NCT07643844
AAVrh10-PCCA Gene Therapy for Propionic Acidemia
Recruiting
PHASE1Ages 6–2InterventionalTreatmentMayo ClinicInvestigator-initiated
~9 participants
Updated 2026-06-23 on ClinicalTrials.gov
What's tested:AAVrh10-PCCA low doseAAVrh10-PCCA middle doseAAVrh10-PCCA high dose
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of treatment-emergent adverse events
Measured over 7 years
Conditions
Where it's being run
1 sites across 1 statesMinnesota1
Study leadership
- David R. Deyle, MD · PRINCIPAL_INVESTIGATOR · Mayo Clinic
Who to contact
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
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
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.
What this trial measures
- Incidence of treatment-emergent adverse events7 years
Total number of treatment-emergency adverse events. Adverse events include serious adverse events, lab safety tests, vital signs, and dose-limiting toxicities.