Retinopathy of Prematurity (ROP) MedDRA version: 17.1 Level: SOC Classification code 10015919 Term: Eye disorders System Organ Class: 10015919 - Eye disorders
Conditions
Interventions
Product Name: mecasermin rinfabate
Product Code: rhIGF-I/rhIGFBP-3
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: mecasermin rinfabate
Current Sponsor code: SHP-607
Other de
Sponsors
Premacure AB, A member of the Shire Group of Companies
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Each subject must meet the following criteria to be enrolled in this study: 1- A signed written informed consent from the subject's parents/guardians prior to any study-related procedures that has been approved by the Institutional Review Board (IRB)/Independent Ethics Committee (IEC) 2- Subject must be between GA of 26 weeks + 0 days and 27 weeks +6 days (Study Section A) or between GA of 23 weeks + 0 days and 27 weeks + 6 days (Study Sections B, C, and D), inclusive Are the trial subjects under 18? yes Number of subjects for this age range: 120 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Subjects who meet any of the following criteria will be excluded from the study: 1- Subjects born small for gestational age (SGA), ie, weight at birth 10 mmol/L at Study Day 0 (day of birth) to exclude severe congenital abnormalities of glucose metabolism 5- Anticipated need of administration of erythropoietin (rhEPO) during treatment with study drug 6- Any maternal diabetes requiring insulin while pregnant 7- Clinically significant neurological disease according to the investigator’s opinion (Stage 1 IVH allowed) 8- Any other condition or therapy that, in the investigator’s opinion, may pose a risk to the subject or interfere with the subject’s ability to be compliant with this protocol or interfere with interpretation of results 9- Monozygotic twins 10- Subject participating or plans to participate in a clinical study of another investigational study drug
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Section A: To establish the dose and sampling schedule to be used in Study Sections B and C Sections B and C: To determine the dose of IMP, administered by continuous IV infusion, required to reach and maintain a physiological range of serum IGF-1 of 20-60 µg/L, defined as the in utero levels of IGF-1 for corresponding GA in a normal population To determine serum concentrations of IGF-1 and associated pharmacokinetic parameters after continuous IV infusion of IMP Section D: To determine the effect of IMP on the severity of ROP as compared to the severity of ROP in an untreated control population To evaluate the dose of IMP administered by continuous IV infusion, required to reach and maintain a physiological range of serum IGF-1 of 28-109 µg/L To determine serum concentrations of IGF-1 and associated pharmacokinetic parameters after continuous IV infusion of IMP To determine serum concentration of IGFBP-3 and acid labile subunit (ALS) after continuous IV infusion of IMP;Secondary Objective: To determine the effect of rhIGF-1/rhIGFBP-3 on other efficacy parameters and determine the safety profile of rhIGF-1/rhIGFBP-3 when compared with standard neonatal care in preterm infants;Primary end point(s): Sections A, B, C: Primary endpoint: Severity of ROP Sections D: Primary endpoint: Maximum severity of ROP stage across all retinal examinations;Timepoint(s) of evaluation of this end point: First ROP examination shall occur at 5 to 6 weeks or PMA 31 weeks. Follow-up examination should occur at least 1 to 2 weeks depending upon the clinical evaluation of the paediatric ophtalmologist. Final ROP assessment will occur at 40 weeks +/- 4 days. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The key secondary endpoint of this study is in Section D: time to discharge from the neonatal intensive care (TDNIC).;Timepoint(s) of evaluation of this end point: TDNIC will be calculated from the day of birth to the day of the discharge | — |
Countries
Canada, Italy, Netherlands, Poland, Sweden, United Kingdom, United States
Contacts
Public ContactEmily Jochim
Shire Human Genetic Therapies Inc.
Outcome results
None listed