Stránka klinických studií Nct

Summary
EudraCT Number:2013-005429-21
Sponsor's Protocol Code Number:D20496
Clinical Trial Type:Outside EU/EEA
Date on which this record was first entered in the EudraCT database:2015-09-01
Trial results
A. Protocol Information
A.2EudraCT number2013-005429-21
A.3Full title of the trial
Multicentre randomised controlled trials of minimally-invasive surfactant therapy in preterm infants 25 – 28 weeks gestation on continuous positive airway pressure.
A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
Premature infants between 25 and 28 weeks gestation may develop lung disease and require respiratory support of continuous positive airway pressure (CPAP). The trial is looking at if there is a benefit from giving a medication called surfactant at an early stage, delivered via a narrow tube briefly placed into the windpipe.
A.3.2Name or abbreviated title of the trial where available
OPTIMIST-A
A.4.1Sponsor's protocol code numberD20496
A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02140580
A.5.4Other Identifiers
Name:Australia New Zealand Clinical Trial RegistryNumber:12611000916943
A.7Trial is part of a Paediatric Investigation Plan No
A.8EMA Decision number of Paediatric Investigation Plan
B. Sponsor Information
B.Sponsor: 1
B.1.1Name of SponsorMenzies Institute of Medical Research University of Tasmania
B.1.3.4CountryAustralia
B.3.1 and B.3.2Status of the sponsorNon-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1Name of organisation providing supportNational Health and Medical Research Council
B.4.2CountryAustralia
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1Name of organisationRoyal Hobart Hospital
B.5.2Functional name of contact pointProf Peter Dargaville
B.5.3 Address:
B.5.3.1Street AddressLiverpool St
B.5.3.2Town/ cityHobart
B.5.3.3Post code7000
B.5.3.4CountryAustralia
B.5.6E-mailpeter.dargaville@dhhs.tas.gov.au
D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3IMP RoleComparator
D.2 Status of the IMP to be used in the clinical trial
D.2.1IMP to be used in the trial has a marketing authorisation Yes
D.2.1.1.1Trade name Curosurf
D.2.1.1.2Name of the Marketing Authorisation holderAngelini Farmaceutica, Lda., Cruz Quebrada-Dafundo
D.2.1.2Country which granted the Marketing AuthorisationPortugal
D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
D.2.5.1Orphan drug designation number
D.3 Description of the IMP
D.3.1Product nameCurosurf
D.3.4Pharmaceutical form Endotracheopulmonary instillation, solution
D.3.4.1Specific paediatric formulation Yes
D.3.7Routes of administration for this IMPEndotracheopulmonary use
D.3.11 The IMP contains an:
D.3.11.1Active substance of chemical origin No
D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
The IMP is a:
D.3.11.3Advanced Therapy IMP (ATIMP) No
D.3.11.3.1Somatic cell therapy medicinal product No
D.3.11.3.2Gene therapy medical product No
D.3.11.3.3Tissue Engineered Product No
D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
D.3.11.5Radiopharmaceutical medicinal product No
D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
D.3.11.7Plasma derived medicinal product No
D.3.11.8Extractive medicinal product No
D.3.11.9Recombinant medicinal product No
D.3.11.10Medicinal product containing genetically modified organisms No
D.3.11.11Herbal medicinal product No
D.3.11.12Homeopathic medicinal product No
D.3.11.13Another type of medicinal product No
D.8 Information on Placebo
E. General Information on the Trial
E.1 Medical condition or disease under investigation
E.1.1Medical condition(s) being investigated
Respiratory Distress Syndrome in Premature Babies.
E.1.1.1Medical condition in easily understood language
Premature baby immature lung disease.
E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
MedDRA Classification
E.1.3Condition being studied is a rare disease No
E.2 Objective of the trial
E.2.1Main objective of the trial
To evaluate in a randomised controlled trial the efficacy of surfactant delivery via a minimally invasive technique in preterm infants 25-28 weeks gestation with RDS treated with CPAP.
E.2.2Secondary objectives of the trial
To evaluate that early surfactant administration via a minimally-invasive technique to preterm infants on CPAP will result in a lesser duration of mechanical respiratory support, and a higher incidence of survival without bronchopulmonary dysplasia.
E.2.3Trial contains a sub-study No
E.3Principal inclusion criteria
1. Requiring CPAP or nasal IPPV because of respiratory distress.
2. CPAP pressure of 5-8 cm H2O and FiO2 ≥0.30.
3. Less than 6 hours of age.
4. Agreement of the Treating Physician in charge of the infant’s care.
5. Signed parental consent.
E.4Principal exclusion criteria
1. Previously intubated, or in imminent need of intubation because of respiratory distress, apnoea or persistent acidosis.
2. Congenital anomaly or condition that might adversely affect breathing.
3. Identifiable alternative cause for respiratory distress (e.g. congenital pneumonia or pulmonary hypoplasia).
4. Lack of availability of an OPTIMIST treatment team.
E.5 End points
E.5.1Primary end point(s)
Incidence of composite outcome of death or physiological BPD
E.5.1.1Timepoint(s) of evaluation of this end point
36 week gestational age
E.5.2Secondary end point(s)
• Physiological BPD
• Clinical BPD (requirement for oxygen or any form of positive pressure support at 36
weeks corrected gestation).
• Mild/moderate/severe BPD
• Death
• Death or BPD (clinical definition)
• Intraventricular haemorrhage (IVH) (all grades)
• IVH grades III and IV
• Periventricular leukomalacia
• Retinopathy of prematurity > stage II
• Major morbidity (any of IVH grade III or IV, periventricular leukomalacia, ROP >stage II, physiological BPD)
• Death or major morbidity
• NEC (Modified Bell stage 2 or greater)
• NEC or spontaneous intestinal perforation requiring surgery
• Requirement for intubation in the first 72 hours of life
• Requirement for intubation at any time
• Need for additional surfactant therapy
• Overall number of surfactant doses (including that given by MIST)
• Duration of intubation (all episodes)
• Duration of CPAP/NIPPV (all episodes)
• Duration of intubation and CPAP
• Duration of high flow nasal cannula (HFNC), minimum flow rate 2 L/min
• Duration of respiratory support
• Duration of oxygen therapy
• Requirement for oxygen at home
• Length of stay in intensive care
• Length of hospital stay
• Total hospital billings
• Calculated cost of hospitalisation
• Pneumothorax requiring drainage
• Pulmonary haemorrhage
• Patent ductus arteriosus (PDA) requiring anti-prostaglandin therapy
• PDA requiring ligation
• Late onset sepsis (positive bacterial or fungal culture from a normally sterile site)
• Time to regain birth weight
• Incidence of successful surfactant administration via MIST
• Number of catheterisation attempts
• Duration of bradycardia and hypoxaemia
• Requirement for, and duration of, positive pressure ventilation by mask
• Incidence of apparent discomfort
E.5.2.1Timepoint(s) of evaluation of this end point
Discharge of baby from hospital (first admission)
E.6 and E.7 Scope of the trial
E.6Scope of the trial
E.6.1Diagnosis Yes
E.6.2Prophylaxis No
E.6.3Therapy Yes
E.6.4Safety No
E.6.5Efficacy Yes
E.6.6Pharmacokinetic No
E.6.7Pharmacodynamic No
E.6.8Bioequivalence No
E.6.9Dose response No
E.6.10Pharmacogenetic No
E.6.11Pharmacogenomic No
E.6.12Pharmacoeconomic No
E.6.13Others No
E.7Trial type and phase
E.7.1Human pharmacology (Phase I) No
E.7.1.1First administration to humans No
E.7.1.2Bioequivalence study No
E.7.1.3Other No
E.7.1.3.1Other trial type description
E.7.2Therapeutic exploratory (Phase II) No
E.7.3Therapeutic confirmatory (Phase III) No
E.7.4Therapeutic use (Phase IV) Yes
E.8 Design of the trial
E.8.1Controlled Yes
E.8.1.1Randomised Yes
E.8.1.2Open No
E.8.1.3Single blind No
E.8.1.4Double blind Yes
E.8.1.5Parallel group Yes
E.8.1.6Cross over No
E.8.1.7Other No
E.8.2 Comparator of controlled trial
E.8.2.1Other medicinal product(s) No
E.8.2.2Placebo No
E.8.2.3Other Yes
E.8.2.3.1Comparator description
A sham procedure will be performed.
E.8.2.4Number of treatment arms in the trial2
E.8.3 Will this trial be conducted at a single site globally? No
E.8.4 Will this trial be conducted at multiple sites globally? Yes
E.8.6 Trial involving sites outside the EEA
E.8.6.2Trial being conducted completely outside of the EEA Yes
E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
Australia
Israel
New Zealand
Slovenia
Turkey
United States
E.8.7Trial has a data monitoring committee Yes
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
LVLS
E.8.9 Initial estimate of the duration of the trial
E.8.9.2In all countries concerned by the trial years7
F. Population of Trial Subjects
F.1 Age Range
F.1.1Trial has subjects under 18 Yes
F.1.1Number of subjects for this age range: 606
F.1.1.1In Utero No
F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Yes
F.1.1.2.1Number of subjects for this age range: 606
F.1.1.3Newborns (0-27 days) No
F.1.1.4Infants and toddlers (28 days-23 months) No
F.1.1.5Children (2-11years) No
F.1.1.6Adolescents (12-17 years) No
F.1.2Adults (18-64 years) No
F.1.3Elderly (>=65 years) No
F.2 Gender
F.2.1Female Yes
F.2.2Male Yes
F.3 Group of trial subjects
F.3.1Healthy volunteers No
F.3.2Patients No
F.3.3Specific vulnerable populations Yes
F.3.3.1Women of childbearing potential not using contraception No
F.3.3.2Women of child-bearing potential using contraception No
F.3.3.3Pregnant women No
F.3.3.4Nursing women No
F.3.3.5Emergency situation No
F.3.3.6Subjects incapable of giving consent personally Yes
F.3.3.6.1Details of subjects incapable of giving consent
25-28 week gestation babies
F.3.3.7Others No
F.4 Planned number of subjects to be included
F.4.2 For a multinational trial
F.4.2.2In the whole clinical trial 606
F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
Normal standard treatment.
G. Investigator Networks to be involved in the Trial
H.4 Third Country in which the Trial was first authorised
H.4.1Third Country in which the trial was first authorised: Australia
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