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

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ClinicalTrials.gov Identifier: NCT01433354
Recruitment Status : Terminated (The study treatment failed to demonstrate efficacy in target population in two other clinical studies (CAFQ056B2214 and CAFQ056A2212).)
First Posted : September 13, 2011
Results First Posted : October 12, 2015
Last Update Posted : March 24, 2016
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fragile X Syndrome
Intervention Drug: AFQ056
Enrollment 119
Recruitment Details The study was conducted at 28 centres in 13 countries.
Pre-assignment Details 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
Arm/Group Title AFQ056
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 119 [1]
Completed 0
Not Completed 119
Reason Not Completed
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
Arm/Group Title AFQ056
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 119
Hide Baseline Analysis Population Description
All enrolled participants who received at least one dose of the study drug (AFQ056) during the study.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 119 participants
15.2  (1.75)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 119 participants
Female
13
  10.9%
Male
106
  89.1%
1.Primary Outcome
Title Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Hide Description

Adverse events were summarized for the open-label treatment period, where the open-label treatment period is defined based on how AEs were collected and reported according to the manner in which participants entered the current study and which treatment (AFQ056 or placebo) they were receiving in the previous study.

AEs which were continuing from the core study or that started after the end of core study but prior to first dose of open-label study medication in the extension study for Category 1 participants are shown under ‘Prior to Ext. first dose’.

AEs which started during the open-label treatment period are presented based on the last AFQ056 dose taken on or before the onset date of the AE (25 mg bid; 50 mg bid; 75 mg bid; or 100 mg bid). No efficacy data presented as study was terminated.

Time Frame Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis was performed in the safety set (SS) population, defined as participants who received at least one dose of study medication and had at least one safety assessment occurring after first dose of extension study medication. Here, ‘Number of Participants Analyzed’ signifies those participants who were evaluable for this outcome measure.
Arm/Group Title AFQ056 Prior to Ext. First Dose AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 75 mg Bid AFQ056 100 mg Bid
Hide Arm/Group Description:
Total
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 119 31 119 118 116 108
Measure Type: Number
Unit of Measure: participants
At least one AE 110 10 36 41 44 90
At least one severe AE 8 0 1 1 1 5
Any serious or significant AE 4 0 0 1 0 3
SAEs 4 0 0 1 0 3
Discontinued due to AEs 6 1 2 2 0 3
Discontinued due to SAEs 1 0 0 0 0 1
Discontinued due to non serious AE 5 1 2 2 0 2
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Prior to Ext.First Dose AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 75 mg Bid AFQ056 100 mg Bid Total
Hide Arm/Group Description Prior to Ext.first dose AFQ056 25 mg bid AFQ056 50 mg bid AFQ056 75 mg bid AFQ056 100 mg bid Total
All-Cause Mortality
Prior to Ext.First Dose AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 75 mg Bid AFQ056 100 mg Bid Total
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Prior to Ext.First Dose AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 75 mg Bid AFQ056 100 mg Bid Total
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/31 (0.00%)   0/119 (0.00%)   1/118 (0.85%)   0/116 (0.00%)   3/108 (2.78%)   4/119 (3.36%) 
Infections and infestations             
Appendicitis  1  0/31 (0.00%)  0/119 (0.00%)  0/118 (0.00%)  0/116 (0.00%)  1/108 (0.93%)  1/119 (0.84%) 
Respiratory tract infection  1  0/31 (0.00%)  0/119 (0.00%)  0/118 (0.00%)  0/116 (0.00%)  1/108 (0.93%)  1/119 (0.84%) 
Injury, poisoning and procedural complications             
Foreign body  1  0/31 (0.00%)  0/119 (0.00%)  1/118 (0.85%)  0/116 (0.00%)  0/108 (0.00%)  1/119 (0.84%) 
Joint dislocation  1  0/31 (0.00%)  0/119 (0.00%)  0/118 (0.00%)  0/116 (0.00%)  1/108 (0.93%)  1/119 (0.84%) 
Psychiatric disorders             
Aggression  1  0/31 (0.00%)  0/119 (0.00%)  0/118 (0.00%)  0/116 (0.00%)  1/108 (0.93%)  1/119 (0.84%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 17
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Prior to Ext.First Dose AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 75 mg Bid AFQ056 100 mg Bid Total
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   6/31 (19.35%)   23/119 (19.33%)   30/118 (25.42%)   33/116 (28.45%)   74/108 (68.52%)   103/119 (86.55%) 
Gastrointestinal disorders             
Diarrhoea  1  1/31 (3.23%)  2/119 (1.68%)  4/118 (3.39%)  0/116 (0.00%)  5/108 (4.63%)  12/119 (10.08%) 
Vomiting  1  1/31 (3.23%)  2/119 (1.68%)  5/118 (4.24%)  0/116 (0.00%)  8/108 (7.41%)  13/119 (10.92%) 
General disorders             
Fatigue  1  0/31 (0.00%)  2/119 (1.68%)  2/118 (1.69%)  3/116 (2.59%)  5/108 (4.63%)  11/119 (9.24%) 
Pyrexia  1  0/31 (0.00%)  0/119 (0.00%)  1/118 (0.85%)  2/116 (1.72%)  4/108 (3.70%)  7/119 (5.88%) 
Infections and infestations             
Ear infection  1  0/31 (0.00%)  2/119 (1.68%)  0/118 (0.00%)  1/116 (0.86%)  5/108 (4.63%)  8/119 (6.72%) 
Gastroenteritis viral  1  0/31 (0.00%)  0/119 (0.00%)  2/118 (1.69%)  1/116 (0.86%)  5/108 (4.63%)  8/119 (6.72%) 
Influenza  1  0/31 (0.00%)  1/119 (0.84%)  0/118 (0.00%)  2/116 (1.72%)  6/108 (5.56%)  8/119 (6.72%) 
Nasopharyngitis  1  0/31 (0.00%)  7/119 (5.88%)  3/118 (2.54%)  8/116 (6.90%)  25/108 (23.15%)  35/119 (29.41%) 
Upper respiratory tract infection  1  1/31 (3.23%)  3/119 (2.52%)  5/118 (4.24%)  3/116 (2.59%)  9/108 (8.33%)  17/119 (14.29%) 
Investigations             
Weight increased  1  1/31 (3.23%)  1/119 (0.84%)  0/118 (0.00%)  0/116 (0.00%)  5/108 (4.63%)  7/119 (5.88%) 
Metabolism and nutrition disorders             
Decreased appetite  1  2/31 (6.45%)  0/119 (0.00%)  2/118 (1.69%)  1/116 (0.86%)  0/108 (0.00%)  5/119 (4.20%) 
Nervous system disorders             
Headache  1  0/31 (0.00%)  4/119 (3.36%)  1/118 (0.85%)  4/116 (3.45%)  8/108 (7.41%)  15/119 (12.61%) 
Psychomotor hyperactivity  1  0/31 (0.00%)  2/119 (1.68%)  3/118 (2.54%)  1/116 (0.86%)  3/108 (2.78%)  9/119 (7.56%) 
Psychiatric disorders             
Aggression  1  0/31 (0.00%)  3/119 (2.52%)  3/118 (2.54%)  4/116 (3.45%)  12/108 (11.11%)  19/119 (15.97%) 
Agitation  1  0/31 (0.00%)  1/119 (0.84%)  1/118 (0.85%)  0/116 (0.00%)  4/108 (3.70%)  6/119 (5.04%) 
Anxiety  1  0/31 (0.00%)  3/119 (2.52%)  1/118 (0.85%)  0/116 (0.00%)  11/108 (10.19%)  15/119 (12.61%) 
Initial insomnia  1  0/31 (0.00%)  3/119 (2.52%)  2/118 (1.69%)  4/116 (3.45%)  9/108 (8.33%)  18/119 (15.13%) 
Insomnia  1  1/31 (3.23%)  4/119 (3.36%)  10/118 (8.47%)  5/116 (4.31%)  12/108 (11.11%)  25/119 (21.01%) 
Irritability  1  0/31 (0.00%)  3/119 (2.52%)  1/118 (0.85%)  3/116 (2.59%)  9/108 (8.33%)  15/119 (12.61%) 
Respiratory, thoracic and mediastinal disorders             
Cough  1  0/31 (0.00%)  1/119 (0.84%)  1/118 (0.85%)  4/116 (3.45%)  7/108 (6.48%)  12/119 (10.08%) 
Epistaxis  1  0/31 (0.00%)  0/119 (0.00%)  2/118 (1.69%)  2/116 (1.72%)  3/108 (2.78%)  6/119 (5.04%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 17
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).
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 terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single­ site are postponed until the publication of the pooled data (i.e, data from all sites) in the clinical trial.
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 )
First Submitted: August 31, 2011
First Posted: September 13, 2011
Results First Submitted: September 14, 2015
Results First Posted: October 12, 2015
Last Update Posted: March 24, 2016