Stanford, CANCT04159103Now EnrollingIRB Ready

Propionic Acidemia Clinical Trial in Stanford, CA

Access cutting-edge propionic acidemia treatment through this clinical trial at a research site in Stanford. Study-provided care at no cost to qualified participants.

Sponsored by ModernaTX, Inc.

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Expert Care in Stanford

Access propionic acidemia specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related propionic acidemia treatment provided free

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Check if you qualify for this propionic acidemia clinical trial in Stanford, CA

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Why Participate?

  • No-Cost Study Care

  • Local to Stanford

    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 Stanford site if eligible
  4. 4Begin participation

About This Propionic Acidemia Study in Stanford

This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determinat

Sponsor: ModernaTX, Inc.

Who Can Participate

Inclusion Criteria

Participants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply:
≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants in Part 1.
≥1 year of age at the time of consent/assent if enrolled after the first 2 participants in Part 1.
Confirmed diagnosis of PA based on diagnosis by molecular genetic testing via central laboratory (PCCA and/or PCCB mutations).
Part 2 only: At least one documented MDE in the 12-month period before consent. Participants \<1 Year of Age :
Identification by newborn screening shortly after birth or having suspected PA by presenting with a spectrum of metabolic symptoms, and having a sibling diagnosed with PA. Participant may enter the Screening Period while awaiting genetic testing results, provided that all other eligibility criteria are met but would not be enrolled until diagnosis of PA is confirmed.
For infants in the neonatal intensive ca

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 Stanford?

Yes, this clinical trial (NCT04159103) has an active research site in Stanford, 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.

Propionic Acidemia Treatment Options in Stanford, CA

If you're searching for propionic acidemia treatment options in Stanford, CA, this clinical trial (NCT04159103) 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 Stanford research site is actively enrolling participants for this clinical trial. You'll receive care from experienced propionic acidemia 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 propionic acidemia clinical trials near you to find additional studies recruiting in your area.

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