Clinical trial NCT02047461: Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Molybdenum Cofactor Deficiency, Type A
Keywords: Molybdenum cofactor deficiency (MoCD)
molybdenum cofactor (MoCo) biosynthesis
sulfite oxidase (SO)
xanthine dehydrogenase
aldehyde oxidase
S sulfocysteine (SSC)
Lead sponsor: Origin Biosciences
Sponsor class: INDUSTRY
First posted: 2014-01-28
Enrollment: 8
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Safety of ORGN001 (Formerly ALXN1101)
timeFrame: Baseline to Month 24 for all patients plus additional follow-up up to Month 90
Secondary outcomes:
measure: Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)
timeFrame: First 6 months at each dose level, where available
measure: S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time
timeFrame: Baseline to Month 24 for all patients plus additional follow-up to Month 90
measure: Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination
timeFrame: Baseline to Month 24 for all patients plus additional follow-up until Month 30
measure: Long-term Safety of ORGN001 (Formerly ALXN1101)
timeFrame: Baseline to Month 24 for all patients plus additional follow up until Month 72
Other outcomes:
Locations:
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
city: Melbourne
country: Australia
facility: Monash Medical Centre
city: Groningen
country: Netherlands
facility: Beatrix Children's Hospital
city: Tunis
country: Tunisia
facility: Unité des maladies métaboliques
city: Glasgow
country: United Kingdom
facility: Royal Hospital for Sick Children
city: Manchester
country: United Kingdom
facility: Manchester University Hospitals NHS Foundation Trust
Study officials:
References: