Adults 32 Months to 6, any sex, with Fragile X Syndrome. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Change in Weighted Child Intentional Communication ScorePrimary· 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 ch
Group
Value
95% CI
Placebo Comparator: Double-Blind Placebo With Language Intervention
0.14
0.05 – 0.22
Experimental: Double-Blind AFQ056 With Language Intervention
0.04
-0.05 – 0.12
Change in Mullen Scales of Early Learning (MSEL) Developmental Quotient (DQ)Secondary· 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.
Group
Value
95% CI
Placebo Comparator: Double-Blind Placebo With Language Intervention
-0.7
-1.90 – 0.50
Experimental: Double-Blind AFQ056 With Language Intervention
-2.35
-3.55 – -1.16
Change in Mullen Scales of Early Learning (MSEL) Expressive Language SubscoreSecondary· 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 s
Group
Value
95% CI
Placebo Comparator: Double-Blind Placebo With Language Intervention
1.99
1.19 – 2.79
Experimental: Double-Blind AFQ056 With Language Intervention
1.32
0.52 – 2.11
Change in Vineland Adaptive Behavior Scale (Vineland-3) Composite ScoreSecondary· 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
Group
Value
95% CI
Placebo Comparator: Double-Blind Placebo With Language Intervention
0.68
-0.51 – 1.88
Experimental: Double-Blind AFQ056 With Language Intervention
0.39
-0.81 – 1.60
Change in Vineland Adaptive Behavior Scale (Vineland-3) Communication SubscoreSecondary· 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
Group
Value
95% CI
Placebo Comparator: Double-Blind Placebo With Language Intervention
0.54
-0.97 – 2.05
Experimental: Double-Blind AFQ056 With Language Intervention
0.63
-0.90 – 2.16
Change in Preschool Language Scale (PLS-5) Expressive Communication ScoreSecondary· 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
Group
Value
95% CI
Placebo Comparator: Double-Blind Placebo With Language Intervention
1.78
1.10 – 2.46
Experimental: Double-Blind AFQ056 With Language Intervention
0.93
0.25 – 1.60
MacArthur-Bates Communicative Development Inventory (CDI) Number of Spoken WordsSecondary· 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.
Group
Value
95% CI
Placebo Comparator: Double-Blind Placebo With Language Intervention
319
0 – 677
Experimental: Double-Blind AFQ056 With Language Intervention
413.5
0 – 680
Number of Participants With a Positive Response as Defined by Improvement in Clinical Global Impression - Improvement (CGI-I) Overall Function ScoresSecondary· 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,
Group
Value
95% CI
Placebo Comparator: Double-Blind Placebo With Language Intervention
10
Experimental: Double-Blind AFQ056 With Language Intervention
8
Adverse events — posted to ClinicalTrials.gov
Time frame: Participants were monitored for AEs from the time they signed the consent until the final Study visit, up to 21 months. If an AE was ongoing or discovered at a subject's final Study visit, the AE was followed until resolution, or for a minimum of 30 days, whichever comes first..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Placebo Lead-In Period
Serious: 1/110 (1%)
Deaths: 0/110
Double-Blind Period: Placebo With Language Intervention
Serious: 1/49 (2%)
Deaths: 0/49
Double-Blind Period: AFQ056 With Language Intervention
Serious: 0/50 (0%)
Deaths: 0/50
Open-Label Period: AFQ056 With Language Intervention
NCT04771143 — Study to Assess Safety, Tolerability, and Interactions of Cocaine and Oral AFQ056
· Phase 1
· withdrawn
NCT03242928 — Study to Investigate Whether AFQ056 Reduces Cocaine Use in Patients Diagnosed With Cocaine Use Disorder (CUD)
· Phase 2
· completed
Other recruiting trials for Fragile X Syndrome
Currently open trials in the same condition.
NCT06279858 — Probiotic Intervention for Microbiome Modifications and Clinical Improvements in Fragile X Syndrome
· NA
· recruiting
NCT06088589 — Speech-in-noise Perception in Autism and Fragile X
· NA
· recruiting
NCT06227780 — Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders
· NA
· recruiting
NCT05367960 — An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome
· Phase 3
· active not recruiting
NCT06560242 — Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG- Infant
· NA
· recruiting
Other Elizabeth Berry-Kravis trials
Trials by the same sponsor.
NCT05030129 — ERG/5-HTP in Fragile X Syndrome (FXS)
· Phase 2
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Elizabeth Berry-Kravis
Last refreshed: 10 October 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02920892.