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

Assistance Publique - Hôpitaux de Paris

Status and phase

Completed

Phase 3

Phase 2

Conditions

SMA

Treatments

Drug: Riluzole

Study type

Interventional

Funder types

Other

Identifiers

NCT00774423

P040904

Details and patient eligibility

About

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).

Full 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.

Enrollment

141 patients

Sex

All

Ages

6 to 20 years old

Volunteers

No Healthy Volunteers

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