Treatment of Indolent Systemic Mastocytosis With PA101
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|ClinicalTrials.gov Identifier: NCT02478957|
Recruitment Status : Completed
First Posted : June 23, 2015
Last Update Posted : September 29, 2016
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.
|Condition or disease||Intervention/treatment||Phase|
|Mastocytosis Systemic Mastocytosis Indolent Systemic Mastocytosis||Drug: PA101 Drug: Placebo||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||41 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Randomized, Double-blind, Placebo-controlled, Crossover Design, Efficacy and Safety Study With PA101 in Patients With Indolent Systemic Mastocytosis|
|Study Start Date :||June 2015|
|Actual Primary Completion Date :||March 2016|
|Actual Study Completion Date :||June 2016|
PA101, 40 mg administered via inhalation three times daily for 6 weeks
40 mg PA101 administered via inhalation three times daily for 6 weeks
Placebo Comparator: Placebo
Placebo, administered via inhalation three times daily for 6 weeks
matching placebo administered via inhalation three times daily for 6 weeks
- Overall symptom score [ Time Frame: 6 weeks ]Mastocytosis Activity of Symptoms (MAS Plus) questionnaire for daily symptom score using eDiary
- Disease specific quality of life (QoL) [ Time Frame: 6 weeks ]Mastocytosis Impact Questionnaire (MIQ) for quality of life
- Patient Global Impression of Change (PGIC) scale [ Time Frame: 6 weeks ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02478957
|Hopital Necker - Enfants Malades|
|Charité - Universitätsmedizin Berlin|
|University Medical Center Mainz|
|Technical University München|
|University of Salerno|
|University Medical Center of Groningen|
|Hospital Universitario de Fuenlabrada|
|Principal Investigator:||Frank Siebenhaar, MD||Charite University, Berlin, Germany|