Chronic Sinusitis With Nasal Polyps and EosinophiliaPhase 2CS201NCT02217332

Study of Dexpramipexole Chronic Sinusitis With Nasal Polyps and Eosinophilia

A Phase 2 study testing dexpramipexole in people with Chronic Sinusitis With Nasal Polyps and Eosinophilia, run by Knopp Biosciences at 7 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

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. Read more about this study

Who is it for?: Ages 18 to 70 years
Conditions the study is for: Chronic Sinusitis With Nasal Polyps and Eosinophilia
What is being tested?: dexpramipexole
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 20 participants
Where: 7 sitesUnited States
Study dates: Aug 2014to 20 Jan 2017
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 70 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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)

Who cannot join

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

Show all 31Show fewer

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

Trial locations and contacts

7 sites in 1 country.

City, postcode or hospital

7 sites in 1 country

United States7 sites

Johns Hopkins University
Baltimore, Maryland, 21205
ENT Associates of South Florida
Boca Raton, Florida, 33487
Brigham and Women's Hospital
Boston, Massachusetts, 02115
University of Virginia
Charlottesville, Virginia, 22903
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, 15213
Wake Research Associates
Raleigh, North Carolina, 27612

Other trials in United States

About this study

Official title: Safety and Preliminary Efficacy of Dexpramipexole in Patients With Chronic Sinusitis With Nasal Polyps and Eosinophilia (CSNP-E)

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.

Read the detailed description

This open-labelled study will evaluate the safety and preliminary efficacy of dexpramipexole for reducing the number of eosinophils in the peripheral blood and in improving nasal polyp score when administered to 20 subjects with chronic sinusitis with nasal polyps and eosinophilia.

Subjects will received dexpramipexole for up to 6 months and will have safety tests performed monthly and will have efficacy evaluations performed at month 1, month 3, and month 6 after beginning study drug.

Conditions: Chronic Sinusitis With Nasal Polyps and Eosinophilia
Being tested: dexpramipexole
Keywords: Sinusitis, Rhinosinusitis, CRS, CRSwNP, CSNP-E, Nasal polyps, Nasal polyposis, Eosinophilia, Asthma
Medical subject headings: Eosinophilia, Sinusitis, Rhinosinusitis, Nasal Polyps, Asthma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

dexpramipexoleDrug
Also called KNS-760704, BIIB050, RPPX
Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)

Study groups in NCT02217332
Study group	Role	What they receive
dexpramipexole	Experimental	dexpramipexole 150 mg BID Receives: Drug: dexpramipexole

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Baseline and Month 6
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.
Measured: Baseline and Month 6

Secondary outcomes (5)

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
Measured: 6 months
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
Measured: 6 months
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
Measured: 6 months
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.
Measured: Baseline and Month 3
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.
Measured: Baseline and Month 3

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Aug 201420 Jan 2017

First posted15 Aug 2014 (estimated)
Study startAug 2014
Primary completion14 Dec 2016
Study completion20 Jan 2017

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Chronic Sinusitis With Nasal Polyps and EosinophiliaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 20 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02217332
Sponsor's protocol ID: KNS-76704-CS201
Acronym: CS201
Status last verified: Apr 2021
First submitted: 12 Aug 2014
Last updated: 18 May 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Study at a glance

From the registry record, last updated by the sponsor on 18 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Safety and Preliminary Efficacy of Dexpramipexole in Patients With Chronic Sinusitis With Nasal Polyps and Eosinophilia (CSNP-E)
ClinicalTrials.gov ID: NCT02217332
Acronym / sponsor ID: CS201 / KNS-76704-CS201
Conditions: Chronic Sinusitis With Nasal Polyps and Eosinophilia
Keywords: Sinusitis, Rhinosinusitis, CRS, CRSwNP, CSNP-E, Nasal polyps, Nasal polyposis, Eosinophilia
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 20 participants (actual)
Lead sponsor: Knopp BiosciencesIndustry (pharma, biotech, devices)
Study start: Aug 2014
Primary completion: 14 Dec 2016 (actual)
Study completion: 20 Jan 2017 (actual)
First posted: 15 Aug 2014 (estimated)
Results: Posted 18 May 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
dexpramipexole	Experimental	Drug: dexpramipexoledexpramipexole 150 mg BID

Who may qualify

Ages: 18 to 70 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

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

Key exclusion criteria (31 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
dexpramipexoleAlso: KNS-760704, BIIB050, RPPXDexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)	Drug	dexpramipexole

Primary outcome

Ratio to Baseline in Peripheral Blood Eosinophil Counts After 6 Months of Treatment
Time frame: Baseline and Month 6
Change in peripheral blood eosinophil (measured in cells/μL) from baseline to month 6 is presented as ratio to baseline.
Change From Baseline in Total Polyp Score (TPS) After 6 Months of Treatment
Time frame: Baseline and Month 6
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.

5 secondary outcomes

Number of Subjects With Potentially Clinically Significant Values in Clinical Laboratory Evaluations
Time frame: 6 months
Number of Subjects With Potentially Clinically Significant Values in Vital Signs and Weight
Time frame: 6 months
Number of Subjects With Potentially Significant Values in Electrocardiogram Parameters
Time frame: 6 months
Ratio to Baseline in Peripheral Blood Eosinophil Counts After 3 Months of Treatment
Time frame: Baseline and Month 3
Change From Baseline in TPS After 3 Months of Treatment
Time frame: Baseline and Month 3

Results and safety

Results posted to the registry on 18 May 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Dexpramipexole	20	13

Primary outcome: Ratio to Baseline in Peripheral Blood Eosinophil Counts After 6 Months of Treatment

Time frame: Baseline and Month 6. Geometric mean (95% Confidence Interval). Unit: ratio to baseline

Measure	Dexpramipexole n = 16
Value	0.06 (0.023 to 0.154)

Dexpramipexole: p < 0.001, t-test, 2 sided

Population analysed: All subjects who had a Month 3 evaluation (on study drug Day 77 or later) for TPS and peripheral blood eosinophil count. Last observation carried forward (LOCF) was used to replace missing data due to drop out. Only Month 3 data or later (assessment date Day 77 or later and within 7 days of last dose) were used.

Primary outcome: Change From Baseline in Total Polyp Score (TPS) After 6 Months of Treatment

Time frame: Baseline and Month 6. Mean (Standard Deviation). Unit: units on a scale

Measure	Dexpramipexole n = 15
Value	0.07 (1.751)

Dexpramipexole: p = 0.885, t-test, 2 sided

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: 6 months on treatment. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Dexpramipexole	1 of 20 (5%)	18 of 20 (90%)	0 of 20

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

7 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing dexpramipexole in people with Chronic Sinusitis With Nasal Polyps and Eosinophilia, run by Knopp Biosciences at 7 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 31 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 May 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 May 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Ratio to Baseline in Peripheral Blood Eosinophil Counts After 6 Months of Treatment (time frame: Baseline and Month 6). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02217332.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2021.