Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP

Completed · Phase 2

Conditions studied: Molybdenum Cofactor Deficiency, Type A

In brief

This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.

Key facts

Study ID

NCT02047461

Run by

Origin Biosciences

People needed

8

Starts

2014-04-01

Expected to finish

2022-10-01

Last updated by the study team

2023-10-17

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation)
• Currently treated with rcPMP infusions

You may not qualify if…

• Current or planned treatment with another investigational drug or device, with the exception rcPMP treatment through Day -1.

Where it is running

• Children's Hospital of Wisconsin — Milwaukee, Wisconsin, United States
• Monash Medical Centre — Melbourne, Australia
• Beatrix Children's Hospital — Groningen, Netherlands
• Unité des maladies métaboliques — Tunis, Tunisia
• Royal Hospital for Sick Children — Glasgow, United Kingdom
• Manchester University Hospitals NHS Foundation Trust — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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