NCT00718341

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.

Trial Health

85
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Geographic Reach
3 countries

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2008

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

July 17, 2008

Completed
1 day until next milestone

First Posted

Study publicly available on registry

July 18, 2008

Completed
7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2009

Completed
Last Updated

February 11, 2020

Status Verified

May 1, 2010

Enrollment Period

8 months

First QC Date

July 17, 2008

Last Update Submit

February 8, 2020

Conditions

Fragile X Syndrome

Keywords

Fragile X Syndrome, adults, efficacy

Outcome Measures

Primary Outcomes (1)

  • Aberrant-Behavior Checklist- Community Edition

Secondary Outcomes (2)

  • 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…)

Study Arms (2)

1

ACTIVE COMPARATOR
Drug: AF056

2

PLACEBO COMPARATOR
Drug: Placebo

Interventions

AF056DRUG
1
PlaceboDRUG
2

Eligibility Criteria

Age18 Years - 35 Years
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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.)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Novartis Investigator Site

Bron, 69677, France

Location

Novartis Investigator Site

Rome, 00168, Italy

Location

Novartis Investigator Site

Lausanne, 1011, Switzerland

Location

Related Publications (1)

  • 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.

    PMID: 21209411DERIVED

MeSH Terms

Conditions

Fragile X Syndrome

Condition Hierarchy (Ancestors)

X-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesSex Chromosome DisordersChromosome DisordersCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, InbornGenetic Diseases, X-LinkedHeredodegenerative Disorders, Nervous System

Study Officials

  • Novartis

    Novartis investigator site

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

July 17, 2008

First Posted

July 18, 2008

Study Start

June 1, 2008

Primary Completion

February 1, 2009

Last Updated

February 11, 2020

Record last verified: 2010-05

Locations

FR(1)CH(1)IT(1)