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NCT05630066: Aldebaran

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.

Completed Phase 2 Last updated 4 December 2025
What this trial tests

Phase 2 trial testing Alogabat in Angelman Syndrome in 48 participants. Completed in 2 December 2025.

Timeline
27 July 2023
Primary endpoint
2 December 2025
2 December 2025

Quick facts

Lead sponsorHoffmann-La Roche
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationnon randomized
Designsequential
Maskingnone
Primary purposetreatment
Enrollment48
Start date27 July 2023
Primary completion2 December 2025
Estimated completion2 December 2025
Sites19 locations across France, Italy, Germany, Australia, United States, Spain

Drugs / interventions tested

Conditions studied

Sponsor

Hoffmann-La Roche — full company profile →

Who can join

Adults 5 to 17, any sex, with Angelman Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Preclinical pharmacology of alogabat: a novel GABA<sub>A</sub>-α5 positive allosteric modulator targeting neurodevelopmental disorders with impaired GABA<sub>A</sub> signaling.
    Cecere G, Ballard TM, Knoflach F, Honer M, et al · · 2025 · cited 3× · PMID 40761395 · DOI 10.3389/fphar.2025.1626078
  2. Using the Bayley-4 and Vineland-3 in Angelman syndrome: barriers, solutions, and challenging items.
    Halpin SN, Potter SN, Gwaltney A, Sadhwani A, et al · · 2025 · cited 1× · PMID 40462183 · DOI 10.1186/s13023-025-03817-x
  3. Crossing the finish line towards a disease-modifying treatment for Angelman syndrome.
    Judson MC, de Almeida LP, Burdine RD, Chamberlain SJ, et al · · 2026 · PMID 41795090 · DOI 10.1186/s11689-026-09681-5

Verify or expand the search:

Other recruiting trials for Angelman Syndrome

Currently open trials in the same condition.

Other Hoffmann-La Roche trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

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/NCT05630066.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing