Clinical trial NCT02061202: Inhaled Mometasone to Reduce Painful Episodes in Patients With Sickle Cell Disease
Official title: Inhaled Mometasone to Promote Reduction in Vasoocclusive Events
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sickle Cell Disease
Keywords: Sickle Cell Disease
Pain Crisis
Painful Crisis
Sickle Pain
Lead sponsor: Icahn School of Medicine at Mount Sinai
Sponsor class: OTHER
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2014-02-12
Enrollment: 54
Sex: ALL
Minimum age (years): 15
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants Who Completed Follow up
timeFrame: at 2 years
Secondary outcomes:
measure: Change in Exhaled Nitric Oxide (eNO)
timeFrame: Before ICS therapy begins and at 8 weeks post enrollment
measure: Change in Soluble Vascular Cell Adhesion Molecule (sVCAM) Level
timeFrame: Before ICS therapy begins and at 8 weeks post enrollment
measure: Change in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me)
timeFrame: baseline and week 20
measure: The Medication Adherence Report Scale
timeFrame: 20 weeks
measure: Change in the Numerical Rating Scale (NRS) for Pain
timeFrame: baseline and 20 weeks
measure: Asthma Control Test
timeFrame: 8 weeks
measure: Admissions or Visits to the Hospital
timeFrame: baseline through 8 weeks
measure: Change in Reticulocytes Count
timeFrame: baseline and 8 weeks
measure: Change in FEV1/FVC
timeFrame: baseline and 8 weeks
Other outcomes:
Locations:
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
Study officials:
affiliation: Icahn School of Medicine at Mount Sinai
name: Jeffrey Glassberg, MD, MA
role: PRINCIPAL_INVESTIGATOR
References:
citation: Langer AL, Leader A, Kim-Schulze S, Ginzburg Y, Merad M, Glassberg J. Inhaled steroids associated with decreased macrophage markers in nonasthmatic individuals with sickle cell disease in a randomized trial. Ann Hematol. 2019 Apr;98(4):841-849. doi: 10.1007/s00277-019-03635-9. Epub 2019 Feb 20.
citation: Glassberg J, Minnitti C, Cromwell C, Cytryn L, Kraus T, Skloot GS, Connor JT, Rahman AH, Meurer WJ. Inhaled steroids reduce pain and sVCAM levels in individuals with sickle cell disease: A triple-blind, randomized trial. Am J Hematol. 2017 Jul;92(7):622-631. doi: 10.1002/ajh.24742. Epub 2017 Jun 5.