MedTrace Logo

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

NCT05345171 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2026-02-17

No results posted yet for this study

Summary

The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels.

Conditions

  • OTC Deficiency

Interventions

GENETIC

DTX301

non-replicating, self-complementary recombinant adeno-associated virus serotype 8 (AAV8) vector

OTHER

Placebo

normal saline infusion

DRUG

Oral Corticosteroids

Participants who receive DTX301 solution will receive oral corticosteroids.

DRUG

Placebo for oral corticosteroids

Participants who receive Placebo will receive placebo corticosteroids to maintain the study blind

DRUG

Sodium Acetate

A tracer for the Ureagenesis Rate Test (URT)

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-10-18
Primary Completion
2027-09-30
Completion
2031-03-31
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Brazil
  • Canada
  • France
  • Germany
  • Japan
  • Netherlands
  • Portugal
  • Spain

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05345171 on ClinicalTrials.gov