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Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01960413
Recruitment Status : Completed
First Posted : October 10, 2013
Results First Posted : March 26, 2019
Last Update Posted : March 26, 2019
Sponsor:
Collaborators:
Medical College of Wisconsin
Versiti
Information provided by (Responsible Party):
Michael DeBaun, Vanderbilt University Medical Center

Study Description
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Brief Summary:
In this feasibility trial, the investigators will compare participants treated with montelukast and hydroxyurea to those treated with placebo and hydroxyurea for a total of 8 weeks.

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

Detailed Description:

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 (SCD):

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 46 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arms and Interventions
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Arm Intervention/treatment
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


Outcome Measures
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Primary Outcome Measures :
  1. Change in Soluble Vascular Cell Adhesion Molecule-1 (sVCAM) [ Time Frame: baseline to 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.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 1)Diagnosis of HbSS, or HbSβ-thalassemia0, confirmed by hemoglobin analysis
  • 2)Males and females age 16 years to 70 years old
  • 3)Greater than 2 episodes of pain in the last 12 months
  • 4)On a stable dose of hydroxyurea for at least 2 months and a stable hemoglobin

Exclusion Criteria:

  1. Judged not likely to be study compliant by his/her hematologist
  2. History of adverse reaction to montelukast or any of the components of montelukast
  3. Have used medications known to interact with montelukast such as rifampin, phenobarbital, and gemfibrozil within 4 weeks of enrollment
  4. Currently being treated with a leukotriene antagonist (montelukast or zileuton) or have used montelukast/zileuton within the last 60 days
  5. Chronic blood transfusion therapy defined as regularly scheduled transfusions.
  6. Hemoglobin A greater than15% on hemoglobin analysis
  7. Individuals with a current physician diagnosis of asthma (within last 12 months) or requires continuous supplemental oxygen, or predicted or current use of asthma medications (inhaled corticosteroids, but participants taking bronchodilators will be allowed to participate).
  8. Current participation in another therapeutic trial for SCD
  9. Known current pregnancy
  10. Known history of HIV
  11. Serum creatinine greater than 3 times the site's upper limit of normal
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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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
Sponsors and Collaborators
Vanderbilt University Medical Center
Medical College of Wisconsin
Versiti
Investigators
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Principal Investigator: Michael R. DeBaun, MD, MPH Vanderbilt University Medical Center
Principal Investigator: Joshua Field, MD, MS Medical College of Wisconsin
  Study Documents (Full-Text)

Documents provided by Michael DeBaun, Vanderbilt University Medical Center:
Study Protocol and Statistical Analysis Plan  [PDF] October 10, 2016

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Michael DeBaun, Professor of Pediatrics and Medicine, JC Peterson Endowed Chair in Pediatrics, Vice Chair for Clinical Research in Pediatrics, Director, Vanderbilt-Meharry-Matthew Walker Center for Excellence in Sickle Cell Disease, Vanderbilt University Medical Center
ClinicalTrials.gov Identifier: NCT01960413    
Other Study ID Numbers: R01FD004117 ( U.S. FDA Grant/Contract )
First Posted: October 10, 2013    Key Record Dates
Results First Posted: March 26, 2019
Last Update Posted: March 26, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Individual participant data that underlie the results reported in this article, after de-identification (text, tables, figures, and appendices)
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Time Frame: Beginning 9 months and ending 36 months following article publication
Access Criteria: Investigators whose proposed use of the data has been approved by an independent review committee ("learned intermediary") identified for this purpose.
Additional relevant MeSH terms:
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Anemia
Thalassemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Montelukast
Anti-Asthmatic Agents
Respiratory System Agents
 Leukotriene Antagonists
Hormone Antagonists
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Cytochrome P-450 CYP1A2 Inducers
Cytochrome P-450 Enzyme Inducers
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Nucleic Acid Synthesis Inhibitors