SMAPhase 2/3ASIRINCT00774423
Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA)
A Phase 2/3 study testing Riluzole in people with SMA, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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). Read more about this study
- Who is it for?
- Ages 6 to 20 years
- Conditions the study is for
- SMA
- What is being tested?
- Riluzole
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 141 participants
- Where
- 1 siteFrance
- Study dates
- Jan 2006to Dec 2011
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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).
Who cannot join
- 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)
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- 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
Trial locations and contacts
1 site in 1 country.
About this study
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)
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).
Read the 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.
- Conditions
- SMA
- Being tested
- Riluzole
- Keywords
- Spinal muscular atrophies (SMA), Degeneration of the motor neurons, Anterior horn of the spinal cord, Riluzole
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RiluzoleDrug
50 mg per day during 24 months
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | MAIN EXCIPIENT OF THE RILUTEK Receives: Drug: Riluzole |
| Riluzole | Active comparator | RILUTEK Receives: Drug: Riluzole |
What is being measured
The primary outcome is the main question the study is designed to answer.
The drug could stabilize patients condition during 24 months, and especially interrupt paralysis progression: Motor function (MFM scale)
Measured: 6, 12, 18 and 24 months
Secondary outcomes (4)
Forced vital capacity (spirometry)
Measured: 6, 12, 18 and 24 months
Quality of life (OKado questionnaire)
Measured: 12 and 24 months
Measure of functional independence (MFI)
Measured: 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
Measured: every 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2008 (estimated)
- Study startJan 2006
- Primary completionSep 2011
- Study completionDec 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SMAStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 141 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- BRIGITTE ESTOURNET, PU-PH, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00774423
- Sponsor's protocol ID
- P040904
- Acronym
- ASIRI
- Status last verified
- Sep 2011
- First submitted
- 16 Oct 2008
- Last updated
- 15 Feb 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT00774423
- Acronym / sponsor ID
- ASIRI / P040904
- Conditions
- SMA
- Keywords
- Spinal muscular atrophies (SMA), Degeneration of the motor neurons, Anterior horn of the spinal cord, Riluzole
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 141 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Jan 2006
- Primary completion
- Sep 2011 (actual)
- Study completion
- Dec 2011 (actual)
- First posted
- 17 Oct 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: RiluzoleMAIN EXCIPIENT OF THE RILUTEK |
| Riluzole | Active comparator | Drug: RiluzoleRILUTEK |
Who may qualify
- Ages
- 6 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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).
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Riluzole50 mg per day during 24 months | Drug | Placebo, Riluzole |
Primary outcome
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
4 secondary outcomes
Forced vital capacity (spirometry)
Time frame: 6, 12, 18 and 24 months
Quality of life (OKado questionnaire)
Time frame: 12 and 24 months
Measure of functional independence (MFI)
Time frame: 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
Time frame: every 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Feb 2013.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
BRIGITTE ESTOURNET, PU-PH principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Riluzole in people with SMA, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Feb 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Feb 2013.