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NCT06270316 · ClinicalTrials.gov registry record · Phase 1
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
A Phase 1 study of Fabry Disease, sponsored by UniQure Biopharma B.V..
- Recruiting
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 8
- Study locations
NCT06270316: Recruiting Phase 1 study of Fabry Disease, sponsored by UniQure Biopharma B.V..
NCT06270316 is a Phase 1 study of Fabry Disease that is actively recruiting participants, run by UniQure Biopharma B.V.. The registered enrollment target is 12 participants, below the 542-participant average among 32 other Fabry Disease trials with a reported enrollment target (98% lower). The trial reports 8 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06270316, a Phase 1 study of Fabry Disease, is actively recruiting participants, sponsored by UniQure Biopharma B.V..
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 8
- Study locations
Study Summary
The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.
Conditions Studied
Interventions
- DRUG AMT-191
Study Locations (8)
Alabama
- The Kirklin Clinic Of university of Alabama Birmingham Hospital - Birmingham
Georgia
- Emory University School of Medicine - Atlanta
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Minnesota
- MHealth Fairview University of Minnesota Medical Center East Bank - Minneapolis
New York
- NYC Health + Hospitals/Metropolitan - New York
Pennsylvania
- UPMC Children's Hospital of Pittsburgh - Pittsburgh
Utah
- University of Utah, Clinical and Translational Sciences Institute - Salt Lake City
Virginia
- Lysosomal & Rare Disorders Research and Treatment Center, Inc - Fairfax
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2024-06-05 |
| Est. Completion | 2031-04-30 |
| Phase | Phase 1 |
What NCT06270316 shows while recruiting
NCT06270316 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 542-participant average among 32 other Fabry Disease trials with a reported enrollment target (98% lower).
The record links to 1 condition, with Fabry Disease appearing as the primary indexed condition, and to 1 intervention - of which AMT-191 is the first listed.
NCT06270316 lists 8 locations in 8 states (Alabama, Georgia, Illinois).
Frequently Asked Questions
What is clinical trial NCT06270316 about?
NCT06270316 is a clinical study titled "Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease". The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.
What is the current status of trial NCT06270316?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2024-06-05. Estimated completion is 2031-04-30.
What conditions does trial NCT06270316 study?
This clinical trial studies the following conditions: Fabry Disease.
What interventions are being tested in trial NCT06270316?
The interventions under investigation include: AMT-191 (DRUG).
Who is sponsoring clinical trial NCT06270316?
This trial is sponsored by UniQure Biopharma B.V., which has 4 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06270316 being conducted?
This trial has 8 study locations across Alabama, Georgia, Illinois, Minnesota, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT06270316's enrollment target sits among peer trials
12 29th of 32 higher than 3 of 32 other Fabry Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Fabry Disease 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.
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