MastocytosisPhase 2NCT02478957
Treatment of Indolent Systemic Mastocytosis With PA101
A Phase 2 study testing PA101 in people with Mastocytosis and Systemic Mastocytosis, run by Patara Pharma at 8 sites in 5 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Mastocytosis, Systemic Mastocytosis, Indolent Systemic Mastocytosis
- What is being tested?
- PA101
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 41 participants
- Where
- 8 sitesin 5 countries
- Study dates
- Jun 2015to Jun 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 13Show fewer
- 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
Trial locations and contacts
8 sites in 5 countries.
8 sites in 5 countries
Germany4 sites
Charité - Universitätsmedizin Berlin
Berlin
Klinikum Darmstadt
Darmstadt
University Medical Center Mainz
Mainz
Technical University München
Munich
About this study
Official title: Randomized, Double-blind, Placebo-controlled, Crossover Design, Efficacy and Safety Study With PA101 in Patients With Indolent Systemic Mastocytosis
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.
Read the detailed description
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.
- Being tested
- PA101, Placebo
- Keywords
- mastocytosis, cromolyn sodium
- Medical subject headings
- Mastocytosis, Mastocytosis, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PA101Drug
40 mg PA101 administered via inhalation three times daily for 6 weeks
PlaceboDrug
matching placebo administered via inhalation three times daily for 6 weeks
| Study group | Role | What they receive |
|---|---|---|
| PA101 | Experimental | PA101, 40 mg administered via inhalation three times daily for 6 weeks Receives: Drug: PA101 |
| Placebo | Placebo comparator | Placebo, administered via inhalation three times daily for 6 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall symptom score
Mastocytosis Activity of Symptoms (MAS Plus) questionnaire for daily symptom score using eDiary
Measured: 6 weeks
Secondary outcomes (2)
Disease specific quality of life (QoL)
Mastocytosis Impact Questionnaire (MIQ) for quality of life
Measured: 6 weeks
Patient Global Impression of Change (PGIC) scale
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2015 (estimated)
- Study startJun 2015
- Primary completionMar 2016
- Study completionJun 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MastocytosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Patara Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Frank Siebenhaar, MD, Principal investigator, Charite University, Berlin, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02478957
- Sponsor's protocol ID
- PA101-SM-02
- Status last verified
- Sep 2016
- First submitted
- 19 Jun 2015
- Last updated
- 29 Sep 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Double-blind, Placebo-controlled, Crossover Design, Efficacy and Safety Study With PA101 in Patients With Indolent Systemic Mastocytosis
- ClinicalTrials.gov ID
- NCT02478957
- Acronym / sponsor ID
- PA101-SM-02
- Keywords
- mastocytosis, cromolyn sodium
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Patara PharmaIndustry (pharma, biotech, devices)
- Study start
- Jun 2015
- Primary completion
- Mar 2016 (actual)
- Study completion
- Jun 2016 (actual)
- First posted
- 23 Jun 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PA101 | Experimental | Drug: PA101PA101, 40 mg administered via inhalation three times daily for 6 weeks |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo, administered via inhalation three times daily for 6 weeks |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PA10140 mg PA101 administered via inhalation three times daily for 6 weeks | Drug | PA101 |
| Placebomatching placebo administered via inhalation three times daily for 6 weeks | Drug | Placebo |
Primary outcome
Overall symptom score
Time frame: 6 weeks
Mastocytosis Activity of Symptoms (MAS Plus) questionnaire for daily symptom score using eDiary
2 secondary outcomes
Disease specific quality of life (QoL)
Time frame: 6 weeks
Patient Global Impression of Change (PGIC) scale
Time frame: 6 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Frank Siebenhaar, MD principal investigator
Charite University, Berlin, Germany
8 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PA101 in people with Mastocytosis and Systemic Mastocytosis, run by Patara Pharma at 8 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Germany, France, Italy, Netherlands, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall symptom score (time frame: 6 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2016.