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A Clinical Efficacy and Safety Study of SHP607 in Preventing Chronic Lung Disease in Extremely Premature Infants

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ClinicalTrials.gov Identifier: NCT03253263
Recruitment Status : Recruiting
First Posted : August 17, 2017
Last Update Posted : October 4, 2019
Sponsor:
Information provided by (Responsible Party):
Shire

Brief Summary:
The purpose of this study is to determine if an investigational drug can reduce the burden of chronic lung disease in extremely premature babies through 12 months corrected age (CA), as compared to extremely premature babies receiving standard neonatal care alone.

Condition or disease Intervention/treatment Phase
Retinopathy of Prematurity (ROP) Intraventricular Hemorrhage Bronchopulmonary Dysplasia Chronic Lung Disease of Prematurity Drug: SHP607 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 600 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: A Phase 2b, Multicenter, Randomized, Open-label, Controlled, 3-Arm Study to Evaluate the Clinical Efficacy and Safety of SHP607 in Preventing Chronic Lung Disease Through 12 Months Corrected Age Compared to Standard Neonatal Care in Extremely Premature Infants
Actual Study Start Date : May 9, 2019
Estimated Primary Completion Date : August 31, 2022
Estimated Study Completion Date : February 22, 2023


Arm Intervention/treatment
Experimental: SHP607 250 mcg/kg/24 hours
Participants will receive continuous intravenous (IV) infusion of SHP607 250 micrograms per kilogram per 24 hours (mcg/kg/24 hours) from birth up to postmenstrual age (PMA) 29 weeks +6 days.
Drug: SHP607
Participants will receive intravenous infusion of SHP607 at doses of 250 mcg/kg/24 hours and 400 mcg/kg/24 hours from birth up to PMA 29 weeks + 6 days.
Other Name: Mecasermin Rinfabate

Experimental: SHP607 400 mcg/kg/24 hours
Participants will receive continuous IV infusion of SHP607 400 mcg/kg/24 hours through from birth up to PMA 29 weeks +6 days.
Drug: SHP607
Participants will receive intravenous infusion of SHP607 at doses of 250 mcg/kg/24 hours and 400 mcg/kg/24 hours from birth up to PMA 29 weeks + 6 days.
Other Name: Mecasermin Rinfabate

No Intervention: Standard Neonatal Care
Standard neonatal care alone will be provided.



Primary Outcome Measures :
  1. Time to Final Weaning off Respiratory Technology Support (RTS) From Day 1 Through 12 Months Corrected age (CA) [ Time Frame: Baseline through 12 months Corrected Age (CA) ]
    RTS is defined as any one of the following: (1) any fraction of inspired oxygen (FiO2) greater than (>) 21 percent (%), (2) noninvasive respiratory support delivered via a nasal interface (example [e.g.], continuous positive airway pressure [CPAP], bilevel positive airway pressure [BiPAP], high flow therapy, nasal intermittent positive pressure ventilation [NIPPV], nasal cannula), (3) invasive respiratory support (mechanical ventilation) via an endotracheal tube or tracheostomy. Time to final weaning off RTS as compared to a standard neonatal care group will be reported.


Secondary Outcome Measures :
  1. Incidence of Bronchopulmonary Dysplasia (BPD) or Death Through Postmenstrual age (PMA) 36 Weeks [ Time Frame: Baseline through 36 weeks postmenstrual age (PMA) ]
    BPD is diagnosed by need for oxygen/respiratory support, and confirmed by oxygen challenge test.

  2. Total Number of Days on Respiratory Technology Support (RTS) From Birth Through 12 Months Corrected age (CA) [ Time Frame: Birth through 12 months CA ]
    RTS is defined as any one of the following: (1) any fraction of inspired oxygen (FiO2) greter than (>) 21 percent (%), (2) noninvasive respiratory support delivered via a nasal interface (e.g., continuous positive airway pressure [CPAP], bilevel positive airway pressure [BiPAP], high flow therapy, nasal intermittent positive pressure ventilation [NIPPV], nasal cannula), (3) invasive respiratory support (mechanical ventilation) via an endotracheal tube or tracheostomy. Total number of days on RTS from birth through 12 months CA will be reported.

  3. Duration of Re-hospitalizations [ Time Frame: From neonatal intensive care unit (NICU) discharge through 12 months CA ]
    Duration of re-hospitalizations due to respiratory diagnoses will be reported.

  4. Number of Emergency Room Visits [ Time Frame: From NICU discharge through 12 months CA ]
    Number of emergency room visits associated with a respiratory diagnosis will be reported.

  5. Number of Days of Respiratory Medication use [ Time Frame: From NICU discharge through 12 months CA ]
    Number of days of respiratory medication use (for example, bronchodilators, steroids, leukotriene inhibitors, diuretics) will be reported.

  6. Incidence of Signs and Symptoms of Respiratory Disease as Assessed by a 28-day Caregiver-administered Diary Ending at 12 Months Corrected age (CA) [ Time Frame: 11 months CA through 12 months CA ]
    Incidence of signs and symptoms of respiratory disease (yes/no) at 12 months CA as assessed by a 28-day caregiver-administered diary recording episodes of wheezing, coughing, and respiratory medication will be reported.

  7. Incidence of Chronic Respiratory Morbidity (CRM1) Through 12 Months Corrected age (CA) [ Time Frame: From NICU discharge through 12 months CA ]
    A participant will be defined as having CRM1 if he or she experienced/required at least 1 of the 3 following clinical/treatment events, as reported by parents/caregivers and captured by the pulmonary morbidity assessment on at least two 3-month quarters over a 12-month time period: 1. Emergency room visit or hospitalization associated with a respiratory diagnosis, 2. Home RTS, 3. Daily use of respiratory medications (e.g., bronchodilators, steroids, leukotriene inhibitors, diuretics) as reported by caregivers on the pulmonary morbidity assessment. Incidence of CRM1 through 12 months CA will be reported.

  8. Incidence of Chronic Respiratory Morbidity Including Symptoms of Respiratory Disease (CRM2) Through 12 Months Corrected age (CA) [ Time Frame: From NICU discharge through 12 months CA ]
    A participant will be defined as having CRM2 if he or she experienced/required at least 1 of the 4 following clinical/treatment events, as reported by parents/caregivers and captured by the pulmonary morbidity assessment on at least two 3-month quarters over a 12-month time period: 1. Emergency room visit or hospitalization associated with a respiratory diagnosis, 2. Home RTS, 3. Daily use of respiratory medications (e.g., bronchodilators, steroids, leukotriene inhibitors, diuretics) as reported by caregivers on the pulmonary morbidity assessment, 4. Symptoms of respiratory disease as defined by presence of cough without cold, or wheeze at least once per week. Incidence of CRM2 through 12 months CA will be reported.

  9. Severity of Chronic Respiratory Morbidity (CRM3) Through 12 Months Corrected age (CA), as Determined by the Chronic Lung Disease (CLD) of Infancy Severity Score [ Time Frame: From NICU discharge through 12 months CA ]
    Severity of chronic respiratory morbidity (CRM3) through 12 months CA will be reported, as determined by the CLD of infancy severity score that will include components such as respiratory hospitalizations, RTS use, and use of respiratory medications.

  10. Incidence of Intraventricular Haemorrhage (IVH) Through Postmenstrual age (PMA) 40 weeks, as Assessed by Cranial Ultrasound [ Time Frame: Baseline through 40 Weeks PMA ]
    Incidence of IVH through PMA 40 weeks, as assessed by cranial ultrasound, will be reported.

  11. Motor Function at 12 Months Corrected age (CA), as Measured by Alberta Infant Motor Scales (AIMS) [ Time Frame: At 12 months CA ]
    The AIMS is a measure of early motor maturation used for assessment of infants at risk for motor delay, focusing on attainment of motor milestones and development of postural control. It consists of 58 items, including assessments in 4 postural positions: prone (21 items), supine (9 items), sitting (12 items) and standing (16 items). Each item is scored as 'observed' or 'not observed'. The scorer identifies the least and most mature item observed. Motor function at 12 months CA, as measured by AIMS will be reported.

  12. Functional Status as Assessed by PREMature Infant Index (PREMII) at Postmenstrual Age (PMA) 36 Weeks [ Time Frame: At 36 weeks PMA ]
    PREMII is a Clinician-Reported Outcome (ClinRO) assessment used to capture overall functional maturation of extremely preterm neonates. Functional Status is defined as what the infant can do with respect to 8 key functional areas (feeding, weight gain, thermoregulation, respiratory support, apnea, bradycardia, desaturation events, and oxygen administration), as a reflection of the infant's overall health and development.

  13. Incidence of Retinopathy of Prematurity (ROP) [ Time Frame: 31 weeks through 40 weeks PMA ]
    The ROP examination will consist of a dilated fundus examination. Typically ROP is classified according to the International Classification and is subdivided into Stage 1 (demarcation line), Stage 2 (ridge), Stage 3 (ridge with extraretinal fibrovascular proliferations), Stage 4 (subtotal retinal detachment), and Stage 5 (total retinal detachment). Incidence of ROP will be reported.

  14. Incidence of Mortality [ Time Frame: From birth through 12 months CA ]
    Mortality from birth through 12 months CA will be reported.



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Ages Eligible for Study:   up to 1 Day   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Written informed consents and/or assents must be signed and dated by the participant's parent(s) or legally authorized representative(s), if applicable, 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).
  2. Written informed consents and/or assents must be signed and dated by the participant's birth mother or her legally authorized representative(s), if applicable, prior to providing study-related information related to birth mother medical history, pregnancy and the birth of the participant. The informed consent and any assents for underage birth mothers must be approved by the IRB/IEC.
  3. Initially, participants must be between gestational age (GA) of 26 weeks +0 days and 27 weeks +6 days, inclusive. After approximately 75 participants (approximately 25 participants in each treatment group) have completed the postmenstrual age (PMA) 40 weeks visit, an independent data monitoring committee (DMC) will assess safety data and may authorize enrollment of participants of GA between 23 weeks +0 days and 27 weeks +6 days, inclusive.

Exclusion Criteria:

  1. Detectable major (or severe) congenital malformation identified before randomization.
  2. Known or suspected chromosomal abnormality, genetic disorder, or syndrome, identified before randomization, according to the investigator's opinion.
  3. Hypoglycemia at Baseline (blood glucose less than (<) 45 milligrams per deciliter [mg/dL] or 2.5 milli moles per liter [mmol/L]) which persists in spite of glucose supplementation, to exclude severe congenital abnormalities of glucose metabolism.
  4. Clinically significant neurological disease identified before randomization according to cranial ultrasound (hemorrhages confined to the germinal matrix are allowed) and investigator's opinion.
  5. Any other condition or therapy that, in the investigator's opinion, may pose a risk to the participant or interfere with the participant's potential compliance with this protocol or interfere with interpretation of results.
  6. Current or planned participation in a clinical study of another investigational study drug, device, or procedure (participation in observational studies is permitted on a case-by-case basis).
  7. The participant or participant's parent(s) or legally authorized representative(s) is/are unable to comply with the protocol or is unlikely to be available for long-term follow-up 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): NCT03253263


Contacts
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Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

Locations
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United States, Indiana
Memorial Hospital of South Bend Recruiting
South Bend, Indiana, United States, 46601
Contact: Site Contact    574-647-3048      
Principal Investigator: Basharat Buchh, MD         
United States, Mississippi
University of Mississippi Medical Center Recruiting
Jackson, Mississippi, United States, 39216
Contact: Site Contact    601-815-3070      
Principal Investigator: Abhay Bhatt, MD         
United States, West Virginia
West Virginia University Recruiting
Morgantown, West Virginia, United States, 26506
Contact: Site Contact    304-293-1202    mpolak@hsc.wvu.edu   
Principal Investigator: Mark Polak, MD         
Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Shire

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT03253263     History of Changes
Other Study ID Numbers: SHP607-202
First Posted: August 17, 2017    Key Record Dates
Last Update Posted: October 4, 2019
Last Verified: October 2019
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 in order to minimize the risk that individual patients could be re-identified, given that there are limited numbers of study participants at each study site per year.

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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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Lung Diseases
Bronchopulmonary Dysplasia
Retinopathy of Prematurity
Premature Birth
Hemorrhage
Pathologic Processes
Obstetric Labor, Premature
Obstetric Labor Complications
Pregnancy Complications
Respiratory Tract Diseases
Retinal Diseases
Eye Diseases
Ventilator-Induced Lung Injury
Lung Injury
Infant, Premature, Diseases
Infant, Newborn, Diseases
Mecasermin
Growth Substances
Physiological Effects of Drugs