Adults 18 to 74, any sex, with Eosinophilic Asthma or Asthma. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Change in Blood Absolute Eosinophil Count From Baseline to Week 12Primary· Baseline, 12 Weeks
The primary endpoint of this study was the change in AEC from Baseline to Week 12 on a ratio scale. The analysis used a mixed effects model repeated-measures (MMRM) with terms for log10 transformed baseline, GINA treatment step, treatment, visit, treatment by visit interaction, and log10 transformed baseline by visit interaction as fixed effects, and subject as a random effect. An unstructured covariance matrix was used. The response variable was the log10 transformed post-baseline value minus the log10 transformed baseline value. The estimates of Geometric LS Means and their ratios were obtai
Group
Value
95% CI
Placebo BID
0.8980
± 1.26
37.5 mg BID Dexpramipexole
0.4031
± 1.28
75 mg BID Dexpramipexole
0.3056
± 1.27
150 mg BID Dexpramipexole
0.2051
± 1.25
Change in Pre-bronchodilator FEV1 (Liters) From Baseline to Week 12Secondary· Baseline, 12 Weeks
FEV1 is defined as the amount of air that can be forcibly exhaled from the lungs in the first second of a forced exhalation.
Group
Value
95% CI
Placebo BID
0.0700
± 0.08329
37.5 mg BID Dexpramipexole
0.208
± 0.08387
75 mg BID Dexpramipexole
0.0557
± 0.07848
150 mg BID Dexpramipexole
0.247
± 0.07769
Combined 150 mg BID and 75 mg BID Arms
0.151
± 0.05746
Change in Asthma Control Questionnaire (ACQ-6) Score From Baseline to Week 12Secondary· Baseline, 12 Weeks
ACQ-6 is simple questionnaire to measure the adequacy of asthma control and change in asthma control which occurs either spontaneously or as a result of treatment. The 6-point self-administered scale has items measuring asthma symptoms and rescue inhaler use. The ACQ score is the mean of the questions and therefore between 0 (totally controlled) and 6 (severely uncontrolled). The original protocol planned to analyze the ACQ-7 score. As a result of FEV1 testing restrictions imposed on the study during the COVID-19 pandemic, the analysis was prospectively modified to the ACQ-6 score prior to dat
Group
Value
95% CI
Placebo BID
-0.391
± 0.1866
37.5 mg BID Dexpramipexole
-0.419
± 0.1974
75 mg BID Dexpramipexole
-0.437
± 0.1924
150 mg BID Dexpramipexole
-0.655
± 0.1803
Change in Post-bronchodilator FEV1 From Baseline to Week 12Secondary· Baseline, 12 Weeks
Post-bronchodilator FEV1 is defined as the amount of air that can be forcibly exhaled from the lungs in the first second of a forced exhalation, after treatment with inhaled albuterol.
Group
Value
95% CI
Placebo BID
-0.00546
± 0.07208
37.5 mg BID Dexpramipexole
0.0932
± 0.07455
75 mg BID Dexpramipexole
-0.000717
± 0.06977
150 mg BID Dexpramipexole
0.176
± 0.07182
Change in Quality of Life, as Measured by the Asthma Quality of Life Questionnaire (AQLQ) From Baseline to Week 12Secondary· Baseline, 12 Weeks
The AQLQ is a 32-item asthma specific questionnaire designed to measure functional impairments that are most important to patients with asthma. The 32 questions in the AQLQ are divided into four domains; activity limitations, symptoms, emotional function, and environmental stimuli. Individual questions are equally weighted. The overall AQLQ score is the mean of the responses to each of the 32 questions and ranges from 1 to 7. A score 7.0 indicates that the patient has no impairments due to asthma and score 1.0 indicates severe impairment.
Group
Value
95% CI
Placebo BID
0.376
± 0.1999
37.5 mg BID Dexpramipexole
0.531
± 0.2112
75 mg BID Dexpramipexole
0.312
± 0.2055
150 mg BID Dexpramipexole
0.584
± 0.1979
Number of Participants With Potentially Clinically Significant Hematology Results by Treatment Group Post Randomization Through Week 12Secondary· Immediately post-baseline up to Week 12
Number of Participants with Potentially Clinically Significant Hematology Results by Treatment Group. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
Eosinophils >1.6 x 10^9/L
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Basophils >1.6 x 10^9/L
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Erythrocytes ≤3.5 x 10^12/L
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
1
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
2
Erythrocytes ≥6.4 x 10^12/L
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Hematocrit ≤32% - Females
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
2
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
1
Hematocrit ≥54% - Females
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Hematocrit ≤37% - Males
Group
Value
95% CI
Placebo BID
2
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
1
150 mg BID Dexpramipexole
1
Hematocrit ≥60% - Males
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Number of Participants With Potentially Clinically Significant Blood Chemistry Results by Treatment Group Post Randomization Through Week 12Secondary· Immediately post-baseline up to Week 12
Number of Participants with Potentially Clinically Significant Blood Chemistry Results by Treatment Group. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
ALT ≥ 3 x ULN
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
1
150 mg BID Dexpramipexole
0
Albumin ≤ 2.5 g/dL
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Alkaline Phosphatase ≥ 1.5 x ULN
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
1
150 mg BID Dexpramipexole
1
AST ≥ 3 x ULN
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Bicarbonate ≤ 16 mEq/L
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
1
150 mg BID Dexpramipexole
0
Bicarbonate ≥ 35 mEq/L
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Bilirubin > 2 X ULN and (ALT or AST ≥ 3 X ULN)
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Bilirubin ≥ 1.5 x ULN
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Number of Participants With Potentially Clinically Significant Urinalysis Results by Treatment Group Post Randomization Through Week 12Secondary· Immediately post-baseline up to Week 12
Number of Participants with Potentially Clinically Significant Urinalysis Results (glycosuria, ketonuria, or proteinuria) by Treatment Group. Percentages based on number of patients with at least one non-missing post-baseline urinalysis value in each treatment group. Patients are only counted once per criterion per laboratory test. The number of participants with potential clinical important urinalysis findings at any post-baseline visit were reported.
glycosuria (glucose in urine ++++)
Group
Value
95% CI
Placebo BID
1
37.5 mg BID Dexpramipexole
2
75 mg BID Dexpramipexole
1
150 mg BID Dexpramipexole
0
ketonuria (ketones in urine ≥ ++++)
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
proteinuria (protein in urine ≥ ++)
Group
Value
95% CI
Placebo BID
1
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Number of Participants With Potentially Clinically Significant Vital Signs Results by Treatment Group Post Randomization Through Week 12Secondary· Immediately post-baseline up to Week 12
Number of Participants with Potentially Clinically Significant Vital Signs Results by Treatment Group. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
Systolic blood pressure: >180 mmHg
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Systolic blood pressure: Increase >40 mmHg
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Systolic blood pressure: <90 mmHg
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Systolic blood pressure: Decrease >30 mmHg
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Diastolic blood pressure: >105 mmHg
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Diastolic blood pressure: Increase >30 mmHg
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
1
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Diastolic blood pressure: <50 mmHg
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Diastolic blood pressure: Decrease >20 mmHg
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Number of Participants With Potentially Clinically Significant ECG Results by Treatment Group Post Randomization Through Week 12Secondary· Immediately post-baseline up to Week 12
Number of Participants with Potentially Clinically Significant ECG Results by Treatment Group. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
Heart Rate >120 bpm
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Heart Rate Increase from Baseline >30 bpm
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
1
QT Interval: >450 ms
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
QT Interval: >480 ms
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
QT Interval: >500 ms
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
QT Interval: Increase from Baseline >30 ms
Group
Value
95% CI
Placebo BID
7
37.5 mg BID Dexpramipexole
3
75 mg BID Dexpramipexole
2
150 mg BID Dexpramipexole
0
QT Interval: Increase from Baseline >60 ms
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
QTcF Interval: >450 ms
Group
Value
95% CI
Placebo BID
0
37.5 mg BID Dexpramipexole
0
75 mg BID Dexpramipexole
0
150 mg BID Dexpramipexole
0
Change in Nasal Eosinophil Peroxidase (Presented as Ratio to Protein) From Baseline to Week 12Secondary· Baseline, Week 12
The EPX:protein ratio was used to normalize the EPX for the quantity of sample, yielding the values in ng EPX per mg protein. The ratio of nasal Eosinophil Peroxidase to Protein is a biomarker for airway eosinophils. A lower ratio to Baseline represents a lowering in airway eosinophilia, which is a marker of successful drug therapy.
Group
Value
95% CI
Placebo BID
0.833
0.258 – 2.99
37.5 mg BID Dexpramipexole
0.645
0.275 – 1.18
75 mg BID Dexpramipexole
0.174
0 – 0.708
150 mg BID Dexpramipexole
0.110
0 – 0.943
Change in Blood Absolute Blood Basophil Count From Baseline to Week 12Secondary· Baseline, Week 12
The analysis used a mixed effects model repeated-measures MMRM with terms for baseline, GINA treatment step, treatment, visit, treatment by visit interaction, and baseline by visit interaction as fixed effects, and subject as a random effect. An unstructured covariance matrix was used. The response variable was the post-baseline value minus the baseline value. Basophils were enumerated as part of the WBC automated differential performed by the Central Laboratory.
Group
Value
95% CI
Placebo BID
-0.00439
± 0.006323
37.5 mg BID Dexpramipexole
-0.00655
± 0.006674
75 mg BID Dexpramipexole
-0.0250
± 0.006487
150 mg BID Dexpramipexole
-0.0277
± 0.006082
Adverse events — posted to ClinicalTrials.gov
Time frame: Placebo Run-in Period = from enrollment to Randomization (2-4 weeks); Primary Assessment Period = from Randomization through the Week 12 visit, plus 30 days following the last dose; Eosinophil Recovery Period = all visits occurring from 30 days following the last dose through the end of study (Week 24 post-Randomization).
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Screening Period
Serious: 0/103 (0%)
Deaths: 0/103
Primary Assessment Period: Placebo BID
Serious: 0/27 (0%)
Deaths: 0/27
Primary Assessment Period: 37.5 mg BID
Serious: 0/22 (0%)
Deaths: 0/22
Primary Assessment Period: 75 mg BID
Serious: 0/26 (0%)
Deaths: 0/26
Primary Assessment Period: 150 mg BID
Serious: 0/28 (0%)
Deaths: 0/28
Eosinophil Recovery Period: Assigned to Placebo BID During Primary Assessment Period
Serious: 0/25 (0%)
Deaths: 0/25
Eosinophil Recovery Period: Assigned to 37.5 mg BID During Primary Assessment Period
Serious: 0/22 (0%)
Deaths: 0/22
Eosinophil Recovery Period: Assigned to 75 mg BID During Primary Assessment Period
Serious: 0/24 (0%)
Deaths: 0/24
Eosinophil Recovery Period: Assigned to 150 mg BID During Primary Assessment Period
This is a randomized, double-blind, placebo-controlled, parallel-group, dose-ranging, multi-center study to evaluate the clinical effects of oral administration of dexpramipexole for 12 weeks on peripheral blood eosinophil count in subjects with eosinophilic asthma.
Publications & conference data
5 peer-reviewed publications reference this trial (live from Europe PMC):
NCT02101138 — Study to Evaluate Safety and Efficacy of Dexpramipexole (KNS-760704) in Subjects With Hypereosinophilic Syndrome
· Phase 2
· unknown
Other recruiting trials for Eosinophilic Asthma
Currently open trials in the same condition.
NCT06750289 — Randomised Clinical Trial to Investigate Efficacy and Safety of Benralizumab 30 mg SC as an add-on Therapy in Uncontroll
· Phase 3
· recruiting
NCT04538937 — Prospective Registry of Eosinophilia With Respiratory Manifestations With Translational Research Identifying and Charact
· recruiting
Other Knopp Biosciences trials
Trials by the same sponsor.
NCT00647296 — Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS)
· Phase 2
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Knopp Biosciences
Last refreshed: 13 April 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04046939.