Clinical trial NCT03636438: Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
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
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Ornithine Transcarbamylase (OTC) Deficiency
Keywords: gene therapy
OTC Deficiency
Urea Cycle Disorder
Lead sponsor: Ultragenyx Pharmaceutical Inc
Sponsor class: INDUSTRY
First posted: 2018-08-17
Enrollment: 11
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects 18 years of age or older with OTC previously enrolled in Study 301OTC01

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

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.

Eligibility 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.

Primary outcomes:
measure: Number of Participants with Adverse Events and Serious Adverse Events
timeFrame: Up to 416 weeks
Secondary outcomes:
measure: Change from Baseline Over Time in the Ureagenesis Rate
timeFrame: Baseline (average of Screening and Day 1) up to 416 weeks following DTX301 administration
measure: Change from Baseline Over Time in 24-Hour Area Under the Curve for Plasma Ammonia
timeFrame: Baseline (Day 0 of Study 301OTC01) up to 208 weeks following DTX301 administration
Other outcomes:
Locations:
city: Aurora
country: United States
facility: The Children's Hospital Colorado
state: Colorado
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: New York
country: United States
facility: Icahn School of Medicine
state: New York
city: Cleveland
country: United States
facility: University Hospital Cleveland Medical Center/Case Western Reserve University
state: Ohio
city: Calgary
country: Canada
facility: Alberta Children's Hospital
state: Alberta
city: Bron
country: France
facility: Hopital Femme Mere Enfant
state: Rhone
city: Santiago De Compostela
country: Spain
facility: Hospital Clinico Universitario de Santiago
state: Coruna
city: Barakaldo
country: Spain
facility: Hospital Universitario de Cruces. Servicio de Pediatria
state: Vizcaya
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital, Department of Endocrinology
Study officials:
affiliation: Ultragenyx Pharmaceuticals Inc
name: Medical Director
role: STUDY_DIRECTOR
References: