Riluzole in Fragile X Syndrome

NCT00895752 | Completed Phase 4 Results DMC

• 1 other identifier: 0809-11
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to investigate the effectiveness and tolerability of riluzole in adults with Fragile X Syndrome.

Conditions

Fragile X Syndrome

Outcome Measures

Primary Outcomes (2)

• 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

  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.

  Obtained at Baseline and Week 6

Secondary Outcomes (6)

• Aberrant Behavior Checklist

  Week 6

• The ADHD Rating Scale

  Week 6

• The Clinical Global Impression - Severity Scale

  Week 6

• The Peabody Picture Vocabulary Test

  Week 6

• The Social Reciprocity Scale

  Week 6

Study Arms (1)

Riluzole

EXPERIMENTAL

Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.

Drug: Riluzole

Interventions

Riluzole DRUG

Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.

Riluzole

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Indiana University: lead
• Indiana Clinical and Translational Sciences Institute: collaborator

Study Sites (1)

Riley Child and Adolescent Psychiatry Clinic - Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Related Publications (1)

• 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.

  PMID: 21059347 RESULT

MeSH Terms

Conditions

Fragile X Syndrome

Interventions

Riluzole

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System

Intervention Hierarchy (Ancestors)

Thiazoles; Sulfur Compounds; Organic Chemicals; Benzothiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Results Point of Contact

• Title: Craig Erickson MD
• Organization: Cincinnati Childrens Hospital

craig.erickson@cchmc.org

5136366265

Study Officials

• Craig A. Erickson, MD

  Indiana University School of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 6, 2009
• First Posted: May 8, 2009
• Study Start: April 1, 2009
• Primary Completion: July 1, 2010
• Study Completion: July 1, 2010
• Last Updated: April 18, 2017
• Results First Posted: April 18, 2017

Record last verified: 2017-03

Locations

US (1)