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About
Randomized, double blind placebo controlled clinical trial to evaluate effectiveness and safety of inhaled nitric oxide for the treatment of sickle cell painful crisis in pediatric patients with sickle cell disease.
Full description
The specific aim of this study is to evaluate the clinical effectiveness of inhaled nitric oxide (INO) for the treatment of acute vaso-occlusive pain crisis in pediatric patients with sickle cell disease. Nitric oxide (NO) deficiency is known to be central to the pathophysiology of vaso-occlusion. The aim is unchanged from the original application. The study is a randomized, double blind, placebo controlled, clinical trial with eligible patients randomized to receive either NO (with 21% O2 final concentration) for 16 hrs with 8 hr wean (80 ppm 0-8 hrs, 40 ppm 9-16 hrs, 20 ppm 17-20 hrs, 10 ppm 21-24 hrs) or placebo (21% O2 alone) for 24 hrs. The null hypothesis is that there is no difference in change in mean pain score after 16 hours between patients treated with NO and placebo. The primary outcome measure of the study remains the difference in mean change in pain scores between groups as assessed using a 10 cm visual analogue pain scale (VAS). Secondary outcome measures also remain the same. The study is a next step to our completed FDA Orphan Product Development Grant funded study (FD-R-001686) that evaluated safety and efficacy of NO used for 4 hrs for treatment of vaso-occlusive crisis in pediatric patients with sickle cell disease.
Enrollment
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Inclusion criteria
Exclusion criteria
> 24 pain crises in the last 12 months. Patients with very frequent pain crisis may have biologic and/or psychosocial pathophysiology that differs from those with fewer pain crises.
Pain crisis treated at a medical facility within the last 12 hours.
Use of investigational drugs other than hydroxyurea within the last 30 days.
Significant respiratory compromise (initial SaO2 < 90%) and/or patients likely to have acute chest syndrome (chest pain and infiltrate) will be eliminated.
Clinically significant acute or chronic cardiac dysfunction.
Acute priapism.
New focal neurologic symptoms.
Concurrent documented or suspected bacterial or parvovirus infection.
Temperature > 38.4ºC. These patients may have concomitant infection.
Transfusion within 30 days or chronic transfusion therapy.
Pregnant female
Cigarette smoker > 1/2 ppd.
Allergy to morphine
Primary purpose
Allocation
Interventional model
Masking
18 participants in 2 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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