A Non-interventional Post Authorisation Safety Study (PASS) of Patients with MoCD Type A Treated with NULIBRY (fosdenopterin)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  NULIBRY
• Medicinal product name, other:
  Fosdenopterin
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AX19) fosdenopterin

  fosdenopterin
• Medical condition to be studied:
  Molybdenum cofactor deficiency
• Additional medical condition(s):
  MoCD Type A

Population studied

• Short description of the study population:
  Male and female patients who have been diagnosed with MoCD Type A and have been prescribed and treated with NULIBRY will be approached for formal consent to participate in the study. Due to the life-threatening nature of the disease, and because NI-PASS will consider patients being treated and who previously received NULIBRY, patients of any age will be considered for NI-PASS inclusion.
• Age groups:
  • Paediatric Population (< 18 years)

    • Preterm newborn infants (0 – 27 days)
    • Term newborn infants (0 – 27 days)
    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)

Study design details

• Study design:
  This is a multicentre, non-interventional post-authorisation safety study (NI-PASS). The purpose of this study is to describe contemporary, real-world presentation, and clinical outcomes in patients with MoCD Type A.
• Main study objective:
  Safety is the primary objective of this NI-PASS. Safety analyses will be performed for all patients who consented and received at least one dose of NULIBRY. The secondary objective of this NI-PASS is to evaluate the effectiveness of NULIBRY in MoCD Type A patients.
• Setting:
  Routine clinical setting
• Outcomes:
  Primary safety outcomes ; Primary safety outcomes will be derived from the ID, collected prospectively, and reconciled ; with clinician notes at the time of periodic chart abstraction, and include: ;  Occurrence of adverse events, serious adverse events, adverse events of special interest, pregnancy/lactation ;  Overall survival ; ; Secondary efficacy outcomes ; Secondary efficacy outcomes will be collected (via chart abstraction) by the HCP and include: ;  Growth parameters: ; o Body weight ; o Height ; o Head circumference ;  Feeding methods