Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children

Sickle Cell Disease Clinical Trial

— INNOSTAPED  

Official title:

Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children: Randomized, Double Blind Placebo-controlled Concept-proof Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01089439
• Other study ID #: P071003
• Status: Completed
• Phase: Phase 2
• First received: October 21, 2009
• Last updated: December 3, 2014
• Start date: June 2010
• Est. completion date: November 2013

Study information

• Verified date: August 2013
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: November 2013
• Est. primary completion date: November 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 18 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Acute Chest Syndrome
• Anemia, Sickle Cell
• Sickle Cell Disease
• Syndrome

Intervention

Drug:
• Nitric oxide by inhalation INOMAX
Nitric oxide by inhalation INOMAX 800 ppm 40 ppm during 24 hours then 20 ppm during 24 hours then 10 ppm during 24 hours
• Placebo
placebo

Locations

Country	Name	City	State
France	Hoipital Robert Debre	Paris

Sponsors (2)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris	INO Therapeutics

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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% )		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	No
Secondary	Number of blood transfusions and total transfused blood volume		7 to 10 days	No
Secondary	Quantity of Pain-killer drugs required and particularly OPIOIDS		7 to 10 days	No
Secondary	Duration of Nitric oxide therapy		after 7 to 10 days	No
Secondary	Duration of OXYGENOTHERAPY		7 to 10 days	No