Trial · NCT02991144
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
NCT02991144, Phase 1, Phase 2, Completed, Ultragenyx Pharmaceutical Inc; results first posted 2022-12-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency. Trials, record NCT02991144. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02991144
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
- NCT number
- NCT02991144
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 16 participants, actual
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- Ornithine Transcarbamylase (OTC) Deficiency
- Interventions
- scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen
- First posted
- 2016-12-13
- Last updated
- 2023-01-26
- Other registries' IDs
- eudract:2016-001057-40
- Results first posted
- 2022-12-23
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation | AEs Prior to Dosing: From signing the informed consent form (ICF) to first dose of study drug. TEAEs: From first dose of study drug up to End of Study (Week 52). | AE: any untoward medical occurrence regardless of its causal relationship to study product. TEAE: any event not present before exposure to study product or any event already present that worsens in either intensity or frequency after exposure to study product. SAE: any event that results in death; is immediately life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; is an important medical event, according to the investigator. AE intensity was rated as Grade 1 (mild), 2 (moderate), 3 (severe), 4 (life threatening), or 5 (death) according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE). The relationship or association of the study product in causing or contributing to the AE was characterized as: unrelated; possible; probably; definite. |
| Secondary | Change From Baseline Over Time in Rate of Ureagenesis | Baseline (Day 0), Weeks 6, 12, 20, 24, End of Study (Week 52). AUC was derived based on the following time points: 0.5, 1, 1.5, 2, 3, and 4 hours postdose. | The change from baseline in the rate of ureagenesis (as measured by the generation of \[13C\]urea over 4 hours) as determined by gas chromatography mass spectrometry over time to 52 weeks after the IV administration of DTX301. Sodium acetate was used as a tracer to measure the rate of ureagenesis. Rate of ureagenesis was derived in the following manner: 1. Derive area under the curve from time zero to 240 minutes (AUC0-240min) of absolute 13C-urea (µmol/l/min) estimated by the linear trapezoidal rule 2. Derive percent of normal AUC0-240min of absolute 13C-urea by dividing AUC0-240min of absolute 13C-urea (µmol\*min/L) by 669.56 µmol\*min/L (i.e. the AUC0-240min of absolute 13C-urea for an adult control) 3. Derive rate of ureagenesis by multiplying % of normal AUC0-240min of absolute 13C-urea by 300 µmol\*h/kg (i.e. the approximate rate of ureagenesis in healthy adults). |
| Secondary | Change From Baseline Over Time in Area Under the Curve From Time Zero to 24 Hours (AUC0-24) of Plasma Ammonia | Baseline (Day 0), Weeks 6, 12, 24, End of Study (Week 52). AUC was derived based on predose (time 0) and approximately 2, 4, 8, 12, 16, 20, 24 hours (±5 minutes) postdose. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation | AEs Prior to Dosing: From signing the informed consent form (ICF) to first dose of study drug. TEAEs: From first dose of study drug up to End of Study (Week 52). | Safety Set: all participants who received DTX301. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline Over Time in Rate of Ureagenesis | Baseline (Day 0), Weeks 6, 12, 20, 24, End of Study (Week 52). AUC was derived based on the following time points: 0.5, 1, 1.5, 2, 3, and 4 hours postdose. | Safety Set: all participants who received DTX301. | μmol*h/kg | MEAN |
| SECONDARY | Change From Baseline Over Time in Area Under the Curve From Time Zero to 24 Hours (AUC0-24) of Plasma Ammonia | Baseline (Day 0), Weeks 6, 12, 24, End of Study (Week 52). AUC was derived based on predose (time 0) and approximately 2, 4, 8, 12, 16, 20, 24 hours (±5 minutes) postdose. | Safety Set: all participants who received DTX301. Participants with an assessment at given time point. | μmol*h/L | MEAN |
Source ClinicalTrials.gov
Results by group80
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation | Cohort 1: DTX301 2.0 × 10^12 GC/kg | Any AE prior to dosing | 1 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation | Cohort 2: DTX301 6.0 × 10^12 GC/kg | Any AE prior to dosing | 1 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation | Cohort 3: DTX301 1.0 × 10^13 GC/kg | Any AE prior to dosing | 0 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation | Cohort 4: DTX301 1.0x10^13 GC/kg + Prophylactic Corticosteroids | Any AE prior to dosing | 1 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation | Cohort 1: DTX301 2.0 × 10^12 GC/kg | Any TEAE | 3 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation | Cohort 2: DTX301 6.0 × 10^12 GC/kg | Any TEAE | 3 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation | Cohort 3: DTX301 1.0 × 10^13 GC/kg | Any TEAE | 3 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation | Cohort 4: DTX301 1.0x10^13 GC/kg + Prophylactic Corticosteroids | Any TEAE | 2 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation | Cohort 1: DTX301 2.0 × 10^12 GC/kg | Any serious TEAE | 0 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation | Cohort 2: DTX301 6.0 × 10^12 GC/kg | Any serious TEAE | 0 | Participants |
1 to 10 of 80Source ClinicalTrials.gov
See all 80 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: DTX301 2.0 × 10^12 GC/kg | 3 | |
| Overall Study | STARTED | Cohort 2: DTX301 6.0 × 10^12 GC/kg | 3 | |
| Overall Study | STARTED | Cohort 3: DTX301 1.0 × 10^13 GC/kg | 3 | |
| Overall Study | STARTED | Cohort 4: DTX301 1.0x10^13 GC/kg + Prophylactic Corticosteroids | 2 | |
| Overall Study | COMPLETED | Cohort 1: DTX301 2.0 × 10^12 GC/kg | 3 | |
| Overall Study | COMPLETED | Cohort 2: DTX301 6.0 × 10^12 GC/kg | 3 | |
| Overall Study | COMPLETED | Cohort 3: DTX301 1.0 × 10^13 GC/kg | 3 | |
| Overall Study | COMPLETED | Cohort 4: DTX301 1.0x10^13 GC/kg + Prophylactic Corticosteroids | 2 | |
| Overall Study | NOT COMPLETED | Cohort 1: DTX301 2.0 × 10^12 GC/kg | 0 | |
| Overall Study | NOT COMPLETED | Cohort 2: DTX301 6.0 × 10^12 GC/kg | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events275
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Extrasystoles | Cardiac disorders | DTX301 2.0 × 10^12 GC/kg | 0 | 3 | |
| other | Extrasystoles | Cardiac disorders | DTX301 6.0 × 10^12 GC/kg | 0 | 3 | |
| other | Extrasystoles | Cardiac disorders | DTX301 1.0 × 10^13 GC/kg | 0 | 3 | |
| other | Extrasystoles | Cardiac disorders | DTX301 1.0x10^13 GC/kg + Prophylactic Corticosteroids | 1 | 2 | |
| other | Extrasystoles | Cardiac disorders | Total | 1 | 11 | |
| other | Cushingoid | Endocrine disorders | DTX301 2.0 × 10^12 GC/kg | 1 | 3 | |
| other | Cushingoid | Endocrine disorders | DTX301 6.0 × 10^12 GC/kg | 0 | 3 | |
| other | Cushingoid | Endocrine disorders | DTX301 1.0 × 10^13 GC/kg | 1 | 3 | |
| other | Cushingoid | Endocrine disorders | DTX301 1.0x10^13 GC/kg + Prophylactic Corticosteroids | 0 | 2 | |
| other | Cushingoid | Endocrine disorders | Total | 2 | 11 |
1 to 10 of 275Source ClinicalTrials.gov
See all 275 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DTX301 2.0 × 10^12 GC/kg | 0 | 3 |
| Total, all-cause mortality | DTX301 6.0 × 10^12 GC/kg | 0 | 3 |
| Total, all-cause mortality | DTX301 1.0 × 10^13 GC/kg | 0 | 3 |
| Total, all-cause mortality | DTX301 1.0x10^13 GC/kg + Prophylactic Corticosteroids | 0 | 2 |
| Total, all-cause mortality | Total | 2 | 16 |
| Total, other adverse events | DTX301 2.0 × 10^12 GC/kg | 3 | 3 |
| Total, other adverse events | DTX301 6.0 × 10^12 GC/kg | 3 | 3 |
| Total, other adverse events | DTX301 1.0 × 10^13 GC/kg | 3 | 3 |
| Total, other adverse events | DTX301 1.0x10^13 GC/kg + Prophylactic Corticosteroids | 2 | 2 |
| Total, other adverse events | Total | 11 | 11 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT02991144
GET https://conductscience.org/api/trials/v1/NCT02991144/results?page=2