2-STEP: A single-centre, phase II study to evaluate the safety, tolerability and pharmacokinetics of 2-Iminobiotin (2-IB) in neonates with gestational age of *36 weeks with moderate to severe perinatal asphyxia treated with therapeutic hypothermia

Perinatal asphyxia is a rare but life-threatening condition. The introduction
of therapeutic hypothermia as standard care has improved the outcome of these
patients, but still almost half of the treated neonates die or have serious
long term morbidity. For that reason additional
neuroprotective treatment is warranted. 2-IB has shown to be effective in
multiple animal models in substantially diminishing the neurological damage
after perinatal
asphyxia. Also it has shown to be safe and well tolerated in both juvenile and
adult animal models, in adult human volunteers and in a small group of neonates
suffering from perinatal asphyxia but not treated with hypothermia.

To explore the safety, tolerability and the pharmacokinetic profile of 2-IB
when given on top of therapeutic hypothermia.

Open label phase II study

None, all subjects are treated with 2-Iminobiotin in addition to standard care

The additional burden placed on subjects in this trial is minimal and does not
interfere with standard care. The risks associated with administration of
2-Iminobiotin are judged to be minimal since no relevant adverse reaction were
reported to date. Based on the vulnerable population, the overall risk
classification is upgraded to *moderate* with a subsequent monitoring plan.
On an individual level, administration of 2-Iminobiotin could have a positive
therapeutic effect. On a group level, information will become available
regarding pharmacokinetics and safety, which is of great importance for further
clinical research.