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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03636438
Recruitment Status : Enrolling by invitation
First Posted : August 17, 2018
Last Update Posted : September 17, 2021
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

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

Condition or disease Intervention/treatment
Ornithine Transcarbamylase (OTC) Deficiency 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.

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Study Type : Observational
Estimated Enrollment : 18 participants
Observational Model: Other
Time Perspective: Prospective
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
Actual Study Start Date : August 30, 2018
Estimated Primary Completion Date : December 2025
Estimated Study Completion Date : December 2025

Intervention Details:
  • Other: No Intervention
    No Intervention

Primary Outcome Measures :
  1. Number of Participants with Adverse Events and Serious Adverse Events [ Time Frame: Up to 260 weeks following DTX301 administration ]

Secondary Outcome Measures :
  1. Change from Baseline Over Time in the Ureagenesis Rate [ Time Frame: Baseline (average of Screening and Day 1) up to 260 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 260 weeks following DTX301 administration ]

Biospecimen Retention:   Samples With DNA

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Sampling Method:   Non-Probability Sample
Study Population
Subjects 18 years of age or older with OTC previously enrolled in Study 301OTC01

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03636438

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United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, New York
Icahn School of Medicine
New York, New York, United States, 10029
United States, Ohio
University Hospital Cleveland Medical Center/Case Western Reserve University
Cleveland, Ohio, United States, 44106
Canada, Alberta
Alberta Children's Hospital
Calgary, Alberta, Canada, T3B 6A8
Hopital Femme Mere Enfant
Bron, Rhone, France, 69677
Hospital Clinico Universitario de Santiago
Santiago De Compostela, Coruna, Spain, 15706
Hospital Universitario de Cruces. Servicio de Pediatria
Barakaldo, Vizcaya, Spain, 48903
United Kingdom
Queen Elizabeth Hospital, Department of Endocrinology
Birmingham, United Kingdom, B15 2TH
Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
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Study Director: Medical Director Ultragenyx Pharmaceuticals Inc
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Responsible Party: Ultragenyx Pharmaceutical Inc Identifier: NCT03636438    
Other Study ID Numbers: 301OTC02
2018-000156-18 ( EudraCT Number )
First Posted: August 17, 2018    Key Record Dates
Last Update Posted: September 17, 2021
Last Verified: September 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ultragenyx Pharmaceutical Inc:
gene therapy
OTC Deficiency
Urea Cycle Disorder
Additional relevant MeSH terms:
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Ornithine Carbamoyltransferase Deficiency Disease
Urea Cycle Disorders, Inborn
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases