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

Origin Biosciences

Status and phase: Completed; Phase 2

Conditions: Molybdenum Cofactor Deficiency, Type A

Treatments: Drug: ORGN001 (formerly ALXN1101)

Study type: Interventional

Funder types: Industry

Identifiers

NCT02047461

ALXN1101-MCD-201

2013-002701-56 (EudraCT Number)

Details and patient eligibility

About

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

Full 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.

Enrollment: 8 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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8 participants in 1 patient group

ORGN001 (formerly ALXN1101)

Experimental group

Description:

daily IV infusions

Treatment:

Drug: ORGN001 (formerly ALXN1101)