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

NCT01089439 | Completed Phase 2 DMC

• 1 other identifier: P071003
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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.

Conditions

Acute Chest Syndrome; Sickle Cell Disease

Keywords

Nitric oxide

Outcome Measures

Primary Outcomes (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 Outcomes (4)

• Number of blood transfusions and total transfused blood volume

  7 to 10 days

• Quantity of Pain-killer drugs required and particularly OPIOIDS

  7 to 10 days

• Duration of Nitric oxide therapy

  after 7 to 10 days

• Duration of OXYGENOTHERAPY

  7 to 10 days

Study Arms (2)

1: INOMAX

ACTIVE COMPARATOR

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

Drug: Nitric oxide by inhalation INOMAX

2: Placebo

PLACEBO COMPARATOR

placebo arm treated with placebo at the same conditions

Drug: Placebo

Interventions

Nitric oxide by inhalation INOMAX 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

Also known as: INOMAX

1: INOMAX

Placebo DRUG

placebo

2: Placebo

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• Mallinckrodt: collaborator

Study Sites (1)

Hoipital Robert Debre

Paris, 75019, France

Location

MeSH Terms

Conditions

Acute Chest Syndrome; Anemia, Sickle Cell

Interventions

Endothelium-Dependent Relaxing Factors

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Respiration Disorders; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Vasodilator Agents; Cardiovascular Agents; Therapeutic Uses; Pharmacologic Actions; Chemical Actions and Uses

Study Officials

• Malika Benkerrou, Dr.

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 21, 2009
• First Posted: March 18, 2010
• Study Start: June 1, 2010
• Primary Completion: November 1, 2013
• Study Completion: November 1, 2013
• Last Updated: December 4, 2014

Record last verified: 2013-08

Locations

FR (1)