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NCT Number: NCT00774423

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

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).

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Key information

Conditions

SMA

Age range

6 year–20 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Hopital Raymond Poincare

Garches, 92380, France

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Riluzole

Drug

50 mg per day during 24 months

Primary outcomes

  1. 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 outcomes

  1. Forced vital capacity (spirometry)

    Time frame: 6, 12, 18 and 24 months

  2. Quality of life (OKado questionnaire)

    Time frame: 12 and 24 months

  3. Measure of functional independence (MFI)

    Time frame: 6,12,18 and 24 months

  4. 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

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Registry information

Official study 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)

Acronym: ASIRI

Important dates

Study start
2006
Primary completion
2011
Study completion
2011
First posted
Oct 17, 2008
Registry last updated
Feb 15, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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