Trial Outcomes & Findings for Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia (NCT NCT01960413)
NCT ID: NCT01960413
Last Updated: 2019-03-26
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
46 participants
Primary outcome timeframe
baseline to eight weeks
Results posted on
2019-03-26
Participant Flow
Participant milestones
| Measure |
Montelukast Added to Hydroxyurea
Oral montelukast therapy taken daily for eight weeks with current hydroxyurea regiment
Montelukast added to Hydroxyurea
|
Placebo Added to Hydroxyurea
Oral placebo taken daily for eight weeks with current hydroxyurea regiment
Placebo added to Hydroxyurea
|
|---|---|---|
|
Overall Study
STARTED
|
24
|
22
|
|
Overall Study
COMPLETED
|
20
|
21
|
|
Overall Study
NOT COMPLETED
|
4
|
1
|
Reasons for withdrawal
| Measure |
Montelukast Added to Hydroxyurea
Oral montelukast therapy taken daily for eight weeks with current hydroxyurea regiment
Montelukast added to Hydroxyurea
|
Placebo Added to Hydroxyurea
Oral placebo taken daily for eight weeks with current hydroxyurea regiment
Placebo added to Hydroxyurea
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
1
|
|
Overall Study
Physician Decision
|
1
|
0
|
|
Overall Study
Lost to Follow-up
|
2
|
0
|
Baseline Characteristics
Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia
Baseline characteristics by cohort
| Measure |
Montelukast Added to Hydroxyurea
n=24 Participants
Oral montelukast therapy taken daily for eight weeks with current hydroxyurea regiment
Montelukast added to Hydroxyurea
|
Placebo Added to Hydroxyurea
n=22 Participants
Oral placebo taken daily for eight weeks with current hydroxyurea regiment
Placebo added to Hydroxyurea
|
Total
n=46 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
29.25 years
STANDARD_DEVIATION 10.46 • n=99 Participants
|
30.76 years
STANDARD_DEVIATION 10.24 • n=107 Participants
|
29.95 years
STANDARD_DEVIATION 10.27 • n=206 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=99 Participants
|
14 Participants
n=107 Participants
|
27 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
19 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
23 Participants
n=99 Participants
|
22 Participants
n=107 Participants
|
45 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
24 Participants
n=99 Participants
|
18 Participants
n=107 Participants
|
42 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
24 participants
n=99 Participants
|
22 participants
n=107 Participants
|
46 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: baseline to eight weeksThe 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.
Outcome measures
| Measure |
Montelukast Added to Hydroxyurea
n=20 Participants
Oral montelukast therapy taken daily for eight weeks with current hydroxyurea regiment
Montelukast added to Hydroxyurea
|
Placebo Added to Hydroxyurea
n=21 Participants
Oral placebo taken daily for eight weeks with current hydroxyurea regiment
Placebo added to Hydroxyurea
|
|---|---|---|
|
Change in Soluble Vascular Cell Adhesion Molecule-1 (sVCAM)
|
-3.2 ng/mL
Interval -13.6 to 11.6
|
4.17 ng/mL
Interval -3.3 to 12.1
|
Adverse Events
Montelukast Added to Hydroxyurea
Serious events: 4 serious events
Other events: 10 other events
Deaths: 0 deaths
Placebo Added to Hydroxyurea
Serious events: 2 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Montelukast Added to Hydroxyurea
n=24 participants at risk
Oral montelukast therapy taken daily for eight weeks with current hydroxyurea regiment
Montelukast added to Hydroxyurea
|
Placebo Added to Hydroxyurea
n=22 participants at risk
Oral placebo taken daily for eight weeks with current hydroxyurea regiment
Placebo added to Hydroxyurea
|
|---|---|---|
|
Blood and lymphatic system disorders
Pain
|
8.3%
2/24 • Number of events 2 • 8 weeks
|
9.1%
2/22 • Number of events 2 • 8 weeks
|
|
Blood and lymphatic system disorders
Acute Chest Syndrome
|
8.3%
2/24 • Number of events 2 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
Other adverse events
| Measure |
Montelukast Added to Hydroxyurea
n=24 participants at risk
Oral montelukast therapy taken daily for eight weeks with current hydroxyurea regiment
Montelukast added to Hydroxyurea
|
Placebo Added to Hydroxyurea
n=22 participants at risk
Oral placebo taken daily for eight weeks with current hydroxyurea regiment
Placebo added to Hydroxyurea
|
|---|---|---|
|
Blood and lymphatic system disorders
Pain
|
12.5%
3/24 • Number of events 4 • 8 weeks
|
27.3%
6/22 • Number of events 9 • 8 weeks
|
|
Gastrointestinal disorders
diarrhea
|
4.2%
1/24 • Number of events 1 • 8 weeks
|
9.1%
2/22 • Number of events 3 • 8 weeks
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
0.00%
0/24 • 8 weeks
|
4.5%
1/22 • Number of events 1 • 8 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pain
|
4.2%
1/24 • Number of events 1 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
|
General disorders
headache
|
0.00%
0/24 • 8 weeks
|
4.5%
1/22 • Number of events 1 • 8 weeks
|
|
Ear and labyrinth disorders
ear injury
|
0.00%
0/24 • 8 weeks
|
4.5%
1/22 • Number of events 2 • 8 weeks
|
|
Eye disorders
scleral disorder
|
4.2%
1/24 • Number of events 1 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
|
Nervous system disorders
headache, migraine
|
0.00%
0/24 • 8 weeks
|
4.5%
1/22 • Number of events 2 • 8 weeks
|
|
Reproductive system and breast disorders
vaginal discharge
|
4.2%
1/24 • Number of events 1 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
|
Renal and urinary disorders
urinary frequency
|
4.2%
1/24 • Number of events 1 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
|
Infections and infestations
pharyngitis
|
4.2%
1/24 • Number of events 1 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
|
Infections and infestations
flu like symptoms
|
0.00%
0/24 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
|
Gastrointestinal disorders
stomach pain
|
4.2%
1/24 • Number of events 1 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
|
Reproductive system and breast disorders
penile pain/priapsm
|
4.2%
1/24 • Number of events 1 • 8 weeks
|
4.5%
1/22 • Number of events 1 • 8 weeks
|
|
Respiratory, thoracic and mediastinal disorders
sore throat
|
4.2%
1/24 • Number of events 1 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
|
Respiratory, thoracic and mediastinal disorders
nasal congestion
|
0.00%
0/24 • 8 weeks
|
4.5%
1/22 • Number of events 1 • 8 weeks
|
|
Respiratory, thoracic and mediastinal disorders
upper respiratory infection
|
8.3%
2/24 • Number of events 2 • 8 weeks
|
9.1%
2/22 • Number of events 2 • 8 weeks
|
|
Respiratory, thoracic and mediastinal disorders
dyspnea
|
4.2%
1/24 • Number of events 1 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
|
Respiratory, thoracic and mediastinal disorders
pain,dyspnea
|
4.2%
1/24 • Number of events 1 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
|
Cardiac disorders
systolic murmur
|
4.2%
1/24 • Number of events 1 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
|
Infections and infestations
Breast Infection
|
0.00%
0/24 • 8 weeks
|
4.5%
1/22 • Number of events 1 • 8 weeks
|
|
Injury, poisoning and procedural complications
Injury,laceration
|
0.00%
0/24 • 8 weeks
|
4.5%
1/22 • Number of events 1 • 8 weeks
|
|
Respiratory, thoracic and mediastinal disorders
productive cough
|
4.2%
1/24 • Number of events 2 • 8 weeks
|
0.00%
0/22 • 8 weeks
|
|
Injury, poisoning and procedural complications
Pain
|
12.5%
3/24 • Number of events 3 • 8 weeks
|
9.1%
2/22 • Number of events 2 • 8 weeks
|
Additional Information
Michael DeBaun, MD MPH
Vanderbilt University Medical Center
Phone: (615) 875-3040
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place