At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 8 enrolled
Drug / intervention
ORGN001 (formerly ALXN1101)drug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ORGN001 (Formerly ALXN1101) in Pediatric Patients With Molybdenum Cofactor Deficiency (MoCD) Type A Currently Treated With Recombinant Escherichia Coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)
In Brief
A Phase 2 clinical trial evaluating ORGN001 (formerly ALXN1101) for Molybdenum Cofactor Deficiency, Type A. Completed, enrolled 8 participants across 6 sites in 5 countries.
Detailed Summary
This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMolybdenum Cofactor Deficiency, Type A
CountriesAustralia, Netherlands, Tunisia, United Kingdom, United States
Collaborators--
Timeline
Phase 2CompletedFinished
2014201520162017201820192020202120222023202420252026
First PostedJan 2014
Enrollment StartApr 2014
Primary CompletionAug 2022
Study CompletionOct 2022
TodayJul 2026
First PostedJan 28, 2014
Enrollment StartApr 1, 2014
Primary CompletionAug 1, 2022
Study CompletionOct 1, 2022
TodayJul 2, 2026
Enrollment to primary: 8.3 yearsPosted 12.4 years ago
Interventions
ORGN001 (formerly ALXN1101)drug
IV infusion