Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT01736293 · ClinicalTrials.gov registry record
Natural History of Eye Diseases Related to ABCA4 Mutations
A clinical trial of Retinal Degeneration and ABCA4-Related Retinopathies, sponsored by National Eye Institute (NEI).
- Active
- Registry status
- 68
- Enrollment target
- 1
- Study location
NCT01736293: Active study of Retinal Degeneration and ABCA4-Related Retinopathies, sponsored by National Eye Institute (NEI).
NCT01736293 is a study of Retinal Degeneration and ABCA4-Related Retinopathies that is active but no longer recruiting, run by National Eye Institute (NEI). The registered enrollment target is 68 participants, below the 337-participant average among 17 other Retinal Degeneration trials with a reported enrollment target (80% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01736293, a study of Retinal Degeneration and ABCA4-Related Retinopathies, is active but no longer recruiting, sponsored by National Eye Institute (NEI).
- ACTIVE NOT RECRUITING
- Registry status
- 68 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: \- The ABCA4 gene contains a blueprint for the ABCA4 protein. When this protein is absent or faulty (such as in Stargardt s disease), waste material from dead cells collects in the eye. The waste material may cause other cells in the eye to die. This can lead to the loss of vision. Researchers want to look at blood and skin samples from people with ABCA4 gene mutations to study related eye diseases. Objectives: \- To study eye diseases that are related to mutations in the ABCA4 gene. Eligibility: \- Individuals at least 12 years of age who have ABCA4 gene mutations. Design: * The study requires 12 visits to the National Eye Institute clinic over 10 years. In the first year, there will be three visits. After the first year, participants will have one visit a year for 9 more years. * Participants will be screened with a physical exam, full eye exam, and medical history. The eye exam will check eye pressure, light and color sensitivity, and retina function. * Participants will provide a blood sample and a skin tissue sample for study. * No treatment will be provided as part of this study.
Primary Outcome
Establish a cohort of participants with ABCA4 retinopathies in anticipation of future clinical trials.
Conditions Studied
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 68 participants |
| Start Date | 2012-10-09 |
What the registry record for NCT01736293 still lists
NCT01736293 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 68 participants, a relatively small participant target, below the 337-participant average among 17 other Retinal Degeneration trials with a reported enrollment target (80% lower).
The record links to 2 conditions, with Retinal Degeneration appearing as the primary indexed condition, and to 0 interventions.
NCT01736293 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT01736293 about?
NCT01736293 is a clinical study titled "Natural History of Eye Diseases Related to ABCA4 Mutations". Background: \- The ABCA4 gene contains a blueprint for the ABCA4 protein. When this protein is absent or faulty (such as in Stargardt s disease), waste material from dead cells collects in the eye. The waste material may cause other cells in the eye to die. This can lead to the loss of vision. Rese...
What is the current status of trial NCT01736293?
This trial is currently active not recruiting. The enrollment target is 68 participants. The study started on 2012-10-09.
What conditions does trial NCT01736293 study?
This clinical trial studies the following conditions: Retinal Degeneration, ABCA4-Related Retinopathies.
Who is sponsoring clinical trial NCT01736293?
This trial is sponsored by National Eye Institute (NEI), which has 221 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01736293 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Retinal Degeneration
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT01736293's enrollment target sits among peer trials
68 7th of 17 higher than 11 of 17 other Retinal Degeneration trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Retinal Degeneration trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
SGLT2 Inhibitors in Geographic Atrophy
RECRUITING · Phase 2
-
Cell Collection to Study Eye Diseases
RECRUITING
-
A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001
RECRUITING · Phase 1
-
Adaptive Optics Imaging of Outer Retinal Diseases
RECRUITING
-
Rod and Cone Mediated Function in Retinal Disease
RECRUITING
-
Oral Metformin for Treatment of ABCA4 Retinopathy
RECRUITING · Phase 1
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02044497 · 68 participants · Phase 4
Population Pharmacokinetics and Pharmacogenomics of Oral Oxycodone in Pediatric Surgical Patients
- NCT03779854 · 68 participants · Phase 2
Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant
- NCT03821246 · 68 participants · Phase 2
Neoadjuvant Atezolizumab-Based Combination Therapy in Men With Localized Prostate Cancer Prior to Radical Prostatectomy
- NCT04115514 · 68 participants · Phase 2
Treatment of ARDS With Instilled T3
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI