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

Sickle Cell Disease Clinical Trial

— IMPROVE  

Official title:

Inhaled Mometasone to Promote Reduction in Vasoocclusive Events

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02061202
• Other study ID #: GCO 12-1565
• Secondary ID: K23HL119351
• Status: Completed
• Phase: Phase 2
• Start date: March 2014
• Est. completion date: November 2017

Study information

• Verified date: March 2019
• Source: Icahn School of Medicine at Mount Sinai
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 54
• Est. completion date: November 2017
• Est. primary completion date: November 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 15 Years and older

Eligibility

Inclusion Criteria:

• Age 15 or older

• Sever SCD phenotypes (Hb SS and Sßthalassemia0)

• 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

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• Mometasone Furoate
inhaled cortico-steroid (ICS) with a dosage of 220mcg once daily for 16 weeks
• Placebo
placebo training inhaler with the same instructions as the experimental group.

Locations

Country	Name	City	State
United States	Icahn School of Medicine at Mount Sinai	New York	New York

Sponsors (2)

Lead Sponsor	Collaborator
Jeffrey Glassberg	National Heart, Lung, and Blood Institute (NHLBI)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants Who Completed Follow up	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.	at 2 years
Secondary	Change in Exhaled Nitric Oxide (eNO)	Change in effects of inhaled corticosteroids (ICS) as measured by exhaled nitric oxide levels, which is the primary marker of pulmonary inflammation.	Before ICS therapy begins and at 8 weeks post enrollment
Secondary	Change in Soluble Vascular Cell Adhesion Molecule (sVCAM) Level	Mean Change in effects of inhaled corticosteroids vascular injury, assessed by biomarker sVCAM as a surrogate for vascular injury.	Before ICS therapy begins and at 8 weeks post enrollment
Secondary	Change in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me)	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.	baseline and week 20
Secondary	The Medication Adherence Report Scale	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	20 weeks
Secondary	Change in the Numerical Rating Scale (NRS) for Pain	Mean change in patient reported pain NRS score, full scale range 0- 10, higher score indicate more pain	baseline and 20 weeks
Secondary	Asthma Control Test	Asthma control test, total score from 0-25, with higher score indicating more symptoms	8 weeks
Secondary	Admissions or Visits to the Hospital	Number of times participant visited the Emergency Department (ED) or was admitted to the hospital	baseline through 8 weeks
Secondary	Change in Reticulocytes Count	Mean change in reticulocytes count - the number of new red blood cells.	baseline and 8 weeks
Secondary	Change in FEV1/FVC	Mean change in FEV1/FVC at 8 weeks compared to baseline	baseline and 8 weeks