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

Phase 2

Completed

Conditions: Sickle Cell Disease

Interventions: Drug: Placebo
Drug: Mometasone Furoate

Registration Number: NCT02061202

Lead Sponsor: Jeffrey Glassberg

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.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 54

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 & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo).
Mometasone Furoate	Mometasone Furoate	1 puff daily (220mcg) for 16 weeks

Primary Outcome Measures

Name	Time	Method
Number of Participants Who Completed Follow up	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 Outcome Measures

Name	Time	Method
Change in Exhaled Nitric Oxide (eNO)	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.
Change in Soluble Vascular Cell Adhesion Molecule (sVCAM) Level	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.
Change in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me)	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.
The Medication Adherence Report Scale	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
Change in the Numerical Rating Scale (NRS) for Pain	baseline and 20 weeks	Mean change in patient reported pain NRS score, full scale range 0- 10, higher score indicate more pain
Asthma Control Test	8 weeks	Asthma control test, total score from 0-25, with higher score indicating more symptoms
Admissions or Visits to the Hospital	baseline through 8 weeks	Number of times participant visited the Emergency Department (ED) or was admitted to the hospital
Change in Reticulocytes Count	baseline and 8 weeks	Mean change in reticulocytes count - the number of new red blood cells.
Change in FEV1/FVC	baseline and 8 weeks	Mean change in FEV1/FVC at 8 weeks compared to baseline