Salt Lake City, UTNCT04808505Now EnrollingIRB Ready

Pompe Disease Clinical Trial in Salt Lake City, UT

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

Sponsored by Amicus Therapeutics

Quick Self-Assessment

See if you qualify for this Salt Lake City location

Preparing your pre-screening questions...

Expert Care in Salt Lake City

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

Apply for This Salt Lake City Location

Check if you qualify for this pompe disease clinical trial in Salt Lake City, UT

Secure & Confidential

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

Why Participate?

  • No-Cost Study Care

  • Local to Salt Lake City

    Convenient for UT 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 Salt Lake City site if eligible
  4. 4Begin participation

About This Pompe Disease Study in Salt Lake City

This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.

Sponsor: Amicus Therapeutics

Who Can Participate

Inclusion Criteria

Cohort 1: 1. Male or female subjects who are aged 6 months to \< 18 years on Day 1 2. Subject must have documentation of IOPD genotype 3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis 4. Subject must have received ERT for at least 6 months immediately before enrollment. For subjects whose ERT dosage has been modified, the subject must have been on the modified dosage and regimen for at least 3 months before enrollment 5. Subjects aged ≥ 12 to \< 18 years must perform one valid 6-minute walk test (6MWT) (≥ 75 meters) at screening; Subjects aged ≥ 5 to \< 12 years must perform one valid 6MWT (≥ 40 meters) at screening; Subjects aged 18 months to \< 5 years must be ambulatory and assessed to be likely to be able to perform 6MWT (≥ 40 meters) when they turn 5 years old 6. Subjects must have experienced a clinical decline on their current rhGAA dose and frequency Cohort 2: 1. Male or female subjects who are aged 0 to \<6 months at Day 1 2

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 Salt Lake City?

Yes, this clinical trial (NCT04808505) has an active research site in Salt Lake City, UT 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 Salt Lake City, UT

If you're searching for pompe disease treatment options in Salt Lake City, UT, this clinical trial (NCT04808505) 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 Salt Lake City 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.

More Pulmonary Embolism Trials in Salt Lake City, UT

See all pulmonary embolism clinical trials recruiting in Salt Lake City — not just this study.

Browse Pulmonary Embolism Trials in Salt Lake City →

Browse More Trials by Condition

Pulmonary Embolism TrialsAnkylosing Spondylitis TrialsPompe Disease TrialsAll Conditions →

Ready to Join in Salt Lake City?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Salt Lake City, UT