Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency (CAPtivate)
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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
Ornithine Transcarbamylase (OTC) Deficiency
Other: No Intervention
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.
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 :
Estimated Study Completion Date :
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Layout table for eligibility information
Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
Subjects 18 years of age or older with OTC previously enrolled in Study 301OTC01
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.
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