Irvine, CANCT07354724Now EnrollingIRB Ready

Late-onset Pompe Disease Clinical Trial in Irvine, CA

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

Sponsored by Denali Therapeutics Inc.

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Expert Care in Irvine

Access late-onset pompe disease specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

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

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Check if you qualify for this late-onset pompe disease clinical trial in Irvine, CA

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Why Participate?

  • No-Cost Study Care

  • Local to Irvine

    Convenient for CA 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 Irvine site if eligible
  4. 4Begin participation

About This Late-onset Pompe Disease Study in Irvine

This is a Phase 1, multicenter, open-label study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL952 in adult participants with late-onset Pompe disease. The principal aim of this study is to obtain safety and tolerability data across varous dose levels of DNL952 in participants with late-onset Pompe disease (LOPD).

Sponsor: Denali Therapeutics Inc.

Who Can Participate

Inclusion Criteria

Body weight ≥40 kg
Diagnosis of LOPD
Upright FVC ≥ 30% of predicted normal value
Able to ambulate ≥ 40 meters (use of assistive devices is acceptable)
\[Cohorts A1-A4 only\] Have received avalglucosidase alfa or cipaglucosidase alfa at a dose of 20 mg/kg every 2 weeks for at least 12 months prior to screening
\[Cohorts B1-B2 only\] Must not have received any enzyme-replacement therapy for Pompe disease in the 12 months prior to screening Key

Exclusion Criteria

Any ongoing, clinically significant, unstable, or poorly controlled neurological, psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematological, immunological, allergic, or ophthalmic disease not related to Pompe disease, or other major disorders. Well-controlled conditions are permitted if investigator and Sponsor agree.
Wheelchair-dependent
Require noninvasive ventilation for an average of more than 6 hours per day while awake or any invasive ventilation. Use of noninvasive ventilation during sleep is acceptable.
Received an experimental gene therapy at any time or participation in any other investigational drug trial or use of investigational drug within 60 days or 5 half-lives, whichever is longer, before screening

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 Irvine?

Yes, this clinical trial (NCT07354724) has an active research site in Irvine, CA 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.

Late-onset Pompe Disease Treatment Options in Irvine, CA

If you're searching for late-onset pompe disease treatment options in Irvine, CA, this clinical trial (NCT07354724) 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 Irvine research site is actively enrolling participants for this clinical trial. You'll receive care from experienced late-onset 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 late-onset pompe disease clinical trials near you to find additional studies recruiting in your area.

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