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Trial record 1 of 1 for:    NCT02386839
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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)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02386839
Recruitment Status : Completed
First Posted : March 12, 2015
Results First Posted : April 25, 2022
Last Update Posted : May 18, 2022
Sponsor:
Information provided by (Responsible Party):
Takeda ( Shire )

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).

Condition or disease Intervention/treatment Phase
Retinopathy of Prematurity (ROP) Drug: rhIGF-1/rhIGFBP-3 Phase 2

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)

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 76 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : March 26, 2015
Actual Primary Completion Date : September 28, 2021
Actual Study Completion Date : September 28, 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Mecasermin

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 Name: 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 Name: Mecasermin rinfabate




Primary Outcome Measures :
  1. Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) [ Time Frame: At 6 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.

  2. Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA [ Time Frame: 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.

  3. Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA [ Time Frame: 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.

  4. Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA [ Time Frame: 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.

  5. Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA [ Time Frame: 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.

  6. Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA [ Time Frame: 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.

  7. Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA [ Time Frame: 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.

  8. Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA [ Time Frame: 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.

  9. Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA [ Time Frame: 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.

  10. Number of Participants With Nystagmus at 12 Months CA [ Time Frame: At 12 Months CA ]
    Nystagmus was observed during the ocular alignment assessments. Presence and absence of nystagmus was reported at 12 Months CA.

  11. Number of Participants With Nystagmus at 24 Months CA [ Time Frame: At 24 Months CA ]
    Nystagmus was observed during the ocular alignment assessments. Presence and absence of nystagmus was reported at 24 Months CA.

  12. Number of Participants With Nystagmus at 5 Years CA [ Time Frame: At 5 Years CA ]
    Nystagmus was observed during the ocular alignment assessments. Presence and absence of nystagmus was reported at 5 Years CA.

  13. Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA [ Time Frame: 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'.

  14. Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA [ Time Frame: At 12 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'.

  15. Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA [ Time Frame: At 20 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'.

  16. Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA [ Time Frame: 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'.

  17. Number of Participants With Stereoacuity as Assessed With the Lang Stereotest At 5 Years CA [ Time Frame: 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.

  18. Number of Participants With Adverse Events (AEs) [ Time Frame: From start of study up to end of study (up to 6.5 years) ]
    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.


Secondary Outcome Measures :
  1. Change From Baseline in Body Weight Z-score [ Time Frame: Baseline, 6 Months CA, 12 Months CA, 24 Months CA and 5 Years CA ]
    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.

  2. Change From Baseline in Height Z-score [ Time Frame: Baseline, 6 Months CA, 12 Month CA, 24 Months CA and 5 Years CA ]
    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.

  3. Change From Baseline in Head Circumference Z-score [ Time Frame: Baseline, 6 Months CA, 12 Months CA and 24 Months CA ]
    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.

  4. 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 [ Time Frame: Baseline (12 Months CA), 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.

  5. Cognitive Development as Assessed by Wechsler Preschool and Primary Scale of Intelligence (WPPSI-IV) Full Scale at 5 Years CA [ Time Frame: 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.

  6. Number of Participants With Abnormal Physical Examination [ Time Frame: From start of study drug administration up to end of study (up to 6.5 years) ]
    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.

  7. Number of Participants Diagnosed With Neurological Examination for Cerebral Palsy (CP) by Neurological Examination at 24 Months CA [ Time Frame: 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.

  8. Number of Participants With Normal and Abnormal Hearing Screening Status [ Time Frame: At 6 Months CA and 5 Years CA ]
    Number of participants with any hearing status (normal and abnormal) were reported.

  9. Change From Baseline (6 Months CA) in Child Behavior as Assessed by Vineland Adaptive Behaviour Scales (VABS-II) [ Time Frame: Baseline (6 Months CA), 12 Months CA, 24 Months CA, and 5 Year CA ]
    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.

  10. Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories [ Time Frame: At 24 Months CA and 5 Years CA ]
    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.

  11. Child Behavior as Assessed by Attention-Deficit/Hyperactivity Disorder Rating Scale (ADHD-RS) Based on Total Score [ Time Frame: At 5 Years CA ]
    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.

  12. Child Behavior as Assessed by Social Communication Questionnaire (SCQ) [ Time Frame: At 5 Years CA ]
    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.

  13. Number of Participants With Pulmonary Morbidity [ Time Frame: At 6 Months CA, 12 Months CA and 24 Months CA ]
    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.

  14. Number of Participants With Survival Status [ Time Frame: From start of study drug administration up to end of study (up to 6.5 years) ]
    Survival status was assessed by number participants who died and were censored during the study.

  15. Change From Baseline (3 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Infant Scale [ Time Frame: Baseline (3 Months CA) up to 6 Months CA, 12 Months CA and 24 Months CA ]
    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.

  16. Change From Baseline (24 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Generic Core Scale (GCS) Total Score [ Time Frame: Baseline (24 Months CA), up to 3 Years CA, 4 Years CA and 5 Years CA ]
    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.

  17. Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) [ Time Frame: At 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA ]
    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.

  18. Health Status Measured by the Health Utilities Index (HUI) Mark 2 and 3 [ Time Frame: At 5 Years CA ]
    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.

  19. Health Care Resource Use (HCRU) [ Time Frame: At 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA ]
    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.

  20. Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department [ Time Frame: At 3 Months CA, 6 Months CA, 12 Months CA , 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA ]
    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.

  21. Health Care Resource Utilization (HCRU): Duration of Hospitalization [ Time Frame: At 3 Months CA, 6 Months CA, 12 Months CA , 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA ]
    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.

  22. Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support [ Time Frame: At 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA ]
    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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   40 Weeks to 108 Weeks   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02386839


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

Documents provided by Takeda ( Shire ):
Study Protocol  [PDF] April 9, 2018
Statistical Analysis Plan  [PDF] September 1, 2020

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02386839    
Other Study ID Numbers: SHP607-201
2014-003556-31 ( EudraCT Number )
First Posted: March 12, 2015    Key Record Dates
Results First Posted: April 25, 2022
Last Update Posted: May 18, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
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)

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Retinal Diseases
Retinopathy of Prematurity
Premature Birth
Eye Diseases
Obstetric Labor, Premature
Obstetric Labor Complications
Pregnancy Complications
Infant, Premature, Diseases
Infant, Newborn, Diseases
Mecasermin
Growth Substances
Physiological Effects of Drugs