New York, NYNCT07282847Now EnrollingIRB Ready

Pompe Disease (Late-onset) Clinical Trial in New York, NY

Access cutting-edge pompe disease (late-onset) treatment through this clinical trial at a research site in New York. Study-provided care at no cost to qualified participants.

Sponsored by AskBio Inc

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Expert Care in New York

Access pompe disease (late-onset) specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related pompe disease (late-onset) treatment provided free

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Check if you qualify for this pompe disease (late-onset) clinical trial in New York, NY

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Why Participate?

  • No-Cost Study Care

  • Local to New York

    Convenient for NY 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 New York site if eligible
  4. 4Begin participation

About This Pompe Disease (Late-onset) Study in New York

This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with late-onset Pompe disease (LOPD).

Sponsor: AskBio Inc

Who Can Participate

Inclusion Criteria

Participant must be ≥18 years of age at the time of signing the informed consent form.
Confirmed GAA enzyme deficiency from any tissue source and/or confirmed biallelic GAA gene mutations.
Undergone enzyme replacement treatment (ERT) (either alglucosidase alfa (Lumizyme®) or avalglucosidase alfa-ngpt (Nexviazyme®)), for at least 6 months (at least 10 infusions) before signing the initial informed consent form. During the screening process, participants need to remain on their current ERT until close to dosing;
FVC in the upright position ≥30% and ≤80% of predicted;
Capable of walking at least 100 meters in the 6MWT (use of a cane, quad cane, or standard walker is permitted);
Contraceptive/barrier use by men and women requirements as per protocol.
Capable of giving informed consent and able to understand and comply with all study procedures.

Exclusion Criteria

Severe cardiomyopathy, defined as left ventricular ejection fraction (LVEF) \<40% or New York Heart Association (NYHA) functional class 3 or above;
Require invasive mechanical ventilation, or rely on noninvasive ventilation during the day;
Intolerance to ERT or investigator-assessed intolerance to ERT, prior experience of serious ERT-related infusion-associated reactions (IARs);
Have known intrinsic liver diseases, including hepatitis, HIV-related liver disease, prior diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy, severe fatty liver, cirrhosis or liver fibrosis ≥stage 2, ultrasound-identified liver neoplasms, or laboratory tests suggesting elevated alpha-fetoprotein. Patients with liver function tests including ALT or AST \>3× upper limit of normal (ULN) or any total bilirubin above ULN during screening will also be excluded;
Prior or ongoing medical condition(s), physical finding(s), assessment findings, or laboratory abnormality that, in the investigator's opinion, would impact participant's safety and compliance with the study procedures.
Have received gene therapy prior to screening;
Have received any systemic immunosuppressants (except inhalation or topical use) other than glucocorticoids or investigator-recommended immunosuppressants 30 days prior to screening through completion of screening, and/or known intolerance to immunosuppressants such as glucocorticoids;
Use of investigational drugs or drugs that could affect this study as evaluated by the investigator within 30 days prior to screening through completion of Week 52 or within 5 half-lives of the investigational drug (whichever is longer);
Have received any vaccine within 30 days prior to dosing;
Other conditions that make the participant not eligible for the study according to 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 New York?

Yes, this clinical trial (NCT07282847) has an active research site in New York, NY 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 (Late-onset) Treatment Options in New York, NY

If you're searching for pompe disease (late-onset) treatment options in New York, NY, this clinical trial (NCT07282847) 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 New York research site is actively enrolling participants for this clinical trial. You'll receive care from experienced pompe disease (late-onset) 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 (late-onset) clinical trials near you to find additional studies recruiting in your area.

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