Study to Evaluate the Efficacy of Riluzole in Children an... | NCT00774423

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

NCT00774423

Sponsor

Assistance Publique - Hôpitaux de Paris

Status

Completed

Last Update Posted

Feb 15, 2013Estimated

Enrollment

141Actual

Phase

Phase 2Phase 3

Conditions

Interventions

Riluzole

Countries

France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Sep 2011

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 2006

Primary Completion Date

Sep 2011Actual

Completion Date

Dec 2011Actual

First Submitted Date

Oct 16, 2008

First Submission Date that Met QC Criteria

Oct 16, 2008

First Posted Date

Oct 17, 2008Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 13, 2013

Last Update Posted Date

Feb 15, 2013Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

141Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Placebo	Placebo Comparator	MAIN EXCIPIENT OF THE RILUTEK	Drug: Riluzole
Riluzole	Active Comparator	RILUTEK	Drug: Riluzole

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Riluzole	Drug	50 mg per day during 24 months	Placebo Riluzole

Outcomes Module

Primary Outcomes

Measure	Description	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 Outcomes

Measure	Description	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)

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Years

Maximum Age

20 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

ID	Term
D013844	Thiazoles
D013457	Sulfur Compounds
D009930	Organic Chemicals
D052160	Benzothiazoles