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Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

Primary Purpose

Fragile X Syndrome

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
AF056
Placebo
Sponsored by
Novartis
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fragile X Syndrome focused on measuring Fragile X Syndrome, adults, efficacy

Eligibility Criteria

18 Years - 35 Years (Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • Male, non-smoking patients between 18 and 35 years of age (both inclusive).
  • Patients with fmr1 full mutation (> 200 CGG repeats)
  • Patients with a Clinical Global Impression Severity Score (CGI-S) of > 4 (moderately ill)
  • Patients with a score of >20 in the ABC-C scale (at screening)
  • Patients with a mental age of ≥ 48 months as measured by the Stanford-Binet test

Exclusion Criteria:

  • Patients with DSM-IV diagnosis of schizophrenia, history and/or presence of psychosis, confusional states and/or repeated hallucinations.
  • Patients with a history of seizures in the past 5 years without any therapeutic treatment controlling the disorders.
  • Patients under stable anti-convulsant therapies that experienced seizures in the 2 years prior to randomization
  • Patients with ECG abnormalities, autonomic dysfunctions, bronchospastic diseases, drug or atopic allergy
  • Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of drugs
  • Patients using (or have used within four weeks before randomization) concomitant medications that are potent inhibitors of CYP3A4 (e.g., ketoconazole, ritonavir, etc.)

Other protocol-defined inclusion/exclusion criteria may apply.

Sites / Locations

  • Novartis Investigator Site
  • Novartis Investigator Site
  • Novartis Investigator Site

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

1

2

Arm Description

Outcomes

Primary Outcome Measures

Aberrant-Behavior Checklist- Community Edition

Secondary Outcome Measures

28 days treatment with AFQ056 on behavior (communication, socialization, daily living, repetitive behaviors, anxiety/avoidance, clinical global improvement)
28 days treatment with AFQ056 on cognition (receptive language, attention, vigilance…)

Full Information

First Posted
July 17, 2008
Last Updated
February 8, 2020
Sponsor
Novartis
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1. Study Identification

Unique Protocol Identification Number
NCT00718341
Brief Title
Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients
Official Title
A Multi-centre, Randomized, Double-blind, Placebo Controlled, Two-period, Crossover Proof-of-concept Study in Male Patients With Fragile X Syndrome to Assess the Efficacy, Safety and Tolerability of Multiple Oral Doses of AFQ056
Study Type
Interventional

2. Study Status

Record Verification Date
May 2010
Overall Recruitment Status
Completed
Study Start Date
June 2008 (undefined)
Primary Completion Date
February 2009 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Novartis

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
This study will evaluate the safety, tolerability and efficacy of multiple doses of AFQ056 in patients with Fragile X Syndrome. The dose range will be 50 to 150 mg b.i.d. The primary read-out of efficacy is reduction in Aberrant-Behavior Checklist score.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fragile X Syndrome
Keywords
Fragile X Syndrome, adults, efficacy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
30 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Active Comparator
Arm Title
2
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
AF056
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Aberrant-Behavior Checklist- Community Edition
Secondary Outcome Measure Information:
Title
28 days treatment with AFQ056 on behavior (communication, socialization, daily living, repetitive behaviors, anxiety/avoidance, clinical global improvement)
Title
28 days treatment with AFQ056 on cognition (receptive language, attention, vigilance…)

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male, non-smoking patients between 18 and 35 years of age (both inclusive). Patients with fmr1 full mutation (> 200 CGG repeats) Patients with a Clinical Global Impression Severity Score (CGI-S) of > 4 (moderately ill) Patients with a score of >20 in the ABC-C scale (at screening) Patients with a mental age of ≥ 48 months as measured by the Stanford-Binet test Exclusion Criteria: Patients with DSM-IV diagnosis of schizophrenia, history and/or presence of psychosis, confusional states and/or repeated hallucinations. Patients with a history of seizures in the past 5 years without any therapeutic treatment controlling the disorders. Patients under stable anti-convulsant therapies that experienced seizures in the 2 years prior to randomization Patients with ECG abnormalities, autonomic dysfunctions, bronchospastic diseases, drug or atopic allergy Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of drugs Patients using (or have used within four weeks before randomization) concomitant medications that are potent inhibitors of CYP3A4 (e.g., ketoconazole, ritonavir, etc.) Other protocol-defined inclusion/exclusion criteria may apply.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis
Organizational Affiliation
Novartis investigator site
Official's Role
Principal Investigator
Facility Information:
Facility Name
Novartis Investigator Site
City
Bron cedex
ZIP/Postal Code
69677
Country
France
Facility Name
Novartis Investigator Site
City
Rome
ZIP/Postal Code
00168
Country
Italy
Facility Name
Novartis Investigator Site
City
Lausanne
ZIP/Postal Code
1011
Country
Switzerland

12. IPD Sharing Statement

Citations:
PubMed Identifier
21209411
Citation
Jacquemont S, Curie A, des Portes V, Torrioli MG, Berry-Kravis E, Hagerman RJ, Ramos FJ, Cornish K, He Y, Paulding C, Neri G, Chen F, Hadjikhani N, Martinet D, Meyer J, Beckmann JS, Delange K, Brun A, Bussy G, Gasparini F, Hilse T, Floesser A, Branson J, Bilbe G, Johns D, Gomez-Mancilla B. Epigenetic modification of the FMR1 gene in fragile X syndrome is associated with differential response to the mGluR5 antagonist AFQ056. Sci Transl Med. 2011 Jan 5;3(64):64ra1. doi: 10.1126/scitranslmed.3001708.
Results Reference
derived

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Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

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