CompletedNot Applicable

A Study to Assess Choroideremia (CHM) Health Outcomes

United States46 participantsStarted 2020-12-15

Plain-language summary

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.

Who can participate

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. At least 60% of time spent in direct participant care.
✓. Board-certified or eligible with a Specialty in Ophthalmology, such as Retinal Specialist, IRD Specialist, Retinal Surgeon.
✓. CHM diagnosis confirmed via genetic testing.
✓. Include any minimal disease severity requirement.
✓. 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.

What they're measuring

Time to Disease Progression by Age in Participants with CHM

Timeframe: Up to 8 months

Best Corrected Visual Acuity by Age

Timeframe: Up to 8 months

Participants with CHM: EuroQol- 5 Dimension- 5 Level (EQ-5D-5L) Score

Timeframe: Up to 8 months

Participants with CHM: Health Utilities Index (HUI3) Score

Timeframe: Up to 8 months

Participants with CHM: Work Productivity and Activity Impairment (WPAI) Score

Timeframe: Up to 8 months

Participants with CHM: Visual Function Questionnaire (VFQ-25) Score

Timeframe: Within 8 months

Resource Utilization in Participants with CHM

Timeframe: Up to 8 months

Caregivers of Participants With CHM: Caregiver-reported Depression Via Patient Health Questionnaire-9 (PHQ-9) Score

Timeframe: Up to 8 months

Trial details

NCT IDNCT04750785

SponsorBiogen

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2021-09-27

View on ClinicalTrials.gov More Choroideremia trials

Plain-language summary

Who can participate

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. At least 60% of time spent in direct participant care.
✓. Board-certified or eligible with a Specialty in Ophthalmology, such as Retinal Specialist, IRD Specialist, Retinal Surgeon.
✓. CHM diagnosis confirmed via genetic testing.
✓. Include any minimal disease severity requirement.
✓. 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.

What they're measuring

Time to Disease Progression by Age in Participants with CHM

Timeframe: Up to 8 months

Best Corrected Visual Acuity by Age

Timeframe: Up to 8 months

Participants with CHM: EuroQol- 5 Dimension- 5 Level (EQ-5D-5L) Score

Timeframe: Up to 8 months

Participants with CHM: Health Utilities Index (HUI3) Score

Timeframe: Up to 8 months

Participants with CHM: Work Productivity and Activity Impairment (WPAI) Score

Timeframe: Up to 8 months

Participants with CHM: Visual Function Questionnaire (VFQ-25) Score

Timeframe: Within 8 months

Resource Utilization in Participants with CHM

Timeframe: Up to 8 months

Caregivers of Participants With CHM: Caregiver-reported Depression Via Patient Health Questionnaire-9 (PHQ-9) Score

Timeframe: Up to 8 months