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

SMA Clinical Trial

— ASIRI  

Official 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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00774423
• Other study ID #: P040904
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: October 16, 2008
• Last updated: February 13, 2013
• Start date: January 2006
• Est. completion date: December 2011

Study information

• Verified date: September 2011
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Ministry of Health
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 141
• Est. completion date: December 2011
• Est. primary completion date: September 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years to 20 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Muscular Atrophy
• Muscular Atrophy, Spinal
• SMA

Intervention

Drug:
• Riluzole
50 mg per day during 24 months

Locations

Country	Name	City	State
France	Hopital Raymond Poincare	Garches

Sponsors (1)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The drug could stabilize patients condition during 24 months, and especially interrupt paralysis progression: Motor function (MFM scale)		6, 12, 18 and 24 months	No
Secondary	Forced vital capacity (spirometry)		6, 12, 18 and 24 months	No
Secondary	Quality of life (OKado questionnaire)		12 and 24 months	No
Secondary	Measure of functional independence (MFI)		6,12,18 and 24 months	No
Secondary	Tolerance evaluation: Somatic symptoms and adverse events Blood pressure and heart rate Weight and size Blood count, hepatic enzymes and bilirubin		every 3 months	Yes