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

NCT ID: NCT01089439

Last Updated: 2014-12-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-06-30

Study Completion Date

2013-11-30

Brief Summary

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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

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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.

Conditions

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Acute Chest Syndrome Sickle Cell Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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1: INOMAX

Nitric oxide by inhalation INOMAX:

active arm treated with nitric oxide

Group Type ACTIVE_COMPARATOR

Nitric oxide by inhalation INOMAX

Intervention Type DRUG

Nitric oxide by inhalation INOMAX 800 ppm

40 ppm during 24 hours then 20 ppm during 24 hours then 10 ppm during 24 hours

2: Placebo

placebo arm treated with placebo at the same conditions

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

placebo

Interventions

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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

Intervention Type DRUG

Placebo

placebo

Intervention Type DRUG

Other Intervention Names

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INOMAX

Eligibility Criteria

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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
Minimum Eligible Age

1 Year

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Mallinckrodt

INDUSTRY

Sponsor Role collaborator

Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Malika Benkerrou, Dr.

Role: PRINCIPAL_INVESTIGATOR

Assistance Publique - Hôpitaux de Paris

Locations

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Hoipital Robert Debre

Paris, , France

Site Status

Countries

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France

Other Identifiers

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P071003

Identifier Type: -

Identifier Source: org_study_id