Ignite Creation Date:
2025-12-24 @ 6:44 PM
Ignite Modification Date:
2025-12-25 @ 4:15 PM
Study NCT ID:
NCT02478957
Status:
COMPLETED
Last Update Posted:
2016-09-29
First Post:
2015-06-19
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Treatment of Indolent Systemic Mastocytosis With PA101
Sponsor:
Patara Pharma
Organization:
Study Overview
Official Title:
Randomized, Double-blind, Placebo-controlled, Crossover Design, Efficacy and Safety Study With PA101 in Patients With Indolent Systemic Mastocytosis
Status:
COMPLETED
Status Verified Date:
2016-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a randomized, placebo- and active-controlled, 2-period crossover, 2 cohort study in adult patients with indolent systemic mastocytosis (ISM).
The purpose of the study is to determine the efficacy and safety profile of PA101 delivered via eFlow high efficiency nebulizer in patients with ISM who are symptomatic despite using standard treatments.
The purpose of the study is to determine the efficacy and safety profile of PA101 delivered via eFlow high efficiency nebulizer in patients with ISM who are symptomatic despite using standard treatments.
Detailed Description:
The symptom scores for determining eligibility will be established during the 2-week Run-in Period using eDiary and the eligible patients will be randomly allocated in a 2:1 ratio to one of two treatment cohorts at the baseline visit.
In Cohort 1 (n=24), patients will receive inhaled 40 mg PA101 three times daily and inhaled placebo three times daily via eFlow for 6 weeks each in a double-blind, 2-period crossover fashion with a 4-week washout period between the treatment periods.
In Cohort 2 (n=12), patients will receive inhaled 40 mg PA101 three times daily via eFlow and oral cromolyn sodium 200 mg four times daily for 6 weeks each in an open label, 2-period crossover fashion with a 4-week washout period between the treatment periods.
Patients will be allowed to use the same daily doses of pre-randomization H1 and H2 antihistamines as well as the same daily doses of any other allowed medications during each treatment period.
Visits during each treatment period will occur at the baseline Visit, and at the end of Weeks 1, 2, 4, and 6.
Blood and urine samples will be collected to test for various biomarkers. In a subset of patients, additional clinical assessments of the skin will be performed and blood samples will be collected for pharmacokinetic assessments. Clinical safety assessments will be performed in all patients at the start and end of each treatment period.
In Cohort 1 (n=24), patients will receive inhaled 40 mg PA101 three times daily and inhaled placebo three times daily via eFlow for 6 weeks each in a double-blind, 2-period crossover fashion with a 4-week washout period between the treatment periods.
In Cohort 2 (n=12), patients will receive inhaled 40 mg PA101 three times daily via eFlow and oral cromolyn sodium 200 mg four times daily for 6 weeks each in an open label, 2-period crossover fashion with a 4-week washout period between the treatment periods.
Patients will be allowed to use the same daily doses of pre-randomization H1 and H2 antihistamines as well as the same daily doses of any other allowed medications during each treatment period.
Visits during each treatment period will occur at the baseline Visit, and at the end of Weeks 1, 2, 4, and 6.
Blood and urine samples will be collected to test for various biomarkers. In a subset of patients, additional clinical assessments of the skin will be performed and blood samples will be collected for pharmacokinetic assessments. Clinical safety assessments will be performed in all patients at the start and end of each treatment period.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: