NCT07439510
A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
At risk: Primary endpoint(s) modified, enrollment reduced 52%.
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Record History
All 2 versions: 2 with changes below. See every version on ClinicalTrials.gov ↗
Notable Enrollment reduced: 248 → 120 participants Show 7 more changesShow less
- Primary endpoint(s) modified
- Start date: Mar 1, 2026 to Dec 1, 2026
- Interventions modified
- Eligibility criteria expanded, about 41 words
- Secondary endpoints: 21 to 25
- Study description expanded, about 15 words
- Study title expanded, about 3 words
- Study First Submitted
- Notable: the plan changed
Endpoints
Primary
Phase 2b: FXS- Change from baseline in Total Cognition Composite Change Sensitive Score from the NIH-TCB
Timeframe: 28 days
Phase 2b: ASD - Change from Baseline at Day 28 on the Vineland Adaptive Behavior Scales in ASD(Vineland-III) Adaptive Behavior Composite Score
Timeframe: 28 days
Phase 3: FXS - Change from baseline in Total Cognition Composite score from the NIH-TCB
Timeframe: 12 weeks
Secondary (25)
Phase 2b: FXS- Change from baseline in Fluid Reasoning Composite Change Sensitive Scores from the NIH-TCB
Phase 2b: FXS- Change from baseline in Flanker Scores from the NIH-TCB
Phase 2b- FXS Change from Baseline at Day 28 on the Vineland Adaptive Behavior Scales (Vineland-III) Adaptive Behavior Composite Score
Phase 2b: FXS and ASD: Change from baseline at Day 28 in Clinical global impression of severity (CGI-S) as assessed by the investigator
Phase 2b: FXS and ASD: Change from baseline at Day 28 in Clinical global impression of improvement as assessed by the investigator
Show 20 moreShow less
Phase 2b: FXS and ASD: Change from baseline at Day 28 in Caregiver global impression of change (CGI-C) as assessed by the caregiver
Phase 2b: FXS and ASD: Change from baseline at Day 28 in the Numerical Rating Scale as completed by the caregiver
Phase 2b: FXS and ASD - Incidence, nature, and severity of adverse events (AEs) and serious adverse events (SAEs)
Phase 2b: FXS and ASD - Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS) throughout the study
Phase 2b: FXS and ASD - The PK parameters of codabakalner concentrations in plasma-Cmax
Phase 2b: FXS and ASD: Change from baseline to Day 28 in EEG resting state relative power in the alpha, theta, and gamma bands during rest
Phase 3: Change from baseline to week 12 in Total Cognition Composite score from the NIH-TCB
Phase 3: Incidence, nature, and severity of adverse events (AEs) and serious adverse events (SAEs)
Phase 3: Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS) throughout the study
Phase 3: Change from baseline to Week 4, Week 8, and Week 12 in Flanker from the NIH-TCB
Phase 3: Change from baseline to Week 4, Week 8, and Week 12 in Fluid Reasoning Composite Change Sensitive Scores from the NIH-TCB
Phase 3: Change from baseline to week 4 and week 8 in Total Cognition Composite score from the NIH-TCB
Phase 3: The PK parameters of codabakalner concentrations in plasma-Cmax
Phase 3: Change in from baseline to Week 4, Week 8, and Week 12, in ABC total score and subscale scores (FXS scoring)
Phase 3: Change from baseline to Week 4, Week 8, and Week 12 in behaviors and other symptoms on NRS by caregiver
Phase 3: Change from baseline to Week 12 in Vineland Adaptive Behavior Scale 3rd Edition
Phase 3: Change from baseline in CGI-S as assessed by investigator
Phase 3: Change from baseline in CGI-I as assessed by investigator
Phase 3: Change from baseline in Caregiver Global Impression of Improvement (CGI-C) assessments
Phase 3: Change from baseline in the Numerical Rating Scale at Week 4, Week 8, and Week 12 as completed by the caregiver
Arms and interventions
Phase 2b: ActiveActive comparator
Phase 2b: Participants with FXS or ASD will be randomized to receive a daily dose of codabakalner daily for 4 weeks
Phase 2b: PlaceboPlacebo comparator
Participants with FXS or ASD will be randomized to receive a daily dose of placebo over 4 weeks.
Phase 3: ActiveActive comparator
Participants with FXS will be randomized to receive a daily dose of codabakalner determined from Phase 2b over 12 weeks
Phase 3: PlaceboPlacebo comparator
Participants with FXS will be randomized to receive a daily dose of placebo for 12 weeks
Drugs: Codabakalner, Placebo