Trial · NCT02920892
AFQ056 for Language Learning in Children With FXS
NCT02920892, Phase 2, Completed, Elizabeth Berry-Kravis; results first posted 2023-10-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. AFQ056 for Language Learning in Children With FXS. Trials, record NCT02920892. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02920892
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- AFQ056 for Language Learning in Children With FXS
- NCT number
- NCT02920892
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 110 participants, actual
- Lead sponsor
- Elizabeth Berry-Kravis
- Conditions
- Fragile X Syndrome
- Interventions
- AFQ056, Placebo, Language Intervention
- First posted
- 2016-09-30
- Last updated
- 2023-10-10
- Results first posted
- 2023-10-10
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Weighted Child Intentional Communication Score | Baseline through Month 8 | The Weighted Child Intentional Communication Score is derived from a 22 minute semi-structured examiner/child play session. Structured and unstructured component scores are found by multiplying each intentional communication act by the following weights: nonverbal = 1; single symbol = 2; and multiple symbols = 3. The two scores are summed together to obtain the total. Higher scores indicate more child-initiated communication. There is no maximum score. The scale was administered at Baseline, Month 2, Month 4, Month 6, and Month 8. A composite score representing the average of the estimated change scores calculated at months 2, 4, 6, and 8 was recorded. A higher least squares mean value indicates a greater increase in WCS score from baseline. |
| Secondary | Change in Mullen Scales of Early Learning (MSEL) Developmental Quotient (DQ) | Baseline through Month 8 | Mullen Scales of Early Learning (MSEL) Developmental Quotient (DQ) is calculated by averaging the developmental age equivalents from 4 domains of the Mullen (visual reception, fine motor, receptive language, and expressive language) to find developmental age in months, dividing by chronological age in months, then multiplying by 100. DQ can range from 1 to 70. A higher DQ indicates better performance on the MSEL. The MSEL was administered at Baseline, Month 2, and Month 8. A composite score representing the average of the estimated change scores calculated at month 2 and month 8 was recorded. A negative least squares mean indicates a net decrease in DQs over time. A more negative least squares mean indicates a larger negative change in DQs over time. |
| Secondary | Change in Mullen Scales of Early Learning (MSEL) Expressive Language Subscore | Baseline through Month 8 | The MSEL measures performance over 5 subscales including expressive language. The range of possible subscores for the expressive language domain is 1-50. A higher score indicates better performance / greater use of expressive language The MSEL was administered at Baseline, Month 2, and Month 8. A composite score representing the average of the estimated change scores calculated at month 2 and month 8 was recorded. A positive least squares mean indicates a net positive change in expressive language scores over time. A higher least squares mean indicates a larger change in expressive language scores over time. |
| Secondary | Change in Vineland Adaptive Behavior Scale (Vineland-3) Composite Score | Baseline through Month 8 | The Vineland-3 composite score is yielded from the subject's level of adaptive functioning in the domains of communication, daily living skills, socialization, and motor skills. The range of possible composite scores is 20-140 with a higher score indicating higher levels of adaptive functioning. The Vineland-3 was administered at Baseline, Month 2, Month 4, Month 6, and Month 8. A composite score representing the average of the estimated change scores calculated at months 2, 4, 6, and 8 was recorded. A positive least squares mean indicates a net increase in composite score over time. A higher least squares mean indicates a greater change in composite score over time. |
| Secondary | Change in Vineland Adaptive Behavior Scale (Vineland-3) Communication Subscore | Baseline and Month 8 | The communication domain of the Vineland-3 examines adaptive functioning in receptive, expressive, and written language. The range of possible subscores for the communication domain is 20-140 with a higher score indicating more advanced use of language. The Vineland-3 was administered at Baseline, Month 2, Month 4, Month 6, and Month 8. A composite score representing the average of the estimated change scores calculated at months 2, 4, 6, and 8 was recorded. A positive least squares mean indicates a net increase in communication score over time. A higher least squares mean indicates a greater change in communication score over time. |
| Secondary | Change in Preschool Language Scale (PLS-5) Expressive Communication Score | Baseline and Month 8 | The PLS-5 is a comprehensive developmental language assessment that requires the child to point to or verbally respond to items. Raw scores for expressive language were collected from this test. It is difficult to specify an upper bound on the score, because there is no clear expectation of a limit on the number of times a child can communicate unless one sets it artificially (e.g., once per second in an 11-minute free play). The lower bound is zero for a child who produces no communication acts. The PLS-5 was administered at Baseline, Month 2, and Month 8. A composite score representing the average of the estimated change scores calculated at month 2 and month 8 was recorded. A higher least squares mean indicates a larger change in PLS-5 communication score over 8 months. |
| Secondary | MacArthur-Bates Communicative Development Inventory (CDI) Number of Spoken Words | Month 8 | The MacArthur-Bates CDI is a parent interview that allows tracking of a child's progress in developing words, sentences, and more complex language. The assessment consists of two parts including "Words Children Use," a 680-word vocabulary checklist in which the parent indicates those vocabulary words the child regularly produces in spoken language, and "Sentences and Grammar" an assessment of several aspects of grammar and word endings. The MacArthur-Bates CDI number of spoken words will be recorded with higher scores indicating more words used / greater language development at Month 8. |
| Secondary | Number of Participants With a Positive Response as Defined by Improvement in Clinical Global Impression - Improvement (CGI-I) Overall Function Scores | Month 8 | Completion of the CGI-I requires the clinician to rate how much the study participant's illness has improved or worsened relative to a baseline state. For this study, two sets of CGI-I are administered at each applicable visit - one associated with Language/Communication and the other to Overall Function. The Overall Function CGI-I considers all areas of function including cognition, adaptive behavior, and maladaptive behavior. The CGI-I is a 7-point scale that includes the following ratings: 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, 7 = very much worse. The CGI-I was rated at all study visits after screening. This measure shows the number of participants with positive CGI-I scores indicating perceived improvement at Month 8. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Weighted Child Intentional Communication Score | Baseline through Month 8 | Comparison of WCS from baseline and from month 8 visit (end of double-blind period) in both AFQ056 \& Language Intervention and Placebo \& Language Intervention groups. | log10(scores on a scale) | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Mullen Scales of Early Learning (MSEL) Developmental Quotient (DQ) | Baseline through Month 8 | Comparison of DQ from baseline and from month 8 visit (end of double-blind period) in both AFQ056 \& Language Intervention and Placebo \& Language Intervention groups. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Mullen Scales of Early Learning (MSEL) Expressive Language Subscore | Baseline through Month 8 | Comparison of MSEL expressive language subscore from baseline and from month 8 visit (end of double-blind period) in both AFQ056 \& Language Intervention and Placebo \& Language Intervention groups. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Vineland Adaptive Behavior Scale (Vineland-3) Composite Score | Baseline through Month 8 | Comparison of Vineland-3 Composite Score from baseline and from month 8 visit (end of double-blind period) in both AFQ056 \& Language Intervention and Placebo \& Language Intervention groups. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Vineland Adaptive Behavior Scale (Vineland-3) Communication Subscore | Baseline and Month 8 | Comparison of Vineland-3 Communication Subscore from baseline and from month 8 visit (end of double-blind period) in both AFQ056 \& Language Intervention and Placebo \& Language Intervention groups. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Preschool Language Scale (PLS-5) Expressive Communication Score | Baseline and Month 8 | Comparison of PLS-5 expressive communication scores from baseline and from month 8 visit (end of double-blind period) in both AFQ056 \& Language Intervention and Placebo \& Language Intervention groups. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | MacArthur-Bates Communicative Development Inventory (CDI) Number of Spoken Words | Month 8 | Number of words spoken at month 8 visit (end of double-blind period) in both AFQ056 \& Language Intervention and Placebo \& Language Intervention groups. | Spoken Words | MEDIAN |
| SECONDARY | Number of Participants With a Positive Response as Defined by Improvement in Clinical Global Impression - Improvement (CGI-I) Overall Function Scores | Month 8 | Patients with positive change in CGI-I scores from baseline to month 8 visit (end of double-blind period) in both AFQ056 \& Language Intervention and Placebo \& Language Intervention groups. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Weighted Child Intentional Communication Score | Placebo Comparator: Double-Blind Placebo With Language Intervention | 0.14 | 90% Confidence Interval 0.05 to 0.22 | log10(scores on a scale) | |
| Change in Weighted Child Intentional Communication Score | Experimental: Double-Blind AFQ056 With Language Intervention | 0.04 | 90% Confidence Interval -0.05 to 0.12 | log10(scores on a scale) | |
| Change in Mullen Scales of Early Learning (MSEL) Developmental Quotient (DQ) | Placebo Comparator: Double-Blind Placebo With Language Intervention | -0.7 | 90% Confidence Interval -1.90 to 0.50 | scores on a scale | |
| Change in Mullen Scales of Early Learning (MSEL) Developmental Quotient (DQ) | Experimental: Double-Blind AFQ056 With Language Intervention | -2.35 | 90% Confidence Interval -3.55 to -1.16 | scores on a scale | |
| Change in Mullen Scales of Early Learning (MSEL) Expressive Language Subscore | Placebo Comparator: Double-Blind Placebo With Language Intervention | 1.99 | 90% Confidence Interval 1.19 to 2.79 | scores on a scale | |
| Change in Mullen Scales of Early Learning (MSEL) Expressive Language Subscore | Experimental: Double-Blind AFQ056 With Language Intervention | 1.32 | 90% Confidence Interval 0.52 to 2.11 | scores on a scale | |
| Change in Vineland Adaptive Behavior Scale (Vineland-3) Composite Score | Placebo Comparator: Double-Blind Placebo With Language Intervention | 0.68 | 90% Confidence Interval -0.51 to 1.88 | scores on a scale | |
| Change in Vineland Adaptive Behavior Scale (Vineland-3) Composite Score | Experimental: Double-Blind AFQ056 With Language Intervention | 0.39 | 90% Confidence Interval -0.81 to 1.60 | scores on a scale | |
| Change in Vineland Adaptive Behavior Scale (Vineland-3) Communication Subscore | Placebo Comparator: Double-Blind Placebo With Language Intervention | 0.54 | 90% Confidence Interval -0.97 to 2.05 | scores on a scale | |
| Change in Vineland Adaptive Behavior Scale (Vineland-3) Communication Subscore | Experimental: Double-Blind AFQ056 With Language Intervention | 0.63 | 90% Confidence Interval -0.90 to 2.16 | scores on a scale |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow27
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Placebo Lead-In Period | STARTED | Placebo Lead-In | 110 | |
| Placebo Lead-In Period | STARTED | Double-Blind Placebo, Then Open-Label AFQ056 | 0 | |
| Placebo Lead-In Period | STARTED | Double-Blind AFQ056, Then Open-Label AFQ056 | 0 | |
| Placebo Lead-In Period | COMPLETED | Placebo Lead-In | 99 | |
| Placebo Lead-In Period | COMPLETED | Double-Blind Placebo, Then Open-Label AFQ056 | 0 | |
| Placebo Lead-In Period | COMPLETED | Double-Blind AFQ056, Then Open-Label AFQ056 | 0 | |
| Placebo Lead-In Period | NOT COMPLETED | Placebo Lead-In | 11 | |
| Placebo Lead-In Period | NOT COMPLETED | Double-Blind Placebo, Then Open-Label AFQ056 | 0 | |
| Placebo Lead-In Period | NOT COMPLETED | Double-Blind AFQ056, Then Open-Label AFQ056 | 0 | |
| Placebo-Controlled Period | STARTED | Placebo Lead-In | 0 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 participant flowAdverse events84
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 | Diarrhea | Gastrointestinal disorders | Placebo Lead-In Period | 7 | 110 | 8 |
| other | Diarrhea | Gastrointestinal disorders | Double-Blind Period: Placebo With Language Intervention | 3 | 49 | 3 |
| other | Diarrhea | Gastrointestinal disorders | Double-Blind Period: AFQ056 With Language Intervention | 7 | 50 | 8 |
| other | Diarrhea | Gastrointestinal disorders | Open-Label Period: AFQ056 With Language Intervention | 10 | 89 | 13 |
| other | Vomiting | Gastrointestinal disorders | Placebo Lead-In Period | 9 | 110 | 16 |
| other | Vomiting | Gastrointestinal disorders | Double-Blind Period: Placebo With Language Intervention | 9 | 49 | 9 |
| other | Vomiting | Gastrointestinal disorders | Double-Blind Period: AFQ056 With Language Intervention | 4 | 50 | 7 |
| other | Vomiting | Gastrointestinal disorders | Open-Label Period: AFQ056 With Language Intervention | 11 | 89 | 17 |
| other | Pyrexia | General disorders | Placebo Lead-In Period | 4 | 110 | 4 |
| other | Pyrexia | General disorders | Double-Blind Period: Placebo With Language Intervention | 1 | 49 | 1 |
1 to 10 of 84Source ClinicalTrials.gov
See all 84 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo Lead-In Period | 0 | 110 |
| Total, all-cause mortality | Double-Blind Period: Placebo With Language Intervention | 0 | 49 |
| Total, all-cause mortality | Double-Blind Period: AFQ056 With Language Intervention | 0 | 50 |
| Total, all-cause mortality | Open-Label Period: AFQ056 With Language Intervention | 0 | 89 |
| Total, other adverse events | Placebo Lead-In Period | 65 | 110 |
| Total, other adverse events | Double-Blind Period: Placebo With Language Intervention | 44 | 49 |
| Total, other adverse events | Double-Blind Period: AFQ056 With Language Intervention | 45 | 50 |
| Total, other adverse events | Open-Label Period: AFQ056 With Language Intervention | 76 | 89 |
| Total, serious adverse events | Placebo Lead-In Period | 1 | 110 |
| Total, serious adverse events | Double-Blind Period: Placebo With Language Intervention | 1 | 49 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39483619 | DERIVED |
Source PubMed
Grants1
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| U01NS096767 | Effects of AFQ056 on Language Learning in Young Children with Fragile X Syndrome (FXS) | BERRY-KRAVIS, ELIZABETH MARA; ABBEDUTO, LEONARD J.; HAGERMAN, RANDI J. | RUSH UNIVERSITY MEDICAL CENTER | NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE | 2016 to 2019 |
Source NIH RePORTER
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
Placebo Comparator: Double-Blind Placebo With Language Intervention vs Experimental: Double-Blind AFQ056 With Language Intervention: Number of Participants With a Positive Response as Defined by Improvement in Clinical Global Impression - Improvement (CGI-I) Overall Function Scores |
Source Trial comparisons
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/NCT02920892
GET https://conductscience.org/api/trials/v1/NCT02920892/flow?page=2