Trial · NCT05345171
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
NCT05345171, Phase 3, Active not recruiting, Ultragenyx Pharmaceutical Inc
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency. Trials, record NCT05345171. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05345171
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
- NCT number
- NCT05345171
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 37 participants, actual
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- OTC Deficiency
- Interventions
- DTX301, Placebo, Oral Corticosteroids, Placebo for oral corticosteroids, Sodium Acetate
- First posted
- 2022-04-25
- Last updated
- 2026-09-09
- Other registries' IDs
- eudract:2020-003384-25
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Plasma Ammonia as Measured by 24-Hour Ammonia (AUC0-24) | Week 36 | |
| Primary | Complete Responder Rate at the Final Study Visit After DTX301 Exposure | Up to 64 Weeks Post DTX301 Infusion | |
| Secondary | Percentage of Complete Responders or Responders After DTX301 Exposure | Up to 64 Weeks Post DTX301 Infusion | |
| Secondary | Annualized Event Rate of Hyperammonemic Crises (HACs) Pre-DTX301 Exposure vs Post-DTX301 Exposure | Pre-enrollment, Baseline, Up to 64 Weeks Post DTX301 Infusion | |
| Secondary | Annualized Event Rate of Interim Clinical Events (ICEs) Pre-DTX301 Exposure vs Post-DTX301 Exposure | Pre-enrollment, Baseline, Up to 64 Weeks Post DTX301 Infusion | |
| Secondary | Change from Baseline in Plasma Ammonia (AUC0-24) | Baseline, Up to Week 36 | |
| Secondary | Change in Plasma Ammonia (AUC0-24) After DTX301 Exposure | Up to 64 Weeks Post DTX301 Infusion | |
| Secondary | Percentage of Participants Who Have Achieved Complete Management Response (CMR) or Management Response (MR) After DTX301 Exposure | Up to 64 Weeks Post DTX301 Infusion | |
| Secondary | Change in Baseline Disease Management (Dietary Protein and Total Scavenger Medication Use) With Plasma Ammonia (AUC0-24) | Up to 64 Weeks Post DTX301 Infusion | |
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs, Related Serious TEAEs and Adverse Events of Special Interest (AESIs) | Up to Week 324 |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 38171926 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05345171
GET https://conductscience.org/api/trials/v1/NCT05345171/registered