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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Last refreshed on: 1 March 2021
Main ID:  NCT02991144
Date of registration: 09/12/2016
Prospective Registration: Yes
Primary sponsor: Ultragenyx Pharmaceutical Inc
Public title: Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency CAPtivate
Scientific title: A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency
Date of first enrolment: January 2017
Target sample size: 11
Recruitment status: Active, not recruiting
Study type:  Interventional
Study design:  Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 1/Phase 2
Countries of recruitment
Canada Spain United Kingdom United States
Name:     Medical Director
Affiliation:  Ultragenyx Pharmaceutical Inc
Key inclusion & exclusion criteria

Key Inclusion Criteria:

1. Males and females =18 years of age with documented diagnosis of late onset (defined as
first manifestation of signs and symptoms at =1 month of age) OTC deficiency,
confirmed via enzymatic, biochemical, or molecular testing

2. Documented history of =1 symptomatic hyperammonemia event with ammonia =100 µmol/L.

3. Subject's OTC deficiency is stable as evidenced by either a) no clinical symptoms of
hyperammonemia OR b) an ammonia level <100 µmol/L within the 4 week period preceding
the Screening visit.

4. On ongoing daily stable dose of ammonia scavenger therapy for =4 weeks.

5. Males and all females of childbearing potential must be willing to use effective
contraception at the time of administration of gene transfer and for the 52 weeks
following administration of DTX301

Key Exclusion Criteria:

1. At Screening or Baseline (Day 0), plasma ammonia level = 100 µmol/L for patients who
historically maintain normal ammonia levels; OR plasma ammonia level = 200 µmol/L for
patients who historically are not able to fully control ammonia levels with baseline
management; OR signs and symptoms of hyperammonemia.

2. Liver transplant, including hepatocyte cell therapy/transplant.

3. History of liver disease

4. Significant hepatic inflammation or cirrhosis

5. Serum creatinine >2.0 mg/dL.

6. Participation in another investigational medicine study (including another gene
transfer trial) within 3 months of Screening

7. Pregnant or nursing

Note additional inclusion/exclusion criteria may apply, per protocol.

Age minimum: 18 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Ornithine Transcarbamylase (OTC) Deficiency
Drug: Oral prednisone
Genetic: scAAV8OTC
Primary Outcome(s)
The incidence of treatment-related adverse events by dosing group [Time Frame: 52 weeks]
Secondary Outcome(s)
Change in baseline 24 hour area under the curve of ammonia [Time Frame: 52 weeks]
Change in baseline in ureagenesis rate [Time Frame: 52 weeks]
Secondary ID(s)
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results available:
Date Posted:
Date Completed:
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