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

December 3, 2014 updated by: Assistance Publique - Hôpitaux de Paris

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

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.

Study Overview

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.

Study Type

Interventional

Enrollment (Actual)

21

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Paris, France, 75019
        • Hoipital Robert Debre

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: 1: INOMAX

Nitric oxide by inhalation INOMAX:

active arm treated with nitric oxide

Nitric oxide by inhalation INOMAX 800 ppm

40 ppm during 24 hours then 20 ppm during 24 hours then 10 ppm during 24 hours

Other Names:
  • INOMAX
Placebo Comparator: 2: Placebo
placebo arm treated with placebo at the same conditions
placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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% )
Time Frame: 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
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 Outcome Measures

Outcome Measure
Time Frame
Number of blood transfusions and total transfused blood volume
Time Frame: 7 to 10 days
7 to 10 days
Quantity of Pain-killer drugs required and particularly OPIOIDS
Time Frame: 7 to 10 days
7 to 10 days
Duration of Nitric oxide therapy
Time Frame: after 7 to 10 days
after 7 to 10 days
Duration of OXYGENOTHERAPY
Time Frame: 7 to 10 days
7 to 10 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Collaborators

Investigators

  • Principal Investigator: Malika Benkerrou, Dr., Assistance Publique - Hôpitaux de Paris

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2010

Primary Completion (Actual)

November 1, 2013

Study Completion (Actual)

November 1, 2013

Study Registration Dates

First Submitted

October 21, 2009

First Submitted That Met QC Criteria

March 17, 2010

First Posted (Estimate)

March 18, 2010

Study Record Updates

Last Update Posted (Estimate)

December 4, 2014

Last Update Submitted That Met QC Criteria

December 3, 2014

Last Verified

August 1, 2013

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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