A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term
- Conditions
- Ornithine transcarbamylase deficiencyMedDRA version: 20.0Level: LLTClassification code 10071107Term: Ornithine transcarbamylase deficiencySystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2018-000156-18-ES
- Lead Sponsor
- ltragenyx Pharmaceutical, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 9
1. Completed the Week 52 visit in Study 301OTC01. Note that the Day 0 visit of Study 301OTC02 may coincide with the Week 52 visit of 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 8
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1
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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Primary end point(s): The incidence of AEs and SAEs for each dosing cohort assessed by severity and relationship to study product.;Timepoint(s) of evaluation of this end point: From the time the subject signs the Informed Consent Form through the end of study/early withdrawal visit.;Main Objective: To determine the long-term safety of DTX301 following a single IV dose in adults with late-onset OTC deficiency.;Secondary Objective: To evaluate the long-term efficacy of DTX301 on AUC0-24 for plasma ammonia following a single IV dose in adults with late-onset OTC deficiency.<br><br>To evaluate the long-term effects of DTX301 on the rate of ureagenesis in adults with late-onset OTC deficiency.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): The change from baseline (Day 0 of Study 301OTC01) in AUC0-24 for plasma ammonia over time to 260 weeks following IV administration of DTX301.<br><br>The change from baseline (average of Screening and Day 1 results of Study 301OTC01) in the rate of ureagenesis (as measured by the generation of [13C]urea over 4 hours) as determined by gas chromatography mass spectrometry over time to 260 weeks following IV administration of DTX301.;Timepoint(s) of evaluation of this end point: Plasma ammonia: Day 0, Week 52, Week 104, Week 156, Week 208<br><br>Ureagenesis: Day 0, Week 26, Week 52, Week 78, Week 104, Week 130, Week 156, Week 182, Week 208