Inhaled Mometasone to Promote Reduction in Vasoocclusive Events 2

NCT03758950 | Completed Phase 2 DMC FDA Drug

• 2 other identifiers: GCO 17-1936R01HL142671
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 2

Brief Summary

The study team proposes a triple-blind, placebo-controlled, phase II clinical trial of once-daily inhaled mometasone for 48 weeks (with 4-week washout at study completion) in individuals with Sickle Cell Disease (SCD) who report episodic cough or wheeze (ECW) but do not have asthma. Patients will be recruited from and followed in SCD clinics at participating sites. The primary endpoint will be a reduction in sVCAM level of 20% or more in comparison to placebo.

Conditions

Sickle Cell Disease

Keywords

Sickle Cell; Inhaled Steroids

Outcome Measures

Primary Outcomes (1)

• Soluble Vascular Cell Adhesion Molecule (sVCAM) level

  Change in sVCAM level at one year compared to baseline. sVCAM is a biomarker used as a surrogate for red blood cell (RBC) adhesivity and overall disease severity in SCD.

  Baseline and one year

Secondary Outcomes (31)

• Reticulocyte Count level

  Baseline and one year

• Plasma Free Hemoglobin level

  Baseline and one year

• LDH level

  Baseline and one year

• Bilirubin (Direct and Indirect) level

  Baseline and one year

• Hemoglobin level

  Baseline and one year

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Once daily inhaled placebo

Drug: Placebo

Mometasone

ACTIVE COMPARATOR

Inhaled Mometasone Furoate 220 mcg DPI

Drug: Mometasone

Interventions

Placebo DRUG

Once daily inhaled placebo for 48 weeks

Placebo

Mometasone DRUG

Inhaled Mometasone for 48 weeks (with 4-week washout at study completion)

Also known as: Inhaled Mometasone

Mometasone

Eligibility Criteria

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

You may qualify if:

• Participants age 18 and older with severe SCD phenotypes (Hb SS and Sβthalassemia0):

You may not qualify if:

• Not currently having a painful crisis (as defined by validated pain diary questions)
• Do not have acute respiratory symptoms
• Report of recent ECW (answers "Yes" to any question in Box 1)
• Participant is already medically optimized (i.e. already on maximum dose hydroxyurea unless contraindicated and not undergoing medication titration).
• Participant screens positive for possible undiagnosed asthma (Box 2)
• Pregnant or planning to become pregnant
• \> 15 ED visits for SCD pain over the previous 12 months (due to concern for multi-factorial pain that may be less responsive to SCD therapies)
• Have been discharged from the hospital within the previous 7 days.

Sponsors & Collaborators

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

Study Sites (2)

Mount Sinai St Luke's

New York, New York, 10025, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

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

Study Officials

• Jeffrey Glassberg, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: November 28, 2018
• First Posted: November 29, 2018
• Study Start: November 29, 2018
• Primary Completion: May 31, 2023
• Study Completion: May 31, 2023
• Last Updated: June 18, 2023

Record last verified: 2023-06

Locations

US (2)