Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
NCT ID: NCT03636438
Last Updated: 2026-02-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
ACTIVE_NOT_RECRUITING
11 participants
OBSERVATIONAL
2018-08-30
2029-12-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
NCT02991144
Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency
NCT00004498
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
NCT06805695
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
NCT03765632
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
NCT02999984
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
OTHER
PROSPECTIVE
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
No Intervention
No Intervention
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
2. Willing and able to provide written informed consent.
3. Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.
Exclusion Criteria
2. Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study.
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Ultragenyx Pharmaceutical Inc
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Medical Director
Role: STUDY_DIRECTOR
Ultragenyx Pharmaceuticals Inc
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
The Children's Hospital Colorado
Aurora, Colorado, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Icahn School of Medicine
New York, New York, United States
University Hospital Cleveland Medical Center/Case Western Reserve University
Cleveland, Ohio, United States
M.A.G.I.C. Clinic
Calgary, Alberta, Canada
Hopital Femme Mere Enfant
Bron, Rhone, France
Hospital Clinico Universitario de Santiago
Santiago de Compostela, Coruna, Spain
Hospital Universitario de Cruces. Servicio de Pediatria
Barakaldo, Vizcaya, Spain
Queen Elizabeth Hospital, Department of Endocrinology
Birmingham, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Related Links
Access external resources that provide additional context or updates about the study.
Ultragenyx Ornithine Transcarbamylase (OTC) Deficiency Research Study Opportunity
Ultragenyx Transparency Commitment
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2018-000156-18
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2022-501146-30-00
Identifier Type: CTIS
Identifier Source: secondary_id
301OTC02
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.