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

This study is currently recruiting participants.
Verified July 2012 by Assistance Publique - Hôpitaux de Paris
Sponsor:
Collaborator:
INO Therapeutics
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT01089439
First received: October 21, 2009
Last updated: August 4, 2012
Last verified: July 2012
History of Changes
  Purpose

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.


Condition Intervention Phase
Acute Chest Syndrome
Sickle Cell Disease
 Drug: Nitric oxide by inhalation INOMAX
Drug: Placebo
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children: Randomized, Double Blind Placebo-controlled Concept-proof Trial

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Assistance Publique - Hôpitaux de Paris:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Number of blood transfusions and total transfused blood volume [ Time Frame: 7 to 10 days ] [ Designated as safety issue: No ]
  • Quantity of Pain-killer drugs required and particularly OPIOIDS [ Time Frame: 7 to 10 days ] [ Designated as safety issue: No ]
  • Duration of Nitric oxide therapy [ Time Frame: after 7 to 10 days ] [ Designated as safety issue: No ]
  • Duration of OXYGENOTHERAPY [ Time Frame: 7 to 10 days ] [ Designated as safety issue: No ]

Estimated Enrollment: 50
Study Start Date: June 2010
Estimated Study Completion Date: June 2014
Estimated Primary Completion Date: November 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 1: INOMAX

Nitric oxide by inhalation INOMAX:

active arm treated with nitric oxide

 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

Other Name: INOMAX
Placebo Comparator: 2: Placebo
placebo arm treated with placebo at the same conditions
 Drug: Placebo
placebo
Other Name: Placebo

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

Ages Eligible for Study:   1 Year to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01089439

Contacts
Contact: Malika Benkerrou, Dr. 01 40 03 57 36 malika.benkerrou@rdb.aphp.fr
Contact: Jean-Chistophe Mercier, Pr. 01 40 03 21 86 jean-christophe.mercier@rdb.aphp.fr

Locations
France
Hoipital Robert Debre Recruiting
Paris, France, 75019
Contact: Malika Benkerrou, Dr.     01 40 03 57 36     malika.benkerrou@rdb.aphp.fr    
Contact: Jean-Christophe Mercier, Pr.     01 40 03 21 86        
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
INO Therapeutics
Investigators
Principal Investigator: Malika Benkerrou, Dr. Assistance Publique - Hôpitaux de Paris
  More Information

No publications provided

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT01089439     History of Changes
Other Study ID Numbers: P071003
Study First Received: October 21, 2009
Last Updated: August 4, 2012
Health Authority: France: Ministry of Health

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Nitric oxide

Additional relevant MeSH terms:
Anemia, Sickle Cell
Acute Chest Syndrome
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Lung Diseases
Respiratory Tract Diseases
Respiration Disorders
Nitric Oxide
Bronchodilator Agents
Autonomic Agents
 Peripheral Nervous System Agents
Physiological Effects of Drugs
Pharmacologic Actions
Anti-Asthmatic Agents
Respiratory System Agents
Therapeutic Uses
Free Radical Scavengers
Antioxidants
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Endothelium-Dependent Relaxing Factors
Vasodilator Agents
Cardiovascular Agents
Protective Agents

ClinicalTrials.gov processed this record on June 18, 2013