Birmingham, ALNCT06270316Now EnrollingIRB Ready

Fabry Disease Clinical Trial in Birmingham, AL

Access cutting-edge fabry disease treatment through this clinical trial at a research site in Birmingham. Study-provided care at no cost to qualified participants.

Sponsored by UniQure Biopharma B.V.

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Expert Care in Birmingham

Access fabry disease specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related fabry disease treatment provided free

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Check if you qualify for this fabry disease clinical trial in Birmingham, AL

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Why Participate?

  • No-Cost Study Care

  • Local to Birmingham

    Convenient for AL residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Birmingham site if eligible
  4. 4Begin participation

About This Fabry Disease Study in Birmingham

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.

Sponsor: UniQure Biopharma B.V.

Who Can Participate

Inclusion Criteria

Male of age ≥ 18 years and ≤50 years
Confirmed clinical diagnosis of classic Fabry disease (FD) defined as: 1. Absent or minimal αGAL A enzyme activity \< 1% of mean normal measured in plasma regardless of variant status; OR 2. α-galactosidase A (GLA) pathogenic or likely pathogenic variant associated with classic FD phenotype identified on molecular genetic testing with plasma αGLA A enzyme activity below lower bound of the reference range (as measured at trough enzyme replacement therapy \[ERT\] levels).
eGFR ≥ 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 ≥ 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.

Frequently Asked Questions

Q:Is this study available in Birmingham?

Yes, this clinical trial (NCT06270316) has an active research site in Birmingham, AL that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

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

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

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

Fabry Disease Treatment Options in Birmingham, AL

If you're searching for fabry disease treatment options in Birmingham, AL, this clinical trial (NCT06270316) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Birmingham research site is actively enrolling participants for this clinical trial. You'll receive care from experienced fabry disease specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all fabry disease clinical trials near you to find additional studies recruiting in your area.

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