Indolent Systemic Mastocytosis MedDRA version: 18.0 Level: PT Classification code 10042949 Term: Systemic mastocytosis System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Interventions
Product Name: Disodium Cromoglycate
Product Code: PA101
Pharmaceutical Form: Nebuliser solution
INN or Proposed INN: SODIUM CROMOGLICATE
CAS Number: 15826-37-6
Current Sponsor code: PA101
Concentratio
Sponsors
Patara Pharma, LLC
Eligibility
Sex/Gender
All
Inclusion criteria
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 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
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; 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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) 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
| Measure | Time frame |
|---|---|
| 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 | — |
Countries
France, Germany, Italy, Netherlands, Spain
Contacts
Public ContactProject Manager
Premier Research
Outcome results
None listed