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

Brief Title

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

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

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.
    


Study Type

Observational


Primary Outcome

Number of Participants with Adverse Events and Serious Adverse Events

Secondary Outcome

 Change from Baseline Over Time in the Ureagenesis Rate

Condition

Ornithine Transcarbamylase (OTC) Deficiency

Intervention

No Intervention


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Other

Estimated Enrollment

11

Start Date

August 30, 2018

Completion Date

December 2027

Primary Completion Date

December 2027

Eligibility Criteria

        Inclusion Criteria:

          1. Completed the Week 52 visit in Study 301OTC01.

          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:

          1. 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.

          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.
      

Gender

All

Ages

18 Years - N/A


Contacts

Medical Director, , 

Location Countries

Canada

Location Countries

Canada

Administrative Informations


NCT ID

NCT03636438

Organization ID

301OTC02

Secondary IDs

2018-000156-18

Responsible Party

Sponsor

Study Sponsor

Ultragenyx Pharmaceutical Inc


Study Sponsor

Medical Director, Study Director, Ultragenyx Pharmaceuticals Inc


Verification Date

March 2022