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

NCT02061202 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: GCO 12-1565K23HL119351
• Study Type: interventional
• Target: 54
• Locations: 1 country
• Sites: 1

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.

Conditions

Sickle Cell Disease

Keywords

Sickle Cell Disease; Pain Crisis; Painful Crisis; Sickle Pain

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (9)

• Change in Exhaled Nitric Oxide (eNO)

  Before ICS therapy begins and at 8 weeks post enrollment

• Change in Soluble Vascular Cell Adhesion Molecule (sVCAM) Level

  Before ICS therapy begins and at 8 weeks post enrollment

• Change in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me)

  baseline and week 20

• The Medication Adherence Report Scale

  20 weeks

• Change in the Numerical Rating Scale (NRS) for Pain

  baseline and 20 weeks

Study Arms (2)

Mometasone Furoate

EXPERIMENTAL

1 puff daily (220mcg) for 16 weeks

Drug: Mometasone Furoate

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

Mometasone Furoate DRUG

inhaled cortico-steroid (ICS) with a dosage of 220mcg once daily for 16 weeks

Mometasone Furoate

Placebo DRUG

placebo training inhaler with the same instructions as the experimental group.

Placebo

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 15 or older
• Sever SCD phenotypes (Hb SS and Sβthalassemia0)
• A positive response to cough/wheeze questions

You may not qualify if:

• 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

Sponsors & Collaborators

• Jeffrey Glassberg: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (1)

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

Related Publications (2)

• Langer AL, Leader A, Kim-Schulze S, Ginzburg Y, Merad M, Glassberg J. Inhaled steroids associated with decreased macrophage markers in nonasthmatic individuals with sickle cell disease in a randomized trial. Ann Hematol. 2019 Apr;98(4):841-849. doi: 10.1007/s00277-019-03635-9. Epub 2019 Feb 20.

  PMID: 30783732 DERIVED

• Glassberg J, Minnitti C, Cromwell C, Cytryn L, Kraus T, Skloot GS, Connor JT, Rahman AH, Meurer WJ. Inhaled steroids reduce pain and sVCAM levels in individuals with sickle cell disease: A triple-blind, randomized trial. Am J Hematol. 2017 Jul;92(7):622-631. doi: 10.1002/ajh.24742. Epub 2017 Jun 5.

  PMID: 28370266 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Mometasone Furoate

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Results Point of Contact

• Title: Dr. Jeffrey Glassberg
• Organization: Icahn School of Medicine at Mount Sinai

jeffrey.glassberg@mountsinai.org

212-241-3650

Study Officials

• Jeffrey Glassberg, MD, MA

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: January 14, 2014
• First Posted: February 12, 2014
• Study Start: March 1, 2014
• Primary Completion: November 1, 2017
• Study Completion: November 1, 2017
• Last Updated: March 11, 2019
• Results First Posted: March 11, 2019

Record last verified: 2019-03

Locations

US (1)