ChoroideremiaNCT02994368
"Natural History" Study of Choroideremia
An observational study of Choroideremia, run by 4D Molecular Therapeutics at 4 sites in 2 countries.
Trial summary
The purpose of this study is to understand the rate of progression of all stages of choroideremia using a variety of assessments performed in the clinic including visual field measures, specialized photography of the eye and participant-reported visual problems Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Choroideremia
- What is being tested?
- Observation
- How many people
- 57 participants
- Where
- 4 sitesin 2 countries
- Study dates
- Dec 2016to Oct 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 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Individuals with a clinical diagnosis of choroideremia
Who may be able to join
- Healthy individuals (at least 14 years) with choroideremia (20/200 or better vision) willing to participate in an observational study and meeting the eligibility criteria.
- Healthy individuals (at least 18 years) with choroideremia (20/200 or better vision in both eyes) willing to participate in an observational study and meeting the eligibility criteria.
Who cannot join
- Prior therapy with an AAV vector-based treatment
- Pre-existing eye conditions that would: (1) preclude future planned treatment in a therapeutic intent clinical trial (i.e. intravitreal injection), (2) interfere with the interpretation of study endpoints, and/ or put patient at risk for surgical complications
- Complicating systemic diseases that would preclude future enrollment in a therapeutic intent clinical trial
- Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study.
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
United States3 sites
Retina Foundation of the Southwest
Dallas, Texas, 75251
Baylor College of Medicine, Ophthalmology-Cullen Eye Center
Houston, Texas, 77030
Moran Eye Center, University of Utah
Salt Lake City, Utah, 84132
About this study
Official title: A Multicenter Prospective Observational "Natural History" Study in Patients With Choroideremia
The purpose of this study is to understand the rate of progression of all stages of choroideremia using a variety of assessments performed in the clinic including visual field measures, specialized photography of the eye and participant-reported visual problems
- Conditions
- Choroideremia
- Being tested
- Observation
- Keywords
- retinal degeneration, eye, REP-1 gene
- Medical subject headings
- Choroideremia, Retinal Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ObservationOther
| Study group | Role | What they receive |
|---|---|---|
| Primary Cohort | No intervention Receives: Other: Observation | |
| Expansion Cohort | No intervention Receives: Other: Observation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of progression of disease
A variety of psychophysical, anatomical and image-based endpoints
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2016 (estimated)
- Study startDec 2016
- Primary completionOct 2021
- Study completionOct 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 4D Molecular TherapeuticsThe 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
- Prospective
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- 4D Molecular Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Schonmei Lee, MD, Study director, 4D Molecular Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02994368
- Sponsor's protocol ID
- 4D-CHM-001-NH-0001
- Status last verified
- Jan 2022
- First submitted
- 13 Dec 2016
- Last updated
- 6 Jan 2022
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Prospective Observational "Natural History" Study in Patients With Choroideremia
- ClinicalTrials.gov ID
- NCT02994368
- Acronym / sponsor ID
- 4D-CHM-001-NH-0001
- Conditions
- Choroideremia
- Keywords
- retinal degeneration, eye, REP-1 gene
- Study type
- Observational
- Recruitment status
- Terminated
- Enrolment
- 57 participants (actual)
- Lead sponsor
- 4D Molecular TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Dec 2016
- Primary completion
- Oct 2021 (actual)
- Study completion
- Oct 2021 (actual)
- First posted
- 15 Dec 2016 (estimated)
- 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 purpose of this study is to understand the rate of progression of all stages of choroideremia using a variety of assessments performed in the clinic including visual field measures, specialized photography of the eye and participant-reported visual problems
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Primary Cohort | – | Other: ObservationNo intervention |
| Expansion Cohort | – | Other: ObservationNo intervention |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Individuals with a clinical diagnosis of choroideremia
Key inclusion criteria (2 listed)
- Healthy individuals (at least 14 years) with choroideremia (20/200 or better vision) willing to participate in an observational study and meeting the eligibility criteria.
- Healthy individuals (at least 18 years) with choroideremia (20/200 or better vision in both eyes) willing to participate in an observational study and meeting the eligibility criteria.
Key exclusion criteria (4 listed)
- Prior therapy with an AAV vector-based treatment
- Pre-existing eye conditions that would: (1) preclude future planned treatment in a therapeutic intent clinical trial (i.e. intravitreal injection), (2) interfere with the interpretation of study endpoints, and/ or put patient at risk for surgical complications
- Complicating systemic diseases that would preclude future enrollment in a therapeutic intent clinical trial
- Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study.
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 |
|---|---|---|
| Observation | Other | Expansion Cohort, Primary Cohort |
Primary outcome
Rate of progression of disease
Time frame: 4 years
A variety of psychophysical, anatomical and image-based endpoints
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jan 2022.
- Individual participant dataSharing planned: No
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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.
Schonmei Lee, MD study director
4D Molecular Therapeutics
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Choroideremia, run by 4D Molecular Therapeutics at 4 sites in 2 countries.
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 2 inclusion and 4 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: stopped early, as of the sponsor's last update on 6 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Canada. 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 was stopped early. Results are not available in the source record.
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?
Rate of progression of disease (time frame: 4 years). 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 6 Jan 2022.