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Phase III Clinical Study of Rugonersen in Angelman Syndrome.

NCT07605429 · Status: NOT_YET_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 165

Last updated 2026-05-26

No results posted yet for this study

Summary

Purpose of the study is to evaluate the efficacy and safety of intrathecally administered rugonersen in pediatric and adult participants with Angelman syndrome.

Conditions

Interventions

DRUG

rugonersen

Study Drug

PROCEDURE

Sham Control

Sham Procedure

Sponsors & Collaborators

  • Medpace, Inc.

    collaborator INDUSTRY

  • OHB Pediatrics Ltd.

    lead INDUSTRY

Principal Investigators

  • Brenda Vincenzi, MD · OHB Pediatrics Ltd.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
1 Year
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-30
Primary Completion
2029-03-31
Completion
2031-03-31
FDA Drug
Yes

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07605429 on ClinicalTrials.gov