A Study to Assess Choroideremia (CHM) Health Outcomes

Biogen

Status: Completed

Conditions: Choroideremia

Treatments: Other: No Intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT04750785

US-CHM-11722

Details and patient eligibility

About

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.

Enrollment: 46 patients

Sex: Male

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key 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.

Key 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.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

46 participants in 1 patient group

All Participants

Description:

Participants who have been diagnosed with CHM will be enrolled.

Treatment:

Other: No Intervention