Trial Outcomes & Findings for Inhaled Mometasone to Reduce Painful Episodes in Patients With Sickle Cell Disease (NCT NCT02061202)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

54 participants

Primary outcome timeframe

at 2 years

Results posted on

2019-03-11

Participant Flow

Recruitment began in February 2014, with first enrollment in March 2014, and last enrollment in October 2016

Participant milestones

Participant milestones
Measure	Mometasone Furoate 1 puff daily (220mcg) for 16 weeks	Placebo 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
Overall Study STARTED	36	18
Overall Study COMPLETED	35	17
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Mometasone Furoate 1 puff daily (220mcg) for 16 weeks	Placebo 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
Overall Study Lost to Follow-up	1	1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Mometasone Furoate n=35 Participants 1 puff daily (220mcg) for 16 weeks	Placebo n=17 Participants 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)	Total n=52 Participants Total of all reporting groups
Age, Continuous	30 years STANDARD_DEVIATION 8.56 • n=35 Participants	36 years STANDARD_DEVIATION 9.81 • n=17 Participants	32 years STANDARD_DEVIATION 9.28 • n=52 Participants
Sex: Female, Male Female	18 Participants n=35 Participants	6 Participants n=17 Participants	24 Participants n=52 Participants
Sex: Female, Male Male	17 Participants n=35 Participants	11 Participants n=17 Participants	28 Participants n=52 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Mometasone Furoate n=36 Participants 1 puff daily (220mcg) for 16 weeks	Placebo n=18 Participants 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
Number of Participants Who Completed Follow up	35 Participants	17 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Mometasone Furoate n=35 Participants 1 puff daily (220mcg) for 16 weeks	Placebo n=17 Participants 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
Change in Exhaled Nitric Oxide (eNO)	0.63 ppb Interval -2.51 to 3.77	2.71 ppb Interval 0.48 to 5.9

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Mometasone Furoate n=35 Participants 1 puff daily (220mcg) for 16 weeks	Placebo n=17 Participants 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
Change in Soluble Vascular Cell Adhesion Molecule (sVCAM) Level	-182.47 ng/mL Standard Deviation 785.21	170.25 ng/mL Standard Deviation 182.39

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Mometasone Furoate n=35 Participants 1 puff daily (220mcg) for 16 weeks	Placebo n=17 Participants 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
Change in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me)	2.8 score on a scale Standard Deviation 22.7	6.9 score on a scale Standard Deviation 24.0

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	Mometasone Furoate n=35 Participants 1 puff daily (220mcg) for 16 weeks	Placebo n=17 Participants 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
The Medication Adherence Report Scale	17.7 score on a scale Standard Deviation 2.25	17.1 score on a scale Standard Deviation 1.78

SECONDARY outcome

Timeframe: baseline and 20 weeks

Mean change in patient reported pain NRS score, full scale range 0- 10, higher score indicate more pain

Outcome measures

Outcome measures
Measure	Mometasone Furoate n=35 Participants 1 puff daily (220mcg) for 16 weeks	Placebo n=17 Participants 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
Change in the Numerical Rating Scale (NRS) for Pain	2.09 score on a scale Standard Deviation 2.55	2.82 score on a scale Standard Deviation 2.21

SECONDARY outcome

Timeframe: 8 weeks

Asthma control test, total score from 0-25, with higher score indicating more symptoms

Outcome measures

Outcome measures
Measure	Mometasone Furoate n=35 Participants 1 puff daily (220mcg) for 16 weeks	Placebo n=17 Participants 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
Asthma Control Test	17.7 score on a scale Standard Deviation 2.25	17.1 score on a scale Standard Deviation 1.78

SECONDARY outcome

Timeframe: baseline through 8 weeks

Number of times participant visited the Emergency Department (ED) or was admitted to the hospital

Outcome measures

Outcome measures
Measure	Mometasone Furoate n=35 Participants 1 puff daily (220mcg) for 16 weeks	Placebo n=17 Participants 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
Admissions or Visits to the Hospital ED visits	0.97 Events Standard Deviation 1.52	1.12 Events Standard Deviation 1.87
Admissions or Visits to the Hospital Observation admits	0.37 Events Standard Deviation 0.77	0.59 Events Standard Deviation 1.18
Admissions or Visits to the Hospital Admissions	0.37 Events Standard Deviation 0.77	0.47 Events Standard Deviation 1.23

SECONDARY outcome

Timeframe: baseline and 8 weeks

Mean change in reticulocytes count - the number of new red blood cells.

Outcome measures

Outcome measures
Measure	Mometasone Furoate n=35 Participants 1 puff daily (220mcg) for 16 weeks	Placebo n=17 Participants 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
Change in Reticulocytes Count	-0.15 10^3 cells/μL Standard Deviation 0.39	0.07 10^3 cells/μL Standard Deviation 0.52

SECONDARY outcome

Timeframe: baseline and 8 weeks

Mean change in FEV1/FVC at 8 weeks compared to baseline

Outcome measures

Outcome measures
Measure	Mometasone Furoate n=35 Participants 1 puff daily (220mcg) for 16 weeks	Placebo n=17 Participants 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
Change in FEV1/FVC	-0.71 ratio Standard Deviation 3.42	-1.41 ratio Standard Deviation 3.39

Adverse Events

Mometasone Furoate

Serious events: 0 serious events

Other events: 15 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Mometasone Furoate n=35 participants at risk 1 puff daily (220mcg) for 16 weeks	Placebo n=17 participants at risk 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo)
Respiratory, thoracic and mediastinal disorders Hoarseness of voice	42.9% 15/35 • 2 years	23.5% 4/17 • 2 years
Respiratory, thoracic and mediastinal disorders Thrush	5.7% 2/35 • 2 years	0.00% 0/17 • 2 years
Respiratory, thoracic and mediastinal disorders Sore Throat	42.9% 15/35 • 2 years	29.4% 5/17 • 2 years

Additional Information

Dr. Jeffrey Glassberg

Icahn School of Medicine at Mount Sinai

Phone: 212-241-3650

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place