatic symptoms and adverse events Blood pressure and heart rate Weight and size Blood count, hepatic enzymes and bilirubin
Time frame: every 3 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.