E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
massive blood loss in surgery for premature fusion of cranial sutures, massive blood loss in fused bones of the skull
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massaal bloedverlies bij chirurgie voor voortijdige vergroeing van schedelnaden, massaal bloedverlies bij de operatie voor aan elkaar groeien van schedelnaden |
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E.1.1.1 | Medical condition in easily understood language |
massive blood loss |
massaal bloedverlies |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10005645 |
E.1.2 | Term | Blood loss of (NOS) |
E.1.2 | System Organ Class | 100000004866 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective is to investigate whether the volume of infused blood products can be reduced, in children undergoing elective craniosynostosis surgery, using fibrinogen infusion at the start of the operative procedure compared to a placebo-control arm. |
Het primaire doel van ons onderzoek is om in een gerandomiseerd, gecontroleerd dubbel-blind onderzoek aan te tonen dat de kinderen die met fibrinogeen concentraat (Haemocomplettan P®) worden behandeld minder bloedtransfusies nodig zullen hebben dan de kinderen die met de placebo NaCl 0.9% worden behandeld. (zie pagina 9 en 10)
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E.2.2 | Secondary objectives of the trial |
Secondary objectives include the following parameters:
- blood loss in ml
- duration of the operation time,
- the number of hours of post-operative intensive care admission
- duration of total hospital stay
- postoperative complications such as thromboembolic events or wound infections.
- the safety of fibrinogen infusion in this patient group
- monitoring of coagulation parameters including TEG |
Het tweede doel van dit onderzoek zal de mate van bloedverlies, de duur van de operatie en postoperatieve complicaties inventariseren, alsmede de veiligheid van de studiemedicatie. (zie pagina 9 en 10) M.b.v monitoring met thromboelastografie 'aan het bed' zal blijken dat snellere interventie tijdens een operatie mogelijk is, waardoor uiteindelijk minder bloedverlies en dus minder transfusie behoefte zal ontstaan. Uiteindelijk zullen de uitkomsten van waarde zijn voor verdere geprotocolleerde diagnostiek en behandeling van elk massaal bloedverlies op de kinderleeftijd. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- primary non-syndromic craniosynostosis undergoing elective repair
- written informed consent
- age older than 5 months and younger than 25 months |
- kinderen die in aanmerking komen voor chirurgie voor een niet-syndromale craniosynostose
- getekend toestemmingsformulier
- leeftijd tussen 5 en jonger dan 25 maanden |
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E.4 | Principal exclusion criteria |
- the presence of congenital bleeding diathesis or congenital prothrombotic risk factors like protein C or S deficiency or antithrombin deficiency. (Congenital Factor F Leiden and the prothrombin F II mutation are no contraindication for surgery).
- known hypersensitivity against Haemocomplettan P®
- known coagulopathy
- presence of a craniofacial malformation syndrome.
- active infectious disease
- patients with anemia or thrombocytopenia before the surgical intervention
- patients with prior thrombo-embolic disease |
- stollingsstoornis hebben
- overgevoeligheid voor Haemomplettan P®
- craniosynostose als onderdeel van een syndromale afwijking
- anemie
- thrombose |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary outcome measure is the amount of transfused blood products in ml. |
Hoeveelheid aan bloedtransfusies in ml |
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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) |
Secondary study parameters are:
- duration of the operation time,
- the number of hours of post-operative intensive care admission
- duration of total hospital stay
- postoperative complications such as thrombo-embolic events or wound infections the next 2 days.
- the safety of fibrinogen infusion in this patient group according to chills, fever, nausea and vomiting, allergic reactions and thrombo-embolic complications during and after surgery. Total observation will amount 72 hours, in case of an adverse event patient will be followed until resolution of the problem.
- monitoring of coagulation parameters including TEG at fixed time points (see section 7.3.2) |
Secundaire eindpunten:
- de duur van de operatie,
- aantal uren postoperatieve hospitalisatie,
- duur van de total hospitalisatie,
- complicaties inventariseren, alsmdede de veiligheid van de studiemedicatie. (zie pagina 9 en 10)
M.b.v monitoring met thromboelastografie 'aan het bed' zal blijken dat snellere interventie tijdens een operatie mogelijk is, waardoor uiteindelijk minder bloedverlies en dus minder transfusie behoefte zal ontstaan. Uiteindelijk zullen de uitkomsten van waarde zijn voor verdere geprotocolleerde diagnostiek en behandeling van elk massaal bloedverlies op de kinderleeftijd. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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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last patient last visit |
laatste patiënt laatste visite |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |