Clinical trial NCT02386839: Long-term Safety and Efficacy Outcome Study Comparing Children Previously Enrolled in Study ROPP-2008-01 for the Prevention of Retinopathy of Prematurity (ROP)
Official title: Long-term Outcome of Children Enrolled in Study ROPP-2008-01 Previously Treated With rhIGF-1/rhIGFBP-3 for the Prevention of Retinopathy of Prematurity (ROP) or Who Received Standard Neonatal Care
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Retinopathy of Prematurity (ROP)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2015-03-12
Enrollment: 76
Sex: ALL
Minimum age (years): 40
Maximum age (years): 108
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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).

Detailed description: Long-term Safety and Efficacy Outcome Study Comparing Children Previously Enrolled in Study ROPP-2008-01 for the Prevention of Retinopathy of Prematurity (ROP)

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.

Primary outcomes:
measure: Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA)
timeFrame: At 6 Months CA
measure: Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA
timeFrame: At 12 Months CA
measure: Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA
timeFrame: At 20 Months CA
measure: Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA
timeFrame: At 24 Months CA
measure: Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA
timeFrame: At 4.75 Years CA
measure: Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA
timeFrame: At 5 Years CA
measure: Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA
timeFrame: At 12 Months CA
measure: Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA
timeFrame: At 24 Months CA
measure: Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA
timeFrame: At 5 Years CA
measure: Number of Participants With Nystagmus at 12 Months CA
timeFrame: At 12 Months CA
measure: Number of Participants With Nystagmus at 24 Months CA
timeFrame: At 24 Months CA
measure: Number of Participants With Nystagmus at 5 Years CA
timeFrame: At 5 Years CA
measure: Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA
timeFrame: At 6 Months CA
measure: Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA
timeFrame: At 12 Months CA
measure: Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA
timeFrame: At 20 Months CA
measure: Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA
timeFrame: At 4.75 Years CA
measure: Number of Participants With Stereoacuity as Assessed With the Lang Stereotest At 5 Years CA
timeFrame: At 5 Years CA
measure: Number of Participants With Adverse Events (AEs)
timeFrame: From start of study up to end of study (up to 6.5 years)
Secondary outcomes:
measure: Change From Baseline in Body Weight Z-score
timeFrame: Baseline, 6 Months CA, 12 Months CA, 24 Months CA and 5 Years CA
measure: Change From Baseline in Height Z-score
timeFrame: Baseline, 6 Months CA, 12 Month CA, 24 Months CA and 5 Years CA
measure: Change From Baseline in Head Circumference Z-score
timeFrame: Baseline, 6 Months CA, 12 Months CA and 24 Months CA
measure: 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
timeFrame: Baseline (12 Months CA), 24 Months CA
measure: Cognitive Development as Assessed by Wechsler Preschool and Primary Scale of Intelligence (WPPSI-IV) Full Scale at 5 Years CA
timeFrame: At 5 Years CA
measure: Number of Participants With Abnormal Physical Examination
timeFrame: From start of study drug administration up to end of study (up to 6.5 years)
measure: Number of Participants Diagnosed With Neurological Examination for Cerebral Palsy (CP) by Neurological Examination at 24 Months CA
timeFrame: At 24 Months CA
measure: Number of Participants With Normal and Abnormal Hearing Screening Status
timeFrame: At 6 Months CA and 5 Years CA
measure: Change From Baseline (6 Months CA) in Child Behavior as Assessed by Vineland Adaptive Behaviour Scales (VABS-II)
timeFrame: Baseline (6 Months CA), 12 Months CA, 24 Months CA, and 5 Year CA
measure: Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories
timeFrame: At 24 Months CA and 5 Years CA
measure: Child Behavior as Assessed by Attention-Deficit/Hyperactivity Disorder Rating Scale (ADHD-RS) Based on Total Score
timeFrame: At 5 Years CA
measure: Child Behavior as Assessed by Social Communication Questionnaire (SCQ)
timeFrame: At 5 Years CA
measure: Number of Participants With Pulmonary Morbidity
timeFrame: At 6 Months CA, 12 Months CA and 24 Months CA
measure: Number of Participants With Survival Status
timeFrame: From start of study drug administration up to end of study (up to 6.5 years)
measure: Change From Baseline (3 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Infant Scale
timeFrame: Baseline (3 Months CA) up to 6 Months CA, 12 Months CA and 24 Months CA
measure: Change From Baseline (24 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Generic Core Scale (GCS) Total Score
timeFrame: Baseline (24 Months CA), up to 3 Years CA, 4 Years CA and 5 Years CA
measure: Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS)
timeFrame: At 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
measure: Health Status Measured by the Health Utilities Index (HUI) Mark 2 and 3
timeFrame: At 5 Years CA
measure: Health Care Resource Use (HCRU)
timeFrame: At 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
measure: Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department
timeFrame: At 3 Months CA, 6 Months CA, 12 Months CA , 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
measure: Health Care Resource Utilization (HCRU): Duration of Hospitalization
timeFrame: At 3 Months CA, 6 Months CA, 12 Months CA , 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
measure: Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support
timeFrame: At 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
Other outcomes:
Locations:
city: Mobile
country: United States
facility: University of South Alabama Children's and Women's Hospital
state: Alabama
city: Greenville
country: United States
facility: Vidant Medical Center
state: North Carolina
city: Oklahoma City
country: United States
facility: University of Oklahoma Health Sciences Center
state: Oklahoma
city: Madison
country: United States
facility: University of Wisconsin - Madison
state: Wisconsin
city: Firenze
country: Italy
facility: Azienda Ospedaliera Universitaria Careggi
city: Genova
country: Italy
facility: Istituto Giannina Gaslini-Istituto Pediatrico di Ricovero e
city: Padua
country: Italy
facility: University of Padua
city: Roma
country: Italy
facility: Policlinico Universitario Agostino Gemelli
city: Amsterdam
country: Netherlands
facility: VU Medical Center
city: Poznań
country: Poland
facility: Ginekologiczno-Położniczy Szpital Kliniczny Uniwersytetu Medycznego w Poznan
city: Lund
country: Sweden
facility: Skanes Universitetssjukhus Lund
city: Stockholm
country: Sweden
facility: Karolinska Universitetssjukhuset Huddinge
city: Cambridge
country: United Kingdom
facility: Addenbrookes Hospital
city: Chertsey
country: United Kingdom
facility: St Peter's Hospital
city: Liverpool
country: United Kingdom
facility: Alder Hey Childrens Hospital
city: London
country: United Kingdom
facility: UCL EGA Institute for Women's Health
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: