Long Term Follow Up to Evaluate DTX301 in Adults With Lat... | NCT03636438

Studies
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

NCT03636438

Sponsor

Ultragenyx Pharmaceutical Inc

Status

Active, not recruiting

Last Update Posted

Jun 1, 2026Actual

Enrollment

11Actual

Phase

Not provided

Conditions

Ornithine Transcarbamylase (OTC) Deficiency

Interventions

No Intervention

Countries

United States
Canada
France
Spain
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
2018-000156-18	EudraCT Number
2022-501146-30-00	EU Trial (CTIS) Number

Status Module

Record Verification Date

May 2026

Overall Recruitment Status or Expanded Access Status

Active, not recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Aug 30, 2018Actual

Primary Completion Date

Dec 2029Estimated

Completion Date

Dec 2029Estimated

First Submitted Date

Aug 15, 2018

First Submission Date that Met QC Criteria

Aug 15, 2018

First Posted Date

Aug 17, 2018Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 28, 2026

Last Update Posted Date

Jun 1, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.

Detailed Description

Study 301OTC02 is a long-term follow-up study to evaluate the safety and efficacy of adeno-associated virus (AAV) serotype 8 (AAV8)-mediated gene transfer of human OTC in adults with late-onset OTC deficiency. Only subjects who complete Study 301OTC01 (NCT02991144) are eligible to participate in Study 301OTC02.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

Retention

Enrollment

11Actual

Name	Type	Description	Arm Group Labels	Other Names
No Intervention	Other	No Intervention

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of Participants with Adverse Events and Serious Adverse Events		Up to 416 weeks

Secondary Outcomes

Measure	Description	Time Frame
Change from Baseline Over Time in the Ureagenesis Rate	Sodium acetate is used as a tracer to measure the rate of ureagenesis	Baseline (average of Screening and Day 1) up to 416 weeks following DTX301 administration
Change from Baseline Over Time in 24-Hour Area Under the Curve for Plasma Ammonia

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Completed the Week 52 visit in Study 301OTC01.
Willing and able to provide written informed consent.
Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.

Exclusion Criteria:

Planned or current participation in another interventional clinical study that may confound the efficacy or safety evaluation of DTX301 during the duration of this study.
Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study.

Accepts Healthy Volunteers

Not provided

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Subjects 18 years of age or older with OTC previously enrolled in Study 301OTC01

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Medical Director	Ultragenyx Pharmaceuticals Inc	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
The Children's Hospital Colorado		Aurora	Colorado	80045	United States
Boston Children's Hospital

References Module

Citations

Not provided

Label	URL
Ultragenyx Ornithine Transcarbamylase (OTC) Deficiency Research Study Opportunity	View source
Ultragenyx Transparency Commitment	View source

ID	Term
D020163	Ornithine Carbamoyltransferase Deficiency Disease
D056806	Urea Cycle Disorders, Inborn

ID	Term
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases
D002493	Central Nervous System Diseases