Rochester, MNNCT07643844Now EnrollingIRB Ready

Propionic Acidemia Clinical Trial in Rochester, MN

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

Sponsored by Mayo Clinic

Quick Self-Assessment

See if you qualify for this Rochester location

Preparing your pre-screening questions…

Expert Care in Rochester

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

Apply for This Rochester Location

Check if you qualify for this propionic acidemia clinical trial in Rochester, MN

Secure & Confidential

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

Why Participate?

  • No-Cost Study Care

  • Local to Rochester

    Convenient for MN 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 Rochester site if eligible
  4. 4Begin participation

About This Propionic Acidemia Study in Rochester

Propionic acidemia is a genetic metabolic disorder characterized by metabolic acidosis, ketosis, vomiting, lethargy, cognitive impairment, and risk of death. It results from loss of function of the mitochondrial enzyme propionyl-CoA carboxylase and can be due to disease-causing variants in the PCCA gene, leading to accumulation of propionyl-CoA and its toxic metabolites. The purpose of this trial is to evaluate the safety and potential therapeutic benefit of an AAV-based gene therapy for propionic acidemia in patients with genetically confirmed biallelic variants in PCCA.

Sponsor: Mayo Clinic

Who Can Participate

Inclusion Criteria

Age six months to 2 years of age at day of vector infusion. For those \<1 year of age they must have been ≥37 weeks gestational age at the time of birth and without other conditions/comorbidities that in the opinion of the Investigator may interfere with the interpretation of study results.
Confirmed diagnosis of propionic acidemia with biallelic PCCA gene mutations based on molecular genetic testing.
Study participants must have a diagnosis of neonatal-onset propionic acidemia with a documented episode of decompensation that can include any of the following findings: lethargy, poor feeding, irritability, vomiting, encephalopathy, respiratory failure, seizures, coma, metabolic acidosis, lactic acidosis, ketonuria, hypoglycemia, hyperammonemia, and cytopenias or history of recurrent hospitalizations.
Parents or legal guardians of study participants must agree to comply in good faith with the conditions of the study, including attending all of the required baseline and follow-up assessments, and parents or legal guardians must give consent for their child's participation.

Exclusion Criteria

Hemoglobin \<10 g/dl
Platelet count \< 100,000 per mm3
Liver Enzyme ALT/AST \>2.5 ULN
Direct Bilirubin \> 1.5
Active viral infection (includes HIV or serology positive for hepatitis B or C).
Previous liver transplant
Subjects with active decompensation as demonstrated by a pH \< 7.3, bicarbonate \< 15 mmol/L, NH3 \> 75 mcmol/L, lactate \> 2.5 mmol/L, urine ketones
Previously received gene therapy or messenger ribonucleic acid (mRNA) treatments for PA.
Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification.
Family does not want to disclose patient's study participation with primary care physician and other medical providers.

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 Rochester?

Yes, this clinical trial (NCT07643844) has an active research site in Rochester, MN 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 Rochester, MN

If you're searching for propionic acidemia treatment options in Rochester, MN, this clinical trial (NCT07643844) 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 Rochester 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.

More Heart Attack Trials in Rochester, MN

See all heart attack clinical trials recruiting in Rochester — not just this study.

Browse Heart Attack Trials in Rochester →

Browse More Trials by Condition

Heart Attack TrialsInterstitial Cystitis TrialsMyocardial Infarction TrialsAll Conditions →

Ready to Join in Rochester?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Rochester, MN