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Angelman Syndrome Video Assessment (ASVA) Source Material Study

NCT ID: NCT05637697

Last Updated: 2023-12-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

73 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-08-31

Study Completion Date

2023-08-01

Brief Summary

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This is a longitudinal, observational, nonrandomized, fully remote study enrolling approximately 55 participant-caregiver dyads. The study includes no treatments or interventions, and participants will not be asked to change their current treatments. The objective of this study is to gather sufficient source material videos to develop a scoring system for ASVA and to perform preliminary validation of that scoring system.

Detailed Description

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Conditions

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Angelman Syndrome

Keywords

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Angelman Syndrome

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Ambulatory

No interventions assigned to this group

Limited Ambulation

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* Neurotypical individual is between 1 and 8 years of age
* Caregiver is willing and able to provide informed consent and oversee study participation and NT individual is willing and able to provide assent, as applicable
* Caregiver is comfortable reading and speaking in English
Minimum Eligible Age

1 Year

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role collaborator

Boston Children's Hospital

OTHER

Sponsor Role collaborator

The Emmes Company, LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Mindy Leffler

Role: PRINCIPAL_INVESTIGATOR

Casimir

Locations

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Casimir

Kingston, Massachusetts, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol

View Document

Other Identifiers

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CAS-CAS006

Identifier Type: -

Identifier Source: org_study_id