Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease
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|ClinicalTrials.gov Identifier: NCT02960503|
Recruitment Status : Withdrawn (Key personnel moved to new location. Therefore, we had insufficient resources to initiate the trial.)
First Posted : November 9, 2016
Last Update Posted : October 2, 2018
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: Azithromycin Other: Placebo||Phase 1 Phase 2|
Specific aim 1: To determine acceptability of a clinical trial in which participants are randomly allocated to either a placebo or azithromycin 500 mg 3 days a week for 6 months for adults with SCD who have FEV1<80%.
To assess acceptability of this intervention, investigators will measurement recruitment rate, retention and adherence to the study medication. Recruitment will be assessed by proportion of eligible participants that agree to be randomized. Retention will be measured as proportion randomly allocated who complete the trial. Dropout due to toxicity will be categorized using a questionnaire. Medication adherence will be assessed using the previously validated 8 item modified Morisky medication adherence scale (MMAS-8), where responses are categorized: high adherence (8 points), average adherence (6-7 points), and poor adherence (0-5 points). If recruitment rate is < 60%, the retention rate < 80%, or average adherence rate is ≤5 points, the original protocol will be examined and alternative strategies to enhance recruitment, retention, and adherence will be considered.
Specific aim 2: To evaluate the effect of 6 months of low dose azithromycin therapy on FEV1 and respiratory symptoms in patients with SCA. Baseline FEV1 testing with a portable, in-office spirometer will be completed at study enrollment and at the end of the study period (6 months). The previously validated American Thoracic Society (ATS-DLD-78 for adults) questionnaire will also be used to evaluate respiratory symptoms at baseline and end of the study. Under a separate protocol, investigators will calculate the coefficient of variation for FEV1% predicted in adults with sickle cell disease in order to define the within-subject variability for tests of respiratory function in this specific population, which has not been previously described within the medical literature. Calculation of the coefficient of variation for FEV1 % predicted will be essential for the interpretation of clinically and statistically meaningful changes in spirometry for participants who are treated with azithromycin to improve their baseline pulmonary function when compared to controls.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease: a Feasibility Trial|
|Estimated Study Start Date :||September 2016|
|Estimated Primary Completion Date :||October 2018|
|Estimated Study Completion Date :||October 2018|
Active Comparator: Treatment arm (azithromycin)
Treatment arm: azithromycin 500 mg three times a week for 6 months
Study participants will be randomized to the treatment arm (azithromycin 500 mg three times a week for 6 months).
Other Name: Zithromax, zmax, z-pack
Placebo Comparator: Placebo arm
Control arm: placebo three times a week for 6 months
Study participants will be randomized to the control arm (placebo three times a week for 6 months).
- Acceptability of the trial will be assessed by the modified Morisky Medication Adherence Scale (MMAS - 8) [ Time Frame: 6 months ]Acceptability of the trial will be measured based on three outcomes: recruitment, retention, and adherence rates to therapy. Retention defined as the number of participants that complete the entire study. Recruitment defined as the number of eligible participants that elect to consent to continue with study evaluations. Adherence rate measured based on MMAS score, which was previously validated in children with sickle cell disease (SCD) and scored as follows: high adherence (8 points), average adherence (6 to 7 points) and poor adherence (0 - 5 points).
- Change in FEV1 % predicted in response to 6 months of low dose azithromycin therapy [ Time Frame: 6 months ]
- Change in respiratory symptom score (by ATS-DLD-78) in response to 6 months of low dose azithromycin therapy [ Time Frame: 6 months ]
- Change in quality of life (by SF-36) in response to 6 months of low dose azithromycin therapy [ Time Frame: 6 months ]
- Number of participants with treatment-related adverse events [ Time Frame: 6 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02960503
|United States, Tennessee|
|Vanderbilt University Medical Center|
|Nashville, Tennessee, United States, 37203|
|Principal Investigator:||Michael R. DeBaun, M.D., M.P.H.||Vanderbilt University Medical Center|