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

Condition(s) targeted: Acute Chest Syndrome; Sickle Cell Disease

Intervention: Nitric oxide by inhalation INOMAX (Drug); Placebo (Drug)

Phase: Phase 2

Status: Recruiting

Sponsored by: Assistance Publique - Hôpitaux de Paris

Official(s) and/or principal investigator(s):
Malika Benkerrou, Dr., Principal Investigator, Affiliation: Assistance Publique - Hôpitaux de Paris

Overall contact:
Malika Benkerrou, Dr., Phone: 01 40 03 57 36, Email: malika.benkerrou@rdb.aphp.fr

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.

Official title: Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children: Randomized, Double Blind Placebo-controlled Concept-proof Trial

Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Primary outcome: 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:

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

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.

Minimum age: 1 Year. Maximum age: 18 Years. Gender(s): Both.

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

Malika Benkerrou, Dr., Phone: 01 40 03 57 36, Email: malika.benkerrou@rdb.aphp.fr

Hoipital Robert Debre, Paris 75019, France; Recruiting
Malika Benkerrou, Dr., Phone: 01 40 03 57 36, Email: malika.benkerrou@rdb.aphp.fr
Jean-Christophe Mercier, Pr., Phone: 01 40 03 21 86

Starting date: June 2010
Last updated: August 4, 2012