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Showing 10 of 10 recruiting trials for “kleefstra-syndrome-due-to-a-point-mutation

RecruitingNCT07417137

A Natural History Study of Angelman Syndrome

👨‍⚕️ Christopher J Keary, MD, Massachusetts General Hospital📍 1 site📅 Started Mar 2026View details ↗
Phase 1, PHASE2RecruitingNCT07181837

A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome

🏥 MavriX Bio, LLC📍 3 sites📅 Started Oct 2025View details ↗
Phase 2RecruitingNCT07157254

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

👨‍⚕️ Medical Director, Ultragenyx Pharmaceutical Inc📍 22 sites📅 Started Oct 2025View details ↗
RecruitingNCT06737718

Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome

👨‍⚕️ Nathalie MD, PhD Boddaert, Assistance Publique - Hôpitaux de Paris📍 1 site📅 Started Feb 2025View details ↗
Phase 3Enrolling by InvitationNCT06415344

Long-term Extension of GTX-102 in Angelman Syndrome

👨‍⚕️ Medical Director, Ultragenyx Pharmaceuticals Inc.📍 23 sites📅 Started Jul 2024View details ↗
RecruitingNCT06115109

Angelman Natural History Study - FAST Spain

👨‍⚕️ BELEN RUIZ ANTORAN, MD, Ph, Clinical Pharmacology Deparment, Hospital Universitario Puerta de Hierro Majadahonda📍 2 sites📅 Started Dec 2023View details ↗
RecruitingNCT06337383

Study of the Prevalence of Autistic Traits in Angelman Syndrome

👨‍⚕️ Martina Baggio, Scientific Institute IRCCS E. Medea, Epilepsy Unit, Conegliano 31015, Italy📍 1 site📅 Started Mar 2022View details ↗
Phase 1, PHASE2RecruitingNCT05127226

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

🏥 Ionis Pharmaceuticals, Inc.📍 11 sites📅 Started Dec 2021View details ↗
RecruitingNCT06229769

Natural History Study for Patients With Angelman Syndrome

👨‍⚕️ Laura Vanden Brande, CHR Citadelle📍 1 site📅 Started Oct 2021View details ↗
RecruitingNCT05293184

The Global Angelman Syndrome Registry

👨‍⚕️ Helen (Honey) Heussler, MBBS, FRACP DM, The University of Queensland📍 1 site📅 Started Sep 2016View details ↗

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