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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Ornithine Transcarbamylase (OTC) Deficiency
• Intervention / Treatment: Other: No Intervention

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
• Enrollment (Actual): 11

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Calgary, Alberta, Canada, T3B 6A8
      • Alberta Children's Hospital
• France
  • Rhone
    • Bron, Rhone, France, 69677
      • Hôpital Femme Mère Enfant
• Spain
  • Coruna
    • Santiago De Compostela, Coruna, Spain, 15706
      • Hospital Clinico Universitario de Santiago
  • Vizcaya
    • Barakaldo, Vizcaya, Spain, 48903
      • Hospital Universitario de Cruces. Servicio de Pediatria
• United Kingdom
  • Birmingham, United Kingdom, B15 2TH
    • Queen Elizabeth Hospital, Department of Endocrinology
• United States
  • Colorado
    • Aurora, Colorado, United States, 80045
      • The Children's Hospital Colorado
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
  • New York
    • New York, New York, United States, 10029
      • Icahn School of Medicine
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • University Hospital Cleveland Medical Center/Case Western Reserve University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Subjects 18 years of age or older with OTC previously enrolled in Study 301OTC01

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants with Adverse Events and Serious Adverse Events	Up to 416 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline Over Time in the Ureagenesis Rate	Sodium acetate is used as a tracer to measure the rate of ureagenesis	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		Baseline (Day 0 of Study 301OTC01) up to 208 weeks following DTX301 administration

Collaborators and Investigators

• Sponsor: Ultragenyx Pharmaceutical Inc
• Investigators: Study Director: Medical Director, Ultragenyx Pharmaceuticals Inc

Publications and helpful links

Helpful Links

• Ultragenyx Ornithine Transcarbamylase (OTC) Deficiency Research Study Opportunity

Study record dates

Study Major Dates

• Study Start (Actual): August 30, 2018
• Primary Completion (Estimated): December 1, 2029
• Study Completion (Estimated): December 1, 2029

Study Registration Dates

• First Submitted: August 15, 2018
• First Submitted That Met QC Criteria: August 15, 2018
• First Posted (Actual): August 17, 2018

Study Record Updates

• Last Update Posted (Estimated): January 25, 2024
• Last Update Submitted That Met QC Criteria: January 23, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: gene therapy; OTC Deficiency; Urea Cycle Disorder
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Metabolism, Inborn Errors; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Amino Acid Metabolism, Inborn Errors; Urea Cycle Disorders, Inborn; Ornithine Carbamoyltransferase Deficiency Disease
• Other Study ID Numbers: 301OTC02; 2018-000156-18 (EudraCT Number); 2022-501146-30 (Other Identifier: EU CT Number)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No