Sickle Cell DiseasePhase 2IMPROVENCT02061202
Inhaled Mometasone to Reduce Painful Episodes in Patients With Sickle Cell Disease
A Phase 2 study testing Mometasone Furoate in people with Sickle Cell Disease, run by Jeffrey Glassberg at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Mometasone Furoate
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 54 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2014to Nov 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age 15 or older
- Sever SCD phenotypes (Hb SS and Sβthalassemia0)
- A positive response to cough/wheeze questions
Who cannot join
- 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 locations and contacts
1 site in 1 country.
United States1 site
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
About this study
Official title: Inhaled Mometasone to Promote Reduction in Vasoocclusive Events
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
- Being tested
- Mometasone Furoate, Placebo
- Keywords
- Sickle Cell Disease, Pain Crisis, Painful Crisis, Sickle Pain
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mometasone FuroateDrug
inhaled cortico-steroid (ICS) with a dosage of 220mcg once daily for 16 weeks
PlaceboDrug
placebo training inhaler with the same instructions as the experimental group.
| Study group | Role | What they receive |
|---|---|---|
| Mometasone Furoate | Experimental | 1 puff daily (220mcg) for 16 weeks Receives: Drug: Mometasone Furoate |
| Placebo | Placebo comparator | 1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo). Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: at 2 years
Secondary outcomes (9)
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.
Measured: Before ICS therapy begins and at 8 weeks post enrollment
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.
Measured: 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.
Measured: 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
Measured: 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
Measured: baseline and 20 weeks
Asthma Control Test
Asthma control test, total score from 0-25, with higher score indicating more symptoms
Measured: 8 weeks
Admissions or Visits to the Hospital
Number of times participant visited the Emergency Department (ED) or was admitted to the hospital
Measured: baseline through 8 weeks
Change in Reticulocytes Count
Mean change in reticulocytes count - the number of new red blood cells.
Measured: baseline and 8 weeks
Change in FEV1/FVC
Mean change in FEV1/FVC at 8 weeks compared to baseline
Measured: baseline and 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2014 (estimated)
- Study startMar 2014
- Primary completionNov 2017
- Study completionNov 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 54 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jeffrey Glassberg
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor-investigator, Jeffrey Glassberg, Icahn School of Medicine at Mount Sinai
- Investigators
- Jeffrey Glassberg, MD, MA, Principal investigator, Icahn School of Medicine at Mount Sinai
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02061202
- Sponsor's protocol ID
- GCO 12-1565
- Other IDs
- K23HL119351
- Acronym
- IMPROVE
- Status last verified
- Mar 2019
- First submitted
- 14 Jan 2014
- Last updated
- 11 Mar 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 30783732Glassberg 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.
PubMed 28370266
Study at a glance
From the registry record, last updated by the sponsor on 11 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Inhaled Mometasone to Promote Reduction in Vasoocclusive Events
- ClinicalTrials.gov ID
- NCT02061202
- Acronym / sponsor ID
- IMPROVE / GCO 12-1565
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Disease, Pain Crisis, Painful Crisis, Sickle Pain
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 54 participants (actual)
- Lead sponsor
- Jeffrey GlassbergUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Mar 2014
- Primary completion
- Nov 2017 (actual)
- Study completion
- Nov 2017 (actual)
- First posted
- 12 Feb 2014 (estimated)
- Results
- Posted 11 Mar 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mometasone Furoate | Experimental | Drug: Mometasone Furoate1 puff daily (220mcg) for 16 weeks |
| Placebo | Placebo comparator | Drug: Placebo1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo). |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Age 15 or older
- Sever SCD phenotypes (Hb SS and Sβthalassemia0)
- A positive response to cough/wheeze questions
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mometasone Furoateinhaled cortico-steroid (ICS) with a dosage of 220mcg once daily for 16 weeks | Drug | Mometasone Furoate |
| Placeboplacebo training inhaler with the same instructions as the experimental group. | Drug | Placebo |
Primary outcome
Number of Participants Who Completed Follow up
Time frame: 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.
9 secondary outcomes
Change in Exhaled Nitric Oxide (eNO)
Time frame: Before ICS therapy begins and at 8 weeks post enrollment
Change in Soluble Vascular Cell Adhesion Molecule (sVCAM) Level
Time frame: Before ICS therapy begins and at 8 weeks post enrollment
Change in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me)
Time frame: baseline and week 20
The Medication Adherence Report Scale
Time frame: 20 weeks
Change in the Numerical Rating Scale (NRS) for Pain
Time frame: baseline and 20 weeks
Asthma Control Test
Time frame: 8 weeks
Admissions or Visits to the Hospital
Time frame: baseline through 8 weeks
Change in Reticulocytes Count
Time frame: baseline and 8 weeks
Change in FEV1/FVC
Time frame: baseline and 8 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Mar 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 30783732Langer 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.
- Derived from this study: PubMed 28370266Glassberg 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jeffrey Glassberg, MD, MA principal investigator
Icahn School of Medicine at Mount Sinai
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Mometasone Furoate in people with Sickle Cell Disease, run by Jeffrey Glassberg at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Mar 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Who Completed Follow up (time frame: at 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02061202, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Mar 2019.