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RECRUITING Phase 1

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCT06270316 · View on ClinicalTrials.gov ↗

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

FieldValue
Enrollment Target 12 participants
Start Date 2024-06-05
Est. Completion 2031-04-30
Phase Phase 1

Sponsor

UniQure Biopharma B.V.

4 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT06270316

The ClinicalTrials.gov registry entry for NCT06270316 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is UniQure Biopharma B.V., which has 4 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT06270316 reports 8 study locations spanning 8 distinct geographic areas — top geographies include Alabama, Georgia, Illinois. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT06270316?

The interventions under investigation include: AMT-191 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

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. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial