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

Phase 2CompletedNCT02047461

Origin Biosciences8 enrolled

Overview

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

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Molybdenum Cofactor Deficiency, Type A

Interventions

ORGN001 (formerly ALXN1101)DRUG

IV infusion

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (6)

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

Outcomes

Primary Outcomes

Safety of ORGN001 (Formerly ALXN1101)

Treatment Emergent Serious Adverse Events

Time frame: Baseline to Month 24 for all patients plus additional follow-up up to Month 90

Secondary Outcomes

Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)

ORGN001 levels by dose at pre-infusion and end of infusion (EOI) at scheduled timepoints

Time frame: First 6 months at each dose level, where available

S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time

Analyses were performed on urine SSC, a biomarker of the MoCD pathway. Levels of SSC measured in urine were normalized to urine creatinine levels. The observed value, change, and percent change in urine and blood SSC levels from baseline were summarized by visit over time.

Time frame: Baseline to Month 24 for all patients plus additional follow-up to Month 90

Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination

Change from baseline on repeated Neurologic examinations such as muscle strength and tone, as well as sensory and reflex exam.

Time frame: Baseline to Month 24 for all patients plus additional follow-up until Month 30

Long-term Safety of ORGN001 (Formerly ALXN1101)

Change from baseline in Seizure frequency

Time frame: Baseline to Month 24 for all patients plus additional follow up until Month 72

Data from ClinicalTrials.gov

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