A Phase 2 interventional study of Alogabat in Angelman Syndrome, sponsored by Hoffmann-La Roche. Completed at 19 sites in 6 countries. Open to participants aged 5 Years to 17 Years. Per ClinicalTrials.gov, last updated 2025-12-04.
Sponsored by Hoffmann-La Roche · Phase 2, Interventional, and Treatment
This is a two-part, Phase IIa, multicenter, 12-week, open-label study. Up to 56 participants with deletion AS aged 5-17 years (inclusive) will be enrolled in the study.
The study will have Part 1-dose confirmations and Part 2 with dose levels to be decided based on the cumulative PK, electroencephalography (EEG), and safety data emerging from Part 1.
The dose levels for the first cohort of Part 2 will be decided based on the cumulative PK, EEG, and safety data emerging from Part 1.
Part 2 will explore the change in EEG beta-band power relative to baseline at Week 2, Week 4 (i.e., approximately 2 weeks after the start of the Dose B), and at the end of the 12-week treatment period after daily administration of alogabat.
46 studies on the registry are indexed under Angelman Syndrome; 16 are open to participants now.
This study's enrollment of 48 is below the median of 67 across 25 interventional studies indexed under Angelman Syndrome.
Browse Angelman Syndrome studies →Hoffmann-La Roche is the lead sponsor of 2,061 studies on the registry; 85 are open to participants now.
Of its 319 completed or terminated interventional studies of FDA-regulated products, 239 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
A female participant is eligible to participate if she is not pregnant, not breastfeeding, and non-childbearing or remain abstinent and/or Hormonal contraceptive methods must be supplemented
-Male participants: Male contraception is not required in this study because of the minimal seminal dose transmitted through sexual intercourse
Exclusion Criteria:
In Part 1 of the study participants will receive alogabat once a day (QD).
Drug: Alogabat
In Part 1 of the study, participants will receive age-adjusted QD doses of alogabat.
Drug: Alogabat
In Part 1 of the study, participants will receive age-adjusted QD doses of alogabat.
Drug: Alogabat
In Part 2 of the study, the dosing will depend upon the results of Part 1 with two different dose levels per cohort. Doses can be age-adjusted.
Drug: Alogabat
In Part 2 of the study, the dosing will depend upon the interim results with two different dose levels per cohort. Doses can be age-adjusted.
Drug: Alogabat
If dose adjustments (e.g., increase or decrease in dose) are required, particularly due to uncertainty of the clearance estimates (e.g., due to high variability) or over-/underprediction of the pediatric clearance versus adult clearance, additional participants may be recruited in any of the of the 3 age-groups in order to confirm the exposure equivalence. A total of two optional cohorts may be utilized in this study, allocated to Part 1 and/or Part 2.
Drug: Alogabat
In Part 2 of the study, the dosing will depend upon the interim results with two different dose levels per cohort. Doses can be age-adjusted.
Drug: Alogabat
Alogabat will be administered QD with dose depending on cohort and age of the participant.
Part 1: Age-group Based Ratio of Plasma PK Parameter, Area Under the Concentration-time Curve (AUC)
Age-group based ratio of plasma PK parameters in pediatric participants with AS versus data collected from adult healthy volunteers and participants with autism spectrum disorder (ASD) (AUC)
Time frame: Up to 12 Weeks
Part 1: Age-group Based Ratio of Plasma PK Parameter, Apparent Clearance (CL/F)
Age-group based ratio of plasma PK parameters in pediatric participants with AS versus data collected from adult healthy volunteers and participants with ASD (CL/F)
Time frame: Up to 12 Weeks
Part 2: Change From Baseline to Week 2, 4, and 12 in Resting State EEG Power in the Beta Band
Time frame: Week 2, 4, and 12
Parts 1 and 2: Plasma PK Parameter of Alogabat, Maximum Concentration (Cmax)
Plasma PK parameter of alogabat Cmax as derived using a population-pharmacokinetic (popPK) model
Time frame: Up to 12 Weeks
Parts 1 and 2: Plasma PK Parameter of Alogabat, AUC
Plasma PK parameter of alogabat AUC as derived using a popPK model
Time frame: Up to 12 Weeks
Parts 1 and 2: Plasma PK Parameter of Alogabat, CL/F
Plasma PK parameter of alogabat CL/F derived using a popPK model
Time frame: Up to 12 Weeks
Parts 1 and 2: Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Incidence and severity of AEs and SAEs
Time frame: Up to 18 Weeks
Parts 1 and 2: Incidence of Treatment Discontinuations due to AEs
Incidence of treatment discontinuations due to AEs
Time frame: Up to 18 Weeks
Parts 1 and 2: Incidence of Daytime Sleepiness Assessed With the Karolinska Sleepiness Scale (KSS), and Incidence of Sudden Onset of Sleep Assessed With Somnolence Diary
Incidence of daytime sleepiness assessed with the KSS, and incidence of sudden onset of sleep assessed with somnolence diary.
Time frame: Up to 12 Weeks
Plan to share: Yes — For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
Supporting information: Study protocol
No publications or documents are linked to this record.
This study is completed, as verified in Dec 2025. You cannot join it, but the record below documents what was studied.
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