Fairfax, VANCT07123155Now EnrollingIRB Ready

Pompe Disease Clinical Trial in Fairfax, VA

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

Sponsored by Shionogi

Quick Self-Assessment

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Expert Care in Fairfax

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 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 Pompe Disease Study in Fairfax

The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.

Sponsor: Shionogi

Who Can Participate

Inclusion Criteria

Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the informed consent.
Participant must have a diagnosis of LOPD based on documentation of 1 of the following:
Deficiency of acid alpha-glucosidase (GAA) enzyme
GAA genotype
Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening; or Participant has ≥10% %FVC drop from upright position to supine position and %FVC ≥20% in a supine position.
Participant performs the 6MWT at screening, as determined by the clinical evaluator, and meets all of the following criteria:
Screening values of 6-minute walk distance (6MWD) are ≥75 meters
Screening values of 6MWD are ≤90% of the predicted value for healthy adults
Participants must be ERT-experienced, defined as currently receiving ERT and having been receiving ERT for ≥24 months, with no regimen change in the last 6 months. Key

Exclusion Criteria

Has a medical condition or any other extenuating circumstance that may pose an undue safety risk to the participant or may compromise his/her ability to comply with or adversely impact protocol requirements.
Has active infections at screening.
Malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.
Current or chronic history of liver disease.
Known biallelic loss of function mutations whether in glycogenin gene (GYG) or in glycogen phosphorylase muscle associated gene(PYGM) .
Has received any investigational therapy or pharmacological treatment for Pompe disease, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before day 1 or is anticipated to do so during the study.
Has received gene therapy or small interfering ribonucleic acid (RNA) therapy for Pompe disease.
Participant, if female, is pregnant or breastfeeding at screening.
Participant, whether male or female, is planning to conceive a child during the study. Note: Other protocol-specified inclusion and exclusion criteria may apply.

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 (NCT07123155) 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.

Pompe Disease Treatment Options in Fairfax, VA

If you're searching for pompe disease treatment options in Fairfax, VA, this clinical trial (NCT07123155) 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 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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Secure · Expert Care · Fairfax, VA