Fairfax, VANCT06904261Now EnrollingIRB Ready

Fabry Disease Clinical Trial in Fairfax, VA

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

Sponsored by Amicus Therapeutics

Quick Self-Assessment

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Expert Care in Fairfax

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

Apply for This Fairfax Location

Check if you qualify for this fabry disease clinical trial in Fairfax, VA

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

Why Participate?

  • No-Cost Study Care

  • Local to Fairfax

    Convenient for VA 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 Fairfax site if eligible
  4. 4Begin participation

About This Fabry Disease Study in Fairfax

An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.

Sponsor: Amicus Therapeutics

Who Can Participate

Inclusion Criteria

Male or female subjects, diagnosed with Fabry disease who are between ages 2 and \< 12 years at randomization (subjects aged 11 years must have birthdays \> 30 days after randomization)
Subject's parent or legally authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable.
Subject has a GLA variant documented in his/her medical record that is amenable to migalastat prior to Visit 2.
Subject has not received ERT (eg, Replagal® \[agalsidase alfa\] or Fabrazyme® \[agalsidase beta\]) for at least 14 days prior to Baseline visit.
Subject has at least 1 documented complication (ie, historical or current laboratory abnormality or sign/symptom) of Fabry disease
If of reproductive potential, both male and female subjects agree to use a medically accepted method of contraception throughout the duration of the study and for up to 30 days after their last dose of migalastat.

Exclusion Criteria

Has moderate or severe renal impairment (eGFR \< 60 mL/min/1.73 m2 at Visit 1 \[screening\]).
Has advanced kidney disease requiring dialysis or kidney transplantation.
History of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).
Has received any investigational/experimental drug, biologic, or device within 30 days or 5 half-lives of the investigational product (whichever is longer) before Visit 1 (screening).
Has received any gene therapy at any time or anticipates starting gene therapy during the study period.
Requires treatment with Glyset (miglitol) or Zavesca (miglustat), within 6 months before Visit 1(screening) or throughout the study.
Has any intercurrent illness or condition at Visit 1 (screening) or Visit 2 (baseline) that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study.
Pregnant or breastfeeding
Otherwise unsuitable for the study in the opinion of the investigator

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 Fairfax?

Yes, this clinical trial (NCT06904261) has an active research site in Fairfax, VA 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 Fairfax, VA

If you're searching for fabry disease treatment options in Fairfax, VA, this clinical trial (NCT06904261) 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 Fairfax 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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