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

Phase 1

Conditions: Indolent Systemic Mastocytosis
MedDRA version: 17.1Level: PTClassification code 10042949Term: Systemic mastocytosisSystem Organ Class: 10005329 - Blood and lymphatic system disorders
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2014-004113-85-ES

Lead Sponsor: Patara Pharma, LLC

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 36

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 H2 antihistamines and 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 H2 antihistamines and 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
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, 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
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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To determine the efficacy profile of PA101 delivered via a high efficiency nebulizer (eFlow®, PARI) in comparison with placebo following 6 weeks of treatment in patients with indolent systemic mastocytosis (ISM) who are symptomatic despite using standard treatments;Secondary Objective: To compare the efficacy and safety profile of PA101 to marketed oral cromolyn sodium (open-label control)<br>To assess the safety, tolerability, and pharmacokinetic (PK) profile of PA101;Primary end point(s): The primary efficacy endpoint is change from baseline in total symptom score recorded in the daily eDiary using the MAS Plus questionnaire at Week 6.;Timepoint(s) of evaluation of this end point: Week 6

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Secondary efficacy endpoints include the changes from baseline in each organ system domain and in each symptom score using the MAS Plus questionnaire and the change from baseline in quality of life as measured using the MIQ and SF-36 Questionnaire at Week 6. Another secondary endpoint is the PGIC score.;Timepoint(s) of evaluation of this end point: Week 6

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