Chronic Sinusitis With Nasal Polyps and Eosinophilia Clinical Trial
— CS201Official title:
Safety and Preliminary Efficacy of Dexpramipexole in Patients With Chronic Sinusitis With Nasal Polyps and Eosinophilia (CSNP-E)
| Verified date | April 2021 |
| Source | Knopp Biosciences |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
Phase 2, open-label, multi-center study to evaluate the clinical effects of oral administration of dexpramipexole for 6 months in subjects with chronic sinusitis with nasal polyps and eosinophilia.
| Status | Completed |
| Enrollment | 20 |
| Est. completion date | January 20, 2017 |
| Est. primary completion date | December 14, 2016 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years to 70 Years |
| Eligibility | Inclusion Criteria: - Male or female >18 or <70 years of age - Willing to practice effective contraception during the study and be willing and able to continue contraception for 1 month (females) or 3 months (males) after the last dose of study treatment - Confirmed diagnosis of chronic sinusitis with nasal polyps - Documented history of nasal eosinophilia - Documented peripheral absolute eosinophil count >300 cells/µL - Bilateral total polyp score of >4 - Sino-nasal outcome test (SNOT-22) score of >7 - Using an intranasal corticosteroid spray or irrigation (< 1000 µg/day beclomethasone or equivalent) Exclusion Criteria: - Acute sinusitis, concurrent nasal infection, or subjects who have had a nasal or upper respiratory tract infection within 2 weeks prior to baseline - CT scan suggestive of allergic fungal rhinosinusitis - Nasal septal deviation that would occlude at least one nostril - Nasal surgery (including polypectomy) within 6 months prior to baseline - History of more than 5 sinonasal surgeries requiring general anesthesia - History of more than 2 sinonasal surgeries that changed the lateral wall of the nose - History of cystic fibrosis, primary ciliary's dysfunction or Kartagener's syndrome - History of diagnosis with a parasitic infection - Hospitalization or emergency treatment for the treatment of asthma two or more times in the 12 months prior to baseline - Hospitalization for an acute asthmatic attack within 4 weeks prior to baseline - Forced expiratory volume (FEV1) of <60% of predicted normal range - Treatment with a systemic corticosteroid or intra-polyp corticosteroid within 8 weeks prior to baseline or anticipated need for systemic corticosteroids during the study treatment period - Utilization of rescue oral corticosteroids for asthma or chronic sinusitis exacerbation more than one time within the past 1 year - Treatment with an investigational drug in the previous 30 days or 5-half-lives, whichever is longer - Treatment with a monoclonal antibody therapy including omalizumab (Xolair®), within 5-half-lives - Treatment with zileuton (Zyflo®) within 4 weeks of baseline - Treatment with pramipexole (Mirapex®) within 4 weeks of baseline - History of malignancy, including solid tumors and hematologic malignancies (except basal cell and squamous cell cancers of the skin that have been completely excised and cured) - History of human immunodeficiency virus (HIV) or hepatitis B or C - History of unstable or severe cardiac, hepatic, or renal disease, or other medically significant illness - Medical or other condition likely to interfere with subject's ability to undergo study procedures, adhere to visit schedule or comply with study requirements - Absolute neutrophil count <2000 cells/µL at screening, or any documented history of neutropenia - Total IgE >1500 IU/ml at any visit prior to baseline - Renal dysfunction, defined as an estimated glomerular filtration rate (eGFR) of =80 mg/dL at screening (estimation of creatinine clearance using the MDRD formula) - History of long QT syndrome or arrhythmia - Prolongation of QT/QTc interval (e.g., repeated demonstration of a QT/QTc interval >450 ms) at screening or pre-dose on day 1 - Clinically important abnormalities in resting ECG that may interfere with the interpretation of QTc interval changes at screening or pre-dose on day 1, including any of the following: - PR interval >210 ms; - QRS >110 ms; - Heart rate <45 bpm or >100 bpm (average of 3 assessments). - Pregnant women or women breastfeeding |
| Country | Name | City | State |
|---|---|---|---|
| United States | Johns Hopkins University | Baltimore | Maryland |
| United States | ENT Associates of South Florida | Boca Raton | Florida |
| United States | Brigham and Women's Hospital | Boston | Massachusetts |
| United States | University of Virginia | Charlottesville | Virginia |
| United States | University of Pennsylvania | Philadelphia | Pennsylvania |
| United States | University of Pittsburgh School of Medicine | Pittsburgh | Pennsylvania |
| United States | Wake Research Associates | Raleigh | North Carolina |
| Lead Sponsor | Collaborator |
|---|---|
| Knopp Biosciences |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Other | Ratio to Baseline in Polyp Tissue Eosinophil Count After 6 Months of Treatment | Change in polyp tissue eosinophil count measured as eosinophils/hpf (high powered field) from baseline to month 6 is presented as ratio to baseline. | Baseline and Month 6 | |
| Primary | Ratio to Baseline in Peripheral Blood Eosinophil Counts After 6 Months of Treatment | Change in peripheral blood eosinophil (measured in cells/µL) from baseline to month 6 is presented as ratio to baseline. | Baseline and Month 6 | |
| Primary | Change From Baseline in Total Polyp Score (TPS) After 6 Months of Treatment | The change in Nasal Polyp Score (NPS) score after 6 months of treatment is presented. A blinded Central Endoscopy Rater determined the NPS for each nare on a scale of 0 to 4, and the bilateral NPS scores were added to generate the Total Polyp Score (TPS) on a scale of 0 to 8. A lower TPS corresponds to a lower polyp burden. | Baseline and Month 6 | |
| Secondary | Number of Subjects With Potentially Clinically Significant Values in Clinical Laboratory Evaluations | Summary of subjects from the safety population who experienced potentially clinically significant values in hand serum chemistry results | 6 months | |
| Secondary | Number of Subjects With Potentially Clinically Significant Values in Vital Signs and Weight | Summary of subjects from the safety population who experienced potentially clinically significant values or changes in vital signs or weight | 6 months | |
| Secondary | Number of Subjects With Potentially Significant Values in Electrocardiogram Parameters | Summary of subjects from the safety population who experienced potentially significant values in electrocardiogram parameters | 6 months | |
| Secondary | Ratio to Baseline in Peripheral Blood Eosinophil Counts After 3 Months of Treatment | Change in peripheral blood eosinophil (measured in cells/µL) from baseline to month 3 is presented as ratio to baseline. | Baseline and Month 3 | |
| Secondary | Change From Baseline in TPS After 3 Months of Treatment | The change in Nasal Polyp Score (NPS) score after 3 months of treatment is presented. A blinded Central Endoscopy Rater determined the NPS for each nare on a scale of 0 to 4, and the bilateral NPS scores were added to generate the Total Polyp Score (TPS) on a scale of 0 to 8. A lower TPS corresponds to a lower polyp burden. | Baseline and Month 3 |