CClinicalTrials.gg
CompletedNCT05630066AldebaranUpdated Dec 4, 2025

A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.

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

Phase
Phase 2
Study type
Interventional
Enrollment
48
Allocation
Non-randomized
Ages
5 Years to 17 Years
Sex
All
01

Study summary

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.

Read the detailed description

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.

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Conditions studied

  • Angelman Syndrome

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03

In context

Angelman Syndrome

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 →

Lead sponsor

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.

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Who can participate

Ages eligible
5 Years to 17 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Clinical diagnosis of AS and a genetic subtype of deletion on chromosome 15q11q13 confirmed by a historical molecular diagnosis
  • The participant's general health status, in the context of the disease under study, allows them to participate in a clinical trial in the opinion of the investigator
  • The reliability of sexual abstinence for male and/or female enrollment eligibility needs to be evaluated in relation to the duration of the clinical study and the preferred and usual lifestyle of the participant. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or post-ovulation methods) and withdrawal are not acceptable methods of preventing drug exposure
  • Female participants:

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

Exclusion Criteria:

  • A molecular diagnosis of AS with genotypic classification of any type besides the molecular diagnosis as specified in Inclusion Criterion
  • Concurrent cardiovascular disease considered not well controlled by drug treatment, including participants with clinically significant hypertension, bradycardia and arrhythmias, myocardial infarction (MI) within 12 months of screening or uncompensated heart failure
  • Confirmed clinically significant abnormality on 12-lead electrocardiogram (ECG), including:
  • a QT corrected for heart rate using the Fridericia's correction factor (QTcF) of >/= 450 ms (based on the average of 3 consecutive measurements) for participants older than 10 years old
  • a QT corrected for heart rate using Bazett's formula (QTcB) of >/= 450 ms (based on the average of 3 consecutive measurements) for participants up to, and including, the age of 10 years old
  • Congenital heart diseases not treated and congenital QT corrected for heart rate (QTc) prolongation or family history of Long QT Syndrome
  • Medical history of malignancy if not considered cured or if occurred within the last 5 years with the exception of fully excised non-melanoma skin cancers or in-situ carcinoma of the cervix that has been successfully treated
  • Concomitant disease, condition, or treatment that would either interfere with the conduct of the study or pose an unacceptable risk to the participant in the opinion of the investigator
  • Known active or uncontrolled bacterial, viral, or other infection (excluding fungal infections of nail beds) or any major episode of infection or hospitalization (relating to the completion of the course of antibiotics) within 6 weeks prior to the start of drug administration. Rescreening is allowed once the infection is cured and if the rescreening criteria are met
  • Any concomitant condition that might interfere with the clinical evaluation of AS and that is not related to AS
  • Known history of human immunodeficiency virus (HIV) or hepatitis B virus (HBV) or hepatitis C virus (HCV)
  • Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks of Screening or planned during the study. Rescreening is allowed not earlier than 12 weeks after the surgery and if the rescreening criteria are met.
  • Use of prohibited medications within 6 weeks or 5 half-lives (t1/2) prior to start of study medication on Day 1 (whichever is longer)
  • Clinically significant loss of blood within 3 months prior to screening defined by participant age and weight per recommendations from Duke University (2012)
  • Any prior or current treatment with an investigational study drug within 6 weeks or 5 times the t1/2 of the investigational molecule (whichever is longer) prior to baseline or prior or current use of an investigational medical device within 6 weeks prior to baseline or if the device is still active. Concurrent or planned concurrent participation in any clinical study (including observational and non-interventional studies) without approval of the Investigator.
  • Previous participation in a cellular therapy, gene therapy, or gene editing clinical study
  • Clinically significant vital sign or ECG abnormalities at Screening
  • Confirmed clinically significant abnormality in hematological, chemistry or coagulation laboratory parameters
  • Uncorrected hypokalemia or hypomagnesaemia
  • Positive test result at screening for hepatitis B surface antigen (HBsAg), HCV (untreated), or HIV-1/2. Participants with HCV who have been successfully treated and who test negative for HCV ribonucleic acid (HCV RNA) may be considered eligible for entry into the study
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
48 participants (actual)

Study arms

  • Experimental
    Part 1 Adult Alogabat Dose (Age 15-17)

    In Part 1 of the study participants will receive alogabat once a day (QD).

    Drug: Alogabat

  • Experimental
    Part 1 Age-adjusted Dose (Age 10-14)

    In Part 1 of the study, participants will receive age-adjusted QD doses of alogabat.

    Drug: Alogabat

  • Experimental
    Part 1 Age-adjusted Dose (Age 5-9)

    In Part 1 of the study, participants will receive age-adjusted QD doses of alogabat.

    Drug: Alogabat

  • Experimental
    Part 2 Cohort 1

    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

  • Experimental
    Part 2 Cohort 2

    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

  • Experimental
    Part 1 Optional Cohort

    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

  • Experimental
    Part 2 Optional Cohort

    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

Interventions

  • DrugAlogabat

    Alogabat will be administered QD with dose depending on cohort and age of the participant.

06

What researchers measure

Primary outcomes

  1. 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

  2. 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

  3. 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

Secondary outcomes

  1. 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

  2. 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

  3. 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

  4. 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

  5. Parts 1 and 2: Incidence of Treatment Discontinuations due to AEs

    Incidence of treatment discontinuations due to AEs

    Time frame: Up to 18 Weeks

  6. 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

07

Study locations

19 sites
  • Rush Medical Center
    Chicago, Illinois 60612, United States
  • Boston Children's Hospital
    Boston, Massachusetts 02115, United States
  • Columbia University Medical Center
    New York, New York 10032, United States
  • Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine
    Carrboro, North Carolina 27510, United States
  • Vanderbilt Children's Hospital
    Nashville, Tennessee 37232-9119, United States
  • Multicare Institute for Research and Innovation
    Tacoma, Washington 98405, United States
  • Queensland Children?s Hospital
    South Brisbane, Queensland 4101, Australia
  • CHRU de Brest
    Brest, 29609, France
  • CHU Dijon Bourgogne Hôpital François Mitterand
    Dijon, 21000, France
  • Hopital la Timone Enfants
    Marseille, 13005, France
  • Groupe Hospitalier Necker Enfants Malades
    Paris, 75015, France
  • Dr. Von Haunersches Kinderspital
    München, 80337, Germany
  • Ospedale Pediatrico Bambino Gesù
    Rome, Lazio 00165, Italy
  • IRCCS Istituto G. Gaslini
    Genoa, Liguria 16147, Italy
  • IRCCS Eugenio Medea
    Conegliano Veneto (TV), Veneto 31015, Italy
  • Hospital Sant Joan de Deu
    Esplugues de Llobregat · Barcelona, Barcelona 08950, Spain
  • Corporacio Sanitaria Parc Tauli
    Sabadell, Barcelona 08208, Spain
  • Hospital Universitario de Navarra;Unidad de Neuropediatría
    Pamploa, Navarre 31008, Spain
  • Hospital Universitario Puerta De Hierro Majadahonda
    Madrid, 28222, Spain
08

References and documents

Individual participant data

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.

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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Dec 4, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT05630066
Lead sponsor
Hoffmann-La Roche
Responsible party
Sponsor
First posted
Nov 29, 2022
Start date
Jul 27, 2023
Primary completion
Dec 2, 2025
Completion
Dec 2, 2025
Last update
Dec 4, 2025

Study contacts

Clinical Trials
study director · Hoffmann-La Roche

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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