Fragile X SyndromePhase 4NCT00895752
Riluzole in Fragile X Syndrome
A Phase 4 study testing Riluzole in people with Fragile X Syndrome, run by Indiana University at 1 site in United States.
Trial summary
The purpose of this study is to investigate the effectiveness and tolerability of riluzole in adults with Fragile X Syndrome. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- Riluzole
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2009to Jul 2010
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females age 18 years or older.
- Confirmed molecular diagnosis of Fragile X Syndrome.
- Clinical Global Impression Severity (CGI-S) score of 3 or greater.
- Significant interfering repetitive behavior as determined by the principal investigator.
- Must be in good health as determined by screening procedures including a detailed medical history, and complete physical and neurological examination.
- Dosing of concomitant medications during the study must remain stable.
Who cannot join
- Pregnancy.
- Concomitant use of another glutamatergic agent (memantine, topiramate, amantadine, among others.
- Evidence of prior trial of riluzole and/or hypersensitivity/allergic reaction to riluzole.
- Abnormal baseline liver function tests at screen or by history; or complete blood count abnormalities at screen or by history.
Trial locations and contacts
1 site in 1 country.
United States1 site
Riley Child and Adolescent Psychiatry Clinic - Riley Hospital for Children
Indianapolis, Indiana, 46202
About this study
Official title: Riluzole in Fragile X Syndrome: A Pilot Study Incorporating Biomarker Assay
The purpose of this study is to investigate the effectiveness and tolerability of riluzole in adults with Fragile X Syndrome.
Read the detailed description
Fragile X Syndrome (FXS) represents the most common inherited form of intellectual disability. FXS is more common in males and the symptoms associated with the disorder are more marked in males. FXS is associated with characteristic physical features, behaviors, and comorbidities. Those with FXS often suffer from behavioral difficulties that include anxiety-related symptoms (shyness, social phobia, obsessive-compulsive disorder (OCD) symptoms), attention deficit hyperactivity symptoms (overarousal, hyperactivity, distractibility, impulsivity) and aggressive/self-injurious behaviors.
Riluzole is approved by the FDA for use in treating amyotrophic lateral sclerosis (ALS) in adults. Recently, riluzole has been the subject of several open-label studies describing the use of the drug in treatment-resistant depression and OCD.
Given the overlap between repetitive behavior in FXS and symptoms of OCD, it is logical to study riluzole in FXS given the compound's promise in ameliorating treatment-refractory symptoms of OCD.
- Conditions
- Fragile X Syndrome
- Being tested
- Riluzole
- Medical subject headings
- Fragile X Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RiluzoleDrug
Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.
| Study group | Role | What they receive |
|---|---|---|
| Riluzole | Experimental | Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day. Receives: Drug: Riluzole |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical Global Impression-Improvement (CGI-I)
The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. and rated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse
Measured: Obtained at Week 6
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
The CY-BOCS PDD has been utilized in a largescale clinical treatment study of repetitive behavior in idiopathic ASDs. CYBOCS-PDD scores range from 0 to 20 and measure repetitive/compulsive behavior and not obsessions. Higher score indicate worse outcome.
Measured: Obtained at Baseline and Week 6
Secondary outcomes (6)
Aberrant Behavior Checklist
The Aberrant Behavior Checklist (ABC) is a 58-item measure of maladaptive behaviors and is used as a measure of drug effects. The ABC has 5 subscales: Social Withdrawal (16 items) ranging from 0 (not at all) to 48 (severe), Irritability (15 items) ranging from 0 (not at all) to 45 (severe), Inappropriate Speech (4 items) ranging from 0 (not at all) to 12 (severe), Hyperactivity (16 items) ranging from 0 (not at all) to 48 (severe), and Stereotypy (7 items) ranging from 0 (not at all) to 21 (severe). Items are rated from 0 (not at all) to 3 (severe).
Measured: Week 6
The ADHD Rating Scale
The ADHD Rating Scale is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder. The ADHD Rating Scale-IV is completed by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. The total score can range from 0 to 54, with a higher score indicating greater severity
Measured: Week 6
The Clinical Global Impression - Severity Scale
The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating 1, normal, not at all ill; 2, borderline mentally ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill
Measured: Week 6
The Peabody Picture Vocabulary Test
The Peabody Picture Vocabulary Test is one of the most commonly used assessment tests that measure verbal ability in standard American English vocabulary. This test has been nationally standardized using examinees from various age groups, from children to adults. Thus, the raw scores are equated to mental age, using the norms obtained from standardization. The total standard scores range from 40 (worse receptive vocabulary) to 160 (better receptive vocabulary). The scores can also be converted to percentile rank.
Measured: Week 6
The Social Reciprocity Scale
The 65-item SRS is a standardized measure of the core symptoms of autism. Each item is scored on a 4-point Likert scale. The score of each individual item is summed to create a total raw score. A total scores results are as follows: 0-62: Within normal limits 63-79: Mild range of impairment 80-108: Moderate range of impairment 109-149: Severe range of impairment.
Measured: Week 6
Extra-cellular Signal-relatedness Kinase (ERK)
ERK activations times, as defined as the time in minutes for ERK phosphorylation to reach the half maximal level.
Measured: Screen and Week 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2009 (estimated)
- Study startApr 2009
- Primary completionJul 2010
- Study completionJul 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fragile X SyndromeStudies of the same condition, open and under way.
- More trials run by Indiana UniversityThe 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Collaborators
- Indiana Clinical and Translational Sciences Institute
- Responsible party
- Sponsor
- Investigators
- Craig A. Erickson, MD, Principal investigator, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00895752
- Sponsor's protocol ID
- 0809-11
- Status last verified
- Mar 2017
- First submitted
- 6 May 2009
- Last updated
- 18 Apr 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Erickson CA, Weng N, Weiler IJ, Greenough WT, Stigler KA, Wink LK, McDougle CJ. Open-label riluzole in fragile X syndrome. Brain Res. 2011 Mar 22;1380:264-70. doi: 10.1016/j.brainres.2010.10.108. Epub 2010 Nov 5.
PubMed 21059347
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Riluzole in Fragile X Syndrome: A Pilot Study Incorporating Biomarker Assay
- ClinicalTrials.gov ID
- NCT00895752
- Acronym / sponsor ID
- 0809-11
- Conditions
- Fragile X Syndrome
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Collaborators
- Indiana Clinical and Translational Sciences Institute
- Study start
- Apr 2009
- Primary completion
- Jul 2010 (actual)
- Study completion
- Jul 2010 (actual)
- First posted
- 8 May 2009 (estimated)
- Results
- Posted 18 Apr 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to investigate the effectiveness and tolerability of riluzole in adults with Fragile X Syndrome.
Detailed description
Fragile X Syndrome (FXS) represents the most common inherited form of intellectual disability. FXS is more common in males and the symptoms associated with the disorder are more marked in males. FXS is associated with characteristic physical features, behaviors, and comorbidities. Those with FXS often suffer from behavioral difficulties that include anxiety-related symptoms (shyness, social phobia, obsessive-compulsive disorder (OCD) symptoms), attention deficit hyperactivity symptoms (overarousal, hyperactivity, distractibility, impulsivity) and aggressive/self-injurious behaviors.
Riluzole is approved by the FDA for use in treating amyotrophic lateral sclerosis (ALS) in adults. Recently, riluzole has been the subject of several open-label studies describing the use of the drug in treatment-resistant depression and OCD.
Given the overlap between repetitive behavior in FXS and symptoms of OCD, it is logical to study riluzole in FXS given the compound's promise in ameliorating treatment-refractory symptoms of OCD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Riluzole | Experimental | Drug: RiluzoleSix week open-label treatment with riluzole, maximum dose of 50 mg twice a day. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Males and females age 18 years or older.
- Confirmed molecular diagnosis of Fragile X Syndrome.
- Clinical Global Impression Severity (CGI-S) score of 3 or greater.
- Significant interfering repetitive behavior as determined by the principal investigator.
- Must be in good health as determined by screening procedures including a detailed medical history, and complete physical and neurological examination.
Key exclusion criteria (4 listed)
- Pregnancy.
- Concomitant use of another glutamatergic agent (memantine, topiramate, amantadine, among others.
- Evidence of prior trial of riluzole and/or hypersensitivity/allergic reaction to riluzole.
- Abnormal baseline liver function tests at screen or by history; or complete blood count abnormalities at screen or by history.
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 |
|---|---|---|
| RiluzoleSix week open-label treatment with riluzole, maximum dose of 50 mg twice a day. | Drug | Riluzole |
Primary outcome
Clinical Global Impression-Improvement (CGI-I)
Time frame: Obtained at Week 6
The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. and rated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
Time frame: Obtained at Baseline and Week 6
The CY-BOCS PDD has been utilized in a largescale clinical treatment study of repetitive behavior in idiopathic ASDs. CYBOCS-PDD scores range from 0 to 20 and measure repetitive/compulsive behavior and not obsessions. Higher score indicate worse outcome.
6 secondary outcomes
Aberrant Behavior Checklist
Time frame: Week 6
The ADHD Rating Scale
Time frame: Week 6
The Clinical Global Impression - Severity Scale
Time frame: Week 6
The Peabody Picture Vocabulary Test
Time frame: Week 6
The Social Reciprocity Scale
Time frame: Week 6
Extra-cellular Signal-relatedness Kinase (ERK)
Time frame: Screen and Week 6
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 21059347Erickson CA, Weng N, Weiler IJ, Greenough WT, Stigler KA, Wink LK, McDougle CJ. Open-label riluzole in fragile X syndrome. Brain Res. 2011 Mar 22;1380:264-70. doi: 10.1016/j.brainres.2010.10.108. Epub 2010 Nov 5.
Related trials for Fragile X Syndrome
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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.
Craig A. Erickson, MD principal investigator
Indiana University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Riluzole in people with Fragile X Syndrome, run by Indiana University at 1 site in United States.
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 6 inclusion and 4 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 18 Apr 2017. 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 United States. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Apr 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Clinical Global Impression-Improvement (CGI-I) (time frame: Obtained at Week 6). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00895752, which also lists 1 linked publication.
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 18 Apr 2017.