Clinical trial NCT04750785: A Study to Assess Choroideremia (CHM) Health Outcomes
Official title: Choroideremia Health Outcomes
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Choroideremia
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2021-02-11
Enrollment: 46
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants who have been diagnosed with CHM. A minimum of approximately 50% of the participants will have an associated caregiver who will participate.

Participating physicians will enroll participants for inclusion in the study.

Brief 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.


Eligibility 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.

Primary outcomes:
measure: Time to Disease Progression by Age in Participants with CHM
timeFrame: Up to 8 months
measure: Best Corrected Visual Acuity by Age
timeFrame: Up to 8 months
measure: Participants with CHM: EuroQol- 5 Dimension- 5 Level (EQ-5D-5L) Score
timeFrame: Up to 8 months
measure: Participants with CHM: Health Utilities Index (HUI3) Score
timeFrame: Up to 8 months
measure: Participants with CHM: Work Productivity and Activity Impairment (WPAI) Score
timeFrame: Up to 8 months
measure: Participants with CHM: Visual Function Questionnaire (VFQ-25) Score
timeFrame: Within 8 months
measure: Resource Utilization in Participants with CHM
timeFrame: Up to 8 months
measure: Caregivers of Participants With CHM: Caregiver-reported Depression Via Patient Health Questionnaire-9 (PHQ-9) Score
timeFrame: Up to 8 months
measure: Caregivers of Participants With CHM: Caregiver-reported Anxiety Via General Anxiety Disorder-7 (GAD-7) Score
timeFrame: Up to 8 months
measure: Caregivers of Participants With CHM: Caregiver Reaction Assessment (CRA) Score
timeFrame: Up to 8 months
measure: Caregivers of Participants With CHM: Work Productivity and Activity Impairment Caregiver (WPAI-CG) Score
timeFrame: Up to 8 months
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Research Site
state: California
city: San Francisco
country: United States
facility: Research Site
state: California
city: Coral Gables
country: United States
facility: Research Site
state: Florida
city: Gainesville
country: United States
facility: Research Site
state: Florida
city: Portland
country: United States
facility: Research Site
state: Oregon
city: Pittsburgh
country: United States
facility: Research Site
state: Pennsylvania
city: Dallas
country: United States
facility: Research Site
state: Texas
city: Madison
country: United States
facility: Research Site
state: Wisconsin
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: