Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia
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|ClinicalTrials.gov Identifier: NCT01960413|
Recruitment Status : Completed
First Posted : October 10, 2013
Last Update Posted : September 5, 2018
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)||Drug: Montelukast added to Hydroxyurea Drug: Placebo added to Hydroxyurea||Phase 2|
The primary hypothesis for this trial is that montelukast adds efficacy to hydroxyurea therapy for improving vaso-occlusion when compared to hydroxyurea alone. The following specific aims will be tested in adolescents and adults with sickle cell disease:
Aim 1. To determine whether montelukast versus placebo added to hydroxyurea will improve markers of vaso-occlusion-associated tissue injury in adolescents and adults with sickle cell disease.
Aim 2. To evaluate physiologic effects of montelukast versus placebo added to hydroxyurea in adolescents and adults with sickle cell disease.
Subaim 2A. To determine if montelukast versus placebo added to hydroxyurea will improve lung function in adolescents and adults with sickle cell disease.
Subaim 2B. To determine if montelukast versus placebo added to hydroxyurea will improve forearm microvascular blood flow in adolescents and adults with sickle cell disease, respectively.
Funding Source - FDA OOPD
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||46 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia (Also Known as the Montelukast Trial in Sickle Cell Anemia)|
|Study Start Date :||November 2013|
|Actual Primary Completion Date :||March 2018|
|Actual Study Completion Date :||March 2018|
Experimental: Montelukast added to Hydroxyurea
Oral montelukast therapy taken daily for eight weeks with current hydroxyurea regiment
Drug: Montelukast added to Hydroxyurea
Placebo Comparator: Placebo added to Hydroxyurea
Oral placebo taken daily for eight weeks with current hydroxyurea regiment
Drug: Placebo added to Hydroxyurea
- Improvement in soluble vascular cell adhesion molecule-1 (sVCAM) [ Time Frame: Eight weeks ]The primary outcome measure is based on a 30% reduction, which would be ~106 ng/ml reduction. The study was designed with 25 in each group in order to explore all three aims and potential confounders. However, if the investigators are not able to accrue 25 subjects in each arm, the investigators would still be able to detect a 30% difference in sVCAM with 17 subjects in each group. The 95% confidence interval for detecting a 30% difference is between 204 ng/ml and 290 ng/ml (or an 18-42% reduction in sVCAM). Importantly, the lower limit of the 95% confidence interval (18%) is still a clinically relevant reduction in sVCAM. Thus, if the investigators detect a 30% or larger difference in sVCAM in this study, the investigators will be assured that, based on the 95% confidence interval, these data are clinically important.
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): NCT01960413
|United States, Tennessee|
|Vanderbilt University Medical Center|
|Nashville, Tennessee, United States, 37232-9000|
|United States, Wisconsin|
|Medical College of Wisconsin|
|Milwaukee, Wisconsin, United States, 53226|
|Principal Investigator:||Michael R. DeBaun, MD, MPH||Vanderbilt University Medical Center|
|Principal Investigator:||Joshua Field, MD, MS||Medical College of Wisconsin|