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

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

Sponsors

Lead Sponsor: Vanderbilt University Medical Center

Collaborator: Medical College of Wisconsin
Versiti

Source Vanderbilt University Medical Center
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.

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

Overall Status Completed
Start Date November 2013
Completion Date March 2018
Primary Completion Date March 2018
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Change in Soluble Vascular Cell Adhesion Molecule-1 (sVCAM) baseline to eight weeks
Enrollment 46
Condition
Intervention

Intervention Type: Drug

Intervention Name: Montelukast added to Hydroxyurea

Arm Group Label: Montelukast added to Hydroxyurea

Intervention Type: Drug

Intervention Name: Placebo added to Hydroxyurea

Arm Group Label: Placebo added to Hydroxyurea

Eligibility

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

Gender: All

Minimum Age: 16 Years

Maximum Age: 70 Years

Healthy Volunteers: No

Overall Official
Location
Facility:
Vanderbilt University Medical Center | Nashville, Tennessee, 37232-9000, United States
Medical College of Wisconsin | Milwaukee, Wisconsin, 53226, United States
Location Countries

United States

Verification Date

March 2019

Responsible Party

Type: Principal Investigator

Investigator Affiliation: Vanderbilt University Medical Center

Investigator Full Name: Michael DeBaun

Investigator Title: 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

Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Montelukast added to Hydroxyurea

Type: Experimental

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

Label: Placebo added to Hydroxyurea

Type: Placebo Comparator

Description: Oral placebo taken daily for eight weeks with current hydroxyurea regiment

Patient Data Yes
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Triple (Participant, Care Provider, Investigator)

Source: ClinicalTrials.gov