Treatment of Indolent Systemic Mastocytosis With PA101

NCT02478957 | Completed Phase 2

• 1 other identifier: PA101-SM-02
• Study Type: interventional
• Target: 41
• Locations: 5 countries
• Sites: 8

Brief Summary

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.

Conditions

Mastocytosis; Systemic Mastocytosis; Indolent Systemic Mastocytosis

Keywords

mastocytosis; cromolyn sodium

Outcome Measures

Primary Outcomes (1)

• Overall symptom score

  Mastocytosis Activity of Symptoms (MAS Plus) questionnaire for daily symptom score using eDiary

  6 weeks

Secondary Outcomes (2)

• Disease specific quality of life (QoL)

  6 weeks

• Patient Global Impression of Change (PGIC) scale

  6 weeks

Study Arms (2)

PA101

EXPERIMENTAL

PA101, 40 mg administered via inhalation three times daily for 6 weeks

Drug: PA101

Placebo

PLACEBO COMPARATOR

Placebo, administered via inhalation three times daily for 6 weeks

Drug: Placebo

Interventions

PA101 DRUG

40 mg PA101 administered via inhalation three times daily for 6 weeks

PA101

Placebo DRUG

matching placebo administered via inhalation three times daily for 6 weeks

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of indolent systemic mastocytosis (ISM) according to the WHO criteria and the consensus proposal (2001)
• Experiencing at least one predefined qualifying symptom in at least two organ systems within 3 months of Screening despite the use of H1 and/or H2 antihistamines and other anti-mediator therapy
• 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 predefined qualifying symptom each from at least two organ systems, despite the use of H1 and/or H2 antihistamines and/or other anti-mediator therapy
• Willing and able to use an eDiary device daily for the duration of the study
• Completed at least 5 eDiary reports during each of two consecutive weeks of the Run-in period
• Willing and able to provide written informed consent prior to any study procedures

You may not qualify if:

• 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 \[SM-AHNMD\] )
• 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
• Use of oral cromolyn sodium within 6 weeks of Screening
• History of systemic corticosteroid, immunosuppressive, or anti-IgE monoclonal antibody therapy (e.g., omalizumab) within 6 months of Screening
• History of anaphylaxis requiring systemic treatment (i.e., corticosteroid or epinephrine) within 12 months of Screening
• Upper or lower respiratory tract infection within 4 weeks of Screening
• History of malignancy within the last 5 years, except basal cell carcinoma or cervix carcinoma in situ
• Major surgery within 6 months of Screening
• Current or recent history (within 12 months) of excessive use or abuse of alcohol
• Current or recent history (within 12 months) of abusing legal drugs or use of illegal drugs or substances
• Pregnant or breastfeeding females, or if of child-bearing potential unwilling to practice acceptable means of birth control or abstinence during the study
• Participation in any other investigational drug study within 4 weeks of Screening
• History of hypersensitivity or intolerance to aerosol medications or cromolyn sodium

Sponsors & Collaborators

• Patara Pharma: lead

Study Sites (8)

Hopital Necker - Enfants Malades

Paris, France

Location

Charité - Universitätsmedizin Berlin

Berlin, Germany

Location

Klinikum Darmstadt

Darmstadt, Germany

Location

University Medical Center Mainz

Mainz, Germany

Location

Technical University München

Munich, Germany

Location

University of Salerno

Salerno, Italy

Location

University Medical Center of Groningen

Groningen, Netherlands

Location

Hospital Universitario de Fuenlabrada

Madrid, Spain

Location

MeSH Terms

Conditions

Mastocytosis; Mastocytosis, Systemic

Condition Hierarchy (Ancestors)

Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Mast Cell Activation Disorders; Immune System Diseases

Study Officials

• Frank Siebenhaar, MD

  Charite University, Berlin, Germany

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 19, 2015
• First Posted: June 23, 2015
• Study Start: June 1, 2015
• Primary Completion: March 1, 2016
• Study Completion: June 1, 2016
• Last Updated: September 29, 2016

Record last verified: 2016-09

Locations

DE (4); FR (1); NL (1); ES (1); IT (1)