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

NCT ID: NCT01253629

Last Updated: 2020-12-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

175 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-11-30

Study Completion Date

2013-08-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective in treating the behavioral symptoms of Fragile X Syndrome.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Fragile X Syndrome

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Fragile X Syndrome Martin-Bell Syndrome Genetic Diseases X-Linked

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

25 mg bid AFQ056

1 capsule of 25 mg and 1 capsule of placebo per intake

Group Type EXPERIMENTAL

AFQ056

Intervention Type DRUG

AFQ056, was provided as hard gelatin capsules, 25mg and 100 mg oral dosage strengths, identical in appearance were used

50 mg bid AFQ056

2 capsules of 25 mg per intake

Group Type EXPERIMENTAL

AFQ056

Intervention Type DRUG

AFQ056, was provided as hard gelatin capsules, 25mg and 100 mg oral dosage strengths, identical in appearance were used

100 mg bid AFQ056

1 capsule of 100 mg and 1 capsule of placebo per intake

Group Type EXPERIMENTAL

AFQ056

Intervention Type DRUG

AFQ056, was provided as hard gelatin capsules, 25mg and 100 mg oral dosage strengths, identical in appearance were used

Placebo

2 capsules of placebo per intake

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo medication identical in appearance to active medication was provided

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

AFQ056

AFQ056, was provided as hard gelatin capsules, 25mg and 100 mg oral dosage strengths, identical in appearance were used

Intervention Type DRUG

Placebo

Placebo medication identical in appearance to active medication was provided

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

45 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Novartis Investigative Site

Phoenix, Arizona, United States

Site Status

Novartis Investigative Site

Sacramento, California, United States

Site Status

Novartis Investigative Site

Decatur, Georgia, United States

Site Status

Novartis Investigative Site

Chicago, Illinois, United States

Site Status

Novartis Investigative Site

Indianapolis, Indiana, United States

Site Status

Novartis Investigative Site

Boston, Massachusetts, United States

Site Status

Novartis Investigative Site

Omaha, Nebraska, United States

Site Status

Novartis Investigative Site

Staten Island, New York, United States

Site Status

Novartis Investigative Site

Media, Pennsylvania, United States

Site Status

Novartis Investigative Site

Greenwood, South Carolina, United States

Site Status

Novartis Investigative Site

Nashville, Tennessee, United States

Site Status

Novartis Investigative Site

Houston, Texas, United States

Site Status

Novartis Investigative Site

Ryde, New South Wales, Australia

Site Status

Novartis Investigative Site

Waratah, New South Wales, Australia

Site Status

Novartis Investigative Site

Caulfield, Victoria, Australia

Site Status

Novartis Investigative Site

Brampton, Ontario, Canada

Site Status

Novartis Investigative Site

Sherbrooke, Quebec, Canada

Site Status

Novartis Investigative Site

Glostrup Municipality, , Denmark

Site Status

Novartis Investigative Site

Bron, , France

Site Status

Novartis Investigative Site

Paris, , France

Site Status

Novartis Investigative Site

Berlin, , Germany

Site Status

Novartis Investigative Site

Mainz, , Germany

Site Status

Novartis Investigative Site

Tübingen, , Germany

Site Status

Novartis Investigative Site

Würzburg, , Germany

Site Status

Novartis Investigative Site

Genova, GE, Italy

Site Status

Novartis Investigative Site

Roma, RM, Italy

Site Status

Novartis Investigative Site

Málaga, Andalusia, Spain

Site Status

Novartis Investigative Site

Sant Cugat del Vallès, Catalonia, Spain

Site Status

Novartis Investigative Site

Lausanne, , Switzerland

Site Status

Novartis Investigative Site

Zurich, , Switzerland

Site Status

Novartis Investigative Site

Edinburgh, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

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

References

Explore related publications, articles, or registry entries linked to this study.

Berry-Kravis E, Des Portes V, Hagerman R, Jacquemont S, Charles P, Visootsak J, Brinkman M, Rerat K, Koumaras B, Zhu L, Barth GM, Jaecklin T, Apostol G, von Raison F. Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. Sci Transl Med. 2016 Jan 13;8(321):321ra5. doi: 10.1126/scitranslmed.aab4109.

Reference Type DERIVED
PMID: 26764156 (View on PubMed)

Related Links

Access external resources that provide additional context or updates about the study.

https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=12423

Results for CAFQ056A2212 from the Novartis Clinical Trial website

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2009-013667-19

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CAFQ056A2212

Identifier Type: -

Identifier Source: org_study_id