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

Primary Purpose

Acute Chest Syndrome, Sickle Cell Disease

Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Nitric oxide by inhalation INOMAX
Placebo
Sponsored by
Assistance Publique - Hôpitaux de Paris
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Acute Chest Syndrome focused on measuring Nitric oxide

Eligibility Criteria

1 Year - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Hoipital Robert Debre

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

1: INOMAX

2: Placebo

Arm Description

Nitric oxide by inhalation INOMAX: active arm treated with nitric oxide

placebo arm treated with placebo at the same conditions

Outcomes

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% )

Secondary Outcome Measures

Number of blood transfusions and total transfused blood volume
Quantity of Pain-killer drugs required and particularly OPIOIDS
Duration of Nitric oxide therapy
Duration of OXYGENOTHERAPY

Full Information

First Posted
October 21, 2009
Last Updated
December 3, 2014
Sponsor
Assistance Publique - Hôpitaux de Paris
Collaborators
Mallinckrodt
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1. Study Identification

Unique Protocol Identification Number
NCT01089439
Brief Title
Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children
Acronym
INNOSTAPED
Official Title
Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children: Randomized, Double Blind Placebo-controlled Concept-proof Trial
Study Type
Interventional

2. Study Status

Record Verification Date
August 2013
Overall Recruitment Status
Completed
Study Start Date
June 2010 (undefined)
Primary Completion Date
November 2013 (Actual)
Study Completion Date
November 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris
Collaborators
Mallinckrodt

4. Oversight

Data Monitoring Committee
Yes

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Chest Syndrome, Sickle Cell Disease
Keywords
Nitric oxide

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
21 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1: INOMAX
Arm Type
Active Comparator
Arm Description
Nitric oxide by inhalation INOMAX: active arm treated with nitric oxide
Arm Title
2: Placebo
Arm Type
Placebo Comparator
Arm Description
placebo arm treated with placebo at the same conditions
Intervention Type
Drug
Intervention Name(s)
Nitric oxide by inhalation INOMAX
Other Intervention Name(s)
INOMAX
Intervention Description
Nitric oxide by inhalation INOMAX 800 ppm 40 ppm during 24 hours then 20 ppm during 24 hours then 10 ppm during 24 hours
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
placebo
Primary Outcome Measure Information:
Title
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
Secondary Outcome Measure Information:
Title
Number of blood transfusions and total transfused blood volume
Time Frame
7 to 10 days
Title
Quantity of Pain-killer drugs required and particularly OPIOIDS
Time Frame
7 to 10 days
Title
Duration of Nitric oxide therapy
Time Frame
after 7 to 10 days
Title
Duration of OXYGENOTHERAPY
Time Frame
7 to 10 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Malika Benkerrou, Dr.
Organizational Affiliation
Assistance Publique - Hôpitaux de Paris
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hoipital Robert Debre
City
Paris
ZIP/Postal Code
75019
Country
France

12. IPD Sharing Statement

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

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