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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. 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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07643844
The ClinicalTrials.gov registry entry for NCT07643844 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 9 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Mayo Clinic, which has 2,844 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 3 interventions - of which AAVrh10-PCCA low dose is the first listed.
NCT07643844 reports 0 study locations.
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.
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