Trial Outcomes & Findings for Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome (NCT NCT01348087)
NCT ID: NCT01348087
Last Updated: 2016-05-25
Results Overview
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
TERMINATED
PHASE2
148 participants
Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial
2016-05-25
Participant Flow
The study was conducted at 28 centers in 10 countries
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
Participant milestones
| Measure |
AFQ056 Total
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 Study
STARTED
|
148
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
148
|
Reasons for withdrawal
| Measure |
AFQ056 Total
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 Study
Unsatisfactory therapeutic effect
|
35
|
|
Overall Study
Administrative problems
|
79
|
|
Overall Study
protocol deviation
|
1
|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Subject withdrew consent
|
7
|
|
Overall Study
Adverse Event
|
25
|
Baseline Characteristics
Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
Baseline characteristics by cohort
| Measure |
AFQ056
n=148 Participants
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
|
|---|---|
|
Age, Continuous
|
26.6 Years
STANDARD_DEVIATION 6.85 • n=99 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
138 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trialPopulation: 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
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
Outcome measures
| Measure |
Prior to Ext First Dose
n=40 Participants
|
AFQ056 25mg Bid
n=147 Participants
|
AFQ056 50mg Bid
n=148 Participants
|
AFQ056 75mg Bid
n=141 Participants
|
AFQ056 100mg Bid
n=135 Participants
|
AFQ056 Total
n=148 Participants
|
|---|---|---|---|---|---|---|
|
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
At least one AE
|
9 Participants
|
49 Participants
|
47 Participants
|
50 Participants
|
112 Participants
|
138 Participants
|
|
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
At least one severe AE
|
1 Participants
|
1 Participants
|
2 Participants
|
5 Participants
|
18 Participants
|
24 Participants
|
|
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
Any serious or significant AE
|
0 Participants
|
1 Participants
|
0 Participants
|
1 Participants
|
6 Participants
|
7 Participants
|
|
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
SAE
|
0 Participants
|
1 Participants
|
0 Participants
|
1 Participants
|
6 Participants
|
7 Participants
|
|
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
Discontinued due to SAE
|
0 Participants
|
1 Participants
|
0 Participants
|
1 Participants
|
1 Participants
|
3 Participants
|
|
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
Discontinued due to non serious AE
|
0 Participants
|
4 Participants
|
5 Participants
|
4 Participants
|
11 Participants
|
22 Participants
|
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
Serious adverse events
| Measure |
Prior to Ext.First Dose
n=40 participants at risk
Prior to Ext.first dose
|
AFQ056 25 mg Bid
n=147 participants at risk
AFQ056 25 mg bid
|
AFQ056 50 mg Bid
n=148 participants at risk
AFQ056 50 mg bid
|
AFQ056 75 mg Bid
n=141 participants at risk
AFQ056 75 mg bid
|
AFQ056 100 mg Bid
n=135 participants at risk
AFQ056 100 mg bid
|
AFQ056 Total
n=148 participants at risk
|
|---|---|---|---|---|---|---|
|
Investigations
Hepatic enzyme increased
|
0.00%
0/40
|
0.00%
0/147
|
0.00%
0/148
|
0.00%
0/141
|
0.74%
1/135
|
0.68%
1/148
|
|
Nervous system disorders
Epilepsy
|
0.00%
0/40
|
0.00%
0/147
|
0.00%
0/148
|
0.00%
0/141
|
0.74%
1/135
|
0.68%
1/148
|
|
Psychiatric disorders
Aggression
|
0.00%
0/40
|
0.00%
0/147
|
0.00%
0/148
|
0.71%
1/141
|
1.5%
2/135
|
2.0%
3/148
|
|
Psychiatric disorders
Agitation
|
0.00%
0/40
|
0.00%
0/147
|
0.00%
0/148
|
0.00%
0/141
|
1.5%
2/135
|
1.4%
2/148
|
|
Psychiatric disorders
Hallucination, auditory
|
0.00%
0/40
|
0.00%
0/147
|
0.00%
0/148
|
0.00%
0/141
|
0.74%
1/135
|
0.68%
1/148
|
|
Psychiatric disorders
Hallucination, visual
|
0.00%
0/40
|
0.68%
1/147
|
0.00%
0/148
|
0.00%
0/141
|
0.74%
1/135
|
0.68%
1/148
|
|
Psychiatric disorders
Panic attack
|
0.00%
0/40
|
0.00%
0/147
|
0.00%
0/148
|
0.00%
0/141
|
0.74%
1/135
|
0.68%
1/148
|
Other adverse events
| Measure |
Prior to Ext.First Dose
n=40 participants at risk
Prior to Ext.first dose
|
AFQ056 25 mg Bid
n=147 participants at risk
AFQ056 25 mg bid
|
AFQ056 50 mg Bid
n=148 participants at risk
AFQ056 50 mg bid
|
AFQ056 75 mg Bid
n=141 participants at risk
AFQ056 75 mg bid
|
AFQ056 100 mg Bid
n=135 participants at risk
AFQ056 100 mg bid
|
AFQ056 Total
n=148 participants at risk
|
|---|---|---|---|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/40
|
0.68%
1/147
|
0.68%
1/148
|
0.71%
1/141
|
5.2%
7/135
|
6.8%
10/148
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/40
|
0.68%
1/147
|
1.4%
2/148
|
1.4%
2/141
|
10.4%
14/135
|
12.2%
18/148
|
|
General disorders
Asthenia
|
0.00%
0/40
|
0.68%
1/147
|
0.00%
0/148
|
3.5%
5/141
|
5.9%
8/135
|
6.1%
9/148
|
|
General disorders
Fatigue
|
0.00%
0/40
|
0.68%
1/147
|
1.4%
2/148
|
2.1%
3/141
|
2.2%
3/135
|
6.1%
9/148
|
|
Infections and infestations
Nasopharyngitis
|
7.5%
3/40
|
1.4%
2/147
|
2.7%
4/148
|
2.1%
3/141
|
15.6%
21/135
|
18.2%
27/148
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/40
|
2.7%
4/147
|
0.68%
1/148
|
2.1%
3/141
|
11.9%
16/135
|
16.2%
24/148
|
|
Nervous system disorders
Dizziness
|
2.5%
1/40
|
3.4%
5/147
|
2.7%
4/148
|
0.71%
1/141
|
3.7%
5/135
|
8.8%
13/148
|
|
Nervous system disorders
Headache
|
0.00%
0/40
|
2.7%
4/147
|
2.0%
3/148
|
5.0%
7/141
|
10.4%
14/135
|
14.2%
21/148
|
|
Psychiatric disorders
Aggression
|
0.00%
0/40
|
4.1%
6/147
|
5.4%
8/148
|
3.5%
5/141
|
8.1%
11/135
|
14.9%
22/148
|
|
Psychiatric disorders
Agitation
|
0.00%
0/40
|
0.00%
0/147
|
0.68%
1/148
|
2.1%
3/141
|
7.4%
10/135
|
8.8%
13/148
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/40
|
0.68%
1/147
|
2.0%
3/148
|
2.1%
3/141
|
7.4%
10/135
|
10.8%
16/148
|
|
Psychiatric disorders
Insomnia
|
2.5%
1/40
|
2.7%
4/147
|
2.0%
3/148
|
5.0%
7/141
|
8.9%
12/135
|
15.5%
23/148
|
|
Psychiatric disorders
Irritability
|
2.5%
1/40
|
0.00%
0/147
|
4.7%
7/148
|
2.8%
4/141
|
4.4%
6/135
|
10.1%
15/148
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
2.5%
1/40
|
2.7%
4/147
|
0.00%
0/148
|
1.4%
2/141
|
6.7%
9/135
|
10.8%
16/148
|
Additional Information
Study Director
Novartis Pharmaceuticals
Results disclosure agreements
- Principal investigator is a sponsor employee 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
- Publication restrictions are in place
Restriction type: OTHER