Active — Not RecruitingNot Applicable

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

United States, Canada11 participantsStarted 2018-08-30

Plain-language summary

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

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

Number of Participants with Adverse Events and Serious Adverse Events

Timeframe: Up to 416 weeks

Trial details

NCT IDNCT03636438

SponsorUltragenyx Pharmaceutical Inc

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2029-12

View on ClinicalTrials.gov More Ornithine Transcarbamylase (OTC) Deficiency trials