Pittsburgh, PANCT03911505Now EnrollingIRB Ready

Pompe Disease Clinical Trial in Pittsburgh, PA

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

Sponsored by Amicus Therapeutics

Quick Self-Assessment

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Expert Care in Pittsburgh

Access pompe disease specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related pompe disease treatment provided free

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Check if you qualify for this pompe disease clinical trial in Pittsburgh, PA

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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 Pittsburgh

    Convenient for PA 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 Pittsburgh site if eligible
  4. 4Begin participation

About This Pompe Disease Study in Pittsburgh

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to \< 18 years

Sponsor: Amicus Therapeutics

Who Can Participate

Inclusion Criteria

Male or female subjects (ERT-naïve \[have never received a dose of rhGAA\] or ERT-experienced \[have received rhGAA every 2 weeks for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 months before enrollment\]) diagnosed with LOPD who are aged 12 to \<18 years at screening (Cohort 1 only) or aged 0 months to \< 12 years at screening (Cohort 2 only) 2. Subject weighs ≤ 115 kg. (Cohort 1 Only) 3. Subject must have a diagnosis of LOPD based on documentation as defined in study protocol 4. If of reproductive potential and if sexually active, female and male subjects agree to use a highly effective method of contraception throughout the duration of the study and for up to 90 days after their last dose of Cipaglucosidase Alfa/Miglustat 5. Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy Adolescents at screening (Cohort 1 only) 6. Subject

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 Pittsburgh?

Yes, this clinical trial (NCT03911505) has an active research site in Pittsburgh, PA 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.

Pompe Disease Treatment Options in Pittsburgh, PA

If you're searching for pompe disease treatment options in Pittsburgh, PA, this clinical trial (NCT03911505) 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 Pittsburgh research site is actively enrolling participants for this clinical trial. You'll receive care from experienced pompe 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 pompe disease clinical trials near you to find additional studies recruiting in your area.

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