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Nitric Oxide Inhalation to Treat Sickle Cell Pain Crises

This study is currently recruiting participants.
Verified by INO Therapeutics, September 2008

Sponsors and Collaborators: INO Therapeutics
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by: INO Therapeutics
ClinicalTrials.gov Identifier: NCT00094887
  Purpose

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.


Condition Intervention Phase
Anemia, Sickle Cell
Drug: Nitric Oxide
Drug: Placebo
Phase II

Genetics Home Reference related topics:   sickle cell disease   

MedlinePlus related topics:   Anemia    Sickle Cell Anemia   

Drug Information available for:   Nitric oxide   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator), Placebo Control, Parallel Assignment, Safety/Efficacy Study
Official Title:   A Prospective, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study of Nitric Oxide for Inhalation in the Acute Treatment of Sickle Cell Pain Crisis

Further study details as provided by INO Therapeutics:

Primary Outcome Measures:
  • Reduction in VAS Pain Score [ Time Frame: baseline, at 2,4,6 and 8 hours after start of drug and every 24 hours while therapy continues. ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Vital Signs [ Time Frame: At baseline, then every hour for the first 8 hours of therapy, followed by every 4 hours of therapy. ] [ Designated as safety issue: No ]
  • Methemoglobin Levels [ Time Frame: at 2,4,6, and 8 hours after the start of therapy and then every 24 hours while on therapy. ] [ Designated as safety issue: Yes ]
  • Need for analgesics [ Time Frame: baseline and throughout treatment. ] [ Designated as safety issue: No ]
  • blood chemistry levels [ Time Frame: every 24 hours for the first 5 days after start of treatment. ] [ Designated as safety issue: No ]
  • Lenght of hospitalization from admissions defined by the time of the discharge order is written [ Time Frame: study duration ] [ Designated as safety issue: No ]
  • Rate of acute chest syndrome/pneumonia requiring blood transfusion [ Time Frame: study duration ] [ Designated as safety issue: No ]

Estimated Enrollment:   150
Study Start Date:   October 2004
Estimated Study Completion Date:   December 2008
Estimated Primary Completion Date:   December 2008 (Final data collection date for primary outcome measure)

Arms Assigned Interventions
1: Experimental
Inhaled nitric oxide
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.
2: Placebo Comparator
Nitrogen gas
Drug: Placebo
Nitrogen gas will be delivered in the same manor as the experimental drug.

Detailed 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.

  Eligibility
Ages Eligible for Study:   10 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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.
  Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00094887

Contacts
Contact: Patient Recruitment and Public Liaison Office     (800) 411-1222     prpl@mail.cc.nih.gov    
Contact: TTY     1-866-411-1010    

Locations
United States, Alabama
University of Alabama     Recruiting
      Birmingham, Alabama, United States
      Sub-Investigator: Thomas Howard, M.D.            
United States, California
Children's Hospital Oakland     Recruiting
      Oakland, California, United States, 94609-1809
      Sub-Investigator: Ward Hager, M.D.            
United States, Colorado
Colorado Sickle Cell Treatement and Research Center     Recruiting
      Aurora, Colorado, United States, 80045
      Sub-Investigator: Rachelle Nuss, M.D.            
United States, District of Columbia
Howard University Hospital     Recruiting
      Washington, District of Columbia, United States, 20060
      Sub-Investigator: Onyinye Onyekwere, M.D.            
United States, Maryland
Johns Hopkins University     Recruiting
      Baltimore, Maryland, United States, 21205
      Sub-Investigator: James Casella, M.D.            
National Institutes of Health Clinical Center, 9000 Rockville Pike     Recruiting
      Bethesda, Maryland, United States, 20892
United States, Massachusetts
Brigham and Women's Hospital     Recruiting
      Boston, Massachusetts, United States, 02115
      Sub-Investigator: Maureen Okam, M.D.            
Childrens Hospital, Boston     Recruiting
      Boston, Massachusetts, United States, 02115
      Sub-Investigator: Debra Weiner, M.D.            
United States, Ohio
Case Western Reserve University Hospital     Recruiting
      Cleveland, Ohio, United States, 44106-2602
      Sub-Investigator: Brian Berman            
United States, Pennsylvania
St. Christopher's Hospital for Children     Recruiting
      Philadelphia, Pennsylvania, United States
      Sub-Investigator: Lewis Hsu, M.D.            
Childrens Hospital, Pittsburgh     Recruiting
      Pittsburgh, Pennsylvania, United States, 15213-2583
      Sub-Investigator: Lakshaman Krishnamurti            

Sponsors and Collaborators

Investigators
Study Director:     James Baldassarre, MD     INO Therapeutics    
  More Information


NIH Clinical Center Detailed Web Page  This link exits the ClinicalTrials.gov site
 

Publications:

Responsible Party:   INO Therapeutics ( James Baldassarre )
Study ID Numbers:   050019, INOT 36, 05-H-0019
First Received:   October 28, 2004
Last Updated:   September 26, 2008
ClinicalTrials.gov Identifier:   NCT00094887
Health Authority:   United States: Food and Drug Administration

Keywords provided by INO Therapeutics:
Blood Flow  
Nitric Oxide  
Pain Crisis  
Sickle Cell Anemia  
Vaso-Occlusive Crisis
Sickle Cell Anemia
Sickle Cell Disease
SCD

Study placed in the following topic categories:
Nitric Oxide
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies
Anemia
Anemia, Hemolytic
Pain
Hemoglobinopathy
Anemia, Sickle Cell
Sickle cell anemia

Additional relevant MeSH terms:
Respiratory System Agents
Vasodilator Agents
Neurotransmitter Agents
Antioxidants
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Anti-Asthmatic Agents
Cardiovascular Agents
Protective Agents
Pharmacologic Actions
Autonomic Agents
Therapeutic Uses
Free Radical Scavengers
Endothelium-Dependent Relaxing Factors
Peripheral Nervous System Agents
Bronchodilator Agents

ClinicalTrials.gov processed this record on November 19, 2008




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