Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT02723448 · ClinicalTrials.gov registry record · Phase 1
Aclarubicin for the Treatment of Retinal Vasculopathy With Cerebral Leukodystrophy
A Phase 1 study, sponsored by Washington University School of Medicine.
- Completed
- Registry status
- Phase 1
- Development phase
- 4
- Enrollment target
NCT02723448: Completed Phase 1 study, sponsored by Washington University School of Medicine.
NCT02723448 is a Phase 1 study that has completed, run by Washington University School of Medicine. The registered enrollment target is 4 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (93% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02723448, a Phase 1 study, has completed, sponsored by Washington University School of Medicine.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 4 participants
- Enrollment target
Study Summary
The goal of the investigator is to utilize Aclarubicin to treat patients with Retinal Vasculopathy with Cerebral Leukodystrophy (RVCL), a rare and devastating genetic disease with no available specific treatment. RVCL results from a mutation in the tail end of the TREX1 (Three Prime Repair Exonuclease 1) gene, a major deoxyribonucleic acid (DNA) repair enzyme. The RVCL-specific mutations cause expression of a truncated and mislocalized protein. RVCL is an inherited disorder whose symptoms begin at middle age and initially predominantly affects the eye and brain. Because it is an 'autosomal dominant' disease, it strikes both males and females equally. A person with RVCL has a 50-50 chance of transmitting the gene to each child. The investigator's published studies demonstrated in a mouse model for RVCL and in vitro studies with patients' cells that defects were corrected by use of Aclarubicin, an anthracycline antibiotic often used to treat cancer. Thus, there is a strong rationale for conducting a clinical trial of aclarubicin in patients with RVCL. The dosage to be initially administered to RVCL patients initially will be \< 10% of that typically used in cancer therapeutics and will be given monthly on four consecutive days for six months. Patients will undergo assessments every six months to determine disease response. Patients that do not have clear objective response may be dose escalated by 1 dose level with permission of the principal investigator permitting the patient has not previously experienced any toxicities requiring dose modifications. We will evaluate the safety and clinical efficacy of Aclarubicin for the treatment of RVCL and evaluate its effects on cellular function. This work will generate the first clinical research data on the investigational product's utility in treating RVCL. Patients are followed for at least 2 years upon completion of Aclarubicin administration completion. We are not longer administering the drug, but are in the post-dru
Primary Outcome
Volume increase in lesions on FLAIR MRI between baseline and six months is assessed
Interventions
- DRUG aclarubicin
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2016-12-05 |
| Est. Completion | 2020-07-23 |
| Phase | Phase 1 |
What the finished NCT02723448 record still lists
NCT02723448 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 4 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (93% lower).
The record links to 0 conditions, and to 1 intervention - of which aclarubicin is the first listed.
NCT02723448 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02723448 about?
NCT02723448 is a clinical study titled "Aclarubicin for the Treatment of Retinal Vasculopathy With Cerebral Leukodystrophy". The goal of the investigator is to utilize Aclarubicin to treat patients with Retinal Vasculopathy with Cerebral Leukodystrophy (RVCL), a rare and devastating genetic disease with no available specific treatment. RVCL results from a mutation in the tail end of the TREX1 (Three Prime Repair Exonuclea...
What is the current status of trial NCT02723448?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2016-12-05. Estimated completion is 2020-07-23.
What interventions are being tested in trial NCT02723448?
The interventions under investigation include: aclarubicin (DRUG).
Who is sponsoring clinical trial NCT02723448?
This trial is sponsored by Washington University School of Medicine, which has 1,502 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02723448's enrollment target sits among peer trials
4 1995th of 2000 higher than 2 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
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