- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01089439
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
Status
Completed
Conditions
Intervention / Treatment
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:
|
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
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Pathologic Processes
- Respiratory Tract Diseases
- Respiration Disorders
- Lung Diseases
- Disease
- Hematologic Diseases
- Genetic Diseases, Inborn
- Anemia
- Anemia, Hemolytic, Congenital
- Anemia, Hemolytic
- Hemoglobinopathies
- Syndrome
- Anemia, Sickle Cell
- Acute Chest Syndrome
- Physiological Effects of Drugs
- Neurotransmitter Agents
- Molecular Mechanisms of Pharmacological Action
- Vasodilator Agents
- Autonomic Agents
- Peripheral Nervous System Agents
- Protective Agents
- Bronchodilator Agents
- Anti-Asthmatic Agents
- Respiratory System Agents
- Antioxidants
- Free Radical Scavengers
- Endothelium-Dependent Relaxing Factors
- Gasotransmitters
- Nitric Oxide
Other Study ID Numbers
- P071003
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
No
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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