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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01089439
• Date of registration: 21/10/2009
• Prospective Registration: Yes
• Primary sponsor: Assistance Publique - Hôpitaux de Paris
• Public title: Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children INNOSTAPED
• Scientific title: Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children: Randomized, Double Blind Placebo-controlled Concept-proof Trial
• Date of first enrolment: June 2010
• Target sample size: 21
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01089439
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

France

Contacts

• Name: Malika Benkerrou, Dr.
• Affiliation: Assistance Publique - Hôpitaux de Paris

Key inclusion & exclusion 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



Age minimum: 1 Year
Age maximum: 18 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Acute Chest Syndrome

Sickle Cell Disease

Intervention(s)

Drug: Placebo

Drug: Nitric oxide by inhalation INOMAX

Primary Outcome(s)

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(s)

Duration of Nitric oxide therapy [Time Frame: after 7 to 10 days]

Quantity of Pain-killer drugs required and particularly OPIOIDS [Time Frame: 7 to 10 days]

Duration of OXYGENOTHERAPY [Time Frame: 7 to 10 days]

Number of blood transfusions and total transfused blood volume [Time Frame: 7 to 10 days]

Secondary ID(s)

P071003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

INO Therapeutics