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| ID | Type | Description | Link |
|---|---|---|---|
| K23HL119351 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Heart, Lung, and Blood Institute (NHLBI) | NIH |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Mometasone Furoate | Experimental | 1 puff daily (220mcg) for 16 weeks |
|
| Placebo | Placebo Comparator | 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Mometasone Furoate | Drug | inhaled cortico-steroid (ICS) with a dosage of 220mcg once daily for 16 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Exhaled Nitric Oxide (eNO) | Change in effects of inhaled corticosteroids (ICS) as measured by exhaled nitric oxide levels, which is the primary marker of pulmonary inflammation. | Before ICS therapy begins and at 8 weeks post enrollment |
| Change in Soluble Vascular Cell Adhesion Molecule (sVCAM) Level |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jeffrey Glassberg, MD, MA | Icahn School of Medicine at Mount Sinai | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30783732 | Derived | 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. | |
| 28370266 | 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. |
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Recruitment began in February 2014, with first enrollment in March 2014, and last enrollment in October 2016
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| ID | Title | Description |
|---|---|---|
| FG000 | Mometasone Furoate | 1 puff daily (220mcg) for 16 weeks |
| FG001 | Placebo | 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo) |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
1 participant in each group was lost to follow up prior to data collection and excluded from analyses.
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| ID | Title | Description |
|---|---|---|
| BG000 | Mometasone Furoate | 1 puff daily (220mcg) for 16 weeks |
| BG001 | Placebo | 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo) |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Count of Participants | Participants | at 2 years |
|
2 years
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Mometasone Furoate | 1 puff daily (220mcg) for 16 weeks | 0 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hoarseness of voice | Respiratory, thoracic and mediastinal disorders | CTCAE (Unspecified) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Jeffrey Glassberg | Icahn School of Medicine at Mount Sinai | 212-241-3650 | jeffrey.glassberg@mountsinai.org |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Nov 18, 2015 | Oct 10, 2018 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D000068656 | Mometasone Furoate |
| ID | Term |
|---|---|
| D011244 | Pregnadienediols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 |
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| Placebo | Drug | placebo training inhaler with the same instructions as the experimental group. |
|
Mean Change in effects of inhaled corticosteroids vascular injury, assessed by biomarker sVCAM as a surrogate for vascular injury. |
| Before ICS therapy begins and at 8 weeks post enrollment |
| Change in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) | 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. | baseline and week 20 |
| The Medication Adherence Report Scale | 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 | 20 weeks |
| Change in the Numerical Rating Scale (NRS) for Pain | Mean change in patient reported pain NRS score, full scale range 0- 10, higher score indicate more pain | baseline and 20 weeks |
| Asthma Control Test | Asthma control test, total score from 0-25, with higher score indicating more symptoms | 8 weeks |
| Admissions or Visits to the Hospital | Number of times participant visited the Emergency Department (ED) or was admitted to the hospital | baseline through 8 weeks |
| Change in Reticulocytes Count | Mean change in reticulocytes count - the number of new red blood cells. | baseline and 8 weeks |
| Change in FEV1/FVC | Mean change in FEV1/FVC at 8 weeks compared to baseline | baseline and 8 weeks |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants |
|
| Units | Counts |
|---|
| Participants |
|
|
| Secondary | Change in Exhaled Nitric Oxide (eNO) | Change in effects of inhaled corticosteroids (ICS) as measured by exhaled nitric oxide levels, which is the primary marker of pulmonary inflammation. | Posted | Mean | 95% Confidence Interval | ppb | Before ICS therapy begins and at 8 weeks post enrollment |
|
|
|
| Secondary | Change in Soluble Vascular Cell Adhesion Molecule (sVCAM) Level | Mean Change in effects of inhaled corticosteroids vascular injury, assessed by biomarker sVCAM as a surrogate for vascular injury. | Posted | Mean | Standard Deviation | ng/mL | Before ICS therapy begins and at 8 weeks post enrollment |
|
|
|
| Secondary | Change in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) | 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. | Posted | Mean | Standard Deviation | score on a scale | baseline and week 20 |
|
|
|
| Secondary | The Medication Adherence Report Scale | 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 | Posted | Mean | Standard Deviation | score on a scale | 20 weeks |
|
|
|
| Secondary | Change in the Numerical Rating Scale (NRS) for Pain | Mean change in patient reported pain NRS score, full scale range 0- 10, higher score indicate more pain | Posted | Mean | Standard Deviation | score on a scale | baseline and 20 weeks |
|
|
|
| Secondary | Asthma Control Test | Asthma control test, total score from 0-25, with higher score indicating more symptoms | Posted | Mean | Standard Deviation | score on a scale | 8 weeks |
|
|
|
| Secondary | Admissions or Visits to the Hospital | Number of times participant visited the Emergency Department (ED) or was admitted to the hospital | Posted | Mean | Standard Deviation | Events | baseline through 8 weeks |
|
|
|
| Secondary | Change in Reticulocytes Count | Mean change in reticulocytes count - the number of new red blood cells. | Posted | Mean | Standard Deviation | 10^3 cells/μL | baseline and 8 weeks |
|
|
|
| Secondary | Change in FEV1/FVC | Mean change in FEV1/FVC at 8 weeks compared to baseline | Posted | Mean | Standard Deviation | ratio | baseline and 8 weeks |
|
|
|
| 35 |
| 0 |
| 35 |
| 15 |
| 35 |
| EG001 | Placebo | 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo) | 0 | 17 | 0 | 17 | 5 | 17 |
| Thrush | Respiratory, thoracic and mediastinal disorders | CTCAE (Unspecified) | Systematic Assessment |
|
| Sore Throat | Respiratory, thoracic and mediastinal disorders | CTCAE (Unspecified) | Systematic Assessment |
|
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| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| Admissions |
|