Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

• Register: EUCTR
• Last refreshed on: 12 May 2014
• Main ID: EUCTR2009-013667-19-DK
• Date of registration: 22/09/2010
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
• Scientific title: A randomized, double-blind, placebo-controlled, parallel group study to evaluate AFQ056 in adult patients with Fragile X Syndrome
• Date of first enrolment: 16/11/2010
• Target sample size: 160
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-013667-19
• 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: 4

Countries of recruitment

Australia, Canada, Denmark, France, Germany, Italy, Spain, Switzerland, United Kingdom, United States

Contacts

• Name: Klinisk forskningsafdeling
• Affiliation: Novartis Healthcare A/S

• Name: Klinisk forskningsafdeling
• Affiliation: Novartis Healthcare A/S

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? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 160
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

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-injurious behavior

Other protocol defined exclusion criteria may apply


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

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Fragile X Syndrome
MedDRA version: 15.1 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)

Main Objective: To assess the efficacy of three doses of AFQ056 versus placebo in reducing the ABC-C Total score (using the FXS specific algorithm - ABC-CFX) after 12 weeks of treatment in FXS patients with fully-methylated FMR1 gene.

Secondary Objective: Key secondary objective:

To assess the efficacy of AFQ056 versus placebo in reducing irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech and social avoidance assessed by the corresponding individual subscales of the ABC-CFX after 12 weeks of treatment.

Other protocol defined secondary objectives may apply

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

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

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: For each secondary endpoint, Timeframe is 12 weeks

Secondary end point(s): - Change from baseline in behavioral symptoms of Fragile X Syndrome using the FXS specific algorithm - ABC-CFX Total score in Stratum II

- Safety and tolerability as measured by changes in vital signs, ECGs, laboratory values and percentages of adverse events and serious adverse events

- Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale

- Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-CFX scale

- The proportion of patients with clinical response, where response is defined as a reduction of at least 25% from baseline in the ABC-CFX total score and a score of 1 (very much improved) or 2 (much improved) on the CGI-I scale

Other protocol defined endpoints may apply

Secondary ID(s)

CAFQ056A2212

NCT01253629

Source(s) of Monetary Support

Novartis Pharma Services AG