ChoroideremiaNCT04795206
Natural Disease Progression in Participants With Choroideremia
An observational study of Choroideremia, run by Biogen at 1 site in United States.
Trial summary
The primary objective of this study is to quantify disease progression measured by best corrected visual acuity (BCVA) in the real-world setting among Choroideremia (CHM) participants. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Choroideremia
- How many people
- 1,178 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Aug 2020to 10 Aug 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 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Main cohort will include all eligible CHM participants in IRIS registry. Trial-matched cohort will include only male CHM participants from Cohort 1 to match Biogen's IST study population using propensity score matching.
Who may be able to join
- Cohort 1: All CHM Participants
- Participants with a documented diagnosis of CHM identified by the presence of International Classification of Diseases (ICD)-9/10 or SNOMED coding:
- ICD-9: 363.55
- ICD-10: H31.2
- SNOMED-CT: 75241009
- Cohort 2: Trial-Matched CHM Participants
Show all 10Show fewer
- Male participants with a documented diagnosis of CHM identified by the presence of ICD-9/10 or SNOMED coding:
- ICD-9: 363.55
- ICD-10: H31.21
- SNOMED-CT: 75241009
Who cannot join
- N/A
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Retrospective Cohort Analysis of the Natural Disease Progression of Patients With Choroideremia in Real-World Clinical Practice
The primary objective of this study is to quantify disease progression measured by best corrected visual acuity (BCVA) in the real-world setting among Choroideremia (CHM) participants. The secondary objectives are to describe demographic and baseline clinical characteristics among CHM participants and to match CHM participants in the Intelligent Research in Sight (IRIS®) Registry to Biogen's investigator sponsored trial (IST) study population using propensity score matching.
Read the detailed description
This study is a retrospective cohort study of CHM eyes and participants which will include all eligible participants and eyes with the first documented diagnosis of CHM from July 1st, 2013 to December 31st, 2019 that meet the study criteria. This study will be conducted using data from American Academy of Ophthalmology's (AAO) IRIS® Registry which captures CHM participant's demographics, clinical characteristics and clinical outcomes from 2013.
- Conditions
- Choroideremia
- Being tested
- No Intervention
- Keywords
- X-linked inherited retinal disorder, Rare disease, Bilateral disease
- Medical subject headings
- Choroideremia, Rare Diseases
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 group | Role | What they receive |
|---|---|---|
| Cohort 1: Main Cohort | All eligible participants with CHM in IRIS Registry will be included. Receives: Other: No Intervention | |
| Cohort 2: Trial-Matched Cohort | Only male participants with CHM from Cohort 1 to match Biogen's IST study population using propensity score matching will be included. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants with Progression of Best Corrected Visual Acuity (BCVA) in Relation to Age
Measured: Up to 6 years
Percentage of Participants with Progression of BCVA Over Time After the Baseline BCVA
Measured: Up to 6 years
Secondary outcomes (2)
Percentage of Participants with Demographics Described at Baseline
The demographics will include age, sex, race, geographic region and treating provider specialty.
Measured: Up to 6 years
Matching Participants with CHM in the IRIS® Registry to Biogen's IST Study Population Using Propensity Score (PS) Matching
A propensity score matching approach will be performed to select eligible male CHM participants from IRIS Registry to match Biogen's IST study population. With the approach, the probability of participating in the IST study given the observed baseline participant characteristics will be estimated for each participant. Propensity score will be estimated using logistic regression and pre-specified baseline covariates including participants demographics and clinical characteristics. The propensity match will be considered adequate if all the variables between the PS-matched CHM cohort and IST study cohort have an absolute value of the standardized mean difference (SMD) in PS-score that is less than 0.1.
Measured: Up to 6 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2021
- Study start26 Aug 2020
- Primary completion7 May 2021
- Study completion10 Aug 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ChoroideremiaStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Retrospective
- Enrolment
- 1,178 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04795206
- Sponsor's protocol ID
- US-CHM-11761
- Status last verified
- Sep 2021
- First submitted
- 9 Mar 2021
- Last updated
- 8 Sep 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/
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Retrospective Cohort Analysis of the Natural Disease Progression of Patients With Choroideremia in Real-World Clinical Practice
- ClinicalTrials.gov ID
- NCT04795206
- Acronym / sponsor ID
- US-CHM-11761
- Conditions
- Choroideremia
- Keywords
- X-linked inherited retinal disorder, Rare disease, Bilateral disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,178 participants (actual)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 26 Aug 2020 (actual)
- Primary completion
- 7 May 2021 (actual)
- Study completion
- 10 Aug 2021 (actual)
- First posted
- 12 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to quantify disease progression measured by best corrected visual acuity (BCVA) in the real-world setting among Choroideremia (CHM) participants. The secondary objectives are to describe demographic and baseline clinical characteristics among CHM participants and to match CHM participants in the Intelligent Research in Sight (IRIS®) Registry to Biogen's investigator sponsored trial (IST) study population using propensity score matching.
Detailed description
This study is a retrospective cohort study of CHM eyes and participants which will include all eligible participants and eyes with the first documented diagnosis of CHM from July 1st, 2013 to December 31st, 2019 that meet the study criteria. This study will be conducted using data from American Academy of Ophthalmology's (AAO) IRIS® Registry which captures CHM participant's demographics, clinical characteristics and clinical outcomes from 2013.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: Main Cohort | – | Other: No InterventionAll eligible participants with CHM in IRIS Registry will be included. |
| Cohort 2: Trial-Matched Cohort | – | Other: No InterventionOnly male participants with CHM from Cohort 1 to match Biogen's IST study population using propensity score matching will be included. |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Main cohort will include all eligible CHM participants in IRIS registry. Trial-matched cohort will include only male CHM participants from Cohort 1 to match Biogen's IST study population using propensity score matching.
Key inclusion criteria (10 listed)
- Cohort 1: All CHM Participants
- Participants with a documented diagnosis of CHM identified by the presence of International Classification of Diseases (ICD)-9/10 or SNOMED coding:
- Cohort 2: Trial-Matched CHM Participants
- Male participants with a documented diagnosis of CHM identified by the presence of ICD-9/10 or SNOMED coding:
Key exclusion criteria (2 listed)
- N/A
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No InterventionAdministered as specified in the treatment arm. | Other | Cohort 1: Main Cohort, Cohort 2: Trial-Matched Cohort |
Primary outcome
Percentage of Participants with Progression of Best Corrected Visual Acuity (BCVA) in Relation to Age
Time frame: Up to 6 years
Percentage of Participants with Progression of BCVA Over Time After the Baseline BCVA
Time frame: Up to 6 years
2 secondary outcomes
Percentage of Participants with Demographics Described at Baseline
Time frame: Up to 6 years
Matching Participants with CHM in the IRIS® Registry to Biogen's IST Study Population Using Propensity Score (PS) Matching
Time frame: Up to 6 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2021.
- Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/
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Choroideremia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Biogen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Choroideremia, run by Biogen at 1 site 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 10 inclusion and 2 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 8 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Percentage of Participants with Progression of Best Corrected Visual Acuity (BCVA) in Relation to Age (time frame: Up to 6 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Sep 2021.