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

March 8, 2019 updated by: Jeffrey Glassberg

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

54

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • New York, New York, United States, 10029
        • Icahn School of Medicine at Mount Sinai

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

15 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Mometasone Furoate
1 puff daily (220mcg) for 16 weeks
Drug: Mometasone Furoate
inhaled cortico-steroid (ICS) with a dosage of 220mcg once daily for 16 weeks
Placebo Comparator: Placebo
1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo).
Drug: Placebo
placebo training inhaler with the same instructions as the experimental group.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
at 2 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Before ICS therapy begins and at 8 weeks post enrollment
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.
Before ICS therapy begins and at 8 weeks post enrollment
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.
baseline and week 20
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
20 weeks
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
baseline and 20 weeks
Asthma Control Test
Time Frame: 8 weeks
Asthma control test, total score from 0-25, with higher score indicating more symptoms
8 weeks
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
baseline through 8 weeks
Change in Reticulocytes Count
Time Frame: baseline and 8 weeks
Mean change in reticulocytes count - the number of new red blood cells.
baseline and 8 weeks
Change in FEV1/FVC
Time Frame: baseline and 8 weeks
Mean change in FEV1/FVC at 8 weeks compared to baseline
baseline and 8 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Jeffrey Glassberg

Collaborators

National Heart, Lung, and Blood Institute (NHLBI)

Investigators

  • Principal Investigator: Jeffrey Glassberg, MD, MA, Icahn School of Medicine at Mount Sinai

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2014

Primary Completion (Actual)

November 1, 2017

Study Completion (Actual)

November 1, 2017

Study Registration Dates

First Submitted

January 14, 2014

First Submitted That Met QC Criteria

February 11, 2014

First Posted (Estimate)

February 12, 2014

Study Record Updates

Last Update Posted (Actual)

March 11, 2019

Last Update Submitted That Met QC Criteria

March 8, 2019

Last Verified

March 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GCO 12-1565
  • K23HL119351 (U.S. NIH Grant/Contract)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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