Inhaled Mometasone to Reduce Painful Episodes in Patients With Sickle Cell Disease
| Status: | Completed |
|---|---|
| Conditions: | Anemia |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | 15 - Any |
| Updated: | 3/13/2019 |
| Start Date: | March 2014 |
| End Date: | November 2017 |
Inhaled Mometasone to Promote Reduction in Vasoocclusive Events
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.
(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.
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
We found this trial at
1
site
1428 Madison Ave
New York, New York 10029
New York, New York 10029
(212) 241-6500
Principal Investigator: Jeffrey Glassberg, MD, MA
Phone: 212-824-8085
Icahn School of Medicine at Mount Sinai Icahn School of Medicine at Mount Sinai is...
Click here to add this to my saved trials
