Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA)

• Status: Completed
• Trial end date: 2011-12-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris
• Treatments: Riluzole

Criteria

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