Primary ObjectivesThe primary objectives of this study are:* To evaluate the long-term efficacy outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (Section D) as assessed by ROP associated…
ID
Source
Brief title
Condition
- Retina, choroid and vitreous haemorrhages and vascular disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary efficacy endpoints of this study are:
* Visual acuity as assessed by an age appropriate method
* Ocular alignment and ocular motor examination in primary gaze and in as many
of 9 positions of gaze as possible as assessed by corneal light reflex and by
the cover test
* Assessment of nystagmus by observation
* Refraction as assessed by retinoscopy with cycloplegia
* Stereoacuity as assessed with the Lang Stereotest
Secondary outcome
The secondary efficacy endpoints of this study are:
* Growth parameters including body weight, body length (or height), and head
circumference
* Cognitive development as assessed by the following standardized,
age-appropriate tools:
o Bayley Scales of Infant and Toddler Development, Third Edition
(BSID-III)
o Wechsler Preschool and Primary Scale of Intelligence, Fourth Edition
(WPPSI-IV)
* Physical development as assessed by standardized, age appropriate tools
including physical exam, neurological examination for assessment of cerebral
palsy, and hearing assessment
* Child behavior as assessed by the following:
* Vineland Adaptive Behavior Scales, Second Edition (VABS-II)
* Child Behavior Checklist (CBCL; 1 * to 5)
* Attention Deficit/Hyperactivity Disorder Rating Scale-fourth edition (ADHD
RS-IV) for the assessment of symptoms of attention deficit/hyperactivity
disorder (ADHD)
* Social Communication Questionnaire (SCQ) for screening of Autism Spectrum
Disorder (ASD)
* Pulmonary morbidity data (eg, hospitalizations, emergency room [ER] visits,
pulmonary medications)
* Survival as assessed by death during the study due to any cause
The health economic outcome research endpoints of this study are:
* Health-related quality of life (HRQoL) will be assessed by the Pediatric
Quality of Life Inventory (PedsQL*) Scales appropriate for the child's age of
development with the Total Scale Score and 5 domains within Physical Health
(Physical Functioning and Physical Symptoms) and Psychosocial Health Scores
(Emotional, Social, and Cognitive Functioning, respectively)
* Health status (eg, health utility) will be measured by the Health Status
Classification System-Preschool (HSCS-PS)
* Resource use associated with inpatient visits, outpatient visits, medical
utilization and pharmacy utilization will be assessed
The safety endpoints of this study are:
* Physical examination including tonsil examination
* Adverse events (AEs), as follows:
o those considered related to rhIGF-1/rhIGFBP-3 (as administered in
Study ROPP-2008-01, Section D)
o those considered related to procedures performed in this study (Study
SHP-607-201)
o specified targeted medical events regardless of causality
o fatal SAEs regardless of causality
* Cardiac size as assessed by echocardiogram
* Kidney and spleen size and any other gross abnormalities as assessed by
abdominal ultrasound
Exploratory Endpoints:
The exploratory endpoints of this study are related to the topography of the
retinal layers and optic nerve as assessed by optical coherence tomography
(OCT).
Background summary
The finding in both a mouse model of ROP and preterm infants that development
of ROP is associated with low levels of IGF-1 after premature birth, indicates
a possible role for replacement of IGF-1 to levels found in utero as a strategy
to potentially decrease abnormal retinal vascularization and abnormal retinal
neural development, and ultimately, ROP. Mecasermin rinfabate
(rhIGF-1/rhIGFBP-3) is the human recombinant form of the naturally occurring
protein complex of IGF-1 and insulin-like growth factor binding protein-3
(IGFBP-3). rhIGF-1/rhIGFBP-3 was developed to enhance the systemic exposure of
administered rhIGF-1 and to improve the safety profile of rhIGF-1 therapy.
rhIGF-1/rhIGFBP-3 was approved by the
Food and Drug Administration (FDA) in 2005 for the treatment of growth failure
in children with severe primary IGF-1 deficiency or with growth hormone (GH)
gene deletion who have developed neutralizing antibodies to GH.
Although the rhIGF-1/rhIGFBP-3 therapy in Section D of the Phase II study (Study
ROPP-2008-01) represents a short-term exposure (< 2 months for each subject),
rhIGF-1/rhIGFBP-3 may have long-lasting effects on visual outcomes as well as
other potential outcomes related to complications of prematurity such as
neurodevelopment, pulmonary function, and growth. In addition, it is critical
to understand any long term safety effects from short term exposure to
rhIGF-1/rhIGFBP-3.
The long-term outcomes assessed in this study will require utilization of
different assessment tools than are utilized in the Phase II study,
ROPP-2008-01 Section D, given the changes in physical and cognitive development
that will occur in the subjects as they age during their participation in this
study.
Study objective
Primary Objectives
The primary objectives of this study are:
* To evaluate the long-term efficacy outcomes following short-term exposure to
rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (Section
D) as assessed by ROP associated visual outcomes
* To evaluate the long-term safety outcomes following short-term exposure to
rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (Section
D)
Secondary Objectives
The secondary objectives of this study are to evaluate the effect following
short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study
ROPP-2008-01 (Section D) on:
* Growth parameters
* Cognitive development
* Physical development
* Child behavior
* Pulmonary morbidity
* Survival
* Health-related quality of life (HRQoL)
* Health utility
* Health care resource use (HCRU)
Exploratory Objective
The exploratory objective of this study is to evaluate the effect following
short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study
ROPP-2008-01 (Section D) on the architecture of the retina and optic nerve.
Study design
This is a Phase II, multicenter, long-term developmental outcome study
enrolling subjects who were randomized in Section D of Study ROPP-2008-01.
Enrolled subjects in this study will be followed through age 5 years corrected
age (CA). This study will enroll both subjects who were in the treated
(received rhIGF-1/rhIGFBP-3) and control (received standard neonatal care)
groups of Study ROPP-2008-01 (Section D) to enable assessment of
rhIGF-1/rhIGFBP-3 long-term efficacy and safety outcomes versus standard
neonatal care. No investigational product will be administered in this study.
Study burden and risks
There are minimal risks associated with thus study, as there are no invasive
procedures and no investigational product is administered. The subjects may
experience discomfort or inconvenience from the study procedures. With 6 clinic
visits and 3 phone visits in 5 years, the burden for the subjects is minimal.
Svärdvägen 11D
Danderyd SE-182 33
SE
Svärdvägen 11D
Danderyd SE-182 33
SE
Listed location countries
Age
Inclusion criteria
1. Subject was randomized in Study ROPP-2008-01, Section D
2. Subject*s 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 subject.
Exclusion criteria
1. Any other condition or therapy that, in the Investigator*s opinion, may pose a risk to the subject or interfere with the subject*s ability to be compliant with this protocol or interfere with the interpretation of results
2. The subject or subject*s parent or legally authorized representative(s) is unable to comply with the protocol as determined by the Investigator
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2014-003556-31-NL |
| CCMO | NL52409.029.15 |