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Completed

NCT Number: NCT01960413

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

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.

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Key information

Conditions

Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0) Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

16 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Vanderbilt University Medical Center, Nashville, Tennessee, United States

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

  • Judged not likely to be study compliant by his/her hematologist
  • History of adverse reaction to montelukast or any of the components of montelukast
  • Have used medications known to interact with montelukast such as rifampin, phenobarbital, and gemfibrozil within 4 weeks of enrollment
  • Currently being treated with a leukotriene antagonist (montelukast or zileuton) or have used montelukast/zileuton within the last 60 days
  • Chronic blood transfusion therapy defined as regularly scheduled transfusions.
  • Hemoglobin A greater than15% on hemoglobin analysis
  • 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).
  • Current participation in another therapeutic trial for SCD
  • Known current pregnancy
  • Known history of HIV
  • Serum creatinine greater than 3 times the site's upper limit of normal

Treatment and study plan

Montelukast added to Hydroxyurea

Drug

Placebo added to Hydroxyurea

Drug

Primary outcomes

  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.

Sponsors and collaborators

Lead sponsor

Vanderbilt University Medical Center

Other

Collaborators

  • Medical College of Wisconsin
  • Versiti Blood Health

Registry information

Official study title

Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia (Also Known as the Montelukast Trial in Sickle Cell Anemia)

Important dates

Study start
2013
Primary completion
2018
Study completion
2018
First posted
Oct 10, 2013
Registry last updated
Mar 26, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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