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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00094887
Other study ID # INOT 36
Secondary ID 05-H-0019
Status Completed
Phase Phase 2
First received
Last updated
Start date October 2004
Est. completion date December 2008

Study information

Verified date October 2017
Source Mallinckrodt
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Description:

The object of this study is to determine the safety and efficacy of nitric oxide for inhalation in the treatment of vaso-occlusive pain crisis (VOC) in patients with sickle cell disease. The study population will include patients with sickle cell disease (SS, S-beta-Thalassemia) presenting with vaso-occlusive pain crisis. Patients will be administered either placebo or inhaled nitric oxide to see if the experimental agent, inhaled nitric oxide, can reduce the time it takes for resolution of the vaso-occlusive crisis.


Other known NCT identifiers
  • NCT00652535

Recruitment information / eligibility

Status Completed
Enrollment 150
Est. completion date December 2008
Est. primary completion date December 2008
Accepts healthy volunteers No
Gender All
Age group 10 Years and older
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Nitric Oxide
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.
Placebo
Nitrogen gas will be delivered in the same manor as the experimental drug.

Locations

Country Name City State
United States Colorado Sickle Cell Treatement and Research Center Aurora Colorado
United States Johns Hopkins University Baltimore Maryland
United States National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda Maryland
United States University of Alabama Birmingham Alabama
United States Brigham and Women's Hospital Boston Massachusetts
United States Childrens Hospital, Boston Boston Massachusetts
United States Case Western Reserve University Hospital Cleveland Ohio
United States Children's Hospital Oakland Oakland California
United States St. Christopher's Hospital for Children Philadelphia Pennsylvania
United States Childrens Hospital, Pittsburgh Pittsburgh Pennsylvania
United States Howard University Hospital Washington District of Columbia

Sponsors (2)

Lead Sponsor Collaborator
Mallinckrodt National Heart, Lung, and Blood Institute (NHLBI)

Country where clinical trial is conducted

United States, 

References & Publications (1)

Sanders DY, Severance HW, Pollack CV Jr. Sickle cell vaso-occlusive pain crisis in adults: alternative strategies for management in the emergency department. South Med J. 1992 Aug;85(8):808-11. — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary Time to Vaso-occlusive Pain Crisis (VOC) Resolution VOC resolution was defined by all of the following conditions:
Pain relief - Visual Analog Scale (VAS) pain scores of 6 or less, (6 as worst and 0 as best)
Freedom from parenteral narcotic use,
Ability to walk unless the subject was not able to walk for any reason other than acute VOC prior to the onset of crisis,
Subject and/or family's belief that the painful crisis could be managed at home with or without oral analgesic use, and the physician concurred with that assessment.
within 30 days
Secondary Length of Hospitalization Length of hospitalization is defined as the length of time from admission to discharge order within 40 days
Secondary Number of Participants Discharged to Home Within the First 24 Hours within 24 hours
Secondary Total Dose of Opioids Received The total dose (mg) of opioid medications received during the trial within 8 hours and within 40 days
Secondary Number of Participants With Acute Chest Syndrome/Pneumonia Requiring Blood Transfusion Number of participants who required a blood transfusion before discharge because of acute chest syndrome/pneumonia within 40 days
Secondary Number of Participants Readmitted to Hospital Within 30 Days After Discharge The number of participants readmitted to the hospital for any reason within 30 days after discharge during first 24 hours and during 30 day follow-up
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