AAVrh10-PCCA Gene Therapy for Propionic Acidemia

Part of paid clinical trials in Rochester, Minnesota.

Sponsor
Mayo Clinic
Study ID
NCT07643844
Phase
PHASE1
Status
Not Yet Recruiting

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Conditions

  • Propionic Acidemia

Eligibility Criteria

Sex
ALL
Age
6 Months - 2 Years
Healthy Volunteers
Not accepted

Interventions

  • AAVrh10-PCCA low dose — DRUG
    AAVrh10-PCCA (Dose of 2 x 10\^12 vg per kg body weight) is an adeno-associated viral vector containing the adeno-associated virus terminal repeat sequences flanking a transgene cassette harboring the cytomegalovirus (CMV) immediate-early enhancer and beta actin promoter, the human PCCA cDNA, and the bovine growth hormone polyadenylation sequence.
  • AAVrh10-PCCA middle dose — DRUG
    AAVrh10-PCCA (Dose of 8 x 10\^12 vg per kg body weight) is an adeno-associated viral vector containing the adeno-associated virus terminal repeat sequences flanking a transgene cassette harboring the cytomegalovirus (CMV) immediate-early enhancer and beta actin promoter, the human PCCA cDNA, and the bovine growth hormone polyadenylation sequence.
  • AAVrh10-PCCA high dose — DRUG
    AAVrh10-PCCA (Dose of 3.2 x 10\^13 vg per kg body weight) is an adeno-associated viral vector containing the adeno-associated virus terminal repeat sequences flanking a transgene cassette harboring the cytomegalovirus (CMV) immediate-early enhancer and beta actin promoter, the human PCCA cDNA, and the bovine growth hormone polyadenylation sequence.

Study Details

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.

Key Dates

Start date
Jul 31, 2026
Status verified
Jun 2026
Primary completion
Dec 31, 2032
Completion
Dec 31, 2033

Study Design

Enrollment
9 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Gene Therapy First Cohort (3 patients)
    AAVrh10-PCCA, single dose of 2 x 10\^12 vg per kilogram of body weight (first three patients), IV administration
  • Experimental: Gene Therapy Second Cohort (3 patients)
    AAVrh10-PCCA, single dose of 8 x 10\^12 vg per kilogram of body weight (middle three patients), IV administration
  • Experimental: Gene Therapy Third Cohort (3 patients)
    AAVrh10-PCCA, single dose of 3.2 x 10\^13 vg per kilogram of body weight (last three patients), IV administration

Primary Outcome Measure

Incidence of treatment-emergent adverse events [ Time Frame: 7 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Mayo ClinicRochesterMinnesota55905-

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By research site
Mayo Clinic· Rochester, MN

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