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

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

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).

Study Overview

• Status: Completed
• Conditions: Retinopathy of Prematurity (ROP)
• Intervention / Treatment: Drug: rhIGF-1/rhIGFBP-3

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)

• Study Type: Interventional
• Enrollment (Actual): 76
• Phase: Phase 2

Contacts and Locations

Study Locations

• Italy
  • Firenze, Italy, 50134
    • Azienda Ospedaliera Universitaria Careggi
  • Genova, Italy, 16147
    • Istituto Giannina Gaslini-Istituto Pediatrico di Ricovero e
  • Padua, Italy, 35128
    • University of Padua
  • Roma, Italy, 00168
    • Policlinico Universitario Agostino Gemelli
• Netherlands
  • Amsterdam, Netherlands, 1081 HZ
    • VU Medical Center
• Poland
  • Poznań, Poland, 60-535
    • Ginekologiczno-Położniczy Szpital Kliniczny Uniwersytetu Medycznego w Poznan
• Sweden
  • Lund, Sweden, SE-22185
    • Skånes universitetssjukhus Lund
  • Stockholm, Sweden, 141 86
    • Karolinska Universitetssjukhuset Huddinge
• United Kingdom
  • Cambridge, United Kingdom, CB2 0QQ
    • Addenbrookes Hospital
  • Chertsey, United Kingdom, KT16 0PZ
    • St Peter's Hospital
  • Liverpool, United Kingdom, L8 7SS
    • Alder Hey Childrens Hospital
  • London, United Kingdom, WC1E 6AU
    • UCL EGA Institute for Women's Health
• United States
  • Alabama
    • Mobile, Alabama, United States, 36604
      • University of South Alabama Children's and Women's Hospital
  • North Carolina
    • Greenville, North Carolina, United States, 27834
      • Vidant Medical Center
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • University of Oklahoma Health Sciences Center
  • Wisconsin
    • Madison, Wisconsin, United States, 53715
      • University of Wisconsin - Madison

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 9 months to 2 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Antecedent Standard of Care; Participants who were treated with standard neonatal care in study ROPP-2008-01 (NCT01096784) were enrolled in this group for assessment of rhIGF-1/rhIGFBP-3 long-term efficacy and safety outcomes.	Drug: rhIGF-1/rhIGFBP-3; Participants who received "rhIGF-1/rhIGFBP-3" in study ROPP-2008-01 (NCT01096784) will be enrolled to this study. No investigational product will be administered in this study.; Other Names: Mecasermin rinfabate
Experimental: Antecedent rhIGF-1/rhIGFBP-3; Participants who were treated with rhIGF-1/rhIGFBP-3 in study ROPP-2008-01 (NCT01096784) were enrolled in this group for assessment of rhIGF-1/rhIGFBP-3 long-term efficacy and safety outcomes.	Drug: rhIGF-1/rhIGFBP-3; Participants who received "rhIGF-1/rhIGFBP-3" in study ROPP-2008-01 (NCT01096784) will be enrolled to this study. No investigational product will be administered in this study.; Other Names: Mecasermin rinfabate

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA)	Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity >= 20/40 or >= 15 cycles/degree); below normal (20/200 <= measurable acuity < 20/40 or 3 cycles/degree <= measurable acuity < 15 cycles/degree); poor (measurable acuity <= 20/200 or <= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study.	At 6 Months CA
Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA	Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity >= 20/40 or >= 15 cycles/degree); below normal (20/200 <= measurable acuity < 20/40 or 3 cycles/degree <= measurable acuity < 15 cycles/degree); poor (measurable acuity <= 20/200 or <= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study.	At 12 Months CA
Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA	Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity >= 20/40 or >= 15 cycles/degree); below normal (20/200 <= measurable acuity < 20/40 or 3 cycles/degree <= measurable acuity < 15 cycles/degree); poor (measurable acuity <= 20/200 or <= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study.	At 20 Months CA
Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA	Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity >= 20/40 or >= 15 cycles/degree); below normal (20/200 <= measurable acuity < 20/40 or 3 cycles/degree <= measurable acuity < 15 cycles/degree); poor (measurable acuity <= 20/200 or <= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study.	At 24 Months CA
Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA	Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity >= 20/40 or >= 15 cycles/degree); below normal (20/200 <= measurable acuity < 20/40 or 3 cycles/degree <= measurable acuity < 15 cycles/degree); poor (measurable acuity <= 20/200 or <= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study.	At 4.75 Years CA
Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA	Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity >= 20/40 or >= 15 cycles/degree); below normal (20/200 <= measurable acuity < 20/40 or 3 cycles/degree <= measurable acuity < 15 cycles/degree); poor (measurable acuity <= 20/200 or <= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study.	At 5 Years CA
Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA	Ocular alignment was assessed in primary gaze by comparing the position of the corneal light reflection in left eye and right eye (corneal light reflection assessment). Presence or absence of strabismus was recorded in primary gaze and in as many of the 9 positions of gaze as feasible with the cover test assessment of refixation movement. Extraocular muscle over action or deficiency was recorded. Ocular motility referred to eye movements governed by the 6 extraocular muscles in each eye. It was assessed by examiner observation of the participants ability to abduct, adduct, supra, and inferoduct each eye. Ocular alignment and motility included the presence or absence of strabismus (classified as Esotropia [inward turn of the eye], Exotropia [outward turn of the eye], Hypertropia [upward turn of the eye], Hypotropia [downward turn of the eye]) was recorded. Number of participants with combined data for ocular alignment and oculomotor examination (motility) at 12 months CA were reported.	At 12 Months CA
Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA	Ocular alignment was assessed in primary gaze by comparing the position of the corneal light reflection in left eye and right eye (corneal light reflection assessment). Presence or absence of strabismus was recorded in primary gaze and in as many of the 9 positions of gaze as feasible with the cover test assessment of refixation movement. Extraocular muscle over action or deficiency was recorded. Ocular motility referred to eye movements governed by the 6 extraocular muscles in each eye. It was assessed by examiner observation of the participants ability to abduct, adduct, supra, and inferoduct each eye. Ocular alignment and motility included the presence or absence of strabismus (classified as Esotropia, Exotropia, Hypertropia, Hypotropia) was recorded. Number of participants with combined data for ocular alignment and oculomotor examination (motility) assessed by corneal light reflex and by the cover test at 24 months CA were reported.	At 24 Months CA
Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA	Ocular alignment was assessed in primary gaze by comparing the position of the corneal light reflection in left eye and right eye (corneal light reflection assessment). Presence or absence of strabismus was recorded in primary gaze and in as many of the 9 positions of gaze as feasible with the cover test assessment of refixation movement. Extraocular muscle over action or deficiency was recorded. Ocular motility referred to eye movements governed by the 6 extraocular muscles in each eye. It was assessed by examiner observation of the participants ability to abduct, adduct, supra, and inferoduct each eye. Ocular alignment and motility included the presence or absence of strabismus (classified as Esotropia, Exotropia, Hypertropia, Hypotropia) was recorded. Number of participants with combined data for ocular alignment and oculomotor examination (motility) assessed by corneal light reflex and by the cover test at 5-Years CA were reported.	At 5 Years CA
Number of Participants With Nystagmus at 12 Months CA	Nystagmus was observed during the ocular alignment assessments. Presence and absence of nystagmus was reported at 12 Months CA.	At 12 Months CA
Number of Participants With Nystagmus at 24 Months CA	Nystagmus was observed during the ocular alignment assessments. Presence and absence of nystagmus was reported at 24 Months CA.	At 24 Months CA
Number of Participants With Nystagmus at 5 Years CA	Nystagmus was observed during the ocular alignment assessments. Presence and absence of nystagmus was reported at 5 Years CA.	At 5 Years CA
Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA	Refraction was a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia was measured and recorded in diopters for each eye individually (left eye and right eye). Refraction with cycloplegia performed as part of the corrective lens determination procedure included measurements of sphere, cylinder, axis, and prism for each individual eye (left eye and right eye). For sphere, the negative values are summarized as 'nearsighted', and the positive values (including sphere of 0.00) are summarized as 'farsighted'.	At 6 Months CA
Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA	Refraction was a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia was measured and recorded in diopters for each eye individually (left eye and right eye). Refraction with cycloplegia performed as part of the corrective lens determination procedure included measurements of sphere, cylinder, axis, and prism for each individual eye (left eye and right eye). For sphere, the negative values are summarized as 'nearsighted', and the positive values (including sphere of 0.00) are summarized as 'farsighted'.	At 12 Months CA
Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA	Refraction was a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia was measured and recorded in diopters for each eye individually (left eye and right eye). Refraction with cycloplegia performed as part of the corrective lens determination procedure included measurements of sphere, cylinder, axis, and prism for each individual eye (left eye and right eye). For sphere, the negative values are summarized as 'nearsighted', and the positive values (including sphere of 0.00) are summarized as 'farsighted'.	At 20 Months CA
Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA	Refraction was a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia was measured and recorded in diopters for each eye individually (left eye and right eye). Refraction with cycloplegia performed as part of the corrective lens determination procedure included measurements of sphere, cylinder, axis, and prism for each individual eye (left eye and right eye). For sphere, the negative values are summarized as 'nearsighted', and the positive values (including sphere of 0.00) are summarized as 'farsighted'.	At 4.75 Years CA
Number of Participants With Stereoacuity as Assessed With the Lang Stereotest At 5 Years CA	Stereoacuity, a measure of depth perception, was assessed using the Lang Stereotest. Number of participants with presence and absence of stereopsis (the ability to perceive depth and 3-dimensional structure) was reported.	At 5 Years CA
Number of Participants With Adverse Events (AEs)	An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment.	From start of study up to end of study (up to 6.5 years)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Body Weight Z-score	Body weight collected using calibrated scales (type of scale was dependent upon participant's age). Measure recorded to the nearest 0.1 kilogram (kg). Z-score based on participants chronological age and utilizing World Health Organization child growth standards by adjusting age and sex- matched means and standard deviations (norm). Z-score is standard score that gives idea of how far from the mean a data point is. If Z-score is 0, it indicates that the data point's score is identical to the mean score. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Baseline is first assessment in the antecedent study (ROPP-2008-01 [NCT01096784]). Change from baseline in body weight Z-score at 6-month, 12-month, 24-month and 5-year CA were reported.	Baseline, 6 Months CA, 12 Months CA, 24 Months CA and 5 Years CA
Change From Baseline in Height Z-score	Height was measured using Z-score. Z-score was calculated based on the participants chronological age and utilizing World Health Organization child growth standards by adjusting age and sex- matched means and standard deviations (norm). Z-score is a standard score that gives an idea of how far from the mean a data point is. If a Z-score is 0, it indicates that the data point's score is identical to the mean score. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Baseline was defined as the first assessment in the antecedent study (ROPP-2008-01 [NCT01096784]). Change from baseline in Height Z-score at 6-month, 12-month, 24-month and 5-year CA were reported.	Baseline, 6 Months CA, 12 Month CA, 24 Months CA and 5 Years CA
Change From Baseline in Head Circumference Z-score	Head circumference measured for all participants using a "lasso" type, non-stretchable measuring tape such as the Lasso-o tape. Z-score calculated based on participants chronological age and utilizing World Health Organization child growth standards by adjusting age, sex matched means and standard deviations (norm).Z-score is standard score that gives idea of how far from mean a data point is. If a Z-score is 0, it indicates that data point's score is identical to mean score. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Baseline was defined as the first assessment in the antecedent study (ROPP-2008-01 [NCT01096784]). Change from baseline in Head Circumference Z-score at 6, 12 and 24-months reported.	Baseline, 6 Months CA, 12 Months CA and 24 Months CA
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	BSID-III was to assess cognitive, motor, and language skills, and applicable to children aged 1-42 months. There are 5 subscales, cognitive subscale (Ranges: 55-145) stands alone while 2 language subscales (expressive and receptive) combine to make a total language score (Ranges: 47-153) and 2 motor subtests (fine and gross motor) form combined motor scale (Ranges: 46-154). Higher scores represent greater cognitive, language and motor abilities. Positive value indicates improvement and negative value indicate worsening in cognitive development. The 12 Months CA considered as baseline for this outcome measure. Composite scores derived from various sums of subtest scaled scores and scaled to a metric with a mean of 100 and a standard deviation of 15 and range from 40 to 160. Higher values denote stronger skills and abilities in the domain, indicating better outcomes.	Baseline (12 Months CA), 24 Months CA
Cognitive Development as Assessed by Wechsler Preschool and Primary Scale of Intelligence (WPPSI-IV) Full Scale at 5 Years CA	The WPPSI-IV is a measure of general cognitive development in children that has components of both verbal and non-verbal tasks. It is applicable to preschoolers and young children aged 2 years +6 months to 7 years +7 months and is a direct assessment of a child's cognitive skills. The test framework of the WPPSI-IV is organized into five Primary Index sub scales: Verbal Comprehension, Visual Spatial, Fluid Reasoning, Working Memory, and Processing Speed. The Full Scale includes all of the sub scales at the Primary Index scale level, as well as any additional supplemental subtests that may be used to derive the Full Scale IQ. Composite scores are derived from the sum of sub scaled scores with a mean of 100 and a standard deviation of 15. Composite score for Full Scale IQ and subscales (Verbal Comprehension, Visual Spatial, Fluid Reasoning, Working Memory, and Processing Speed) ranges from 40 (extremely low) to 160 (very superior). Data for Full scale IQ was reported here.	At 5 Years CA
Number of Participants With Abnormal Physical Examination	Physical examination includes general appearance; head and neck, eyes, ears, nose and throat; chest and lungs; endocrine, cardiovascular system, abdomen, genitourinary, skin and musculoskeletal system.	From start of study drug administration up to end of study (up to 6.5 years)
Number of Participants Diagnosed With Neurological Examination for Cerebral Palsy (CP) by Neurological Examination at 24 Months CA	A comprehensive neurological examination for the diagnosis of CP was conducted. The Amiel-Tison neurological examination framework was utilized for this assessment and it was conducted by trained medical professionals. Number of Participants diagnosed with CP by neurological examination at 24 Months CA were reported.	At 24 Months CA
Number of Participants With Normal and Abnormal Hearing Screening Status	Number of participants with any hearing status (normal and abnormal) were reported.	At 6 Months CA and 5 Years CA
Change From Baseline (6 Months CA) in Child Behavior as Assessed by Vineland Adaptive Behaviour Scales (VABS-II)	The VABS-II was used to measure the personal and social skills of participants serially over time. This test measures the following 4 key domains: Communication, Daily Living Skills, Socialization, and Motor skills. The domains have the same range and directionality as the adaptive behavior composite score (20-160). Higher scores indicate higher level of cognitive ability. A positive change value indicates improvement and negative value indicates worsening in adaptive functioning. The 6 months CA was considered as baseline for this outcome measure. Change from baseline (6 Months CA) in child behavior as assessed by VABS-II adaptive behavior composite was reported here.	Baseline (6 Months CA), 12 Months CA, 24 Months CA, and 5 Year CA
Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories	The CBCL (1 ½ to 5) was parent-reported outcome measure used to assess behavioral, emotional, and social functioning of toddlers and preschool children aged 18-60 months. It is composed of 99 items rated on Likert scale and includes 7 syndrome scales under 2 domains (Internalizing and Externalizing Problems): Internalizing includes syndromes of Emotionally Reactive, Anxious/Depressed, Somatic Complaints, and Withdrawn. Externalizing includes syndromes of Aggressive Behavior and Attention Problems. The 2 domains of all 7 scales were combined to form total score range of 23 to 100. For each question, raw scores of all subscales are converted to standardized T-scores. Higher T score indicates more behavior problems. For Internalization, Externalization and Total Behavior Problem, T-scores of less than 60 are considered non-clinical, 60-63 are borderline, and 64 or more are considered clinical.	At 24 Months CA and 5 Years CA
Child Behavior as Assessed by Attention-Deficit/Hyperactivity Disorder Rating Scale (ADHD-RS) Based on Total Score	The ADHD-RS measures the behaviors of children with Attention-Deficit/Hyperactivity Disorder (ADHD). It consisted of 18 items designed to reflect current symptomatology of ADHD based on DSM-IV criteria. Each item was scored on a 4-point scale ranging from 0 (reflecting no symptoms) to 3 (reflecting severe symptoms) with total scores ranging from 0 to 54. Higher scores represent greater severity of ADHD symptoms.	At 5 Years CA
Child Behavior as Assessed by Social Communication Questionnaire (SCQ)	The SCQ is a brief instrument evaluate communication skills and social functioning in children used for screening for autism or autism spectrum disorders (ASD). The SCQ has 40 dichotomous (yes, no) items, and each item scored 1 point for "abnormal behavior" and 0 point for "absence of abnormal behavior/normal behavior." It yields total score ranging from 0 to 39, higher scores represent more social communication impairment. The first item, "Is she/he now able to talk using short phrases or sentences?", is not scored, but determines if six items relating to abnormal language are assigned. Only "verbal" children (children with "yes" response to first question) are assigned the six items relating to abnormal language and can score a total of 0 to 39 points; "non-verbal" children (children with "no" response to first question) are not assigned six items in relation to abnormal language and score a total of 0 to 33 points where higher scores represent more social communication impairment.	At 5 Years CA
Number of Participants With Pulmonary Morbidity	Pulmonary morbidity was assessed with questions related to family history and smoking status as well as diagnosis of select pulmonary symptoms, conditions and related hospitalizations. Anyone living in the same home with participant smoke, Participants had asthma, wheezing, bronchopulmonary dysplasia (BPD) exacerbation or flare-up, Participants had bronchiolitis, bronchitis, or pneumonia diagnosed, Participants had to use oxygen at home, Participants had to visit emergency room or urgent care for respiratory problem, and Participants had to stay in a hospital overnight for respiratory problem were reported.	At 6 Months CA, 12 Months CA and 24 Months CA
Number of Participants With Survival Status	Survival status was assessed by number participants who died and were censored during the study.	From start of study drug administration up to end of study (up to 6.5 years)
Change From Baseline (3 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Infant Scale	The PedsQL was a generic health related quality of life instrument designed specifically for a pediatric population. PedsQL infant scale encompasses 5 dimensions of functioning (Physical Functioning, Physical Symptoms, Emotional Functioning, Social Functioning, and Cognitive Functioning). The infant for ages 1-12 months (36 Items); for ages 13-24 months (45 Items) rated on a 5-point Likert scale (0 to 4). Overall total scores were calculated as average of all dimensional sub-score items of Infant scale, ranging from 0 to 100 where 0=100 (Never), 1=75 (almost never), 2=50 (sometimes), 3=25 (often), and 4=0 (almost always). Higher scores indicate improved quality of life. A negative value indicates decreased quality of life. The 3 months CA was considered as baseline for this outcome measure.	Baseline (3 Months CA) up to 6 Months CA, 12 Months CA and 24 Months CA
Change From Baseline (24 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Generic Core Scale (GCS) Total Score	The PedsQL was a generic health related quality of life instrument designed specifically for a pediatric population. The GCS encompasses 4 dimensions of functioning (physical, emotional, social, school). The GCS that apply to toddler for 2-4 years of age (21 Items) rated on a 5-point Likert scale (0 to 4); and Young Child for 5-7 years of age (23 Items) rated on a 3-point Likert scale (0, 2, 4). Overall total scores were calculated as average of all dimensional sub-score items of GCS (Toddler and young children's) ranging from 0 to 100 where 0=100 (Never), 1=75 (almost never), 2=50 (sometimes), 3=25 (often), and 4=0 (almost always). Higher scores indicate improved quality of life. A negative value indicates decreased quality of life. The 24 month CA was considered as baseline for this outcome measure.	Baseline (24 Months CA), up to 3 Years CA, 4 Years CA and 5 Years CA
Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS)	HSCS-PS was a validated instrument used via parent proxy within age 2.5 - 5 years. The instrument is composed of 12 domains (Vision, Hearing, Speech, Mobility, Dexterity, Self-care, Emotion, Learn/Remember, Think/Problem Solve, Pain, General Health, Behavior) with up to 6 levels (0-5). The 12 dimensions grouped into 4 categories: neurosensory (vision and hearing), motor (mobility, dexterity, and self-care), learning/remembering (speech, learn/remember, think/problem solve), and quality of life (emotion, pain, general health, behavior). For each category, data were recorded into following levels: no problem (scoring 0 on any attribute); a mild problem (scoring 1 on a scale of 0 to 3, or 1 to 2 on a scale of 0 to 5 for any attribute);moderate/severe problem (scoring > 1 on a scale of 0 to 3, or >2 on a scale of 0 to 5 for any attribute). Higher numbers indicating better child's health status.	At 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
Health Status Measured by the Health Utilities Index (HUI) Mark 2 and 3	The HUI is a family of generic health profiles and preference-based systems used for measuring health status, reporting HRQoL, and producing utility scores. The HUI2 score includes 6 attributes: Sensation, Mobility, Cognition, Self-Care, Emotion, and Pain with up to 5 levels of severity (higher numbers indicate worse level of severity). The HUI3 score includes 8 attributes: Vision, Hearing, Speech, Cognition, Ambulation, Dexterity, Emotion, and Pain with up to 6 levels to indicate the severity (with higher numbers indicating worse levels). HUI2/3 single-attribute scores of morbidities are defined on a scale such the worst level has a score of 0.00 and the best level has a score of 1.00. HUI mark 2 and 3 Overall utility score was reported.	At 5 Years CA
Health Care Resource Use (HCRU)	The total number of private office/hospital outpatient visits of health care resource at 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA were reported.	At 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department	Number of visits to emergency department at 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA were reported.	At 3 Months CA, 6 Months CA, 12 Months CA , 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
Health Care Resource Utilization (HCRU): Duration of Hospitalization	The participant's duration of hospitalizations at 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA were reported.	At 3 Months CA, 6 Months CA, 12 Months CA , 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support	Number of participants who required prescription eyeglasses and educational support at 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA were reported.	At 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA

Collaborators and Investigators

• Sponsor: Shire

Study record dates

Study Major Dates

• Study Start (Actual): March 26, 2015
• Primary Completion (Actual): September 28, 2021
• Study Completion (Actual): September 28, 2021

Study Registration Dates

• First Submitted: March 4, 2015
• First Submitted That Met QC Criteria: March 6, 2015
• First Posted (Estimate): March 12, 2015

Study Record Updates

• Last Update Posted (Actual): May 18, 2022
• Last Update Submitted That Met QC Criteria: April 26, 2022
• Last Verified: April 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Infant, Newborn, Diseases; Pregnancy Complications; Obstetric Labor Complications; Obstetric Labor, Premature; Infant, Premature, Diseases; Retinal Diseases; Premature Birth; Retinopathy of Prematurity; Physiological Effects of Drugs; Growth Substances; Mecasermin
• Other Study ID Numbers: SHP607-201; 2014-003556-31 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No
• IPD Plan Description: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No