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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT00774423
First received: October 16, 2008
Last updated: February 13, 2013
Last verified: September 2011
History of Changes
  Purpose

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).


Condition Intervention Phase
SMA
 Drug: Riluzole
 Phase 2
Phase 3

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
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)

Resource links provided by NLM:

Drug Information available for: Riluzole
U.S. FDA Resources

Further study details as provided by Assistance Publique - Hôpitaux de Paris:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Forced vital capacity (spirometry) [ Time Frame: 6, 12, 18 and 24 months ] [ Designated as safety issue: No ]
  • Quality of life (OKado questionnaire) [ Time Frame: 12 and 24 months ] [ Designated as safety issue: No ]
  • Measure of functional independence (MFI) [ Time Frame: 6,12,18 and 24 months ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: Yes ]

Enrollment: 141
Study Start Date: January 2006
Study Completion Date: December 2011
Primary Completion Date: September 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo
MAIN EXCIPIENT OF THE RILUTEK
 Drug: Riluzole
50 mg per day during 24 months
Other Name: Riluzole
Active Comparator: Riluzole
RILUTEK
 Drug: Riluzole
50 mg per day during 24 months
Other Name: 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.

  Eligibility

Ages Eligible for Study:   6 Years to 20 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00774423

Locations
France
Hopital Raymond Poincare
Garches, France, 92380
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
Principal Investigator: BRIGITTE ESTOURNET, PU-PH Assistance Publique - Hôpitaux de Paris
  More Information

No publications provided

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT00774423     History of Changes
Other Study ID Numbers: P040904
Study First Received: October 16, 2008
Last Updated: February 13, 2013
Health Authority: France: Ministry of Health

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Spinal muscular atrophies (SMA)
Degeneration of the motor neurons
Anterior horn of the spinal cord
Riluzole

Additional relevant MeSH terms:
Muscular Atrophy
Muscular Atrophy, Spinal
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Atrophy
Pathological Conditions, Anatomical
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases
 Riluzole
Excitatory Amino Acid Antagonists
Excitatory Amino Acid Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs
Neuroprotective Agents
Protective Agents
Central Nervous System Agents
Therapeutic Uses
Anticonvulsants

ClinicalTrials.gov processed this record on May 21, 2013