search
Back to results

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

Primary Purpose

SMA

Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Riluzole
Sponsored by
Assistance Publique - Hôpitaux de Paris
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for SMA focused on measuring Spinal muscular atrophies (SMA), Degeneration of the motor neurons, Anterior horn of the spinal cord, Riluzole

Eligibility Criteria

6 Years - 20 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Hopital Raymond Poincare

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Active Comparator

Arm Label

Placebo

Riluzole

Arm Description

MAIN EXCIPIENT OF THE RILUTEK

RILUTEK

Outcomes

Primary Outcome Measures

The drug could stabilize patients condition during 24 months, and especially interrupt paralysis progression: Motor function (MFM scale)

Secondary Outcome Measures

Forced vital capacity (spirometry)
Quality of life (OKado questionnaire)
Measure of functional independence (MFI)
Tolerance evaluation: Somatic symptoms and adverse events Blood pressure and heart rate Weight and size Blood count, hepatic enzymes and bilirubin

Full Information

First Posted
October 16, 2008
Last Updated
February 13, 2013
Sponsor
Assistance Publique - Hôpitaux de Paris
search

1. Study Identification

Unique Protocol Identification Number
NCT00774423
Brief Title
Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA)
Acronym
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)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2011
Overall Recruitment Status
Completed
Study Start Date
January 2006 (undefined)
Primary Completion Date
September 2011 (Actual)
Study Completion Date
December 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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).
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
SMA
Keywords
Spinal muscular atrophies (SMA), Degeneration of the motor neurons, Anterior horn of the spinal cord, Riluzole

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
141 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
MAIN EXCIPIENT OF THE RILUTEK
Arm Title
Riluzole
Arm Type
Active Comparator
Arm Description
RILUTEK
Intervention Type
Drug
Intervention Name(s)
Riluzole
Intervention Description
50 mg per day during 24 months
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
Forced vital capacity (spirometry)
Time Frame
6, 12, 18 and 24 months
Title
Quality of life (OKado questionnaire)
Time Frame
12 and 24 months
Title
Measure of functional independence (MFI)
Time Frame
6,12,18 and 24 months
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
BRIGITTE ESTOURNET, PU-PH
Organizational Affiliation
Assistance Publique - Hôpitaux de Paris
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hopital Raymond Poincare
City
Garches
ZIP/Postal Code
92380
Country
France

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs