A Study With RO4917523 in Patients With Fragile X Syndrome

Fragile X Syndrome Clinical Trial

Official title:

A Randomized, Double-blind, Placebo-controlled, Pharmacokinetic, Safety and Tolerability, and Exploratory Efficacy and Pharmacodynamic Effects Study of RO4917523 in Adult Patients With Fragile X Syndrome.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01015430
• Other study ID #: NP22578
• Status: Completed
• Phase: Phase 2
• First received: November 17, 2009
• Last updated: November 1, 2016
• Start date: November 2009
• Est. completion date: January 2011

Study information

• Verified date: November 2016
• Source: Hoffmann-La Roche
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

This randomized, double-blind multiple ascending dose study will evaluate the safety and tolerability, pharmacokinetics and efficacy of RO4917523 in patients with Fragile X Syndrome. The patients will be randomized to receive either active drug or placebo. The anticipated time on study treatment is 6 weeks. The target sample size is <100 patients.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: January 2011
• Est. primary completion date: January 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 50 Years

Eligibility

Inclusion Criteria:

• Adult patients, 18 to 50 years of age

• Fragile X Syndrome

• IQ less than 75

• Reliable caregiver

Exclusion Criteria:

• Current psychosis or presumption of psychosis

• History of suicidal behavior or considered a high suicidal risk

• Severe self-injurious behavior

• Any unstable medical condition other than Fragile X Syndrome (e.g. hypertension, diabetes)

• Current seizure disorder

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• Placebo (for RO4917523 ascending doses)
Placebo daily po
• Placebo (for RO4917523 fixed dose)
Placebo daily po
• RO4917523
RO4917523 ascending doses daily po
• RO4917523
RO4917523 fixed dose daily po

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Hoffmann-La Roche

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety, Tolerability and Pharmacokinetics: AEs, laboratory parameters		AEs throughout study; laboratory assessments: days 1, 8, 15, 29, 43	No
Secondary	Efficacy: Behavior and cognition assessments		Every 2 weeks throughout study	No