Nitric Oxide Inhalation to Treat Sickle Cell Pain Crises
Condition: Anemia, Sickle Cell
MeSH terms: Anemia, Sickle Cell
Intervention: Nitric Oxide; Placebo
Intervention description: Nitric oxide will be delivered for 4 hours at 80 ppm through a face mask. The dose will then be reduced to 40 ppm for 4 hours. After a total of 8 hours of treatment through face mask, the patient will get 6 mL/puls/breath of NO at 800 ppm or 3 m//pulse/breath, depending on patient weight.; Nitrogen gas will be delivered in the same manor as the experimental drug.
Intervention MeSH terms: Nitric Oxide
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 150.0
Sex: All
Minimum age: 10 Years
Healthy volunteers: No
Keywords: Blood Flow; Nitric Oxide; Pain Crisis; Sickle Cell Anemia; Vaso-Occlusive Crisis; Sickle Cell Disease; SCD

Summary:

      This study will examine whether nitric oxide (NO) gas can reduce the time it takes for pain
      to go away in patients who are in sickle cell crisis. NO is important in regulating blood
      vessel dilation, and consequently, blood flow. The gas is continuously produced by cells that
      line the blood vessels. It is also transported from the lungs by hemoglobin in red blood
      cells.

      Patients 10 years of age or older with sickle cell disease (known SS, S-beta-thalassemia or
      other blood problems causing sickle cell disease) may be eligible for this study. Patients
      whose disease is due to hemoglobin (Hgb) SC are excluded. Candidates are screened with blood
      tests and a chest x-ray to look at the lungs and heart.

      Participants are admitted to the hospital in a pain crisis. They are evaluated and then
      randomly assigned to receive one of two treatments: 1) standard treatment plus NO, or 2)
      standard treatment plus placebo. The placebo used in this study is nitrogen, a gas that makes
      up most of the air we breathe and is not known to help in sickle cell disease.

      For the first 8 hours of the study, patients receive placebo or NO through a facemask. The
      mask may be taken off for 5 minutes every hour and for not more than 20 minutes to eat a
      meal. After the first 8 hours, the gas is delivered through a nasal cannula (small plastic
      tubing that rests under the nose) that may be taken off only while showering or using the
      restroom. Patients are questioned about the severity of their pain when they start the study
      and then every few hours while they are in the hospital. Their vital signs (temperature,
      breathing rate, and blood pressure) and medicines are checked. Patients will breathe the gas
      for a maximum of 3 days, but will stay hospitalized until the patient feels well enough to go
      home. Patients are followed up about 1 month after starting the study by a return visit to
      the hospital or by a phone call.
    

Eligibility criteria:

        Inclusion Criteria:

        Each subject must meet all of the following inclusion criteria during the screening process
        in order to participate in the study:

          -  Patient must have a diagnosis of SCD (known SS, S-Beta-thalassemia or other
             hemoglobinopathies causing sickle cell disease). Patients with disease due to Hgb SC
             are not permitted.

          -  Must present to the ED/EC or other appropriate unit in VOC.

          -  Greater than or equal to 10 years old.

          -  Written informed consent/assent has been obtained.

        Exclusion Criteria:

        Subjects meeting any of the following criteria during baseline evaluation will be excluded
        from entry into the study:

          -  Exposure to therapeutic nitric oxide within the past 12 hours.

          -  Patient has received sildenafil or other phosphodiesterase 5 inhibitors, therapeutic
             L-arginine, nitroprusside or nitroglycerine within the past 12 hours.

          -  Patient has received previous ED/EC or other appropriate unit treatment for a
             vaso-occlusive crisis less than 48 hours or hospitalization less than 14 days ago
             (patients transferred directly from another ED or clinic may be enrolled).

          -  Patient has visited the ED/EC or other appropriate unit greater than 10 times in the
             past year having a vaso-occlusive crisis.

          -  Patients presenting with clinically diagnosed bacterial infection (e.g.,
             osteomyelitis, pneumonia, sepsis or meningitis).

          -  Patients who are currently enrolled in any other investigational drug study except for
             hydroxyurea studies.

          -  Pregnant women (urine HCG + )/ nursing mothers.

          -  Patients who have received an exchange transfusion (not simple transfusion) in the
             last 30 days or are on a chronic simple or exchange transfusion program.

          -  Suspected splenic sequestration.

          -  Acute chest syndrome or pneumonia: Abnormal new pulmonary infiltrate (alveolar
             infiltration and not atelectasis) and one or more pulmonary signs and/or symptoms
             (fever, rales, wheezing, cough, shortness of breath, retractions).

          -  Previous participation in this study.