Trial record 12 of 120 for:    oxygen therapy OR supplemental oxygen | Open Studies | NIH, U.S. Fed

Inhaled Nitric Oxide for Pediatric Painful Sickle Crisis

The recruitment status of this study is unknown because the information has not been verified recently.
Verified July 2011 by Children's Hospital Boston.
Recruitment status was  Recruiting
Sponsor:
Collaborator:
Information provided by:
Children's Hospital Boston
ClinicalTrials.gov Identifier:
NCT00142051
First received: August 31, 2005
Last updated: July 15, 2011
Last verified: July 2011
  Purpose

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.


Condition Intervention Phase
Sickle Cell Disease
Drug: nitric oxide
Drug: oxygen
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Inhaled Nitric Oxide for Pediatric Painful Sickle Crisis

Resource links provided by NLM:


Further study details as provided by Children's Hospital Boston:

Primary Outcome Measures:
  • Difference in change in mean pain score (visual analog scale) after 16 hours of treatment between patients treated with INO and placebo. [ Time Frame: every 4 hrs x duration of hospitalization ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Secondary outcome measures to evaluate efficacy [ Time Frame: Duration of hospitalization, followup ] [ Designated as safety issue: No ]
  • Longitudinal analyses of change in VAS pain score over 16 hours. [ Time Frame: every 4 hrs, duration of hospitalization ] [ Designated as safety issue: No ]
  • Change in pain score using a 5 point descriptive scale and a 5 point relief scale. [ Time Frame: every 4 hours duration of hospitalization ] [ Designated as safety issue: No ]
  • Time to pain score less than 5 cm for 2 consecutive VAS pain assessments 4 hours apart and not using parenteral narcotics. [ Time Frame: every 4 hrs, duration of hospitalization ] [ Designated as safety issue: No ]
  • Use of pain medication: cumulative dose of parenteral narcotic pain medications. [ Time Frame: While patient on parenteral narcotic ] [ Designated as safety issue: No ]
  • Duration of hospitalization. [ Time Frame: Time of discharge ] [ Designated as safety issue: No ]
  • Inflammatory markers/mediators. [ Time Frame: 0, 16 and q 24 hrs during hospitalization ] [ Designated as safety issue: No ]
  • Secondary outcome measures to evaluate safety are: [ Time Frame: 4, 8, 16, 24 hrs methb, constant NO2, 02 during inhalation ] [ Designated as safety issue: Yes ]
  • Maximum concentration of methemoglobin. [ Time Frame: 0, 4, 8, 16, 24 hrs ] [ Designated as safety issue: Yes ]
  • Maximum concentration of nitrogen dioxide (NO2) delivered. [ Time Frame: continuous over 24 hrs of inhalaiton ] [ Designated as safety issue: Yes ]
  • Minimum percent oxygen saturation of hemoglobin (SpO2 by pulse oximetry). [ Time Frame: continuous over 24 hrs of inhalation then every 4 hrs for duration of hospitalization ] [ Designated as safety issue: Yes ]
  • Maximum and minimum vital signs: pulse, respiratory rate, blood pressure. [ Time Frame: every 4 hrs during hospitalization ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 20
Study Start Date: April 2005
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
inhaled NO
Drug: nitric oxide
80 ppm 8 hrs, 40 ppm 8 hrs, 20 ppm 4 hrs, 10 ppm 4 hrs
Placebo Comparator: 2
room air inhalation
Drug: oxygen
oxygen fi02 21% (room air)

Detailed 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.

  Eligibility

Ages Eligible for Study:   9 Years to 22 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. HbSS, HbSß0thal or HbSC documented by prior hemoglobin electrophoresis.
  2. Age 9 years or greater, age 22 years or less; pediatric age range, old enough to comply with mask and give reliable pain assessment score.
  3. Acute pain crisis defined as pain in abdomen, back and/or extremities that cannot be explained by a diagnosis other than sickle cell disease.
  4. Initial pain score at least 6 cm; to optimize the likelihood of observing a significant difference in change in pain score between INO treated and placebo groups. Based on data from our previous study, it is anticipated that patients will have an average pre-inhalation pain score of approximately 8 cm.

Exclusion Criteria:

  1. > 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.
  2. Pain crisis treated at a medical facility within the last 12 hours.
  3. Use of investigational drugs other than hydroxyurea within the last 30 days.
  4. Significant respiratory compromise (initial SaO2 < 90%) and/or patients likely to have acute chest syndrome (chest pain and infiltrate) will be eliminated.
  5. Clinically significant acute or chronic cardiac dysfunction.
  6. Acute priapism.
  7. New focal neurologic symptoms.
  8. Concurrent documented or suspected bacterial or parvovirus infection.
  9. Temperature > 38.4ºC. These patients may have concomitant infection.
  10. Transfusion within 30 days or chronic transfusion therapy.
  11. Pregnant female
  12. Cigarette smoker > 1/2 ppd.
  13. Allergy to morphine

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  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00142051

Contacts
Contact: Debra Weiner, MD, PhD 617-355-6624 debra.weiner@childrens.harvard.edu
Contact: Pamela Boardman, MPH 617-355-2901 pamela.boardman@childrens.harvard.edu

Locations
United States, Massachusetts
Children's Hospital Boston Recruiting
Boston, Massachusetts, United States, 02115
Contact: Debra Weiner, MD, PhD    617-355-4144    debra.weiner@childrens.harvard.edu   
Contact: Pamela Boardman, MPH    617-355-2901    pamela.boardman@childrens.harvard.edu   
Sub-Investigator: Carlo Brugnara, MD         
Sub-Investigator: Patricia Hibberd, MD, PhD         
Sub-Investigator: Matthew Heeney, MD         
Sub-Investigator: Nancy Craig, RRT         
Sub-Investigator: Eric Fleegler, MD         
Sponsors and Collaborators
Children's Hospital Boston
Investigators
Principal Investigator: Debra Weiner, MD, PhD Children's Hospital Boston
  More Information

No publications provided

Responsible Party: Debra Weiner, Children's Hospital Boston
ClinicalTrials.gov Identifier: NCT00142051     History of Changes
Other Study ID Numbers: 04-09-119, FD-R-002560-01
Study First Received: August 31, 2005
Last Updated: July 15, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Hospital Boston:
sickle cell disease
pain crisis
vaso-occlusive crisis
nitric oxide

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Nitric Oxide
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Pharmacologic Actions
Anti-Asthmatic Agents
Respiratory System Agents
Therapeutic Uses
Free Radical Scavengers
Antioxidants
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Endothelium-Dependent Relaxing Factors
Vasodilator Agents
Cardiovascular Agents
Protective Agents

ClinicalTrials.gov processed this record on July 10, 2014