Clinical Trial: Safety & Efficacy Study of ALXN1101 in Pediatric Patients With MoCD Type A Currently Treated With rcPMP

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ALXN1101 in Pediatric Patients With Molybdenum Cofactor Deficiency

Brief Summary: This study will include a screening period, a 6-month treatment period, and a 60-month, long-term extension period.

Detailed Summary: Patients will receive daily IV infusions of 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 through Month 66 at their last tolerated dose.
Sponsor: Alexion Pharma GmbH

Current Primary Outcome: Safety of ALXN1101 [ Time Frame: First 6 months of treatment ]

  • The type, number and frequency of adverse events and severity of adverse events (AEs) and serious adverse events (SAEs)
  • Change from baseline in: clinical laboratory assessments, findings on physical examination, vital sign measurements, and EEG results


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Pharmacokinetics parameters of ALXN1101 [ Time Frame: First 6 months of treatment ]
    Plasma concentration (Cmax), time to maximum observed plasma concentration (tmax), area under the plasma concentration-time curve (AUC), and terminal half-life (t½)
  • Effect of ALXN1101 on urine and blood biomarkers [ Time Frame: Up to Month 66 ]
    Change from baseline in urine and blood biomarkers.
  • Effect of ALXN1101 on neurologic function including motor examination [ Time Frame: Up to Month 66 ]
    Change from baseline on repeated Neurologic examinations such as muscle strength and tone, as well as sensory and reflex exam.
  • Long-term safety of ALXN1101 [ Time Frame: Up to Month 66 ]
    • The type, number and frequency of adverse events and serious adverse events (SAEs)
    • Change from baseline in: clinical laboratory assessments, findings on physical examination, vital sign measurements, and EEG results
  • Effect of ALXN1101 on cognitive functions [ Time Frame: Up to Month 66 ]
    Change from baseline in cognitive function using age appropriate measures of cognition, eg. Wechsler Preschoool and Primary Scale of intelligence


Original Secondary Outcome:

  • Pharmacokinetics parameters of ALXN1101 [ Time Frame: First 6 months of treatment ]
    Plasma concentration (Cmax), time to maximum observed plasma concentration (tmax), area under the plasma concentration-time curve (AUC), and terminal half-life (t½)
  • Effect of ALXN1101 on urine and blood biomarkers [ Time Frame: Up to Month 36 ]
    Change from baseline in urine and blood biomarkers.
  • Effect of ALXN1101 on neurologic function including motor examination [ Time Frame: Up to Month 36 ]
    Change from baseline on repeated Neurologic examinations such as tone, strengthen and reflexes.
  • Long-term safety of ALXN1101 [ Time Frame: Up to Month 36 ]
    • The type, number and frequency of adverse events and serious adverse events (SAEs)
    • Change from baseline in: clinical laboratory assessments, findings on physical examination, vital sign measurements, and EEG results
  • Effect of ALXN1101 on cognitive functions [ Time Frame: Up to Month 36 ]
    Change from baseline in cognitive function using age appropriate measures of cognition, eg. Wechsler Preschoool and Primary Scale of intelligence


Information By: Alexion Pharma GmbH

Dates:
Date Received: January 9, 2014
Date Started: April 2014
Date Completion: June 2020
Last Updated: February 6, 2017
Last Verified: February 2017