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

Jeffrey Glassberg

Status and phase: Completed; Phase 2

Conditions: Sickle Cell Disease

Treatments: Drug: Placebo; Drug: Mometasone Furoate

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT02061202

K23HL119351 (U.S. NIH Grant/Contract)

GCO 12-1565

Details and patient eligibility

About

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.

Enrollment: 54 patients

Sex: All

Ages: 15+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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54 participants in 2 patient groups, including a placebo group

Mometasone Furoate

Experimental group

Description:

1 puff daily (220mcg) for 16 weeks

Treatment:

Drug: Mometasone Furoate

Placebo

Placebo Comparator group

Description:

1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo).

Treatment:

Drug: Placebo