Clinical trial NCT04506619: Safety and Efficacy Outcomes Following Previously Administered Short-Term Treatment With SHP607 in Extremely Premature Infants
Official title: Long-Term Safety and Efficacy Outcomes Following Previously Administered Short-Term Treatment With SHP607 in Extremely Premature Infants
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Retinopathy of Prematurity (ROP)
Intraventricular Hemorrhage
Bronchopulmonary Dysplasia
Chronic Lung Disease of Prematurity
Lead sponsor: Oak Hill Bio Ltd
Sponsor class: INDUSTRY
First posted: 2020-08-10
Enrollment: 26
Sex: ALL
Minimum age (years): 12
Maximum age (years): 24
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants who previously received SHP607 or standard neonatal care in Study SHP607-202 (NCT03253263) are planned to be enrolled in Study SHP607-203.

Brief summary: The purpose of this study is to evaluate long-term safety and efficacy outcomes following previously administered short-term exposure to SHP607, as compared to a standard neonatal care group.

Detailed description: In this long-term follow-up study, participants who enrolled in SHP607-202 (NCT03253263) will be followed from 12 months corrected age (CA) through 60 months CA. Participants will not receive any investigational product in this study.

Eligibility criteria: Inclusion Criteria:

* Participants who were randomized into Study SHP607-202 (NCT03253263). Participants who were randomized, but did not complete Study SHP607-202 (NCT03253263) must be at least 12 months CA.
* Written informed consents (and assents, if applicable) must be signed and dated by the participant's parent(s)/legally authorized representative(s) prior to any study-related procedures. The informed consent and any assents for underage parents must be approved by the institutional review board (IRB)/independent ethics committee (IEC).

Exclusion Criteria:

- Participants are excluded from the study if the participant or participant's parent(s)/legally authorized representative(s) is/are unable to comply with the protocol or is/are unlikely to be available for long-term follow-up as determined by the investigator.

Primary outcomes:
measure: Number of Emergency Room Visits
timeFrame: 12 months CA through 60 months CA
measure: Number of Hospitalizations
timeFrame: 12 months CA through 60 months CA
measure: Incidence of Signs and Symptoms of Respiratory Disease
timeFrame: 12 months CA through 60 months CA
measure: Number of Days of Respiratory Medication Use
timeFrame: 12 months CA through 60 months CA
measure: Total Number of Days on Home Respiratory Technology use
timeFrame: 12 months CA through 60 months CA
measure: Number of Participants With Adverse Events (AEs)
timeFrame: 12 months CA through 60 months CA
Secondary outcomes:
measure: Growth Parameters: Body Weight
timeFrame: At 12, 24, and 60 months CA
measure: Growth Parameters: Body Length
timeFrame: At 12 and 24 months CA
measure: Growth Parameters: Height
timeFrame: At 60 months CA
measure: Growth parameters: Head circumference
timeFrame: At 12 and 24 months CA
measure: Physical Development as Assessed by Physical Examination
timeFrame: At 12, 24, and 60 months CA
measure: Physical Development as Assessed by Neurological Examination for Assessment of Cerebral Palsy
timeFrame: At 12, 24, and 60 months CA
measure: Physical Development as Assessed by Visual Acuity
timeFrame: At 12, 24, and 60 months CA
measure: Cognitive Development Assessed by Bayley Scales of Infant and Toddler Development (BSID-III)
timeFrame: At 24 months CA
measure: Cognitive Development Quotient Assessed by Kyoto Scale of Psychological Development (KSPD)
timeFrame: At 24 months CA
measure: Cognitive Development Assessed by Wechsler Preschool and Primary Scale of Intelligence (WPPSI)
timeFrame: At 60 months CA
measure: Gross Motor Function Assessed by Gross Motor Function Measure-88 (GMFM-88)
timeFrame: At 24 and 60 months CA
measure: Childhood Behavior Assessed by Vineland Adaptive Behavior Scales (VABS-III)
timeFrame: At 24 and 60 months CA
measure: Childhood Behavior Assessed by Attention-Deficit/Hyperactivity Disorder Rating Scale (ADHD-RS-V)
timeFrame: At 60 months CA
measure: Childhood Behavior Assessed by Social Communication Questionnaire (SCQ)
timeFrame: At 60 months CA
measure: Health Related Quality of Life (HRQoL) Assessed by the Pediatric Quality of Life Inventory (PedsQL) Scales
timeFrame: At 24, 36, 48, and 60 months CA
measure: Health Status Measured by the Health Utilities Index 2/3
timeFrame: At 60 months CA
measure: Healthcare Resource Utilization
timeFrame: 12 months CA through 60 months CA
measure: Number of Participants With Targeted Medical Events and Fatal Serious Adverse Events (SAEs)
timeFrame: 12 months CA through 60 months CA
Other outcomes:
Locations:
city: South Bend
country: United States
facility: Memorial Hospital of South Bend
state: Indiana
city: Jackson
country: United States
facility: University of Mississippi Medical Center
state: Mississippi
city: Jackson
country: United States
facility: University of Mississippi Medical Center
state: Mississippi
city: New Brunswick
country: United States
facility: Robert Wood Johnson Medical School
state: New Jersey
city: Firenze
country: Italy
facility: Azienda Ospedaliera Universitaria Careggi
city: Genova
country: Italy
facility: Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN
city: Treviso
country: Italy
facility: Presidio Ospedaliero Di Treviso Ca' Foncello
city: Chertsey
country: United Kingdom
facility: Ashford and St. Peter's Hospitals NHS Trust - St. Peter*s Hospital
state: Surrey
Study officials:
References: