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COMPLETEDPHASE2

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 IIa Multicenter, Open-label, 12-week Study to Investigate the Pharmacokinetics 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

NCT05630066•Hoffmann-La Roche

View on ClinicalTrials.gov

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.

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.

Eligibility

Age

5 - 17 years

Sex

ALL

Healthy Volunteers

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

•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
+10 more criteria

Locations (19)

Rush Medical Center

Chicago, Illinois, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Columbia University Medical Center

New York, New York, United States

Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine

Carrboro, North Carolina, United States

Vanderbilt Children's Hospital

Nashville, Tennessee, United States

Multicare Institute for Research and Innovation

Tacoma, Washington, United States

Queensland Children?s Hospital

South Brisbane, Queensland, Australia

CHRU de Brest

Brest, France

CHU Dijon Bourgogne Hôpital François Mitterand

Dijon, France

Hopital la Timone Enfants

Marseille, France

Key Dates

Start Date

July 27, 2023

Primary Completion Date

December 2, 2025

Completion Date

December 2, 2025

Last Updated

December 4, 2025

Enrollment

ACTUAL participants

Conditions

Angelman Syndrome

Interventions

Alogabat

DRUG

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

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Angelman Syndrome

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COMPLETEDPHASE1

A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)

NCT04428281

Angelman Syndrome

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COMPLETEDPHASE2

A Study in Adults and Adolescents With Angelman Syndrome (STARS)

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 4, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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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.

Inclusion Criteria

Exclusion Criteria

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)

A Study in Adults and Adolescents With Angelman Syndrome (STARS)

Angelman Syndrome Natural History Study-FAST UK

A Study of OV101 in Individuals With Angelman Syndrome (AS)

Study on the Brain Network of Angelman Syndrome