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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00774423
• Date of registration: 16/10/2008
• Prospective Registration: No
• Primary sponsor: Assistance Publique - Hôpitaux de Paris
• Public title: Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA) ASIRI
• Scientific title: 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)
• Date of first enrolment: January 2006
• Target sample size: 141
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00774423
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 2/Phase 3

Countries of recruitment

France

Contacts

• Name: BRIGITTE ESTOURNET, PU-PH
• Affiliation: Assistance Publique - Hôpitaux de Paris

Key inclusion & exclusion 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



Age minimum: 6 Years
Age maximum: 20 Years
Gender: Both

Health Condition(s) or Problem(s) studied

SMA

Intervention(s)

Drug: Riluzole

Primary Outcome(s)

The drug could stabilize patients condition during 24 months, and especially interrupt paralysis progression: Motor function (MFM scale) [Time Frame: 6, 12, 18 and 24 months]

Secondary Outcome(s)

Quality of life (OKado questionnaire) [Time Frame: 12 and 24 months]

Tolerance evaluation: Somatic symptoms and adverse events Blood pressure and heart rate Weight and size Blood count, hepatic enzymes and bilirubin [Time Frame: every 3 months]

Forced vital capacity (spirometry) [Time Frame: 6, 12, 18 and 24 months]

Measure of functional independence (MFI) [Time Frame: 6,12,18 and 24 months]

Secondary ID(s)

P040904

Source(s) of Monetary Support

Please refer to primary and secondary sponsors