Ornithine Transcarbamylase (OTC) DeficiencyCAPtivateNCT03636438
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
An observational study of Ornithine Transcarbamylase (OTC) Deficiency, run by Ultragenyx Pharmaceutical Inc at 9 sites in 5 countries.
Trial summary
Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Ornithine Transcarbamylase (OTC) Deficiency
- How many people
- 11 participants
- Where
- 9 sitesin 5 countries
- Study dates
- 30 Aug 2018to Dec 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
Study population: Subjects 18 years of age or older with OTC previously enrolled in Study 301OTC01
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
9 sites in 5 countries.
9 sites in 5 countries
United States4 sites
The Children's Hospital Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
University Hospital Cleveland Medical Center/Case Western Reserve University
Cleveland, Ohio, 44106
Icahn School of Medicine
New York, New York, 10029
Spain2 sites
Hospital Universitario de Cruces. Servicio de Pediatria
Barakaldo, Vizcaya, 48903
Hospital Clinico Universitario de Santiago
Santiago de Compostela, Coruna, 15706
United Kingdom1 site
Queen Elizabeth Hospital, Department of Endocrinology
Birmingham, B15 2TH
About this study
Official title: A Long-Term Follow-up Study to Evaluate Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency
Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.
Read the 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.
- Being tested
- No Intervention
- Keywords
- gene therapy, OTC Deficiency, Urea Cycle Disorder
- Medical subject headings
- Ornithine Carbamoyltransferase Deficiency Disease, Urea Cycle Disorders, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
No Intervention
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Adverse Events and Serious Adverse Events
Measured: Up to 416 weeks
Secondary outcomes (2)
Change from Baseline Over Time in the Ureagenesis Rate
Sodium acetate is used as a tracer to measure the rate of ureagenesis
Measured: 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
Measured: Baseline (Day 0 of Study 301OTC01) up to 208 weeks following DTX301 administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2018
- Study start30 Aug 2018
- Primary completionDec 2029 (estimated)
- Study completionDec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ornithine Transcarbamylase (OTC) DeficiencyStudies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceuticals Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03636438
- Sponsor's protocol ID
- 301OTC02
- Other IDs
- 2018-000156-18, 2022-501146-30-00, 2018
- Acronym
- CAPtivate
- Status last verified
- May 2026
- First submitted
- 15 Aug 2018
- Last updated
- 1 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-Term Follow-up Study to Evaluate Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency
- ClinicalTrials.gov ID
- NCT03636438
- Acronym / sponsor ID
- CAPtivate / 301OTC02
- Keywords
- gene therapy, OTC Deficiency, Urea Cycle Disorder
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- 30 Aug 2018 (actual)
- Primary completion
- Dec 2029 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 17 Aug 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- Subjects 18 years of age or older with OTC previously enrolled in Study 301OTC01
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No InterventionNo Intervention | Other | – |
Primary outcome
Number of Participants with Adverse Events and Serious Adverse Events
Time frame: Up to 416 weeks
2 secondary outcomes
Change from Baseline Over Time in the Ureagenesis Rate
Time frame: 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
Time frame: Baseline (Day 0 of Study 301OTC01) up to 208 weeks following DTX301 administration
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jun 2026.
- Ultragenyx Ornithine Transcarbamylase (OTC) Deficiency Research Study OpportunityLink provided by the sponsor.
- Ultragenyx Transparency CommitmentLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Ultragenyx Pharmaceuticals Inc
9 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Ornithine Transcarbamylase (OTC) Deficiency, run by Ultragenyx Pharmaceutical Inc at 9 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 1 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Spain, Canada, France, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Adverse Events and Serious Adverse Events (time frame: Up to 416 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jun 2026.