Last updated 32 days ago

Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency

50 patients around the world

Available in Argentina, United States, Brazil, Spain

This study is a Phase 3, randomized, double-blind, placebo-controlled study of DTX301 in patients with late-onset OTC deficiency 12 years of age and older. Participants will be randomized 1:1 to DTX301 or placebo group and followed closely for 64 weeks. At week 64 eligible patients will crossover and receive DTX301 if they had previously received placebo or placebo if they had previously received DTX301. The planned study duration is up to 324 weeks. Upon completion of this study or early withdrawal, all participants who received DTX301 are invited to enroll in the Disease Monitoring Program (DMP) for follow-up for up to an additional 5 years.

Ultragenyx Pharmaceutical Inc

50Patients around the world

Requirements for the patient

From 12 Years

All Gender

Medical requirements

: Confirmed clinical diagnosis of late-onset OTC deficiency with historical documentation via enzymatic (ie, liver biopsy), biochemical (ie, hyperammonemia in the presence of elevated plasma glutamine, low citrulline, and elevated spot urine orotic acid), or molecular testing (ie, OTC analysis)
Free from symptomatic hyperammonemia and has not required emergent active intervention for hyperammonemia within 4 weeks before screening/baseline
Plasma spot ammonia level on Day 1 (predose) is ≤ 200 µmol/L
If on ongoing daily ammonia scavenger therapy, must be at stable daily dose(s) for ≥ 4 weeks prior to screening
If on a protein-restricted diet, must be on a stable total daily protein intake that does not vary more than 20% for ≥ 4 weeks prior to screening
From the time written informed consent through Week 128, females of childbearing potential and fertile males must consent to use highly effective contraception. If female, agree not to become pregnant. If male, agree not father a child or donate sperm

: Significant hepatic inflammation or cirrhosis
Estimated glomerular filtration rate < 60 mL/min/1.73 m2 at screening by the CKD EPI creatinine-based formula (Levey et al., 2009) for subjects ≥ 18 years of age or the Schwartz bedside formula (Schwartz and Work, 2009) for subjects < 18 years of age
Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection, documented by current use of antiviral therapy for HBV or HCV or by hepatitis B surface antigen (HBsAg) or HCV RNA positivity
Active infection (viral or bacterial)
Detectable pre-existing antibodies to the AAV8 capsid
Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results
Participation (current or previous) in another gene transfer study

SponsorUltragenyx Pharmaceutical Inc

Study typeInterventional

Requirements

From 12 YearsAll Gender

Unique study IDclinicaltrials.gov

NCT05345171