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Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

This study has been terminated.
(The study treatment failed to demonstrate efficacy in target population in two other clinical studies (CAFQ056B2214 and CAFQ056A2212).)
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01433354
First received: August 31, 2011
Last updated: February 24, 2016
Last verified: February 2016
Results First Received: September 14, 2015  
Study Type: Interventional
Study Design: Endpoint Classification: Safety Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Fragile X Syndrome
Intervention: Drug: AFQ056

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
The study was conducted at 28 centres in 13 countries.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
A total of 120 patients were enrolled, of which 119 received the study medication. Category 1 patients received AFQ056 in the core study and enrolled in the extension within 7 days of the core study; Category 2 included all other patients who were enrolled into the extension study

Reporting Groups
  Description
AFQ056 Participants from a previous AFQ056 study who entered the open-label extension study were administered AFQ056 capsules at a starting dose of 25 milligram (mg) twice daily (bid) and then titrated to 50 mg bid, 75 mg bid and 100 mg bid at weekly intervals.

Participant Flow:   Overall Study
    AFQ056  
STARTED     119 [1]
COMPLETED     0  
NOT COMPLETED     119  
Adverse Event                 6  
Lost to Follow-up                 1  
Administrative problems                 90  
Protocol Violation                 2  
Lack of Efficacy                 17  
Subject Withdrew Consent                 3  
[1] 120 enrolled of which 119 were administered drug



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
All enrolled participants who received at least one dose of the study drug (AFQ056) during the study.

Reporting Groups
  Description
AFQ056 Participants from a previous AFQ056 study who entered the open-label extension study were administered AFQ056 capsules at a starting dose of 25 milligram (mg) twice daily (bid) and then titrated to 50 mg bid, 75 mg bid and 100 mg bid at weekly intervals.

Baseline Measures
    AFQ056  
Number of Participants  
[units: participants]
  119  
Age  
[units: years]
Mean (Standard Deviation)
  15.2  (1.75)  
Gender  
[units: participants]
 
Female     13  
Male     106  



  Outcome Measures

1.  Primary:   Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)   [ Time Frame: Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
The sponsor decided to terminate this study prematurely, as the study treatment failed to demonstrate efficacy in target population in two other clinical studies: CAFQ056B2214 (NCT01357239) and CAFQ056A2212 (NCT01253629).


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
phone: 862­-778­-8300



Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01433354     History of Changes
Other Study ID Numbers: CAFQ056B2278
2011-002379-40 ( EudraCT Number )
Study First Received: August 31, 2011
Results First Received: September 14, 2015
Last Updated: February 24, 2016
Health Authority: United States: Food and Drug Administration
Denmark: Danish Medicines Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Drugs and Medical Devices
Italy: Ethics Committee
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Australia: National Health and Medical Research Council
Switzerland: Swissmedic
Sweden: Medical Products Agency
Canada: Health Canada
Israel: Ministry of Health
Netherlands: Medicines Evaluation Board (MEB)
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Turkey: Ministry of Health
Belgium: Federal Agency for Medicinal Products and Health Products