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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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)

Conditions
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Molybdenum Cofactor Deficiency, Type A

Intervention / Treatment
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* Drug: ORGN001 (formerly ALXN1101)


Participation Criteria
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Eligibility Criteria
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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.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ORGN001 (formerly ALXN1101)<br>daily IV infusions | Drug: ORGN001 (formerly ALXN1101)<br>* IV infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety of ORGN001 (Formerly ALXN1101) | Treatment Emergent Serious Adverse Events | Baseline to Month 24 for all patients plus additional follow-up up to Month 90 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | ORGN001 levels by dose at pre-infusion and end of infusion (EOI) at scheduled timepoints | 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. | 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. | 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 | Baseline to Month 24 for all patients plus additional follow up until Month 72 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Molybdenum cofactor deficiency (MoCD), molybdenum cofactor (MoCo) biosynthesis, sulfite oxidase (SO), xanthine dehydrogenase, aldehyde oxidase, S sulfocysteine (SSC)