Ignite Creation Date:
2025-12-24 @ 12:07 PM
Ignite Modification Date:
2025-12-27 @ 9:08 PM
Study NCT ID:
NCT02047461
Status:
COMPLETED
Last Update Posted:
2023-10-17
First Post:
2014-01-09
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP
Sponsor:
Origin Biosciences
Organization:
Study Overview
Official Title:
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)
Status:
COMPLETED
Status Verified Date:
2023-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
Detailed Description:
Patients will receive daily IV infusions of ORGN001 (formerly ALXN1101) starting on Day 1. After a prescribed period, dosing will increase monthly based on defined patient safety measures. After Month 6, patients will continue daily dosing at their last tolerated dose.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 2013-002701-56 | EUDRACT_NUMBER | None | View |