Clinical trial NCT01089439: Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children
Official title: Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children: Randomized, Double Blind Placebo-controlled Concept-proof Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Acute Chest Syndrome
Sickle Cell Disease
Keywords: Nitric oxide
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
Collaborators: name: Mallinckrodt
First posted: 2010-03-18
Enrollment: 21
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death. Nitric oxide is a potent vasodilator that could reverse pulmonary vascular occlusion and restore normal oxygenation. The randomized trial will test that hypothesis.

Detailed description: Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death. Nitric oxide is a potent vasodilator that could reverse pulmonary vascular occlusion and restore normal oxygenation. The randomized trial will test that hypothesis in a prospective randomized double-blind placebo controlled study. 50 children in two years will be included: 25 in each arm.

Eligibility criteria: Inclusion Criteria:

* child between 1 and 18 years old
* Sickle cell anemia or equivalent (sickle beta 0 thalassemia)whoe weight is between 10kg and 65kg
* presenting acute chest syndrome as defined a new radiological infiltrate with tachypnea, respiratory discomfort, cough, chest wall pain and fever more than 38.5°C
* hypoxaemia with transcutaneous oxygen saturation equal or less than 92%
* informed consent signed by parents and approved by the child able to express his consent
* insured by the National social security system or by the universal medical insurance
* previous medical physical examination

Exclusion Criteria:

* respiratory distress with hypoxaemia with transcutaneous oxygen saturation equal or less than 92% under more than 5l/min of oxygen or 40% oxygen inhaled, hypercapnia signs 'sweating, altered consciousness, paCO2 more than 60mmHg) with need of emergency exchange transfusion and/or tracheal intubation with mechanical ventilation
* Isolated acute asthmatic crisis
* stroke or priapism with emergency acute transfusion needed
* acute anemia with hemoglobin drop of more than 20% as compared to steady state hemoglobin
* chronic long term transfusion therapy
* nitric oxyde hypersensitivity
* patients with right-left extra-pulmonary cardiac shunt
* patient previously included in the protocol
* patient participating in another interventional protocol
* pregnancy or breast feeding

Primary outcomes:
measure: We will observe the patient's transfusional needs under nitric oxide (MONOXYDE AZOTE) versus placebo inhaled therapy to evaluate inhaled MOXYDE AZOTE efficacy on improving oxygenation (transcutaneous O2 superior to 92% )
timeFrame: Oxygenation improvement (transcutaneous O2 superior to 92%) after Gas inhalation will be evaluate 2hours after inclusion and therafter every 2 hours until 12 hours therapy and then every six hours for 3days and then once a day till hospital discharge
Secondary outcomes:
measure: Number of blood transfusions and total transfused blood volume
timeFrame: 7 to 10 days
measure: Quantity of Pain-killer drugs required and particularly OPIOIDS
timeFrame: 7 to 10 days
measure: Duration of Nitric oxide therapy
timeFrame: after 7 to 10 days
measure: Duration of OXYGENOTHERAPY
timeFrame: 7 to 10 days
Other outcomes:
Locations:
city: Paris
country: France
facility: Hoipital Robert Debre
Study officials:
affiliation: Assistance Publique - Hôpitaux de Paris
name: Malika Benkerrou, Dr.
role: PRINCIPAL_INVESTIGATOR
References: