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NCT06270316RECRUITINGIRB Ready

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

Sponsored by UniQure Biopharma B.V.

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...
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1 of 12 participants interested
8% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

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.

Who Can Participate

Inclusion Criteria

Male of age β‰₯ 18 years and ≀50 years
Confirmed clinical diagnosis of classic Fabry disease (FD) defined as: 1. Absent or minimal Ξ±GAL A enzyme activity \< 1% of mean normal measured in plasma regardless of variant status; OR 2. Ξ±-galactosidase A (GLA) pathogenic or likely pathogenic variant associated with classic FD phenotype identified on molecular genetic testing with plasma Ξ±GLA A enzyme activity below lower bound of the reference range (as measured at trough enzyme replacement therapy \[ERT\] levels).
eGFR β‰₯ 40 mL/min/1.73 m2
Suboptimal response after at least 12 months of enzyme replacement therapy (ERT) treatment. Suboptimal response is defined as plasma lyso-Gb3 β‰₯ 2.3 nanograms per milliliter (ng/mL) at Screening and one or both of the following:
Persistent moderate or severe neuropathic pain (intermittent or continuous) over a period of at least 3 months prior to consent
Presence of gastrointestinal symptoms (abdominal cramping, consti

❓ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

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Study Type

INTERVENTIONAL

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Target Enrollment

12 participants

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Age Range

18 Years - 50 Years

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Gender

MALE

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Sponsor

UniQure Biopharma B.V.

Study Locations

Choose your preferred location or select flexible during enrollment

RECRUITING

Birmingham

Alabama

Location available
View Birmingham location page
RECRUITING

Atlanta

Georgia

Location available
View Atlanta location page
RECRUITING

Chicago

Illinois

Location available
View Chicago location page
RECRUITING

Minneapolis

Minnesota

Location available
View Minneapolis location page
RECRUITING

New York

New York

Location available
View New York location page
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Pittsburgh

Pennsylvania

Location available
View Pittsburgh location page
RECRUITING

Salt Lake City

Utah

Location available
View Salt Lake City location page
RECRUITING

Fairfax

Virginia

Location available
View Fairfax location page

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

πŸ”’ Secure Β· πŸ₯ Expert Care

Looking for Fabry Disease Treatment in Birmingham?

Join others in Alabama exploring innovative treatment options through clinical research

Fabry Disease Treatment Options in Birmingham, Alabama

If you're searching for Fabry Disease treatment in Birmingham, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Birmingham, Atlanta, Chicago and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Fabry Disease. All study-related care is provided at no cost to participants.

Local Sites
3 locations in Alabama
Now Enrolling
Up to 12 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Fabry Disease?

Potential Benefits

  • βœ“Access to new treatment approaches before public availability
  • βœ“Close monitoring by experienced medical professionals
  • βœ“Study-related care provided at no cost
  • βœ“Contribute to medical research for Fabry Disease

What to Expect

  • β†’Initial screening to determine eligibility
  • β†’Regular check-ups and monitoring visits
  • β†’Possible compensation for time and travel
  • β†’You can withdraw at any time

Frequently Asked Questions About This Fabry Disease Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT06270316. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.