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

NCT ID: NCT00774423

Last Updated: 2013-02-15

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 141 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-01-31
• Study Completion Date: 2011-12-31

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.

Conditions

SMA

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Placebo

MAIN EXCIPIENT OF THE RILUTEK

Group Type: PLACEBO_COMPARATOR

Riluzole

Intervention Type: DRUG

50 mg per day during 24 months

Riluzole

RILUTEK

Group Type: ACTIVE_COMPARATOR

Riluzole

Intervention Type: DRUG

50 mg per day during 24 months

Interventions

Riluzole

50 mg per day during 24 months

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 20 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

BRIGITTE ESTOURNET, PU-PH

Role: PRINCIPAL_INVESTIGATOR

Assistance Publique - Hôpitaux de Paris

Locations

Hopital Raymond Poincare

Garches, , France

Countries

France

Other Identifiers

P040904

Identifier Type: -

Identifier Source: org_study_id