Ornithine Transcarbamylase Deficiency Disease
4
1
2
0
Key Insights
Highlights
Treatment Research
4 treatments under study, led by Ecur506
Related Conditions
Often studied with 4 related conditions
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 90/100
50.0%
2 terminated out of 4 trials
0.0%
-86.6% vs benchmark
25%
1 trials in Phase 3/4
0%
0 of 0 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Clinical Trials (4)
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency
Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
Research Network
Common Treatments
- Ecur5061
- Recombinant Adenovirus Containing The Ornithine Transcarbamylase Gene1
- Adenoviral Vectormediated Gene Transfer1
- No Intervention1
Active Sponsors
- iECURE, Inc.2
- University of Pennsylvania1
- Children's National Research Institute1
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)1
Top Investigators
iECURE, Inc.
University of Pennsylvania
Related Conditions
- Urea Cycle Disorders Inborn2
- Ornithine Transcarbamylase Deficiency Otc2
- Ornithine Transcarbamylase Deficiency2
- Ornithine Carbamoyltransferase Deficiency Disorder1