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Trial record 18 of 74 for:    "fragile x syndrome"

Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01348087
Recruitment Status : Terminated (Study treatment AFQ056 failed to demonstrate efficacy in the adult patient with Fragile X Syndrome in 2 other clinical studies (CAFQ056B2214 and CAFQ056A2212))
First Posted : May 5, 2011
Results First Posted : May 25, 2016
Last Update Posted : May 25, 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 148
Recruitment Details The study was conducted at 28 centers in 10 countries
Pre-assignment Details A total of 148 patients were enrolled and treated, including 1 patient who discontinued and was later re-enrolled under a new patient number. Category 1 patients received AFQ056 in the core study and enrolled in the extension within 7 days of completing the core study; Category 2 included all other patients enrolled into the extension study
Arm/Group Title AFQ056 Total
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 148
Completed 0
Not Completed 148
Reason Not Completed
Unsatisfactory therapeutic effect             35
Administrative problems             79
protocol deviation             1
Lost to Follow-up             1
Subject withdrew consent             7
Adverse Event             25
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 148
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 148 participants
26.6  (6.85)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 148 participants
Female
10
   6.8%
Male
138
  93.2%
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 patients 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 patients 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 Prior to Ext First Dose AFQ056 25mg Bid AFQ056 50mg Bid AFQ056 75mg Bid AFQ056 100mg Bid AFQ056 Total
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 40 147 148 141 135 148
Measure Type: Number
Unit of Measure: Participants
At least one AE 9 49 47 50 112 138
At least one severe AE 1 1 2 5 18 24
Any serious or significant AE 0 1 0 1 6 7
SAE 0 1 0 1 6 7
Discontinued due to SAE 0 1 0 1 1 3
Discontinued due to non serious AE 0 4 5 4 11 22
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 AFQ056 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 [Not Specified]
All-Cause Mortality
Prior to Ext.First Dose AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 75 mg Bid AFQ056 100 mg Bid AFQ056 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 AFQ056 Total
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/40 (0.00%)   1/147 (0.68%)   0/148 (0.00%)   1/141 (0.71%)   6/135 (4.44%)   7/148 (4.73%) 
Investigations             
Hepatic enzyme increased  1  0/40 (0.00%)  0/147 (0.00%)  0/148 (0.00%)  0/141 (0.00%)  1/135 (0.74%)  1/148 (0.68%) 
Nervous system disorders             
Epilepsy  1  0/40 (0.00%)  0/147 (0.00%)  0/148 (0.00%)  0/141 (0.00%)  1/135 (0.74%)  1/148 (0.68%) 
Psychiatric disorders             
Aggression  1  0/40 (0.00%)  0/147 (0.00%)  0/148 (0.00%)  1/141 (0.71%)  2/135 (1.48%)  3/148 (2.03%) 
Agitation  1  0/40 (0.00%)  0/147 (0.00%)  0/148 (0.00%)  0/141 (0.00%)  2/135 (1.48%)  2/148 (1.35%) 
Hallucination, auditory  1  0/40 (0.00%)  0/147 (0.00%)  0/148 (0.00%)  0/141 (0.00%)  1/135 (0.74%)  1/148 (0.68%) 
Hallucination, visual  1  0/40 (0.00%)  1/147 (0.68%)  0/148 (0.00%)  0/141 (0.00%)  1/135 (0.74%)  1/148 (0.68%) 
Panic attack  1  0/40 (0.00%)  0/147 (0.00%)  0/148 (0.00%)  0/141 (0.00%)  1/135 (0.74%)  1/148 (0.68%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 17.1
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 AFQ056 Total
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   6/40 (15.00%)   26/147 (17.69%)   28/148 (18.92%)   36/141 (25.53%)   88/135 (65.19%)   117/148 (79.05%) 
Gastrointestinal disorders             
Diarrhoea  1  0/40 (0.00%)  1/147 (0.68%)  1/148 (0.68%)  1/141 (0.71%)  7/135 (5.19%)  10/148 (6.76%) 
Vomiting  1  0/40 (0.00%)  1/147 (0.68%)  2/148 (1.35%)  2/141 (1.42%)  14/135 (10.37%)  18/148 (12.16%) 
General disorders             
Asthenia  1  0/40 (0.00%)  1/147 (0.68%)  0/148 (0.00%)  5/141 (3.55%)  8/135 (5.93%)  9/148 (6.08%) 
Fatigue  1  0/40 (0.00%)  1/147 (0.68%)  2/148 (1.35%)  3/141 (2.13%)  3/135 (2.22%)  9/148 (6.08%) 
Infections and infestations             
Nasopharyngitis  1  3/40 (7.50%)  2/147 (1.36%)  4/148 (2.70%)  3/141 (2.13%)  21/135 (15.56%)  27/148 (18.24%) 
Upper respiratory tract infection  1  0/40 (0.00%)  4/147 (2.72%)  1/148 (0.68%)  3/141 (2.13%)  16/135 (11.85%)  24/148 (16.22%) 
Nervous system disorders             
Dizziness  1  1/40 (2.50%)  5/147 (3.40%)  4/148 (2.70%)  1/141 (0.71%)  5/135 (3.70%)  13/148 (8.78%) 
Headache  1  0/40 (0.00%)  4/147 (2.72%)  3/148 (2.03%)  7/141 (4.96%)  14/135 (10.37%)  21/148 (14.19%) 
Psychiatric disorders             
Aggression  1  0/40 (0.00%)  6/147 (4.08%)  8/148 (5.41%)  5/141 (3.55%)  11/135 (8.15%)  22/148 (14.86%) 
Agitation  1  0/40 (0.00%)  0/147 (0.00%)  1/148 (0.68%)  3/141 (2.13%)  10/135 (7.41%)  13/148 (8.78%) 
Anxiety  1  0/40 (0.00%)  1/147 (0.68%)  3/148 (2.03%)  3/141 (2.13%)  10/135 (7.41%)  16/148 (10.81%) 
Insomnia  1  1/40 (2.50%)  4/147 (2.72%)  3/148 (2.03%)  7/141 (4.96%)  12/135 (8.89%)  23/148 (15.54%) 
Irritability  1  1/40 (2.50%)  0/147 (0.00%)  7/148 (4.73%)  4/141 (2.84%)  6/135 (4.44%)  15/148 (10.14%) 
Respiratory, thoracic and mediastinal disorders             
Cough  1  1/40 (2.50%)  4/147 (2.72%)  0/148 (0.00%)  2/141 (1.42%)  9/135 (6.67%)  16/148 (10.81%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 17.1
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 or disclosure of trial results in their entirety
Results Point of Contact
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: 862-778-8300
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01348087     History of Changes
Other Study ID Numbers: CAFQ056B2279
2011-001952-12 ( EudraCT Number )
First Submitted: May 3, 2011
First Posted: May 5, 2011
Results First Submitted: September 9, 2015
Results First Posted: May 25, 2016
Last Update Posted: May 25, 2016