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Active, Not Recruiting

NCT Number: NCT03636438

Long Term Follow Up to Evaluate DTX301 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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Ornithine Transcarbamylase (OTC) Deficiency Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Ornithine Carbamoyltransferase Deficiency Disease Urea Cycle Disorders, Inborn

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

M.A.G.I.C. Clinic, Calgary, Alberta, Canada

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About this study

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.

Who can participate

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

No intervention

Other

No Intervention

Primary outcomes

  1. Number of Participants with Adverse Events and Serious Adverse Events

    Time frame: Up to 416 weeks

Secondary outcomes

  1. 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

    Sodium acetate is used as a tracer to measure the rate of ureagenesis

  2. 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

Sponsors and collaborators

Lead sponsor

Ultragenyx Pharmaceutical Inc

Industry

Registry information

Official study 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

Acronym: CAPtivate

Important dates

Study start
2018
Primary completion
2029
Study completion
2029
First posted
Aug 17, 2018
Registry last updated
Jun 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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