|- candidate number||22211|
|- NTR Number||NTR5221|
|- ISRCTN||ISRCTN no longer applicable|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||19-mei-2015|
|- Secondary IDs||14-671; NL50996.041.14 METC; ABR|
|- Public Title||A study looking at 2-Iminobiotin as treatment for newborn children suffering from insufficient oxygen supply to the brain in addition to standard treatment with cooling.|
|- Scientific Title||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 ¡Ý36 weeks with moderate to severe perinatal asphyxia treated with therapeutic hypothermia|
|- hypothesis||To explore the short term safety and tolerability of 2-IB and the pharmacokinetic profile of 2-IB when given on top of therapeutic hypothermia|
|- Healt Condition(s) or Problem(s) studied||Perinatal asphyxia|
|- Inclusion criteria||1. Neonates with ≥ 36 and <44 weeks gestation who are eligible to receive therapeutic hypothermia.|
2. Ability to start treatment within 12 hours after birth.
|- Exclusion criteria||1. Inability to insert an indwelling catheter (umbilical venous catheter or percutaneously inserted central catheter, preferably multiple lumen) for administration of the drug or an arterial line for recurrent blood sampling.|
2. Major congenital malformations, specifically malformations that may affect the renal function.
|- mec approval received||yes|
|- multicenter trial||no|
|- Type||Single arm|
|- planned startdate ||1-jun-2015|
|- planned closingdate||1-jan-2017|
|- Target number of participants||12|
|- Interventions||Administration of the study drug, blood sampling|
|- Primary outcome||Safety assessments include: vital signs, clinical laboratory parameters, clinical evaluation and (severe) adverse events and local tolerance.|
Pharmacokinetic assessment: a maximum of five PK samples will be taken. The exact time points to determined before start of the study based on all data available at that moment.
The following pharmacokinetic parameters will be calculated for each patient, using the actual sampling times:
• C-max (observed maximum plasma concentration)
• AUC-0-6h (area under the plasma concentration-time curve from time 0 to 6h after administration)
• AUC-0-∞ (area under the plasma concentration-time curve from time 0 to infinity)
• T-end of infusion (time at maximum plasma concentration).
•t1/2 (terminal elimination half-life)
•Vd (volume of distribution)
|- Secondary outcome||•To gather preliminary signs of short term efficacy as defined by the Lac/NAA ratios using MRS at 3-7 days after birth and the percentage of surviving patients with a normal aEEG at 60h after birth.
|- Timepoints||Safety assessments will be recorded during treatment period of 2-iminobiotin and will continue for at least 96 hours or until discharge from the hospital
Blood sampling for pharmacokinetic assessment will occur during the treatment period of 2-iminobiotin and shortly thereafter|
MRI(incl DWI):3-7 days after birth.
aEEG: will be recorded before start treatment till at least 72 hours after birth.
|- Trial web site|
|- CONTACT FOR PUBLIC QUERIES||Dr.
|- CONTACT for SCIENTIFIC QUERIES||Dr.
|- Sponsor/Initiator ||University Medical Center Utrecht (UMCU)|
(Source(s) of Monetary or Material Support)
|University Medical Center Utrecht (UMCU), Neurophyxia BV|
|- Brief summary|
|- Main changes (audit trail)|
|- RECORD||19-mei-2015 - 30-jul-2015|