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Trial record 2 of 2 for:    dtx 301

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

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ClinicalTrials.gov Identifier: NCT03636438
Recruitment Status : Enrolling by invitation
First Posted : August 17, 2018
Last Update Posted : June 4, 2019
Sponsor:
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 : 12 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 2024
Estimated Study Completion Date : December 2024



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 ]
  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
Blood


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
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

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 ClinicalTrials.gov identifier (NCT number): NCT03636438


Locations
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United States, New York
Icahn School of Medicine
New York, New York, United States, 10029
Canada, Alberta
Alberta Children's Hospital
Calgary, Alberta, Canada, T3B 6A8
Spain
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
Investigators
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Study Director: Medical Director Ultragenyx Pharmaceuticals Inc

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Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT03636438     History of Changes
Other Study ID Numbers: 301OTC02
2018-000156-18 ( EudraCT Number )
First Posted: August 17, 2018    Key Record Dates
Last Update Posted: June 4, 2019
Last Verified: June 2019

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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