Natural History of the Progression of Choroideremia Study

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

319 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-06-30

Study Completion Date

2020-10-01

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The objective of this natural history study is to gain a better understanding of the progression of choroideremia (CHM) and add to the knowledge base for this rare disease.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Natural History Study for Charcot Marie Tooth Disease

NCT05902351

Charcot-Marie-Tooth Disease Charcot-Marie-Tooth Charcot-Marie-Tooth Disease, Type IA +45 more

RECRUITING

Genetic Analysis of Familial Hypertrophic Cardiomyopathy

NCT00005251

Cardiovascular Diseases Heart Diseases Myocardial Diseases +1 more

COMPLETED

DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac Amyloidosis

NCT02252653

Familial Amyloidotic Cardiomyopathy (FAC)

COMPLETED

Danon Disease Natural History Study

NCT06214507

Danon Disease

RECRUITING

Stratification of Arrhythmic Risk and/or Heart Failure Risk in Patients With Hereditary Heart Disease

NCT07257289

Hereditary Heart Diseases

NOT_YET_RECRUITING

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Choroideremia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Are willing and able to provide informed consent for participation in the study.
* Have a clinical phenotype and confirmed genetic diagnosis of CHM.
* Have active disease clinically visible within the macular region.
* Are willing and able to undergo ophthalmic examinations once every 4 months for up to 20 months.
* Have a BCVA better than or equal to 6/60 (20/200; decimal 0.1; LogMAR 1.0; 34-38 ETDRS letters) in at least one eye.

Exclusion Criteria

* Have a history of amblyopia in the eligible eye.
* Have any other significant ocular or non-ocular disease/disorder in the eligible eye which, in the opinion of the investigator, may put the subject at risk because of participation in the study, influence the results of the study or influence the subject's ability to participate in the study.
* Have participated in an interventional research study in the past 6 months.

Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No