Protocol Defined Safety Event Reporting

The following information is for healthcare professionals only. If you are a potential PALomino study patient, please click here.

It is important that all HCPs are aware of the safety events captured in the study, the process for reporting the events and the reporting timelines.

If a reportable safety event is identified, the event should be reported to the Coordinating Center (US) or Coordinating Registry Physician (EU) within 1 business day of the HCP becoming aware of the event.
If an event is not a reportable safety event in the study, it should be reported according to national reporting requirements.

The Coordinating Center (US) or CRP (EU) cannot assess seriousness, severity, causality, or clinical significance of reported lab results for patients that are not under their direct care. The HCP reporting the event will make these assessments and provide the information to the Coordinating Center (US) or CRP (EU) to ensure the safety event is captured accurately.

Maternal safety data collection begins after informed consent is obtained and continues through the completion of the pregnancy.

Fetal/Neonatal/Infant – begins after maternal informed consent is obtained and continues through 1 year post birth.

The following Maternal Safety Events should be reported:

Complications associated with pregnancy and birth:

  • Pregnancy outcome: spontaneous or therapeutic abortions, stillbirth (≥ 20 weeks gestation) are reportable as SAEs
  • Fetal, Neonatal, Infant outcomes (e.g., SGA—Small for Gestational Age, microcephaly, failure to thrive, preterm birth) are reportable as SAEs
  • Unplanned cesarian section is reportable as an SAE
  • Pregnancy and/or delivery complications which meet Seriousness criteria

Reportable Safety Events:

  • All Serious Adverse Events
  • All events of Anaphylaxis
  • All events of Acute Systemic Hypersensitivity Reaction
  • All Adverse Drug Reactions, except Grade 1 or 2 Injection Site Reactions
  • All events of Hypophenylalanemia defined as Blood Phe level ≤ 30 μmol/L for a minimum of 2 consecutive values
  • All device-related events, such as a bent needle (US only)

All Special Situation Events:

  • Unusual Failure in Efficacy
  • Abuse
  • Misuse
  • Medication Error
  • Overdose
  • Accidental/Occupational Exposure

Infant Events:

Non-serious and all Serious Adverse Events including but not limited to:

  • Fetal Anomalies (e.g., SGA, FGR, microcephaly, congenital heart defects)
  • AEs which meet Seriousness criteria (including, but not limited to failure to thrive, developmental delays, or other congenital malformations)
  • Hypersensitivity reaction to pegvaliase via breast milk
  • Skin reactions continuing without improvement for greater than 14 days
  • Anaphylactic reactions
  • Angioedema
  • Serum sickness