A study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome

• Register: EUCTR
• Last refreshed on: 27 July 2020
• Main ID: EUCTR2011-004349-42-GB
• Date of registration: 25/04/2012
• Prospective Registration: Yes
• Primary sponsor: F. Hoffmann-La Roche Ltd.
• Public title: A study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome
• Scientific title: A randomized, double-blind, 12- week, parallel group, placebo-controlled study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome.
• Date of first enrolment: 16/07/2012
• Target sample size: 180
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-004349-42
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Argentina, Canada, Chile, France, Mexico, Peru, Spain, Sweden, United Kingdom, United States

Contacts

• Name: Trial Information Support Line-TISL
• Affiliation: F.Hoffmann-La Roche Ltd.

• Name: Trial Information Support Line-TISL
• Affiliation: F.Hoffmann-La Roche Ltd.

Key inclusion & exclusion criteria

Inclusion criteria:
• Adults and adolescents (14 to 50)
• CGI-S of 3 (mildly ill) or more
• ABC total score of 20 or more
• Diagnosis of FXS with a confirmed FMR1 full mutation

Are the trial subjects under 18? yes
Number of subjects for this age range: 60
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 120
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
• Have previously received treatment with another mGlu5 receptor antagonist within 18 months or RO4917523
• Enrollment/participation in any interventional study (clinical trial) involving an investigational drug (unapproved) or non-drug treatment within the prior 3 months or 5 times the half-life (whichever is longer)
• Any uncontrolled, unstable clinically significant psychiatric condition other than FXS that may interfere with interpretation of safety and efficacy evaluations (e.g. Attention Deficit Hyperactivity Disorder (ADHD))
• Current symptoms or presumption of psychosis or euphoria, history of catatonia, hallucinations or delusional thoughts
• History of suicidal behaviour or otherwise considered a high suicidal risk by the investigator


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03]

Fragile X Syndrome (FXS)
MedDRA version: 14.1 Level: LLT Classification code 10025463 Term: Major depressive disorder, single episode System Organ Class: 100000004873

Intervention(s)

Product Name: mGlu5 antagonist
Product Code: Ro 491-7523/F18
Pharmaceutical Form: Capsule
Current Sponsor code: RO4917523
Other descriptive name: mGlu5 antagonist
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0.5-
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use

Product Name: mGlu5 antagonist
Product Code: Ro 491-7523/F19
Pharmaceutical Form: Capsule
Current Sponsor code: RO4917523
Other descriptive name: mGlu5 antagonist
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1.0-
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: Change from baseline to end of treatment (12 weeks)

Primary end point(s): Change in the ADAMS total score from baseline to end of treatment

Secondary Objective: • Change from baseline in symptoms as measured by the: Aberrant Behaviour Checklist (ABC) total, ABC factor scores, Anxiety Depression and Mood Scale (ADAMS) total, ADAMS factor scores, and Social Responsiveness Scale (SRS)
• Change from baseline in cognitive function as measured by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)
• Clinical Global Impression-Improvement (CGI-I) and change from baseline in the Clinical Global Impression-Severity (CGI-S)
• Clinical response (at least 25% improvement in the ABC total score and a CGI-I score of 1 or 2)
• Change in the caregiver-identified most troubling symptom as measured by the Visual Analogue Scale (VAS)
• Change from baseline in adaptive behaviour skills as measured by the Vineland Adaptive Behavior Scale (VABS-II)

Main Objective: • To evaluate the efficacy of 12-week treatment with RO4917523 in patients with Fragile X Syndrome (FXS) as measured by the ADAMS total score
• To evaluate the safety and tolerability of 12-week treatment with RO4917523 in patients with FXS

Secondary Outcome(s)

Secondary end point(s): • Change from baseline in symptoms as measured by the ABC and ADAMS (total and factor scores), and the SRS
• Change from baseline in cognitive function as measured by the RBANS
• CGI-I and change from baseline in the CGI-S
• Clinical response (at least 25% improvement in the ABC total score and a CGI-I score of 1 or 2)
• Change in the caregiver-identified most troubling symptom as measured by the VAS
• Change from baseline in adaptive behavior skills as measured by the VABS-II
• To investigate the pharmacokinetics and exposure response relationship of RO4917523 in patients with FXS using a population analysis approach.

Timepoint(s) of evaluation of this end point: Change from baseline to end of treatment (12 weeks)

Secondary ID(s)

NP27936

Source(s) of Monetary Support

F. Hoffmann-La Roche Ltd.

Ethics review

Status: Approved
Approval date: 16/07/2012
Contact:

Results

• Results available: Yes
• Date Posted: 07/08/2015
• Date Completed: 04/04/2014
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-004349-42/results