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NCT07643844 · ClinicalTrials.gov registry record · Phase 1
AAVrh10-PCCA Gene Therapy for Propionic Acidemia
A Phase 1 study, sponsored by Mayo Clinic.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 9
- Enrollment target
NCT07643844: Recruiting Phase 1 study, sponsored by Mayo Clinic.
NCT07643844 is a Phase 1 study that is actively recruiting participants, run by Mayo Clinic. The registered enrollment target is 9 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07643844, a Phase 1 study, is actively recruiting participants, sponsored by Mayo Clinic.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 9 participants
- Enrollment target
Study Summary
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.
Primary Outcome
Total number of treatment-emergency adverse events. Adverse events include serious adverse events, lab safety tests, vital signs, and dose-limiting toxicities.
Interventions
- DRUG AAVrh10-PCCA low dose
- DRUG AAVrh10-PCCA middle dose
- DRUG AAVrh10-PCCA high dose
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2026-06 |
| Est. Completion | 2033-12 |
| Phase | Phase 1 |
What NCT07643844 shows while recruiting
NCT07643844 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower).
The record links to 0 conditions, and to 3 interventions - of which AAVrh10-PCCA low dose is the first listed.
NCT07643844 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT07643844 about?
NCT07643844 is a clinical study titled "AAVrh10-PCCA Gene Therapy for Propionic Acidemia". 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 ...
What is the current status of trial NCT07643844?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2026-06. Estimated completion is 2033-12.
What interventions are being tested in trial NCT07643844?
The interventions under investigation include: AAVrh10-PCCA low dose (DRUG), AAVrh10-PCCA middle dose (DRUG), AAVrh10-PCCA high dose (DRUG).
Who is sponsoring clinical trial NCT07643844?
This trial is sponsored by Mayo Clinic, which has 2,844 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT07643844's enrollment target sits among peer trials
9 1955th of 2000 higher than 42 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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