A controlled study in patients with Indolent Systemic Mastocytosis to determine the safety and efficacy of PA101 when compared with an existing drug and a placebo

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 42

Inclusion Criteria

1. Male or female patients 18-75 years of age, inclusive
2. Diagnosed with indolent systemic mastocytosis (ISM) according to the WHO criteria and the consensus proposal (2001)
3. Experiencing at least one qualifying symptom in at least two organ systems during the 3 months preceding the Screening Visit, despite the
use of H1 and/or H2 antihistamines and/or other antimediator therapy
4. Experiencing symptoms with a severity score of at least 4 for at least 7 out of 14 days during the Run-in Period with at least one qualifying symptom each from at least two organ systems, despite the use of H1 and/or H2 antihistamines and/or other anti-mediator therapy
5. Willing and able to use an eDiary device daily for the duration of the study
6. Completed at least 5 eDiary reports during each of two consecutive weeks of the Run-in Period
7. Patients must digitally accept the licensing agreement in the eDiary
software
8. Willingness and ability to provide written informed consent prior to any study procedures performed
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 6

Exclusion Criteria

1. Advanced systemic mastocytosis (i.e., aggressive systemic mastocytosis [ASM], mast cell leukemia [MCL], or systemic mastocytosis with an associated clonal hematologic non-mast cell lineage disease
[SM-AHNMD] )
2. Current or recent history of clinically significant cardiovascular, hematological, renal, neurologic, hepatic, endocrine, psychiatric, malignant, or other illnesses that could put the patient at risk or
compromise the quality of the study data as determined by the Investigator
3. Use of oral cromolyn sodium within 6 weeks of the Screening Visit
4. History of systemic corticosteroid use within 6 weeks, or immunosuppressive, or anti-IgE monoclonal antibody therapy (e.g., omalizumab) within 6 months of the Screening Visit
5. History of anaphylaxis requiring systemic treatment (i.e., corticosteroid or epinephrine) within 12 months of the Screening Visit
6. An upper or lower respiratory tract infection within 4 weeks of the Screening Visit
7. History of malignancy within the last 5 years, except basal cell carcinoma or cervix carcinoma in situ
8. Major surgery within 6 months of the Screening Visit
9. History of excessive use or abuse of alcohol (i.e., more than 3 units per day, or more than 21 units per week) within 12 months of the Screening Visit
10. History of abusing legal drugs or use of illegal drugs or substances within 12 months of the Screening Visit
11.Females who are pregnant or breastfeeding, or if of child-bearing potential unwilling to practice acceptable means of birth control or
abstinence during the study; females of child-bearing potential must use one reliable method of contraception (i.e., Pearl Index of less than 1%)
12.Participation in any other investigational drug study within 4 weeks of the Screening Visit
13.History of hypersensitivity or intolerance to aerosol medications or cromolyn sodium
14.Patients under guardianship, trusteeship, or committed to an institution by order of government or judicial authorities
15.Patients who have a relationship as such that they are rendered dependent to the investigator/study site staff, or the sponsor

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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