Systemic Mast Cell ActivationNCT04811365
(PROSPECTOR) Screening Study Evaluating the Prevalence of the KIT D816V Mutation in Patients With Systemic Mast Cell Activation
An observational study of Systemic Mast Cell Activation and KIT D816V Mutation, run by Blueprint Medicines Corporation at 23 sites in 8 countries.
Trial summary
This is a multi-center screening study with the primary objective to determine the prevalence of KIT D816V mutation in peripheral blood in patients with evidence of systemic mast cell activation (MCA). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Systemic Mast Cell Activation, KIT D816V Mutation
- What is being tested?
- Screening
- How many people
- 379 participants
- Where
- 23 sitesin 8 countries
- Study dates
- 29 Jun 2021to 31 Mar 2023
- Phase
- Observational
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult patients with systemic mast cell activation
Who may be able to join
- Presenting with at least one of the three criteria below as evidence of systemic mast cell activation:
- Involvement of 2 or more organ systems, characterized by skin (pruritus, urticaria, flushing and angioedema), cardiovascular (tachycardia, syncope, and hypotension), gastrointestinal (diarrhea, nausea, vomiting, and gastrointestinal cramping) or respiratory/naso-ocular (wheezing, conjunctival injection, and nasal stuffiness) AND serum basal tryptase levels ≥8 ng/ml. One of the organ systems must be the cardiovascular system.
- Severe anaphylaxis (Ring and Messmer grading ≥II) due to Hymenoptera sting, regardless of serum basal tryptase levels.
- Severe anaphylaxis (Ring and Messmer grading ≥II), with cardiovascular involvement and documented event-related tryptase elevation fitting the formula 20% of baseline plus 2 ng/ml evaluated in at least 1 event.
Who cannot join
- Patient previously diagnosed with any of the following WHO systemic mastocytosis (SM) sub-classifications: mastocytosis in the skin, indolent SM, smoldering SM, SM with associated hematological neoplasm, aggressive SM, mast cell leukemia, mast cell sarcoma
Trial locations and contacts
23 sites in 8 countries.
23 sites in 8 countries
United States10 sites
University of Michigan
Ann Arbor, Michigan, 48105
Clinical Research Center of Alabama
Birmingham, Alabama, 35209
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Roswell Park Cancer Center
Buffalo, New York, 14203
Bernstein Clinical Research Center, LLC
Cincinnati, Ohio, 45236
Duke University: Duke Allergy Asthma Airway Center
Durham, North Carolina, 27705
Albert P. Hirdt D.O. P.C.
New Paltz, New York, 12561
Columbia University Medical Center - Herbert Irving Comprehensive Cancer Center HICCC
New York, New York, 10032
Mayo Clinic
Rochester, Minnesota, 55905
Scripps Clinic Carmel Valley
San Diego, California, 92130
Germany3 sites
Charite Universitatsmedizin Berlin
Berlin, 12203
Medizinische Hochschule Hannover, Dept. of Dermatology and Allergy
Hanover, 30625
Klinik fuer Dermatologie und Allergologie
München, 80337
United Kingdom3 sites
University Hospital of Leicester
Leicester, LE1 5WW
Guy's and St Thomas' NHS Foundation Trust
London, SE1 9RT
University Hospitals Plymouth NHS Trust
Plymouth, PL6 8DH
France2 sites
Pitié-Salpêtrière Hospital
Paris, 75013
CHU Toulouse
Toulouse, 31059
Italy2 sites
AOU San Giovanni di Dio e Ruggi d'Aragona University of Salerno
Salerno, 84100
Azienda Ospedaliera Universitaria Integrata Verona
Verona, 37126
About this study
Official title: Study Evaluating the Prevalence of the KIT D816V Mutation in Peripheral Blood of Patients With Evidence of Systemic Mast Cell Activation (MCA)
This is a multi-center screening study with the primary objective to determine the prevalence of KIT D816V mutation in peripheral blood in patients with evidence of systemic mast cell activation (MCA).
- Conditions
- Systemic Mast Cell Activation, KIT D816V Mutation
- Being tested
- Screening
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ScreeningOther
After providing informed consent, relevant medical history data, and blood and buccal swab samples will be collected at a single visit from patients presenting with systemic mast cell activation symptoms
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of patients with KIT D816V mutation in peripheral blood
Measured: Day 1
Secondary outcomes (2)
Mean KIT D816V mutated allele fraction in peripheral blood
Measured: Day 1
Proportion of patients with increased tryptase Alpha/ Beta 1 (TPSAB1) alpha-tryptase gene copy number (GCN) in buccal mucosa
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2021
- Study start29 Jun 2021
- Primary completion31 Mar 2023
- Study completion31 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic Mast Cell ActivationStudies 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
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 379 participants (actual)
Sponsor and investigators
- Lead sponsor
- Blueprint Medicines Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04811365
- Sponsor's protocol ID
- BLU-SM-1101
- Status last verified
- Apr 2023
- First submitted
- 10 Mar 2021
- Last updated
- 6 Apr 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study Evaluating the Prevalence of the KIT D816V Mutation in Peripheral Blood of Patients With Evidence of Systemic Mast Cell Activation (MCA)
- ClinicalTrials.gov ID
- NCT04811365
- Acronym / sponsor ID
- BLU-SM-1101
- Conditions
- Systemic Mast Cell Activation, KIT D816V Mutation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 379 participants (actual)
- Lead sponsor
- Blueprint Medicines CorporationIndustry (pharma, biotech, devices)
- Study start
- 29 Jun 2021 (actual)
- Primary completion
- 31 Mar 2023 (actual)
- Study completion
- 31 Mar 2023 (actual)
- First posted
- 23 Mar 2021 (actual)
- 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 multi-center screening study with the primary objective to determine the prevalence of KIT D816V mutation in peripheral blood in patients with evidence of systemic mast cell activation (MCA).
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult patients with systemic mast cell activation
Key inclusion criteria (4 listed)
- Presenting with at least one of the three criteria below as evidence of systemic mast cell activation:
Key exclusion criteria (1 listed)
- Patient previously diagnosed with any of the following WHO systemic mastocytosis (SM) sub-classifications: mastocytosis in the skin, indolent SM, smoldering SM, SM with associated hematological neoplasm, aggressive SM, mast cell leukemia, mast cell sarcoma
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 |
|---|---|---|
| ScreeningAfter providing informed consent, relevant medical history data, and blood and buccal swab samples will be collected at a single visit from patients presenting with systemic mast cell activation symptoms | Other | – |
Primary outcome
Proportion of patients with KIT D816V mutation in peripheral blood
Time frame: Day 1
2 secondary outcomes
Mean KIT D816V mutated allele fraction in peripheral blood
Time frame: Day 1
Proportion of patients with increased tryptase Alpha/ Beta 1 (TPSAB1) alpha-tryptase gene copy number (GCN) in buccal mucosa
Time frame: Day 1
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 6 Apr 2023.
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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.
The record lists no contact. Use the official record to reach the sponsor.
23 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Systemic Mast Cell Activation and KIT D816V Mutation, run by Blueprint Medicines Corporation at 23 sites in 8 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 4 inclusion and 1 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 6 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, Germany, United Kingdom, France, Italy and 3 more countries. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
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?
Proportion of patients with KIT D816V mutation in peripheral blood (time frame: Day 1). 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 6 Apr 2023.