Phase 2
N=121
IGF-1/IGFBP3 Prevention of Retinopathy of Prematurity
Retinopathy of Prematurity (ROP)
Bottom Line
View on ClinicalTrials.gov: NCT01096784 ↗Enrolled (actual)
121
Serious AEs
70.3%
Results posted
Jun 2017
Primary outcome: Primary: Severity of Retinopathy of Prematurity (ROP) as Compared to the Severity of ROP in an Untreated Control Population — 14; 24; 4; 4 participants — p=0.0642
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- rhIGF-I/rhIGFBP-3 (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Shire
- Primary completion
- Mar 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Severity of Retinopathy of Prematurity (ROP) as Compared to the Severity of ROP in an Untreated Control Population |
14; 24; 4; 4; 17; 13 | 0.0642 |
| SECONDARY Time to Discharge From Neonatal Intensive Care (TDNIC) |
82.00; 74.00 | — |
| SECONDARY Number of Participants With Bronchopulmonary Dysplasia (BPD) |
4; 4; 23; 16; 9; 5 | — |
| SECONDARY Rate of Change in Body Weight |
0.021; 0.023 | — |
| SECONDARY Rate of Change in Length |
0.141; 0.156 | — |
| SECONDARY Rate of Change in Head Circumference |
0.115; 0.119 | — |
| SECONDARY Brain Development Assessed by Brain Volume at 40 Weeks PMA/EOS |
87.94; 93.70; 206.34; 221.98; 110.23; 117.62 | — |
| SECONDARY Percentage of Participants With Intraventricular Hemorrhage (IVH) |
19.67; 30.0; 80.33; 70.0 | — |
| SECONDARY Area Under Curve for Maximum Severity of ROP Stage (AUC for ROP) |
47.95; 32.17 | — |
| SECONDARY Percentage of Participants With Maximum Severity of ROP Stage Greater Than or Equal to 3 at Any Time During the Study |
25.53; 18.00; 74.47; 82.00 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Event (TEAE) and Treatment Emergent Serious Adverse Event (TESAE) |
60; 60; 48; 37 | — |
| SECONDARY Percentage of Serum IGF-1 Concentrations Falling Within Target Range After Infusion of rhIGF-1/rhIGFBP-3 |
66.23; 6.28 | — |
| SECONDARY Serum Concentrations of IGFBP-3 After Intravenous (IV) Infusion of rhIGF-1/rhIGFBP-3 |
494.2; 469.9; 830.1; 882.1 | — |
| SECONDARY Serum Concentrations of Acid Labile Sub-unit (ALS) After Intravenous (IV) Infusion of rhIGF-1/rhIGFBP-3 |
411.9; 500.3; 1804.6; 2114.3 | — |
Summary
To compare the severity of retinopathy of prematurity (ROP) among treated infants with an untreated control population, matched for gestational age at birth while confirming the dose of rhIGF-1/rhIGFBP-3 is safe and efficacious.
Eligibility Criteria
Inclusion Criteria
- Signed informed consent from parents/guardians;
- 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
Exclusion Criteria
- Subjects born small for gestational age (SGA), ie, body weight at birth 10 mmol/L at Study Day 0 (day of birth) to exclude severe congenital abnormalities of glucose metabolism
- Anticipated need of administration of erythropoietin (rhEPO) during treatment with study drug.
- Any maternal diabetes requiring insulin during the pregnancy
- Clinically significant neurological disease according to the Investigator's opinion(Stage 1 IVH allowed)
- 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
- Monozygotic twins
- Subject participating or plans to participate in a clinical study of another investigational study drug
Data sourced from ClinicalTrials.gov (NCT01096784). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.