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

Sponsor
Assistance Publique - Hôpitaux de Paris (Other)
Overall Status
Completed
CT.gov ID
NCT01089439
Collaborator
Mallinckrodt (Industry)
21
Enrollment
1
Location
2
Arms
41
Duration (Months)
0.5
Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Nitric oxide by inhalation INOMAX
  • Drug: Placebo
 Phase 2

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 Design

Study Type:
Interventional
Actual Enrollment :
21 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Double (Participant, 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
Study Start Date :
Jun 1, 2010
Actual Primary Completion Date :
Nov 1, 2013
Actual Study Completion Date :
Nov 1, 2013

Arms and Interventions

Arm Intervention/Treatment
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 Names:
  • INOMAX

    		•
    Placebo Comparator: 2: Placebo

    placebo arm treated with placebo at the same conditions

    Drug: Placebo
    placebo

    Outcome Measures

    Primary Outcome Measures

    1. 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]

    Secondary Outcome Measures

    1. Number of blood transfusions and total transfused blood volume [7 to 10 days]

    2. Quantity of Pain-killer drugs required and particularly OPIOIDS [7 to 10 days]

    3. Duration of Nitric oxide therapy [after 7 to 10 days]

    4. Duration of OXYGENOTHERAPY [7 to 10 days]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    1 Year to 18 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Locations

    Site City State Country Postal Code
    1 Hoipital Robert Debre Paris France 75019

    Sponsors and Collaborators

    • Assistance Publique - Hôpitaux de Paris
    • Mallinckrodt

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Assistance Publique - Hôpitaux de Paris
    ClinicalTrials.gov Identifier:
    NCT01089439
    Other Study ID Numbers:
    • P071003
    First Posted:
    Mar 18, 2010
    Last Update Posted:
    Dec 4, 2014
    Last Verified:
    Aug 1, 2013
    Keywords provided by Assistance Publique - Hôpitaux de Paris
    Nitric oxide
    Additional relevant MeSH terms:
    Acute Chest Syndrome
    Anemia, Sickle Cell
    Syndrome
    Nitric Oxide

    Study Results

    No Results Posted as of Dec 4, 2014