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

NCT00774423 | Completed Phase 2 DMC

• 1 other identifier: P040904
• Study Type: interventional
• Target: 141
• Locations: 1 country
• Sites: 1

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).

Conditions

SMA

Keywords

Spinal muscular atrophies (SMA); Degeneration of the motor neurons; Anterior horn of the spinal cord; Riluzole

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• 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

Study Arms (2)

Placebo

PLACEBO COMPARATOR

MAIN EXCIPIENT OF THE RILUTEK

Drug: Riluzole

Riluzole

ACTIVE COMPARATOR

RILUTEK

Drug: Riluzole

Interventions

Riluzole DRUG

50 mg per day during 24 months

Placebo; Riluzole

Eligibility Criteria

• Age: 6 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Hopital Raymond Poincare

Garches, 92380, France

Location

MeSH Terms

Interventions

Riluzole

Intervention Hierarchy (Ancestors)

Thiazoles; Sulfur Compounds; Organic Chemicals; Benzothiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• BRIGITTE ESTOURNET, PU-PH

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 16, 2008
• First Posted: October 17, 2008
• Study Start: January 1, 2006
• Primary Completion: September 1, 2011
• Study Completion: December 1, 2011
• Last Updated: February 15, 2013

Record last verified: 2011-09

Locations

FR (1)