Trial · NCT01357239

Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

NCT01357239, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2015-05-12

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome. Trials, record NCT01357239. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01357239

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome
NCT number: NCT01357239
Status: Completed
Phases: Phase 2
Enrollment: 139 participants, actual
Lead sponsor: Novartis Pharmaceuticals
Conditions: Fragile X Syndrome
Interventions: AFQ056, Placebo
First posted: 2011-05-20
Last updated: 2015-05-12
Other registries' IDs: eudract:2010-022638-96
Results first posted: 2015-05-12

Source ClinicalTrials.gov

Registered outcomes13

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the Aberrant Behavior Checklist-Community Edition (ABC-CFX) Total Score in Stratum I Patients Exposed to AFQ056 100 mg Bid	Baseline to week 12	The Aberrant Behavior Checklist-Community edition (ABC-C) is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 ("not at all a problem") to 3 ("problem is severe in degree") and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to the modified FXS ABC-C algorithm (ABC-CFX) for which 55 items and six subscales (irritability, lethargy/withdrawal, stereotypic behavior, hyperactivity, inappropriate speech and social avoidance) plus the total score were considered, and for which the total score ranks from 0 to 165. Stratum I included patients whose Fragile X Mental Retardation 1 (FMR1) gene was fully methylated
Secondary	Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum II Patients Exposed to All 3 Doses of AFQ056	Baseline to week 12	The ABC-C is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 ("not at all a problem") to 3 ("problem is severe in degree") and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to the modified FXS ABC-C algorithm (ABC-CFX) for which 55 items and six subscales (irritability, lethargy/withdrawal, stereotypic behavior, hyperactivity, inappropriate speech and social avoidance) plus the total score were considered, and for which the total score ranks from 0 to 165. Stratum II included patients whose FMR1 gene was partially methylated
Secondary	Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum I Patients Exposed to the Two Lower Doses of AFQ056 (25 mg Bid and 50 mg Bid)	Baseline to week 12	The ABC-C is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 ("not at all a problem") to 3 ("problem is severe in degree") and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to the modified FXS ABC-C algorithm (ABC-CFX) for which 55 items and six subscales (irritability, lethargy/withdrawal, stereotypic behavior, hyperactivity, inappropriate speech and social avoidance) plus the total score were considered, and for which the total score ranks from 0 to 165. Stratum I included patients whose FMR1 gene was fully methylated
Secondary	Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum I Patients	12 weeks	The Clinical Global Impression-Improvement (CGI-I) scale is a clinician rated scale used to assess treatment response in psychiatric patients, and the score ranges from 1 to 7 (with 1 being "very much improved", 4 being "no change" to 7 being "very much worse"). Stratum I included patients whose FMR1 gene was fully methylated.
Secondary	Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum II Patients	12 weeks	The Clinical Global Impression-Improvement (CGI-I) scale is a clinician rated scale used to assess treatment response in psychiatric patients, and the score ranges from 1 to 7 (with 1 being "very much improved", 4 being "no change" to 7 being "very much worse"). Stratum II included patients whose FMR1 gene was partially methylated
Secondary	Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression-Improvement (CGI-I) Scale in Stratum I	12 weeks	The Clinical Global Impression-Improvement (CGI-I) scale is a clinician rated scale used to assess treatment response in psychiatric patients, and the score ranges from 1 to 7 (with 1 being "very much improved", 4 being "no change" to 7 being "very much worse"). Stratum I included patients whose FMR1 gene was fully methylated.
Secondary	Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression-Improvement (CGI-I) Scale in Stratum II	12 weeks	The Clinical Global Impression-Improvement (CGI-I) scale is a clinician rated scale used to assess treatment response in psychiatric patients, and the score ranges from 1 to 7 (with 1 being "very much improved", 4 being "no change" to 7 being "very much worse"). Stratum II included patients whose FMR1 gene was partially methylated
Secondary	Change From Baseline in Irritability, Lethargy/Withdrawal, Stereotypic Behavior, Hyperactivity, Inappropriate Speech and Social Avoidance Assessed by the Individual Subscales of the ABC-CFX Scale in Stratum I	Baseline to week 12	The ABC-C is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 ("not at all a problem") to 3 ("problem is severe in degree") and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to the modified FXS ABC-C algorithm (ABC-CFX) for which 55 items and six subscales: irritability (range 0 to 54); lethargy/withdrawal (range 0 to 39); stereotypic behavior (range 0 to 18); hyperactivity (range 0 to 30); inappropriate speech (range 0 to 12); and social avoidance (range 0 to 12) plus the total score (range 0 to 165) were considered. A negative change represents improvement. Stratum I included patients whose FMR1 gene was fully methylated
Secondary	Change From Baseline in Irritability, Lethargy/Withdrawal, Stereotypic Behavior, Hyperactivity, Inappropriate Speech, and Social Avoidance Assessed by the Individual Subscales of the ABC-CFX Scale in Stratum II	Baseline to week 12	The ABC-C is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 ("not at all a problem") to 3 ("problem is severe in degree") and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to the modified FXS ABC-C algorithm (ABC-CFX) for which 55 items and six subscales: irritability (range 0 to 54); lethargy/withdrawal (range 0 to 39); stereotypic behavior (range 0 to 18); hyperactivity (range 0 to 30); inappropriate speech (range 0 to 12); and social avoidance (range 0 to 12) plus the total score (range 0 to 165) were considered. A negative change represents improvement. Stratum II included patients whose FMR1 gene was partially methylated
Secondary	Proportion of Patients With Clinical Response, Where Response is Defined as a Reduction of at Least 25% From Baseline in the ABC-CFX Total Score and a Score of 1 (Very Much Improved) or 2 (Much Improved) on the CGI-I Scale, Stratum I	12 weeks	The ABC-C is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 ("not at all a problem") to 3 ("problem is severe in degree") and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to ABC-CFX algorithm, for which 55 items and six subscales plus the total score were considered, and for which the total score ranks from 0 to 165. The Clinical Global Impression-Improvement (CGI-I) scale is a clinician rated scale used to assess treatment response in psychiatric patients, and the score ranges from 1 to 7 (with 1 being "very much improved", 4 being "no change" to 7 being "very much worse"). Stratum I included patients whose FMR1 gene was fully methylated; Stratum II included patients whose FMR1 gene was partially methylated.

1 to 10 of 13Source ClinicalTrials.gov

See all 13 registered outcomes

Outcomes13

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the Aberrant Behavior Checklist-Community Edition (ABC-CFX) Total Score in Stratum I Patients Exposed to AFQ056 100 mg Bid	Baseline to week 12	Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included	Score	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum II Patients Exposed to All 3 Doses of AFQ056	Baseline to week 12	Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included	Score	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum I Patients Exposed to the Two Lower Doses of AFQ056 (25 mg Bid and 50 mg Bid)	Baseline to week 12	Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included	Score	LEAST_SQUARES_MEAN
SECONDARY	Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum I Patients	12 weeks	Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included	Score	LEAST_SQUARES_MEAN
SECONDARY	Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum II Patients	12 weeks	Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included	Score	LEAST_SQUARES_MEAN
SECONDARY	Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression-Improvement (CGI-I) Scale in Stratum I	12 weeks	Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included	Participants	NUMBER
SECONDARY	Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression-Improvement (CGI-I) Scale in Stratum II	12 weeks	Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included	Participants	NUMBER
SECONDARY	Change From Baseline in Irritability, Lethargy/Withdrawal, Stereotypic Behavior, Hyperactivity, Inappropriate Speech and Social Avoidance Assessed by the Individual Subscales of the ABC-CFX Scale in Stratum I	Baseline to week 12	Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included	Score	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Irritability, Lethargy/Withdrawal, Stereotypic Behavior, Hyperactivity, Inappropriate Speech, and Social Avoidance Assessed by the Individual Subscales of the ABC-CFX Scale in Stratum II	Baseline to week 12	Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included	Score	LEAST_SQUARES_MEAN
SECONDARY	Proportion of Patients With Clinical Response, Where Response is Defined as a Reduction of at Least 25% From Baseline in the ABC-CFX Total Score and a Score of 1 (Very Much Improved) or 2 (Much Improved) on the CGI-I Scale, Stratum I	12 weeks	Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Total is the number of patients with non-missing baseline ABC-CFX total score and at least one nonmissing post-baseline ABC-CFX total score and CGI-I assessment	Number of participants	NUMBER

1 to 10 of 13Source ClinicalTrials.gov

See all 13 outcomes

Results by group185

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the Aberrant Behavior Checklist-Community Edition (ABC-CFX) Total Score in Stratum I Patients Exposed to AFQ056 100 mg Bid	AFQ056 100 mg Bid	8.6	Standard Error 4.48	Score
Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the Aberrant Behavior Checklist-Community Edition (ABC-CFX) Total Score in Stratum I Patients Exposed to AFQ056 100 mg Bid	Placebo	-9.4	Standard Error 3.88	Score
Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum II Patients Exposed to All 3 Doses of AFQ056	AFQ056 25 mg Bid	-6.8	Standard Error 3.88	Score
Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum II Patients Exposed to All 3 Doses of AFQ056	AFQ056 50 mg Bid	-2.8	Standard Error 4.07	Score
Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum II Patients Exposed to All 3 Doses of AFQ056	AFQ056 100 mg Bid	-5.7	Standard Error 4.06	Score
Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum II Patients Exposed to All 3 Doses of AFQ056	Placebo	-3.5	Standard Error 4.30	Score
Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum I Patients Exposed to the Two Lower Doses of AFQ056 (25 mg Bid and 50 mg Bid)	AFQ056 25 mg Bid	-11.8	Standard Error 6.43	Score
Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum I Patients Exposed to the Two Lower Doses of AFQ056 (25 mg Bid and 50 mg Bid)	AFQ056 50 mg Bid	-3.4	Standard Error 7.40	Score
Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum I Patients Exposed to the Two Lower Doses of AFQ056 (25 mg Bid and 50 mg Bid)	Placebo	-9.4	Standard Error 3.88	Score
Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum I Patients	AFQ056 25 mg Bid	3.3	Standard Error 0.30	Score

1 to 10 of 185Source ClinicalTrials.gov

See all 185 results by group

Participant flow36

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Stratum: All Patients	STARTED	AFQ056 25 mg Bid	31
Stratum: All Patients	STARTED	AFQ056 50 mg Bid	27
Stratum: All Patients	STARTED	AFQ056 100 mg Bid	39
Stratum: All Patients	STARTED	Placebo	42
Stratum: All Patients	COMPLETED	AFQ056 25 mg Bid	31
Stratum: All Patients	COMPLETED	AFQ056 50 mg Bid	27
Stratum: All Patients	COMPLETED	AFQ056 100 mg Bid	37
Stratum: All Patients	COMPLETED	Placebo	40
Stratum: All Patients	NOT COMPLETED	AFQ056 25 mg Bid	0
Stratum: All Patients	NOT COMPLETED	AFQ056 50 mg Bid	0

1 to 10 of 36Source ClinicalTrials.gov

See all 36 participant flow

Adverse events88

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
other	Abdominal pain upper	Gastrointestinal disorders	Placebo	0	42
other	Abdominal pain upper	Gastrointestinal disorders	AFQ056 25 mg Bid	2	31
other	Abdominal pain upper	Gastrointestinal disorders	AFQ056 50 mg Bid	0	27
other	Abdominal pain upper	Gastrointestinal disorders	AFQ056 100 mg Bid	2	39
other	Diarrhoea	Gastrointestinal disorders	Placebo	0	42
other	Diarrhoea	Gastrointestinal disorders	AFQ056 25 mg Bid	3	31
other	Diarrhoea	Gastrointestinal disorders	AFQ056 50 mg Bid	0	27
other	Diarrhoea	Gastrointestinal disorders	AFQ056 100 mg Bid	2	39
other	Vomiting	Gastrointestinal disorders	Placebo	0	42
other	Vomiting	Gastrointestinal disorders	AFQ056 25 mg Bid	2	31

1 to 10 of 88Source ClinicalTrials.gov

See all 88 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo
Total, all-cause mortality	AFQ056 25 mg Bid
Total, all-cause mortality	AFQ056 50 mg Bid
Total, all-cause mortality	AFQ056 100 mg Bid
Total, other adverse events	Placebo	17	42
Total, other adverse events	AFQ056 25 mg Bid	22	31
Total, other adverse events	AFQ056 50 mg Bid	9	27
Total, other adverse events	AFQ056 100 mg Bid	30	39
Total, serious adverse events	Placebo	2	42
Total, serious adverse events	AFQ056 25 mg Bid	0	31

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
26764156	DERIVED	PubMed
26855682	DERIVED	PubMed

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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.

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