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BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.

ConditionAngelman Syndrome
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age1-50
SponsorOHB Pediatrics Ltd.

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Male or female and ≥ 1 year to ≤ 50 years of age at signing of the informed consent form.

Able to consent for the participant according to ICH and local regulations,

At least 18 years of age,

Willing and able to accompany the participant to clinic visits and be available to the investigational site by telephone, email, or other electronic form as needed,

Disqualifiers

Uniparental paternal disomy (UPD) of 15q11-q13,

Imprinting center defect (ICD) within 15q11-q13,

A partial molecular diagnosis of AS, that cannot exclude UPD or ICD despite appropriate genetic testing.

Presence of clinically relevant electrocardiogram (ECG) abnormalities prior to dosing such as QT interval corrected for heart rate using Fredericia's formula (QTcF) > 460 ms, personal or family history of congenital long QT syndrome indicating safety risk in the Investigator's opinion. First-degree atrioventricular block or isolated right bundle branch block is allowed.

Trial design

Design model

Parallel

Treatments tested in this trial

  • rugonersen

    Drug

    Study Drug

  • Sham procedure

    Procedure/Surgery

    Sham Procedure

Treatment groups

165 Participants
are divided into 2 treatment groups
Group A: rugonersenExperimental treatment 1 intervention
Group B: ShamSham comparator 1 intervention

Trial outcomes

Primary outcomes

1

Change from baseline in the Bayley-4 cognition and/or expressive communication raw scores without caregiver input at Week 56.

The Bayley-4 is a performance-based assessment of developmental functioning of five core battery scales: cognitive, language (two subtests: expressive and receptive communication), motor (two subtests: gross and fine motor), social-emotional, and adaptive behavior. Change from baseline in the raw scores of the cognition and/or expressive communication scales of the Bayley-4, without caregiver input, higher change reflects a better outcome of the core scales.

Time frame
Baseline to week 56

Secondary outcomes

1

Symptoms of Angelman Syndrome - Clinician Global Impression of Change (SAS-CGI-C) overall at Week 56.

The SAS-CGI-C is a Symptoms of Angelman Syndrome - Clinician Global Impression of Change that has been developed for Angelman Syndrome. The SAS-CGI-C is a nine domain, clinician-rated measure, which assesses the clinician's impression of the severity of a participant's condition ranging from "very much improved" (1) to "very much worse" (7).

Time frame
Week 56
2

Change from baseline in electroencephalogram (EEG) delta-band power at Week 56.

Change from baseline in electroencephalogram (EEG) delta-band power at Week 56.

Time frame
Week 56
3

Incidence of serious adverse events (SAEs).

Incidence of serious adverse events (SAEs).

Time frame
Week 60

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

OHB Pediatrics Ltd.

Lead sponsor

Medpace, Inc.

Collaborator

Source ClinicalTrials.gov
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