Mastocytosis Clinical Trial
Official title:
Randomized, Double-blind, Placebo-controlled, Crossover Design, Efficacy and Safety Study With PA101 in Patients With Indolent Systemic Mastocytosis
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 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.
;
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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