Phase 2
N=76
Long-term Safety and Efficacy Outcome Study Comparing Children Previously Enrolled in Study ROPP-2008-01 for the Prevention of Retinopathy of Prematurity (ROP)
Retinopathy of Prematurity (ROP)
Bottom Line
View on ClinicalTrials.gov: NCT02386839 ↗Enrolled (actual)
76
Serious AEs
6.6%
Results posted
Apr 2022
Primary outcome: Primary: Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) — 0; 0; 12; 19 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- rhIGF-1/rhIGFBP-3 (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Shire
- Primary completion
- Sep 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) |
0; 0; 12; 19; 12; 13 | — |
| PRIMARY Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA |
0; 1; 21; 26; 0; 5 | — |
| PRIMARY Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA |
0; 2; 17; 23; 2; 4 | — |
| PRIMARY Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA |
0; 2; 17; 25; 3; 4 | — |
| PRIMARY Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA |
11; 15; 4; 3; 0; 0 | — |
| PRIMARY Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA |
9; 11; 5; 6; 0; 0 | — |
| PRIMARY Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA |
1; 4; 24; 32; 0; 0 | — |
| PRIMARY Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA |
1; 5; 22; 29; 0; 1 | — |
| PRIMARY Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA |
1; 1; 13; 16; 0; 0 | — |
| PRIMARY Number of Participants With Nystagmus at 12 Months CA |
1; 2; 24; 34 | — |
| PRIMARY Number of Participants With Nystagmus at 24 Months CA |
0; 2; 23; 32 | — |
| PRIMARY Number of Participants With Nystagmus at 5 Years CA |
0; 2; 14; 15 | — |
| PRIMARY Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA |
-1.833; -1.417; 1.976; 1.838; 0.875; 0.932 | — |
| PRIMARY Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA |
-1.564; -2.438; 2.042; 1.491; 0.783; 0.926 | — |
| PRIMARY Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA |
-2.350; -1.700; 1.903; 1.278; 0.750; 0.778 | — |
| PRIMARY Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA |
-8.500; -0.500; 1.691; 1.383; 0.593; 0.603 | — |
| PRIMARY Number of Participants With Stereoacuity as Assessed With the Lang Stereotest At 5 Years CA |
13; 16; 1; 1 | — |
| PRIMARY Number of Participants With Adverse Events (AEs) |
11; 9 | — |
| SECONDARY Change From Baseline in Body Weight Z-score |
4.40; 4.30; 5.08; 4.88; 5.17; 4.99 | — |
| SECONDARY Change From Baseline in Height Z-score |
6.36; 6.19; 6.79; 7.25; 7.30; 7.78 | — |
| SECONDARY Change From Baseline in Head Circumference Z-score |
7.61; 7.12; 8.29; 7.83; 8.44; 8.05 | — |
| SECONDARY Change From Baseline (12 Months CA) in Cognitive Development as Assessed by Bayley Scales of Infant and Toddler Development (BSID-III) Composite Scores at 24 Months CA |
7.0; 6.6; 2.4; -0.9; 12.2; 10.8 | — |
| SECONDARY Cognitive Development as Assessed by Wechsler Preschool and Primary Scale of Intelligence (WPPSI-IV) Full Scale at 5 Years CA |
97.3; 91.6 | — |
| SECONDARY Number of Participants With Abnormal Physical Examination |
13; 18 | — |
| SECONDARY Number of Participants Diagnosed With Neurological Examination for Cerebral Palsy (CP) by Neurological Examination at 24 Months CA |
1; 5 | — |
| SECONDARY Number of Participants With Normal and Abnormal Hearing Screening Status |
20; 24; 1; 4; 15; 20 | — |
| SECONDARY Change From Baseline (6 Months CA) in Child Behavior as Assessed by Vineland Adaptive Behaviour Scales (VABS-II) |
-0.3; -1.5; 7.8; 0.8; -3.2; -3.9 | — |
| SECONDARY Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories |
23; 32; 0; 0; 0; 2 | — |
| SECONDARY Child Behavior as Assessed by Attention-Deficit/Hyperactivity Disorder Rating Scale (ADHD-RS) Based on Total Score |
11.9; 8.3 | — |
| SECONDARY Child Behavior as Assessed by Social Communication Questionnaire (SCQ) |
5.9; 5.2 | — |
| SECONDARY Number of Participants With Pulmonary Morbidity |
6; 7; 22; 32; 8; 4 | — |
| SECONDARY Number of Participants With Survival Status |
1; 0; 35; 40 | — |
| SECONDARY Change From Baseline (3 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Infant Scale |
-1.10; -0.60; -0.16; -0.57; 7.84; -4.82 | — |
| SECONDARY Change From Baseline (24 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Generic Core Scale (GCS) Total Score |
0.62; -1.13; 1.50; -3.46; -4.96; -4.49 | — |
| SECONDARY Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) |
21; 30; 3; 4; 0; 1 | — |
| SECONDARY Health Status Measured by the Health Utilities Index (HUI) Mark 2 and 3 |
0.929; 0.867; 0.893; 0.852 | — |
| SECONDARY Health Care Resource Use (HCRU) |
9.8; 8.3; 15.5; 10.3; 25.0; 22.5 | — |
| SECONDARY Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department |
2.5; 1.6; 2.2; 1.3; 1.6; 2.2 | — |
| SECONDARY Health Care Resource Utilization (HCRU): Duration of Hospitalization |
12.9; 5.5; 6.4; 7.4; 7.2; 9.0 | — |
| SECONDARY Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support |
0; 0; 32; 39; 0; 0 | — |
Summary
The main purpose of this study is to evaluate the long-term efficacy and safety outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (NCT01096784).
Eligibility Criteria
Inclusion Criteria
- Participant was randomized in Study ROPP-2008-01 Section D (NCT01096784).
- Participants parent or legally authorized representative(s) must provide written informed consent prior to performing any study-related activities. Study-related activities are any procedures that would not have been performed during normal management of the participant.
Exclusion Criteria
- Any other condition or therapy that, in the Investigator's opinion, may pose a risk to the Participant or interfere with the participants ability to be compliant with this protocol or interfere with the interpretation of results.
- The participant or participants parent or legally authorized representative(s) is unable to comply with the protocol as determined by the Investigator.
Data sourced from ClinicalTrials.gov (NCT02386839). 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.