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NCT03591757 · ClinicalTrials.gov registry record · Early Phase 1
Short-term Effects of TOLCAPONE on Transthyretin Stability in Subjects With Leptomeningeal TTR Amyloidosis (ATTR)
A Early Phase 1 study, sponsored by Boston University.
- Completed
- Registry status
- Early Phase 1
- Development phase
- 10
- Enrollment target
NCT03591757 is a Early Phase 1 study that has completed, run by Boston University. The registered enrollment target is 10 participants.
The verdict
NCT03591757, a Early Phase 1 study, has completed, sponsored by Boston University.
- COMPLETED
- Registry status
- Early Phase 1
- Development phase
- 10 participants
- Enrollment target
Study Summary
The purpose of this study is to determine whether Tolcapone crosses from the blood stream into the fluid around the brain and stabilizes the protein that makes leptomeningeal amyloid. Tolcapone is a commercially available generic drug that treats Parkinson's disease. The Investigator plans to evaluate Tolcapone as a treatment for ATTR (Transthyretin Amyloidosis), a rare genetic disease often causing death within 5-15 years after diagnosis. ATTR is characterized by deposition of misfolded protein known as amyloid, in one or more organ systems (including the peripheral and autonomic nervous systems, the heart, the brain and the eyes). The age at which symptoms begin to develop varies widely ranging between 20 to 70 years old. ATTR is progressive, and some variants can have a fatal outcome within a few years of presentation. Treatment options include supportive and symptomatic care that may slow or stop progressive decline in functional state but do not alter the pathological process. Liver transplant can be performed in selected patients but is limited by organ supply, requires lifelong immunosuppression, and may be complicated by progressive heart and nerve amyloid deposition. Importantly, liver transplant does not alter the natural course of central nervous system amyloid disease. To date, no treatment for ATTR penetrates the CNS. At present there is no FDA approved treatment for ATTR amyloidosis in the US. In Europe, Tafamidis has been approved for treatment of stage 1 ATTR-polyneuropathy since 2012. Tafamidis and Tolcapone bind to the thyroxine binding site of TTR (with different drug-transthyretin interactions) and in so doing stabilizes the tetrameric form of TTR, preventing dissociation and amyloid fibril formation The preclinical and clinical data from a variety of experimental systems support the therapeutic activity of TOLCAPONE in TTR mediated disease.
Interventions
- DRUG Tolcapone
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2018-10-30 |
| Est. Completion | 2019-04-26 |
| Phase | Early Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03591757
The ClinicalTrials.gov registry entry for NCT03591757 describes a study currently listed as completed, categorized as Early Phase 1. The registered enrollment target is 10 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Boston University, which has 185 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Tolcapone is the first listed.
NCT03591757 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT03591757 about?
NCT03591757 is a clinical study titled "Short-term Effects of TOLCAPONE on Transthyretin Stability in Subjects With Leptomeningeal TTR Amyloidosis (ATTR)". The purpose of this study is to determine whether Tolcapone crosses from the blood stream into the fluid around the brain and stabilizes the protein that makes leptomeningeal amyloid. Tolcapone is a commercially available generic drug that treats Parkinson's disease. The Investigator plans to evalu...
What is the current status of trial NCT03591757?
This trial is currently completed. It is a Early Phase 1 study. The enrollment target is 10 participants. The study started on 2018-10-30. Estimated completion is 2019-04-26.
What interventions are being tested in trial NCT03591757?
The interventions under investigation include: Tolcapone (DRUG).
Who is sponsoring clinical trial NCT03591757?
This trial is sponsored by Boston University, which has 185 total clinical trials registered on ClinicalTrials.gov.
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