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Completed

NCT Number: NCT02061202

Inhaled Mometasone to Reduce Painful Episodes in Patients With Sickle Cell Disease

The proposed research is designed to test the global hypothesis that inhaled corticosteroids (ICS), a therapy developed to treat asthma, will prevent vasoocclusive painful episodes in adults with Sickle Cell Disease (SCD) who wheeze, but do not meet criteria for a diagnosis of asthma. The specific aims of this proposal are 1) Conduct a feasibility study - a randomized controlled trial of ICS for adults with SCD who do not meet criteria for a diagnosis of asthma but report recurrent cough or wheezing, 2) Measure the effects of ICS on biological correlates of pulmonary inflammation (as determined by exhaled nitric oxide) and vascular injury (as determined by sVCAM) in SCD, and 3) Compare properties of traditional and Bayesian adaptive clinical trial design for therapeutic trials in SCD in preparation for designing a definitive trial of ICS. These aims have the potential to 1) change the standard of care for individuals with SCD and recurrent cough or wheeze, 2) provide insight into the pathogenesis of non-asthmatic wheezing in SCD and its response to treatment, 3) explore the suitability of innovative clinical trial designs to overcome the challenges that have hindered therapeutic innovation for SCD.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

15 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Icahn School of Medicine at Mount Sinai

New York, 10029, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age 15 or older
  • Sever SCD phenotypes (Hb SS and Sβthalassemia0)
  • A positive response to cough/wheeze questions

Exclusion criteria

  • Patient carries a physician diagnosis of asthma
  • Patient is prescribed asthma medications
  • Patient is currently having a painful crisis (as defined by validated pain diary questions)
  • Patient has acute respiratory symptoms
  • Known hypersensitivity to milk proteins
  • Meets criteria for our operational diagnosis of asthma
  • More than 15 ED visits for pain over the preceding 12 months
  • Admitted or discharged from the hospital for SCD pain within the last 7 days

Treatment and study plan

Mometasone furoate

Drug

inhaled cortico-steroid (ICS) with a dosage of 220mcg once daily for 16 weeks

Placebo

Drug

placebo training inhaler with the same instructions as the experimental group.

Primary outcomes

  1. Number of Participants Who Completed Follow up

    Time frame: at 2 years

    Feasibility is determined by calculating the proportion of randomized participants who complete follow up and a minimum of 30 pain diaries with good adherence to the study medication vs. the number enrolled.

Secondary outcomes

  1. Change in Exhaled Nitric Oxide (eNO)

    Time frame: Before ICS therapy begins and at 8 weeks post enrollment

    Change in effects of inhaled corticosteroids (ICS) as measured by exhaled nitric oxide levels, which is the primary marker of pulmonary inflammation.

  2. Change in Soluble Vascular Cell Adhesion Molecule (sVCAM) Level

    Time frame: Before ICS therapy begins and at 8 weeks post enrollment

    Mean Change in effects of inhaled corticosteroids vascular injury, assessed by biomarker sVCAM as a surrogate for vascular injury.

  3. Change in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me)

    Time frame: baseline and week 20

    Mean changes in ASCQ-Me (NHLBI developed a patient-reported Sickle Cell Disease (SCD) quality of life measurement tool) pain impact, at week 20 as compared to baseline. A reduction change on a 100-point scale indicated improved quality of life. ASCQ-Me uses a T-score metric (0-100) in which 50 is the mean of the reference population and 10 is the standard deviation (SD) of that population.

  4. The Medication Adherence Report Scale

    Time frame: 20 weeks

    The medication adherence report scale for asthma is a 10 question tool scored between 0 and 5, with full scale from 0 to 25, with higher scores indicating greater adherence

  5. Change in the Numerical Rating Scale (NRS) for Pain

    Time frame: baseline and 20 weeks

    Mean change in patient reported pain NRS score, full scale range 0- 10, higher score indicate more pain

  6. Asthma Control Test

    Time frame: 8 weeks

    Asthma control test, total score from 0-25, with higher score indicating more symptoms

  7. Admissions or Visits to the Hospital

    Time frame: baseline through 8 weeks

    Number of times participant visited the Emergency Department (ED) or was admitted to the hospital

  8. Change in Reticulocytes Count

    Time frame: baseline and 8 weeks

    Mean change in reticulocytes count - the number of new red blood cells.

  9. Change in FEV1/FVC

    Time frame: baseline and 8 weeks

    Mean change in FEV1/FVC at 8 weeks compared to baseline

Sponsors and collaborators

Lead sponsor

Jeffrey Glassberg

Other

Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

Registry information

Official study title

Inhaled Mometasone to Promote Reduction in Vasoocclusive Events

Acronym: IMPROVE

Important dates

Study start
2014
Primary completion
2017
Study completion
2017
First posted
Feb 12, 2014
Registry last updated
Mar 11, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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