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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04750785
Recruitment Status : Completed
First Posted : February 11, 2021
Last Update Posted : October 14, 2021
Sponsor:
Information provided by (Responsible Party):
Biogen

Tracking Information
First Submitted Date February 8, 2021
First Posted Date February 11, 2021
Last Update Posted Date October 14, 2021
Actual Study Start Date December 15, 2020
Actual Primary Completion Date September 27, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: February 8, 2021)
  • Time to Disease Progression by Age in Participants with CHM [ Time Frame: Up to 8 months ]
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Study to Assess Choroideremia (CHM) Health Outcomes
Official Title Choroideremia Health Outcomes
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.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.

Condition Choroideremia
Intervention Other: No Intervention
Administered as specified in the treatment arm.
Study Groups/Cohorts All Participants
Participants who have been diagnosed with CHM will be enrolled.
Intervention: Other: No Intervention
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: October 13, 2021)
46
Original Estimated Enrollment
 (submitted: February 8, 2021)
50
Actual Study Completion Date September 27, 2021
Actual Primary Completion Date September 27, 2021   (Final data collection date for primary outcome measure)
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.

Sex/Gender
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT04750785
Other Study ID Numbers US-CHM-11722
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
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/
Responsible Party Biogen
Study Sponsor Biogen
Collaborators Not Provided
Investigators
Study Director: Medical Director Biogen
PRS Account Biogen
Verification Date October 2021