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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 ROP-2008-01 for the Prevention of Retinopathy of Prematurity (ROP) (PEDAL)

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ClinicalTrials.gov Identifier: NCT02386839
Recruitment Status : Enrolling by invitation
First Posted : March 12, 2015
Last Update Posted : July 6, 2018
Sponsor:
Information provided by (Responsible Party):
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 (Section D) (NCT01096784).

Condition or disease
Retinopathy of Prematurity (ROP)

Study Type : Observational
Estimated Enrollment : 121 participants
Observational Model: Cohort
Time Perspective: Prospective
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 1, 2015
Estimated Primary Completion Date : August 1, 2021
Estimated Study Completion Date : September 1, 2021

Resource links provided by the National Library of Medicine


Group/Cohort
Group 1
Participants who had short-term exposure to rhIGF-I/rhIGFBP-3 in previous study ROPP-2008-01 (NCT01096784)
Group 2
Participants who received standard neonatal care in previous study ROPP-2008-01(NCT01096784)



Primary Outcome Measures :
  1. Number of Participants With Visual Acuity as Assessed by an age-Appropriate Method [ Time Frame: 6 months to 5.5 years corrected age (CA) ]
    Visual acuity is a measure of how well a participant sees at different distances. It will be categorized as the following: normal (measurable acuity greater than or equal to [>=] 20/40); below normal (20/200 less than or equal to [<=] measurable acuity <20/40); poor (measurable acuity <=20/200); blind/low vision (only the ability to detect the 2.2 centimetre [cm] wide stripes on the low-vision Teller acuity card and at any location in the visual field).

  2. Ocular Alignment Assessed by Corneal Light Reflex and by the Cover Test [ Time Frame: 12 months to 5.5 years (CA) ]
    Ocular alignment will be assessed in primary gaze by comparing the position of the corneal light reflection in left eye (OS) and right eye (OD) (corneal light reflection assessment).

  3. Oculomotor Examination (Motility) Assessed by Corneal Light Reflex and by the Cover Test [ Time Frame: 12 months to 5.5 years (CA) ]
    Ocular motility refers to eye movements, which are governed by the 6 extraocular muscles in each eye. It will be assessed by examiner observation of the participant's ability to abduct, adduct, supra, and inferoduct each eye (to assess for strabismus).

  4. Number of Participants With Nystagmus [ Time Frame: 12 months to 5.5 years (CA) ]
    Nystagmus will be observed during the ocular alignment assessments. Presence or absence of nystagmus will be summarized.

  5. Refraction as Assessed by Retinoscopy With Cycloplegia [ Time Frame: 6 months (CA) ]
    Refraction is a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia will be measured and recorded in diopters for each eye individually (OS and OD).

  6. Number of Participants With Stereoacuity [ Time Frame: 5.5 years (CA) ]
    Stereoacuity is a measure of depth perception and will be assessed using the Lang Stereotest. The presence or absence of stereopsis will be recorded.

  7. Number of Participants With Adverse Events (AEs) [ Time Frame: Baseline to 5.5 years (CA) ]
    An adverse event (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: 6 months to 5.5 years (CA) ]
    Body weight will be assessed to the nearest 0.1 kilogram (kg).

  2. Change From Baseline in Body Length Z-score [ Time Frame: 6 to 24 months (CA) ]
    Body length (supine measurement) will be collected when participants are 24-months CA or younger. Body length will be measured by placing the participant in his or her back lying straight and the shoulders and buttocks are flat against the measuring surface.

  3. Change From Baseline in Height Z-score [ Time Frame: 5.5 years ]
    Height will be collected when participants are older than 24-months CA using a stadiometer.

  4. Change From Baseline in Head Circumference Z-score [ Time Frame: 6 to 24 months (CA) ]
    Head circumference or occipital frontal circumference will be measured over the occiput and just above the supraorbital ridge, which is the largest circumference of the head using a "lasso"-type, non-stretchable measuring tape such as the Lasso-o tape.

  5. Cognitive Development as Assessed by Bayley Scales of Infant and Toddler Development (BSID-III) [ Time Frame: 12 to 24 months (CA) ]
    The BSID-III will be used to assess cognitive, motor, and language skills, and is applicable to children aged 1-42 months. The BSID-III is an assessment tool designed to measure a young child's skills in the 3 core areas of development: cognitive, language, and motor. There are 5 subscales, the cognitive subscale (Score range: 55-145) stands alone while the 2 language subscales (expressive and receptive) combine to make a total language score (Range: 47-153) and the 2 motor subtests (fine and gross motor) form a combined motor scale (Range: 46-154). A positive value indicates improvement.

  6. Cognitive Development as Assessed by Wechsler Preschool and Primary Scale of Intelligence (WPPSI) [ Time Frame: 5.5 years (CA) ]
    The WPPSI is a measure of general cognitive development in children that has components of both verbal and nonverbal 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 standard score For the WPPSI ranges from Below 70 (extremely low) to above 130 (very superior).

  7. Physical Development as Assessed by Physical Examination [ Time Frame: 6 months to 5.5 years (CA) ]
    Physical examinations will include a review of the participant's general appearance.

  8. Physical Development as Assessed by Neurological Examination for Assessment of Cerebral Palsy [ Time Frame: 24 month (CA) ]
    Comprehensive neurological examination for the diagnosis of cerebral palsy (CP) will be conducted. The Amiel-Tison neurological examination framework will be utilized for this assessment and will be conducted by trained medical professionals.

  9. Physical Development as Assessed by Hearing Assessment [ Time Frame: 6 months to 5.5 years (CA) ]
    Results of previously completed hearing assessments will be recorded at the 6-month CA and 5-year CA visits.

  10. Child Behaviour as Assessed by Vineland Adaptive Behaviour Scales (VABS-II) [ Time Frame: 6 months to 5.5 years (CA) ]
    The VABS-II Expanded Interview Form will be used to measure the personal and social skills of participants serially over time; these scales are organized within a 3-domain structure: Communication, Daily Living, and Socialization. In addition, the VABS-II offers a Motor Skills Domain and an optional Maladaptive Behavior Index. The VABS-II Expanded Interview Form assesses what a participant actually does, rather than what he or she is able to do. The domain score ranges from 20 (low) to 140 (high). The mean of all 5 domains is 100 and standard deviation 15.

  11. Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) [ Time Frame: 24 months to 5.5 years (CA) ]
    The CBCL (1 ½ to 5) is a 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 that are rated on a Likert scale and includes the following 7 syndrome scales arranged under 2 domains (that is, Internalizing and Externalizing Problems): Internalizing Problems; Emotionally Reactive; Anxious/Depressed; Somatic Complaints; Withdrawn; Sleep Problems; Attention Problems; Aggressive Behavior. The scores range from 23 to 100. For the Total Problem, Internalization, and Externalization Scales, scores of less than 60 are considered non-clinical, 60-63 are borderline, and 64 or more are considered clinical. For all syndrome scales, scores of 65 or less are considered non-clinical, 66 through 70 are considered borderline, and 71 or greater are considered clinical.

  12. Child Behaviour as Assessed by Attention-Deficit/Hyperactivity Disorder Rating Scale (ADHD-RS) [ Time Frame: 5.5 years (CA) ]
    The ADHD-RS measures the behaviors of children with Attention-Deficit/Hyperactivity Disorder (ADHD). It consists of 18 items designed to reflect current symptomatology of ADHD based on DSM-IV criteria. Each item is scored from a range of 0 (reflecting no symptoms) to 3 (reflecting severe symptoms) with total scores ranging from 0-54. The 18 items are grouped into 2 subscales: hyperactivity-impulsivity (even numbered items 2-18) and inattention ("inattentiveness") (odd numbered items 1-17).

  13. Child Behaviour as Assessed by Social Communication Questionnaire (SCQ) [ Time Frame: 5.5 years (CA) ]
    The SCQ is a brief instrument that helps evaluate communication skills and social functioning in children that can be used for screening for autism or autism spectrum disorders (ASD) in the general population. The score ranges from 0 to 39, with a cut off of greater than (>) 15 indicating ASD.

  14. Number of Participants with Pulmonary Morbidity [ Time Frame: 6 months to 5 years (CA) ]
    Pulmonary morbidity will be assessed with questions related to family history and smoking status as well as diagnosis of select pulmonary symptoms, conditions and related hospitalizations.

  15. Survival Assessment [ Time Frame: 3 weeks to 5.5 years (CA) ]
    Survival status will be assessed as the length of time from the participant's date of birth to death during the study due to any cause.

  16. Health Related Quality of Life (HRQoL) Assessed by the Pediatric Quality of Life Inventory (PedsQL™) Scales [ Time Frame: 3 weeks to 5.5 years (CA) ]
    HRQoL will be assessed via the validated Pediatric Quality of Life Inventory (PedsQL™) Scales appropriate for the child's age of development. The following scales will be used in this study: Infant Scale for ages 1-12 months (36 Items); Infant Scale for ages 13-24 months (45 Items); Toddler Scale for 2-4 years of age (21 Items); Young Child Scale for 5-7 years of age (23 Items). Scores are transformed to a 0 to 100 scale. Higher scores indicate better quality of life.

  17. Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) [ Time Frame: 24 months to 5.5 years (CA) ]
    HSCS-PS is a validated instrument adapted for use via parent proxy within the pediatric population age 2.5 - 5. The instrument is composed of 12 dimensions (Vision, Hearing, Speech, Mobility, Dexterity, Self-care, Emotion, Learn/remember, Think/problem solve, Pain, General Health, and Behavior) intended to provide a comprehensive assessment of a child's health status. The responses will be descriptively summarized as proportions.

  18. Health Status Measured by the Health Utilities Index 2/3 [ Time Frame: 5 years (CA) ]
    The Health Utilities Index (HUI) is a family of generic health profiles and preference-based systems used for measuring health status, reporting HRQoL, and producing utility scores. The HUI 2/3 system contains a number of attributes/domains to classify the level of health status. Each attribute or domain (eg, mobility, cognition, emotion or pain) is rated on a 5-point ordinal scale to indicate the severity level, ranging from 1-5 (higher numbers indicating a more severe level).



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Ages Eligible for Study:   40 Weeks to 108 Weeks   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants randomized in Study ROPP-2008-01, Section D are eligible to enroll in this study; completion of Study ROPP-2008-01 Section D is not required. Participants in Study ROPP-2008-01 are premature infants (gestational age of 23 weeks + 0 days to 27 weeks + 6 days) who are randomized to receive either treatment with rhIGF-1/rhIGFBP-3 or standard neonatal care. Up to 121 participants were planned to be randomized in Study ROPP-2008-01 Section D.
Criteria

Inclusion Criteria:

  1. Participant was randomized in Study ROPP-2008-01, Section D
  2. 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:

  1. 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
  2. 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
United States, Alabama
University of South Alabama Children's and Women's Hospital
Mobile, Alabama, United States, 36604
United States, Missouri
St. John's Mercy Medical Center
Saint Louis, Missouri, United States, 63141
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
Study Director: Shire Study Physician Shire

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02386839     History of Changes
Other Study ID Numbers: SHP607-201
2014-003556-31 ( EudraCT Number )
First Posted: March 12, 2015    Key Record Dates
Last Update Posted: July 6, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Retinal Diseases
Premature Birth
Retinopathy of Prematurity
Eye Diseases
Obstetric Labor, Premature
Obstetric Labor Complications
Pregnancy Complications
Infant, Premature, Diseases
Infant, Newborn, Diseases