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

This study is currently recruiting participants.
Verified August 2013 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: September 2, 2013
Last verified: August 2013

October 21, 2009
September 2, 2013
June 2010
November 2013   (final data collection date for primary outcome 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% ) [ 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 ]
We will observe the patient's transfusional needs under NO versus placebo inhaled therapy to evaluate inhaled NO 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 ]
Complete list of historical versions of study NCT01089439 on ClinicalTrials.gov Archive Site
  • 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 ]
  • 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 opioïds [ 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 ]
Not Provided
Not Provided
 
Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children
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.

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.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
  • Acute Chest Syndrome
  • Sickle Cell Disease
  • 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
  • Drug: Placebo
    placebo
    Other Name: Placebo
  • Active Comparator: 1: INOMAX

    Nitric oxide by inhalation INOMAX:

    active arm treated with nitric oxide

    Intervention: Drug: Nitric oxide by inhalation INOMAX
  • Placebo Comparator: 2: Placebo
    placebo arm treated with placebo at the same conditions
    Intervention: Drug: Placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
50
June 2014
November 2013   (final data collection date for primary outcome measure)

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
Both
1 Year to 18 Years
No
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
France
 
NCT01089439
P071003
Yes
Assistance Publique - Hôpitaux de Paris
Assistance Publique - Hôpitaux de Paris
INO Therapeutics
Principal Investigator: Malika Benkerrou, Dr. Assistance Publique - Hôpitaux de Paris
Assistance Publique - Hôpitaux de Paris
August 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP