Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study to Assess Choroideremia (CHM) Health Outcomes

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04750785
Recruitment Status : Recruiting
First Posted : February 11, 2021
Last Update Posted : July 2, 2021
Sponsor:
Information provided by (Responsible Party):
Biogen

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.

Condition or disease Intervention/treatment
Choroideremia Other: No Intervention

Layout table for study information
Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Choroideremia Health Outcomes
Actual Study Start Date : December 15, 2020
Estimated Primary Completion Date : July 30, 2021
Estimated Study Completion Date : September 30, 2021

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
All Participants
Participants who have been diagnosed with CHM will be enrolled.
Other: No Intervention
Administered as specified in the treatment arm.




Primary Outcome Measures :
  1. Time to Disease Progression by Age in Participants with CHM [ Time Frame: Up to 8 months ]
  2. Best Corrected Visual Acuity by Age [ Time Frame: Up to 8 months ]
    Best corrected visual acuity will be used as an indicator to assess disease severity.

  3. Participants with CHM: EuroQol- 5 Dimension- 5 Level (EQ-5D-5L) Score [ Time Frame: Up to 8 months ]
    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.

  4. Participants with CHM: Health Utilities Index (HUI3) Score [ Time Frame: Up to 8 months ]
    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.

  5. Participants with CHM: Work Productivity and Activity Impairment (WPAI) Score [ Time Frame: Up to 8 months ]
    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.

  6. Participants with CHM: Visual Function Questionnaire (VFQ-25) Score [ Time Frame: Within 8 months ]
    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.

  7. Resource Utilization in Participants with CHM [ Time Frame: Up to 8 months ]
    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.

  8. Caregivers of Participants With CHM: Caregiver-reported Depression Via Patient Health Questionnaire-9 (PHQ-9) Score [ Time Frame: Up to 8 months ]
    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.

  9. Caregivers of Participants With CHM: Caregiver-reported Anxiety Via General Anxiety Disorder-7 (GAD-7) Score [ Time Frame: Up to 8 months ]
    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.

  10. Caregivers of Participants With CHM: Caregiver Reaction Assessment (CRA) Score [ Time Frame: Up to 8 months ]
    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.

  11. Caregivers of Participants With CHM: Work Productivity and Activity Impairment Caregiver (WPAI-CG) Score [ Time Frame: Up to 8 months ]
    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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
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.

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04750785


Contacts
Layout table for location contacts
Contact: US Biogen Clinical Trial Center 866-633-463 clinicaltrials@biogen.com
Contact: Global Biogen Clinical Trial Center clinicaltrials@biogen.com

Locations
Layout table for location information
United States, California
Research Site Recruiting
Los Angeles, California, United States, 90095
Research Site Recruiting
San Francisco, California, United States, 94158
United States, Florida
Research Site Not yet recruiting
Coral Gables, Florida, United States, 33146
Research Site Recruiting
Gainesville, Florida, United States, 32607
United States, Oregon
Research Site Not yet recruiting
Portland, Oregon, United States, 97239
United States, Pennsylvania
Research Site Recruiting
Pittsburgh, Pennsylvania, United States, 15213
United States, Texas
Research Site Recruiting
Dallas, Texas, United States, 97239
United States, Wisconsin
Research Site Recruiting
Madison, Wisconsin, United States, 53705
Sponsors and Collaborators
Biogen
Investigators
Layout table for investigator information
Study Director: Medical Director Biogen
Layout table for additonal information
Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT04750785    
Other Study ID Numbers: US-CHM-11722
First Posted: February 11, 2021    Key Record Dates
Last Update Posted: July 2, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/
URL: https://vivli.org/

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Choroideremia
Eye Diseases, Hereditary
Eye Diseases
Choroid Diseases
Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked