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

Last updated: February 13, 2013
Sponsor: Assistance Publique - Hôpitaux de Paris
Overall Status: Completed

Phase

2/3

Condition

Myasthenia Gravis (Chronic Weakness)

Muscular Dystrophy

Spinal Muscular Atrophy

Treatment

N/A

Clinical Study ID

NCT00774423
P040904
  • Ages 6-20
  • All Genders

Study 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).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patients afflicted with spinal muscular atrophy, type II or III, with genetic defectconfirmed.

  • 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

Exclusion Criteria:

  • Patients already treated with Riluzole

  • Concomitant treatment with: GAPAPENTINE, DEXTROMETHORPHANE, amantadine, anyhepatotoxic medication that cannot be stopped, any other experimental product

  • Hepatic insufficiency: SGPT and/or SGOT levels higher than or equal to twice thenormal 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 contraceptionfor nubile women

  • Any pathology or other circumstance likely to interfere with a regular follow-up

  • No affiliation to any social insurance system

Study Design

Total Participants: 141
Study Start date:
January 01, 2006
Estimated Completion Date:
December 31, 2011

Study 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.

Connect with a study center

  • Hopital Raymond Poincare

    Garches, 92380
    France

    Site Not Available

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