SMA
Conditions
Keywords
Spinal muscular atrophies (SMA), Degeneration of the motor neurons, Anterior horn of the spinal cord, Riluzole
Brief summary
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).
Detailed description
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.
Interventions
DRUGRiluzole
50 mg per day during 24 months
Sponsors
Assistance Publique - Hôpitaux de Paris
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to 20 Years
Healthy volunteers
No
Inclusion criteria
* Patients afflicted with spinal muscular atrophy, type II or III, with genetic defect confirmed. * Age between 6 and 20 years old. * Score MFM at least 12 * Negative pregnancy test for women of child-bearing age * Signing of an informed consent form, after appropriate information has been provided (if the patient is under 18 years old, both parents are required to sign the form too; otherwise, only her (his) agreement is necessary).
Exclusion criteria
* Patients already treated with Riluzole * Concomitant treatment with: GAPAPENTINE, DEXTROMETHORPHANE, amantadine, any hepatotoxic medication that cannot be stopped, any other experimental product * Hepatic insufficiency: SGPT and/or SGOT levels higher than or equal to twice the normal higher limit * Renal insufficiency (creatinine above 115 micromoles/l) * Severe cardiac insufficiency * Current pneumopathy (clinical signs of an acute episode, confirmed by pulmonary X-ray, requiring specific treatment) * Pregnancy or nursing for women; non-abstinence or absence of effective contraception for nubile women * Any pathology or other circumstance likely to interfere with a regular follow-up * No affiliation to any social insurance system
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The drug could stabilize patients condition during 24 months, and especially interrupt paralysis progression: Motor function (MFM scale) | 6, 12, 18 and 24 months |
Secondary
| Measure | Time frame |
|---|---|
| Forced vital capacity (spirometry) | 6, 12, 18 and 24 months |
| Quality of life (OKado questionnaire) | 12 and 24 months |
| Measure of functional independence (MFI) | 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 | every 3 months |
Countries
France
Outcome results
None listed