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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

46 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-11-30

Study Completion Date

2018-03-31

Brief Summary

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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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Detailed Description

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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

Conditions

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Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Montelukast added to Hydroxyurea

Oral montelukast therapy taken daily for eight weeks with current hydroxyurea regiment

Group Type EXPERIMENTAL

Montelukast added to Hydroxyurea

Intervention Type DRUG

Placebo added to Hydroxyurea

Oral placebo taken daily for eight weeks with current hydroxyurea regiment

Group Type PLACEBO_COMPARATOR

Placebo added to Hydroxyurea

Intervention Type DRUG

Interventions

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Montelukast added to Hydroxyurea

Intervention Type DRUG

Placebo added to Hydroxyurea

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

16 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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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

Responsibility Role PRINCIPAL_INVESTIGATOR