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

Sex

MALE

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

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

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

Sex

MALE

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