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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 is a Phase 1 study that has completed, run by Washington University School of Medicine. The registered enrollment target is 4 participants.
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
Interventions
- DRUG aclarubicin
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2016-12-05 |
| Est. Completion | 2020-07-23 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02723448
The ClinicalTrials.gov registry entry for NCT02723448 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Washington University School of Medicine, which has 1,502 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which aclarubicin is the first listed.
NCT02723448 reports 0 study locations.
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.
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