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ChoroideremiaNCT04750785

A Study to Assess Choroideremia (CHM) Health Outcomes

An observational study of Choroideremia, run by Biogen at 8 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Any age, men only
Conditions the study is for
Choroideremia
How many people
46 participants
Where
8 sitesUnited States
Study dates
15 Dec 2020to 27 Sep 2021
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

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.

Who may be able to join

  • Physician Participants
  • 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.
  • Patient Participants
  • 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.

Who cannot join

  • Physician Participants
  • Participating physicians must not be affiliated with an approved management organization.
  • Patient Participants
  • 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 locations and contacts

8 sites in 1 country.

8 sites in 1 country

United States8 sites

  • Research Site

    Coral Gables, Florida, 33146

  • Research Site

    Dallas, Texas, 97239

  • Research Site

    Gainesville, Florida, 32607

  • Research Site

    Los Angeles, California, 90095

  • Research Site

    Madison, Wisconsin, 53705

  • Research Site

    Pittsburgh, Pennsylvania, 15213

  • Research Site

    Portland, Oregon, 97239

  • Research Site

    San Francisco, California, 94158

Other trials in United States

About this study

Official title: Choroideremia Health Outcomes

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.

Conditions
Choroideremia
Being tested
No Intervention
Medical subject headings
Choroideremia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • No InterventionOther

    Administered as specified in the treatment arm.

Study groups in NCT04750785
Study groupRoleWhat they receive
All ParticipantsParticipants who have been diagnosed with CHM will be enrolled.
Receives: Other: No Intervention

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Time to Disease Progression by Age in Participants with CHM

    Measured: Up to 8 months

  2. Best Corrected Visual Acuity by Age

    Best corrected visual acuity will be used as an indicator to assess disease severity.

    Measured: Up to 8 months

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

    EQ-5D is a self-report measure of health for clinical and economic appraisal. Participants complete the 5-level (no problems, slight problems, moderate problems, severe problems, and extreme problems), 5-dimension (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) questionnaire concerning their current health state. A unique EQ-5D-5L health state scale ranges from 0 to 100 and is defined by combining 1 level from each of the 5 dimensions. Participants indicate their current health status by marking on a continuum ranging from 100 (best imaginable health state) to 0 (worst imaginable health state). The questionnaire will be implemented only one time per person in the study duration.

    Measured: Up to 8 months

  4. Participants with CHM: Health Utilities Index (HUI3) Score

    Health Utilities Index (HUI3) is a family of generic preference-based systems for measuring comprehensive health status and health-related quality of life (HRQL). HUI® provides descriptive evidence on multiple dimensions of health status, a score for each dimension of health, and a HRQL score for overall health. Health dimensions include vision, hearing, speech, ambulation/mobility, pain, dexterity, self-care, emotion and cognition. Each dimension has 3- 6 levels. HUI® systems describe almost a million unique health states. HUI3 score ranges from 0.36 (worst) to 1 (best). The questionnaire will be implemented only one time per person in the study duration.

    Measured: Up to 8 months

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

    The Work Productivity and Activity Impairment (WPAI) is a 6 questionnaire scale used to measure lost work productivity and impairment in daily activities over the past seven days. The WPAI yields four types of scores: 1. Absenteeism 2. Presenteesism 3. Work productivity loss 4. Activity Impairment, ranging from 0-100%. Higher numbers indicate greater impairment and less productivity. The questionnaire will be implemented only one time per person in the study duration.

    Measured: Up to 8 months

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

    VFQ-25 is a reliable and valid 25-item version of the 51-item National Eye Institute Visual Function Questionnaire (NEI-VFQ). It is especially useful in settings such as clinical trials, where interview length is a critical consideration. The global score derives from the summary of the scores deriving from each question. The total score has a minimum value "0" considered as the worst visual functionality, and the maximum value "100" considered as the better visual functionality. The questionnaire will be implemented only one time per person in the study duration.

    Measured: Within 8 months

  7. Resource Utilization in Participants with CHM

    Healthcare Resource Utilization will be assessed by number of primary care provider visits, specialist visits, urgent care visits, emergency room (ER) visits, hospitalizations, and rehabilitation visits in past 12 months when stratified by all-cause \& CHM-related.

    Measured: Up to 8 months

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

    PHQ-9 can be used to screen for or diagnose depression, as well as measure depression severity. The PHQ-9 measures frequency of depression symptoms, with items scored on a 4-point scale (not at all=0 to nearly every day=3). The total score ranges from 0-27 where 0 is no depression and 27 is severe depression. The questionnaire will be implemented only one time per person in the study duration.

    Measured: Up to 8 months

  9. Caregivers of Participants With CHM: Caregiver-reported Anxiety Via General Anxiety Disorder-7 (GAD-7) Score

    GAD-7 is a 7-item questionnaire that is used for screening, diagnosing, monitoring, and measuring the severity of anxiety. Each item can be answered on a 4 point scale running from 0= 'not at all sure' to 3= 'nearly every day'. The total score ranges from 0-21 where 0 is no anxiety and 27 is severe anxiety. The questionnaire will be implemented only one time per person in the study duration.

    Measured: Up to 8 months

  10. Caregivers of Participants With CHM: Caregiver Reaction Assessment (CRA) Score

    Caregiver Reaction Assessment is a 24-item instrument assessing positive and negative aspects of caregiving (esteem, lack of family support, finances, schedule, and health). Each item is rated on a 1 to 4 scale. 1 (not at all) 2 (somewhat) 3 (quite a bit) 4 (completely). The questionnaire will be implemented only one time per person in the study duration.

    Measured: Up to 8 months

  11. Caregivers of Participants With CHM: Work Productivity and Activity Impairment Caregiver (WPAI-CG) Score

    WPAI-CG is a 6 questionnaire scale used to measure lost work productivity and impairment in daily activities over the past seven days. The WPAI yields four types of scores: 1. Absenteeism 2. Presenteesism 3. Work productivity loss 4. Activity Impairment, ranging from 0-100%. Higher numbers indicate greater impairment and less productivity. The questionnaire will be implemented only one time per person in the study duration.

    Measured: Up to 8 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Feb 2021
  2. Study start15 Dec 2020
  3. Primary completion27 Sep 2021
  4. Study completion27 Sep 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
46 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04750785
Sponsor's protocol ID
US-CHM-11722
Status last verified
Oct 2021
First submitted
8 Feb 2021
Last updated
14 Oct 2021
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/

Questions about this trial

What is this clinical trial studying?

An observational study of Choroideremia, run by Biogen at 8 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 14 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Time to Disease Progression by Age in Participants with CHM (time frame: Up to 8 months). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04750785.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2021.

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