Safety and efficacy of AFQ056in adolescent patients with Fragile X Syndrome

• Register: EUCTR
• Last refreshed on: 30 June 2019
• Main ID: EUCTR2010-022638-96-GB
• Date of registration: 21/02/2011
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: Safety and efficacy of AFQ056in adolescent patients with Fragile X Syndrome
• Scientific title: A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome
• Date of first enrolment: 15/06/2011
• Target sample size: 160
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-022638-96
• 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

Australia, Belgium, Canada, Denmark, France, Germany, Israel, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom, United States

Contacts

• Name: Medical Collaboration Centre
• Affiliation: Novartis Pharmaceuticals UK Ltd

• Name: Medical Collaboration Centre
• Affiliation: Novartis Pharmaceuticals UK Ltd

Key inclusion & exclusion criteria

Inclusion criteria:
Patients with Fragile X Syndrome, who are at least moderately ill based on a Clinical Global Impression Severity score of at least 4 and have qualifying scores on the ABC-C and IQ test at visit 1

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

Exclusion criteria:
-Advanced, severe or unstable disease that may interfere with the study outcome evaluations
-Cancer within the past 5 years, other than localized skin cancer
-Current treatment with more than two psychoactive medications, excluding anti-epileptics
-History of severe self-injurous behaviour
-Weigh less than 32kg
-Females who are sexually active

Other protocol-defined exclusion criteria may apply


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

Health Condition(s) or Problem(s) studied

Fragile X Syndrome
MedDRA version: 14.1 Level: PT Classification code 10017324 Term: Fragile X syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: AFQ056
Product Code: AFQ056
Pharmaceutical Form: Capsule, hard
CAS Number: 543906-09-8
Current Sponsor code: AFQ056
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 25-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Product Name: AFQ056
Product Code: AFQ056
Pharmaceutical Form: Capsule, hard
CAS Number: 543906-09-8
Current Sponsor code: AFQ056
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: - Timeframe: 12 weeks

At multiple visits:
Visits 1, 2, 2.2, 3, 4, 5, 6 and 7

Primary end point(s): Change from baseline in behavioural symptoms of Fragile X Syndrome using the Aberrant Behaviour Checklist - Community (ABC-CFX) Total score in Stratum I

Secondary Objective: Key secondary objectives:
To assess the efficacy of three doses of AFQ056 versus placebo in reducing the ABC-CFX total score after 12 weeks of treatment in FXS patients with partially methylated (PM) FMR1 gene

To assess the efficacy of two lower doses of AFQ056 (25mg BID and 50mg BID) versus placebo in reducing the ABC-CFX total score after 12 weeks of treatment in FXS patients with FMFMR1 gene

Main Objective: • To assess the efficacy of AFQ056 100mg BID versus placebo in reducing the ABC-C Total score (using the FXS-specific algorithm - ABC-CFX) after 12 weeks of treatment in FXS patients in Stratum I.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: - Timeframe: 12 weeks for each secondary endpoint

- ABC-CFX: Visits 1, 2, 2.2, 3, 4, 5, 6 and 7

- CGI-I: Visits 4, 5, 6 and 7

-RBS-R: Visits 2, 3, 5 and 7

- Safety and tolerability: Timeframe 12 weeks

-Neuropsychiatric Inventory - Questionnaire (NPI-Q) at visits 2.2, 3, 4, 5, 6 and 7 / Early discontinuation

Secondary end point(s): - Abberant Behaviour Checklist - Community Edition (ABC-CFX): Total score and subscales

- Clinical Global Impression - Improvement (CGI-I)

- Repetitive Behaviour Scale - Revised (RBS-R)

- Safety and tolerability as measured by changes in vital signs, ECGs, laboratory values and percentages of adverse events and serious adverse events, Neuropsychiatric Inventory - Questionnaire (NPI-Q)

Secondary ID(s)

NCT01357239

CAFQ056B2214

Source(s) of Monetary Support

Novartis Pharma Services AG

Ethics review

Status: Approved
Approval date:
Contact:

Results

• Results available: Yes
• Date Posted: 17/07/2016
• Date Completed: 06/01/2014
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-022638-96/results