E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10066204 |
E.1.2 | Term | Chronic lung disease of prematurity |
E.1.2 | System Organ Class | 100000004855 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
• To evaluate long-term efficacy outcomes following previously administered short-term exposure to SHP607, as compared to a standard neonatal care group, as assessed by chronic respiratory morbidity (CRM) outcomes.
• To evaluate the long-term safety outcomes following previously administered short-term exposure to SHP607, as compared to a standard neonatal care group.
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E.2.2 | Secondary objectives of the trial |
To evaluate long-term effects following previously administered short term exposure to SHP607 on growth, cognitive and motor development, behavior, and resource utilization, as compared to a standard neonatal care group, by assessing:
•Growth parameters
•Physical development
•Cognitive development
•Gross motor function
•Child behavior
•Health related quality of life (HRQoL)
•Health utility
•Health care resource use |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Subject was randomized into Study SHP607-202. Subjects who were randomized, but did not complete Study SHP607-202 must be at least 12 months CA.
2.Written informed consents (and assents, if applicable) must be signed and dated by the subject'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).
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E.4 | Principal exclusion criteria |
Subjects are excluded from the study if the subject or subject’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. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary Efficacy Endpoints: Incidence of the following indicators of chronic respiratory morbidity through 5 years CA:
1)Emergency room visit or hospitalization associated with a respiratory diagnosis.
2)Presence of coughing/wheezing.
3)Use of respiratory medications (eg, bronchodilators, steroids, leukotriene inhibitors, diuretics).
4)Home respiratory technology use (eg, home oxygen, continuous positive airway pressure [CPAP], tracheostomy).
Primary Safety Endpoint:
•AEs reported during the study period of SHP607-203
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
•Growth parameters including body weight, body length (or height), and head circumference.
•Physical development as assessed by standardized, age appropriate tools including physical exam, neurological examination for assessment of cerebral palsy, and vision assessment. Reports of past vision and hearing assessments will also be collected
•Cognitive development as assessed by the following standardized, age appropriate tools:
-Bayley Scales of Infant and Toddler Development (BSID) (or Kyoto Scale of Psychological Development [KSPD])
-Wechsler Preschool and Primary Scale of Intelligence (WPPSI)
•Gross motor function as assessed by the Gross Motor Function Measure-88 (GMFM-88)
•Child behavior as assessed by the following:
- Vineland Adaptive Behavior Scales (VABS)
- Attention-Deficit/Hyperactivity Disorder Rating Scale fifth edition (ADHD RS-V) for the assessment of symptoms of attention-deficit/hyperactivity disorder (ADHD)
- Social Communication Questionnaire (SCQ) for screening of Autism Spectrum Disorder (ASD)
• Health related quality of life 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).
• Health status (eg, health utility) will be measured by the Health Utilities Index Mark 2 (HUI2) and Mark 3 (HUI3).
• Health care resource use associated with inpatient visits, outpatient visits, emergency room visits, and visits to specialists will be assessed.
Secondary Safety Endpoints:
• Targeted medical events occurring during the period of Study SHP607-203.
• Fatal SAEs.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
throughout the study at the following time points in CA:
18 months (±6 weeks), 24 months (±6 weeks), 3 years (±6 weeks), 3.5 years (±6 weeks), 4y (±6 weeks), 4.5 years (±6 weeks) and 5 years (±6 weeks) . |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 34 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Canada |
Finland |
Germany |
Israel |
Italy |
Japan |
Portugal |
Spain |
Sweden |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 8 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 8 |
E.8.9.2 | In all countries concerned by the trial months | 8 |
E.8.9.2 | In all countries concerned by the trial days | 0 |