Randomizowana, kontrolowana próba RIC-NEC (RIC-NEC)
RIC-NEC Faza II Wykonalności Randomizowane, kontrolowane badanie: Zdalne niedokrwienie warunkowane w martwiczym zapaleniu jelit
Necrotizing enterocolitis (NEC) is a serious intestinal disease of preterm and term neonates which remains a major cause of intestinal failure, and an unsolved clinical challenge in pediatrics. While overall mortality of preterm infants continues to decrease due to improvements in general neonatal care, mortality caused by NEC remains high (up to 30-50%) and survivors suffer from reduced quality of life, and long-term disabilities such as debilitating complications of intestinal failure, poor growth and neurodevelopmental delay. Besides prevention, there have been hardly any innovations in the treatment of NEC which underwent trial evaluation.
NEC pathogenesis is multifactorial, but bowel ischemia is known to play an essential role in the development of NEC. Remote ischemic conditioning (RIC) is a therapeutic maneuver that involves brief cycles of non-lethal ischemia and reperfusion applied to a limb, which protects distant organs (such as the intestine) from ischemic damage. The investigators have shown that in preclinical models of NEC, RIC effectively reduces intestinal damage and prolongs survival. The investigators have also demonstrated the safety of RIC in preterm neonates with NEC. Before the investigators can evaluate the effectiveness of RIC in treating neonates with NEC in a Phase III randomized clinical trial (RCT), a Phase II Feasibility RCT must be conducted to evaluate issues related to the enrollment and randomization of neonates, masking of the RIC intervention, and measurement of clinical outcomes.
The investigators hypothesize that it is feasible to conduct a multicenter RCT to evaluate RIC during the management of neonates with medical NEC.
Przegląd badań
Status
Status
Warunki
Warunki
Interwencja / Leczenie
Interwencja / Leczenie
Szczegółowy opis
Background: Necrotizing enterocolitis (NEC) is a serious intestinal disease of preterm and term neonates which remains a major cause of intestinal failure, and an unsolved clinical challenge in pediatrics resulting in mortality rates as high as 50%, reduced quality of life and long-term disabilities such as short bowel syndrome, poor growth, and neurodevelopmental delay. Experimentally, the investigators have discovered that intestinal and brain damage, as well as mortality following NEC, can be avoided by remote ischemic conditioning (RIC) in the early stage of the disease. Remote ischemic conditioning is a therapeutic maneuver involving brief cycles of non-lethal ischemia and reperfusion applied to a limb that protects distant organs (such as the intestine) from sustained ischemic damage. In the clinical setting, the cycles of ischemia and reperfusion can be administered by inflation and deflation of a blood pressure cuff, similar to routine blood pressure measurements. The investigators have also demonstrated that this non-invasive, simple, and easy-to-use maneuver consisting of inflation/deflation of a blood pressure cuff on the upper arm is safe in preterm human neonates with NEC.
Hypothesis and Objectives: The investigators hypothesize that a masked multi-center randomized controlled trial of RIC in neonates with early-stage NEC is feasible.
Study design: This is a Phase II multicenter, masked, randomized controlled feasibility trial consisting of two arms: RIC (intervention) and no RIC (control).
Study population: Preterm neonates with clinical and radiological evidence of early-stage NEC and receiving medical treatment.
Sample size/power of primary endpoint: In the 12 international collaborating centers, the investigators expect to randomize, in 30 months, 78 patients with NEC receiving medical treatment (39 per arm) which represents 40% of approached eligible neonates.
Typ studiów
Typ studiów
Zapisy (Rzeczywisty)
Zapisy
Faza
Faza
- Faza 2
Kontakty i lokalizacje
Lokalizacje studiów
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Ontario
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Toronto, Ontario, Kanada, M5G 1X5
- Mount Sinai Hospital
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Toronto, Ontario, Kanada, M5G1X8
- The Hospital for Sick Children
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Toronto, Ontario, Kanada, M4Y 3M5
- Sunnybrook Health Sciences Center
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Ohio
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Cincinnati, Ohio, Stany Zjednoczone, 45229
- Cincinnati Children's Hospital Medical Center
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Kryteria uczestnictwa
Kryteria kwalifikacji
Kryteria kwalifikacji
Wiek uprawniający do nauki
Akceptuje zdrowych ochotników
Płeć kwalifikująca się do nauki
Opis
Inclusion Criteria:
- All gestational age at birth.
- Current weight ≥600 g
- Confirmed diagnosis of "medical" NEC based on the joint opinion of two attending experts in the field (two neonatologists or one neonatologist and one pediatric surgeon).
- NEC diagnosis established within the previous 24 hours.
Exclusion Criteria:
- Indication for surgery in the joint opinion of the attending neonatologist and pediatric surgeon (i.e. surgical NEC). This diagnosis is based on the presence of pneumoperitoneum in the abdominal radiograph and/or failure of medical treatment for NEC
- Previous episodes of NEC
- Diagnosis of NEC established >24 hours ago
- Major congenital heart disease which needs surgical repair
- Antecedent limb ischemia/limb thrombotic events, occlusive arterial or venous thrombosis
- Associated gastrointestinal anomalies including gastroschisis or congenital diaphragmatic hernia.
Plan studiów
Jak projektuje się badanie?
Szczegóły projektu
- Główny cel: Leczenie
- Przydział: Randomizowane
- Model interwencyjny: Przydział równoległy
- Maskowanie: Potroić
Liczba ramion
Broń i interwencje
Grupa uczestników / ArmGrupa uczestników / Arm |
Interwencja / LeczenieInterwencja / Leczenie |
|---|---|
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Eksperymentalny: Intervention (RIC + standard of care for NEC)
Neonates randomized to the intervention arm will receive RIC and will continue to receive the standard of care for NEC. |
RIC will consist of 4 cycles of limb ischemia (5 min) followed by reperfusion (5 min), repeated on two consecutive days. An appropriately sized blood pressure cuff (covering 2/3 of the distance between the shoulder and the elbow) will be applied by a trained research fellow or nurse to an arm (or leg if the arm is not available because of medical reasons such as central line insertion). The systolic blood pressure will be measured before the first RIC cycle using a different cuff of same size connected to a monitor. During ischemia time, the cuff will be inflated to a pressure of 15 mmHg above the child's systolic pressure. Neonates in this arm will continue to receive standard of care for NEC. |
|
Pozorny komparator: Control (Standard of care for NEC)
Neonates randomized to the control arm will receive the standard of care for NEC. The research fellow or nurse responsible for performing RIC will be performing sham inflation/deflation of the blood pressure cuff connected to a dummy arm to mimic the noise of the cuff for neonates in the control arm. |
Neonates in this arm (i.e. the control arm) will receive standard of care for NEC. |
Co mierzy badanie?
Podstawowe miary wyniku
Podstawowe miary wyniku
Miara wyniku |
Opis środka |
Ramy czasowe |
|---|---|---|
|
The proportion (% total) of screened patients that are eligible for enrollment in the trial.
Ramy czasowe: 24 hours
|
The investigators will determine the proportion (% total) of screened patients who meet the inclusion criteria and do not meet the exclusion criteria and are therefore eligible for enrollment in this study. |
24 hours
|
|
The proportion (% total) of eligible patients that give consent and are randomized.
Ramy czasowe: 24 hours
|
The investigators will determine the proportion (% total) of eligible patients for whom informed consent from parents/caregivers can be obtained and randomization can be completed within 24 hours from confirmed diagnosis of medical NEC by a neonatologist and pediatric surgeon. |
24 hours
|
|
The proportion (% total) of randomized patients that receive allocated intervention.
Ramy czasowe: 72 hours
|
The investigators will determine the proportion (% total) of patients randomized to each study arm who successfully receive the intervention corresponding to that arm: RIC or no RIC. |
72 hours
|
|
The proportion (% total) of randomized patients receiving masked allocated intervention.
Ramy czasowe: 72 hours
|
The investigators will determine the proportion (% total) of randomized patients that receive the allocated intervention (RIC or no RIC) successfully masked from the circle of care as well as parents/caregivers. |
72 hours
|
|
The proportion (% total) of randomized patients assessed for NEC-related outcomes.
Ramy czasowe: 3 months +/- 1 week
|
The investigators will determine the proportion (% total) of randomized patients that are successfully assessed for the NEC-related outcomes (please see secondary outcomes 6-13 below). |
3 months +/- 1 week
|
Miary wyników drugorzędnych
Miary wyników drugorzędnych
Miara wyniku |
Opis środka |
Ramy czasowe |
|---|---|---|
|
Number of patients surviving without the development of intestinal perforation, necrosis or stricture.
Ramy czasowe: 3 months +/- 1 week
|
The investigators will determine the number of randomized patients who survive at 1 month and 3 months post-randomization without developing intestinal perforation, intestinal necrosis, or intestinal stricture. |
3 months +/- 1 week
|
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Timing and cause of death
Ramy czasowe: 3 months +/- 1 week
|
The time, and if possible, the cause of death will be recorded during the 90 days post-randomization considering whether it was possible to determine if death was related to complications of NEC or to a disease process in other systems including cardiac, neurological, respiratory, renal, metabolic. |
3 months +/- 1 week
|
|
Number, type and times of abdominal operations performed.
Ramy czasowe: 3 months +/- 1 week
|
The number, time(s), and type(s) of abdominal operations (insertion of peritoneal drainage or laparotomy) performed during the 90 days post-randomization will be recorded. |
3 months +/- 1 week
|
|
Number of patients receiving parenteral nutrition
Ramy czasowe: 3 months +/- 1 week
|
The number of patients receiving parenteral nutrition during the 90 days post-randomization will be recorded as a measure of intestinal function. |
3 months +/- 1 week
|
|
Number of patients developing severe neurological injury
Ramy czasowe: 3 months +/- 1 week
|
Development of severe neurological injury will be assessed based on head ultrasound at 1-month and 3-months post-randomization and will be defined as the presence of intraventricular hemorrhage (IVH), ventricular enlargement, parenchymal echogenicity or periventricular leucomalacia (PVL). |
3 months +/- 1 week
|
|
Number of patients developing chronic lung disease (CLD)
Ramy czasowe: 3 months +/- 1 week
|
Development of chronic lung disease (CLD) during the 90 days post-randomization will be defined as respiratory support given at 36 weeks' postmenstrual age or at discharge (if earlier than 36 weeks' postmenstrual age) to level 2 neonatal intensive care unit (NICU) and classified in different degrees of severity from mild to moderate to severe CLD according to the criteria published in the Canadian Neonatal Network (CNN) Annual Report (2019). |
3 months +/- 1 week
|
|
Number of patients developing severe retinopathy of prematurity (ROP)
Ramy czasowe: 3 months +/- 1 week
|
During the 90 days post-randomization, the investigators will assess the development of Stage 3, 4 or 5 retinopathy of prematurity (ROP) as defined by the International Classification of ROP and/or those infants requiring treatment (laser or intraocular injection). ROP will be scored as the highest stage in either eye identified at any time. |
3 months +/- 1 week
|
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Survey of stakeholders' satisfaction
Ramy czasowe: 1 month +/- 1 week
|
Satisfaction of key trial stakeholders (parents and healthcare workers) with the recruitment process, delivery and masking of the intervention will be evaluated by questionnaires, using a scale of 0-4. Higher scores indicate greater satisfaction and lower scores indicate less satisfaction. |
1 month +/- 1 week
|
Współpracownicy i badacze
Sponsor
Sponsor
Współpracownicy
Współpracownicy
Śledczy
Śledczy
- Główny śledczy: Agostino Pierro, OBE, MD, The Hospital for Sick Children
Publikacje i pomocne linki
Publikacje ogólne
- Koike Y, Li B, Ganji N, Zhu H, Miyake H, Chen Y, Lee C, Janssen Lok M, Zozaya C, Lau E, Lee D, Chusilp S, Zhang Z, Yamoto M, Wu RY, Inoue M, Uchida K, Kusunoki M, Delgado-Olguin P, Mertens L, Daneman A, Eaton S, Sherman PM, Pierro A. Remote ischemic conditioning counteracts the intestinal damage of necrotizing enterocolitis by improving intestinal microcirculation. Nat Commun. 2020 Oct 2;11(1):4950. doi: 10.1038/s41467-020-18750-9.
- Chen Y, Koike Y, Chi L, Ahmed A, Miyake H, Li B, Lee C, Delgado-Olguin P, Pierro A. Formula feeding and immature gut microcirculation promote intestinal hypoxia, leading to necrotizing enterocolitis. Dis Model Mech. 2019 Dec 9;12(12):dmm040998. doi: 10.1242/dmm.040998.
- Chen Y, Chang KT, Lian DW, Lu H, Roy S, Laksmi NK, Low Y, Krishnaswamy G, Pierro A, Ong CC. The role of ischemia in necrotizing enterocolitis. J Pediatr Surg. 2016 Aug;51(8):1255-61. doi: 10.1016/j.jpedsurg.2015.12.015. Epub 2016 Jan 8.
- Alganabi M, Lee C, Bindi E, Li B, Pierro A. Recent advances in understanding necrotizing enterocolitis. F1000Res. 2019 Jan 25;8:F1000 Faculty Rev-107. doi: 10.12688/f1000research.17228.1. eCollection 2019.
- Neu J, Walker WA. Necrotizing enterocolitis. N Engl J Med. 2011 Jan 20;364(3):255-64. doi: 10.1056/NEJMra1005408. No abstract available.
- Willan AR, Thabane L. Bayesian methods for pilot studies. Clin Trials. 2020 Aug;17(4):414-419. doi: 10.1177/1740774520914306. Epub 2020 Apr 16.
Daty zapisu na studia
Główne daty studiów
Rozpoczęcie studiów (Rzeczywisty)
Rozpoczęcie studiów
Zakończenie podstawowe (Rzeczywisty)
Zakończenie podstawowe
Ukończenie studiów (Rzeczywisty)
Ukończenie studiów
Daty rejestracji na studia
Pierwszy przesłany
Pierwszy przesłany
Pierwszy przesłany, który spełnia kryteria kontroli jakości
Pierwszy przesłany, który spełnia kryteria kontroli jakości
Pierwszy wysłany (Rzeczywisty)
Pierwszy wysłany
Aktualizacje rekordów badań
Ostatnia wysłana aktualizacja (Rzeczywisty)
Ostatnia wysłana aktualizacja
Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości
Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości
Ostatnia weryfikacja
Ostatnia weryfikacja
Więcej informacji
Terminy związane z tym badaniem
Słowa kluczowe
Dodatkowe istotne warunki MeSH
- Choroby układu moczowo-płciowego
- Choroby układu moczowo-płciowego kobiet i powikłania ciąży
- Choroby jelit
- Poród położniczy, przedwczesny
- Powikłania porodu położniczego
- Powikłania ciąży
- Choroby Układu Pokarmowego
- Choroby przewodu pokarmowego
- Nieżyt żołądka i jelit
- Zapalenie jelit
- Przedwczesny poród
- Zapalenie jelit, martwicze
- Administracja usług zdrowotnych
- Jakość opieki zdrowotnej, dostęp i ocena
- Jakość opieki zdrowotnej
- Wskaźniki jakości, opieka zdrowotna
- Standard opieki
Inne numery identyfikacyjne badania
Inne numery identyfikacyjne badania
- CTO 3802
Plan dla danych uczestnika indywidualnego (IPD)
Planujesz udostępniać dane poszczególnych uczestników (IPD)?
Informacje o lekach i urządzeniach, dokumenty badawcze
Bada produkt leczniczy regulowany przez amerykańską FDA
Bada produkt urządzenia regulowany przez amerykańską FDA
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