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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

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

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

NCT ID

NCT00718341

First Posted

July 17, 2008

Last Updated

February 8, 2020

Sponsor

Novartis

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

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

Arm Type

Active Comparator

Arm Title

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

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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