Trial · NCT01348087
Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
NCT01348087, Phase 2, Terminated, Novartis Pharmaceuticals; results first posted 2016-05-25
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome. Trials, record NCT01348087. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01348087
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
- NCT number
- NCT01348087
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 148 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Fragile X Syndrome
- Interventions
- AFQ056
- First posted
- 2011-05-05
- Last updated
- 2016-05-25
- Other registries' IDs
- eudract:2011-001952-12
- Results first posted
- 2016-05-25
- Limitations and caveats
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).
Source ClinicalTrials.gov
Registered outcomes1
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). | Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial | 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 |
Source ClinicalTrials.gov
Outcomes1
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). | Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial | 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 | Participants | NUMBER |
Source ClinicalTrials.gov
Results by group36
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). | Prior to Ext First Dose | At least one AE | 9 | Participants | |
| Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). | AFQ056 25mg Bid | At least one AE | 49 | Participants | |
| Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). | AFQ056 50mg Bid | At least one AE | 47 | Participants | |
| Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). | AFQ056 75mg Bid | At least one AE | 50 | Participants | |
| Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). | AFQ056 100mg Bid | At least one AE | 112 | Participants | |
| Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). | AFQ056 Total | At least one AE | 138 | Participants | |
| Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). | Prior to Ext First Dose | At least one severe AE | 1 | Participants | |
| Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). | AFQ056 25mg Bid | At least one severe AE | 1 | Participants | |
| Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). | AFQ056 50mg Bid | At least one severe AE | 2 | Participants | |
| Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). | AFQ056 75mg Bid | At least one severe AE | 5 | Participants |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | AFQ056 Total | 148 | |
| Overall Study | COMPLETED | AFQ056 Total | 0 | |
| Overall Study | NOT COMPLETED | AFQ056 Total | 148 |
Source ClinicalTrials.gov
Adverse events126
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | Prior to Ext.First Dose | 0 | 40 | |
| other | Diarrhoea | Gastrointestinal disorders | AFQ056 25 mg Bid | 1 | 147 | |
| other | Diarrhoea | Gastrointestinal disorders | AFQ056 50 mg Bid | 1 | 148 | |
| other | Diarrhoea | Gastrointestinal disorders | AFQ056 75 mg Bid | 1 | 141 | |
| other | Diarrhoea | Gastrointestinal disorders | AFQ056 100 mg Bid | 7 | 135 | |
| other | Diarrhoea | Gastrointestinal disorders | AFQ056 Total | 10 | 148 | |
| other | Vomiting | Gastrointestinal disorders | Prior to Ext.First Dose | 0 | 40 | |
| other | Vomiting | Gastrointestinal disorders | AFQ056 25 mg Bid | 1 | 147 | |
| other | Vomiting | Gastrointestinal disorders | AFQ056 50 mg Bid | 2 | 148 | |
| other | Vomiting | Gastrointestinal disorders | AFQ056 75 mg Bid | 2 | 141 |
1 to 10 of 126Source ClinicalTrials.gov
See all 126 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Prior to Ext.First Dose | ||
| Total, all-cause mortality | AFQ056 25 mg Bid | ||
| Total, all-cause mortality | AFQ056 50 mg Bid | ||
| Total, all-cause mortality | AFQ056 75 mg Bid | ||
| Total, all-cause mortality | AFQ056 100 mg Bid | ||
| Total, all-cause mortality | AFQ056 Total | ||
| Total, other adverse events | Prior to Ext.First Dose | 6 | 40 |
| Total, other adverse events | AFQ056 25 mg Bid | 26 | 147 |
| Total, other adverse events | AFQ056 50 mg Bid | 28 | 148 |
| Total, other adverse events | AFQ056 75 mg Bid | 36 | 141 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01348087
GET https://conductscience.org/api/trials/v1/NCT01348087/results?page=2