A Study to Assess Choroideremia (CHM) Health Outcomes

NCT ID: NCT04750785

Last Updated: 2021-10-14

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

46 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-12-15

Study Completion Date

2021-09-27

Brief Summary

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The primary objectives of this study are to describe disease progression and severity by age in participants with CHM, to assess health-related quality-of-life, resource utilization and work productivity, and to assess quality-of-life, work productivity, and impact on daily activities in caregivers of participants with CHM at different stages of disease progression.

Detailed Description

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Conditions

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Choroideremia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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All Participants

Participants who have been diagnosed with CHM will be enrolled.

No Intervention

Intervention Type OTHER

Administered as specified in the treatment arm.

Interventions

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No Intervention

Administered as specified in the treatment arm.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

Physician Participants

1. At least 60% of time spent in direct participant care.
2. Board-certified or eligible with a Specialty in Ophthalmology, such as Retinal Specialist, IRD Specialist, Retinal Surgeon.

Patient Participants

1. CHM diagnosis confirmed via genetic testing.
2. Include any minimal disease severity requirement.
3. Participant has at least one record of visual acuity measurement in the past 24 months, OR has be assigned by the treating clinician as having severe VA impairment of blind/legally blind by using one of the following methods: finger counting, hand movement, light perception / no light perception.

Exclusion Criteria

Physician Participants

a. Participating physicians must not be affiliated with an approved management organization.

Patient Participants

a. Participants who received gene therapy or any other investigational treatment and participants who do not agree to informed consent.
Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Biogen

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Biogen

Locations

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Research Site

Los Angeles, California, United States

Site Status

Research Site

San Francisco, California, United States

Site Status

Research Site

Coral Gables, Florida, United States

Site Status

Research Site

Gainesville, Florida, United States

Site Status

Research Site

Portland, Oregon, United States

Site Status

Research Site

Pittsburgh, Pennsylvania, United States

Site Status

Research Site

Dallas, Texas, United States

Site Status

Research Site

Madison, Wisconsin, United States

Site Status

Countries

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United States

Other Identifiers

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US-CHM-11722

Identifier Type: -

Identifier Source: org_study_id