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

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)

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).

Study Overview

• Status: Completed
• Conditions: SMA
• Intervention / Treatment: Drug: Riluzole

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.

• Study Type: Interventional
• Enrollment (Actual): 141
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• France
  • Garches, France, 92380
    • Hôpital Raymond Poincaré

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 20 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
PLACEBO_COMPARATOR: Placebo; MAIN EXCIPIENT OF THE RILUTEK	Drug: Riluzole; 50 mg per day during 24 months
ACTIVE_COMPARATOR: Riluzole; RILUTEK	Drug: Riluzole; 50 mg per day during 24 months

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Time Frame
Forced vital capacity (spirometry)	6, 12, 18 and 24 months
Quality of life (OKado questionnaire)	12 and 24 months
Measure of functional independence (MFI)	6,12,18 and 24 months
Tolerance evaluation: Somatic symptoms and adverse events Blood pressure and heart rate Weight and size Blood count, hepatic enzymes and bilirubin	every 3 months

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris
• Investigators: Principal Investigator: BRIGITTE ESTOURNET, PU-PH, Assistance Publique - Hôpitaux de Paris

Study record dates

Study Major Dates

• Study Start: January 1, 2006
• Primary Completion (ACTUAL): September 1, 2011
• Study Completion (ACTUAL): December 1, 2011

Study Registration Dates

• First Submitted: October 16, 2008
• First Submitted That Met QC Criteria: October 16, 2008
• First Posted (ESTIMATE): October 17, 2008

Study Record Updates

• Last Update Posted (ESTIMATE): February 15, 2013
• Last Update Submitted That Met QC Criteria: February 13, 2013
• Last Verified: September 1, 2011

More Information

Terms related to this study

• Keywords: Riluzole; Spinal muscular atrophies (SMA); Degeneration of the motor neurons; Anterior horn of the spinal cord
• Additional Relevant MeSH Terms: Nervous System Diseases; Neurologic Manifestations; Neuromuscular Manifestations; Pathological Conditions, Anatomical; Atrophy; Muscular Atrophy; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Excitatory Amino Acid Antagonists; Excitatory Amino Acid Agents; Neuroprotective Agents; Protective Agents; Anticonvulsants; Riluzole
• Other Study ID Numbers: P040904