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Safety and Efficacy of Sodium Nitrite in Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT01033227
Recruitment Status : Terminated (Low enrollment)
First Posted : December 16, 2009
Results First Posted : February 8, 2017
Last Update Posted : October 17, 2017
Sponsor:
Collaborator:
Hope Pharmaceuticals
Information provided by (Responsible Party):
Thomas Coates, Children's Hospital Los Angeles

Study Description
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Brief Summary:
This study will determine if administration of sodium nitrite is safe and can improve small vessel blood flow and tissue oxygenation when given as an additional treatment in patients with acute vaso-occlusive crisis (pain crisis) associated with sickle cell disease.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: sodium nitrite injection, usp Phase 1 Phase 2

Detailed Description:

Nitric oxide (NO) is a naturally occuring chemical that relaxes blood vessels and helps improve blood flow.

The pain associated with vaso-occlusive crisis (pain crisis) in sickle cell disease is caused in part by lack of oxygen and increased tissue acid because blood flow is blocked by stiff sickle red cells. Administration of sodium nitrite should generate nitric oxide in this area of hypoxia and acidosis and improve blood flow.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Safety and Efficacy Evaluation of Sodium Nitrite Injection for the Treatment of Vaso-Occlusive Crisis Associated With Sickle Cell Disease
Study Start Date : December 2009
Actual Primary Completion Date : June 2012
Actual Study Completion Date : June 2012

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Safety

Arms and Interventions
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Arm Intervention/treatment
No Intervention: No drug
No study drug administered
Experimental: Sodium nitrite injection, USP
Administration if sodium nitrite injection, USP
 Drug: sodium nitrite injection, usp
Sodium nitrite injection, USP will be administered in blocks of six subjects (3 sodium nitrite and 3 no drug). A total of five dose levels are planned, pending safety starting. Drug will be given by continuous infusion infusion for 48 hours starting at 6 nmol/min/kg (10% of the maximal tolerated dose).
Other Name: sodium nitrite


Outcome Measures
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Primary Outcome Measures :
  1. 48 Hour Sodium Nitrite Infusion Safety as Determined by Number of Participants With No Adverse Events [ Time Frame: 48 hours from start of infusion ]
    The primary end points will be to determine if a) a 48-hour sodium nitrite infusion is tolerated without a decrease in mean arterial blood pressure by 15mmHg for greater than 2 hours or development of methemoglobin greater than 5% and b) a 48-hour sodium nitrite infusion is safe as determined by monitoring for adverse events


Secondary Outcome Measures :
  1. Secondary End Point [ Time Frame: 48 hours ]
    a) reduced the duration and intensity of pain; b) reduced total narcotic analgesic consumption; and c) reduced length of hospitalization.


Eligibility Criteria
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Ages Eligible for Study:   8 Years to 23 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Subjects meeting all of the following criteria will be considered for admission to the study:

  1. Male or female subject aged between 8 and 23 years of age; only patients up to the age of 23 will be studied at Childrens Hospital Los Angeles
  2. Electrophoretic diagnosis of sickle cell disease;
  3. Sudden onset of acute pain involving >=1 sites typical of vaso-occlusive crisis (Vaso-occlusive crisis is defined as acute, severe pain in the extremities, chest, abdomen, or back that can not be explained by other complications of sickle cell disease or by a cause other than sickle cell disease.);
  4. Severe pain requiring parenteral analgesics and hospitalization.
  5. Informed consent obtained from a legal representative or from subjects 18 years of age or older before enrollment;
  6. Being willing and able to be followed for at least 30 days for evaluation.

Exclusion Criteria:

Subjects presenting with any of the following will not be included in the study:

  1. Clinically significant bleeding;
  2. Current drug abuse or participation in methadone program;
  3. Episode of pain requiring hospitalization within 2 weeks prior to current admission;
  4. Other complications of sickle cell disease including cerebrovascular accident, pulmonary hypertension, or seizure;
  5. Pregnancy (confirmed by a serum human chorionic gonadotropin pregnancy test) or breast feeding;
  6. Blood pressure less than 25th percentile for the subject's age and sex on admission or at the time of screening;
  7. Methemoglobinemia >3%;
  8. Anemia with hemoglobin level less than 6 g/dL;
  9. Red blood cell G6PD deficiency, by laboratory testing prior to enrollment;
  10. History of allergy to nitrites or allergy to other substances characterized by dyspnea and cyanosis;
  11. Treatment with allopurinol, a medication that could interfere with nitrite metabolism, within the past 30 days before screening;
  12. Treatment with any investigational drug within the past 30 days;
  13. Significant acute or chronic concomitant diseases (including renal, hepatic, cardiovascular, pulmonary, or oncologic disease, sepsis, pulmonary edema, pulmonary embolism) that would be inconsistent with survival for at least 6 months;
  14. Any subject judged by the clinical investigator or study manager to be inappropriate for the study.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01033227


Locations
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United States, California
Childrens Hospital Los Angeles
Los Angeles, California, United States, 90027
Sponsors and Collaborators
Children's Hospital Los Angeles
Hope Pharmaceuticals
Investigators
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Principal Investigator: Thomas Coates, MD Children's Hospital Los Angeles
More Information
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Publications:

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Responsible Party: Thomas Coates, Hematology Division Head, Children's Hospital Los Angeles
ClinicalTrials.gov Identifier: NCT01033227    
Other Study ID Numbers: SN-SC-02-01
First Posted: December 16, 2009    Key Record Dates
Results First Posted: February 8, 2017
Last Update Posted: October 17, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Thomas Coates, Children's Hospital Los Angeles:
sickle cell disease
sickle cell anemia
pain crisis
nitric oxide
nitrite
 vaso occlusive crisis
pain medication
sickled cells
blood flow
microcirculation
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn