NCT07143669

Brief Summary

This is a multicenter screening study to characterize the prevalence of the KIT D816V mutation in participants with suspected clonal mast cell disease.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
450

participants targeted

Target at P75+ for all trials

Timeline
30mo left

Started Oct 2025

Typical duration for all trials

Geographic Reach
1 country

17 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress18%
Oct 2025Oct 2028

First Submitted

Initial submission to the registry

August 20, 2025

Completed
7 days until next milestone

First Posted

Study publicly available on registry

August 27, 2025

Completed
2 months until next milestone

Study Start

First participant enrolled

October 17, 2025

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 31, 2028

Expected
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2028

Last Updated

April 20, 2026

Status Verified

April 1, 2026

Enrollment Period

2.8 years

First QC Date

August 20, 2025

Last Update Submit

April 17, 2026

Conditions

Keywords

Mast Cell Activation DisorderMDS/MPN Overlap SyndromeChronic Myelomonocytic LeukemiaHypermobility Syndrome DisorderPostural Orthostatic Tachycardia SyndromeEarly Onset Osteoporosis

Outcome Measures

Primary Outcomes (2)

  • Proportion of Participants in Cohort 1 with KIT D816V Mutation in Peripheral Blood as Measured by Digital Droplet Polymerase Chain Reaction (ddPCR)

    Day 1

  • Proportion of Participants in Cohort 1 with KIT D816V Mutation in Peripheral Blood as Measured by Ultra-sensitive KIT D816V by Rolling Circle Amplification (RCA) Assay

    Day 1

Study Arms (3)

Cohort 1

Participants with symptoms of mast cell activation (SMAC).

Other: Screening

Cohort 2

Participants with select diseases with suspected clonal mast cell involvement.

Other: Screening

Cohort 3

Participants with chronic myelomonocytic leukemia or myelodysplastic syndrome/myeloproliferative neoplasm not otherwise specified.

Other: Screening

Interventions

After providing informed consent and relevant medical history data, samples will be collected from participants with suspected clonal mast cell disease.

Cohort 1Cohort 2Cohort 3

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Adult participants with suspected clonal mast cell disease

You may qualify if:

  • \. SMAC-A
  • Documented anaphylaxis due to Hymenoptera venom with cardiovascular symptoms or
  • Documented anaphylaxis without known trigger(s) or allergen(s) warranting hospitalization, emergency room visit, and/or epinephrine with cardiovascular symptoms 2. SMAC-B
  • Episodic or recurrent signs and symptoms consistent with mast cell activation without known triggers or allergens in at least 2 of the following organ systems: skin, respiratory/naso-ocular, gastrointestinal tract, or cardiovascular.
  • Any clinical response on one or more optimally dosed therapies intended to mitigate mast cell mediators, as determined by the Investigator.
  • Cohort 2 participants must have confirmed, known diagnosis of 1 of the following criteria:
  • Either hypermobile Ehlers-Danlos syndrome or documented history of hypermobility spectrum disorder.
  • Postural orthostatic tachycardia syndrome with one or more systemic symptoms.
  • Early onset (≤50 years old) osteoporosis or osteopenia.
  • Cohort 3 participants must have documented diagnosis of 1 of the following, according to World Health Organization 5th edition criteria: chronic myelomonocytic leukemia or myelodysplastic syndrome/myeloproliferative neoplasm not otherwise specified.

You may not qualify if:

  • Participants previously diagnosed with any of the following:
  • Monoclonal mast cell activation syndrome with a known KIT mutation
  • Cutaneous mastocytosis only (that is, no documentation of systemic mast cell disease via bone marrow biopsy)
  • Any subtype of systemic mastocytosis
  • Mast cell sarcoma
  • Cohort 2 only: Osteopenia or osteoporosis attributed to known genetic, endocrine, nutritional, or other medical conditions.
  • Note: Additional protocol-defined criteria apply.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

AllerVie Clinical Research

Birmingham, Alabama, 35209, United States

RECRUITING

O'Neal Comprehensive Cancer Center at the UAB

Birmingham, Alabama, 35294, United States

RECRUITING

Kaiser Permanente San Diego

San Diego, California, 92123, United States

RECRUITING

Allergy & Asthma Clinical Research of the Bay Area

Walnut Creek, California, 94598, United States

RECRUITING

Allergy, Asthma, & Immunology Associates of Tampa Bay

Tampa, Florida, 33613, United States

RECRUITING

Emory University

Atlanta, Georgia, 30322, United States

RECRUITING

Midwest Allergy Sinus Asthma

Normal, Illinois, 61761, United States

RECRUITING

Tulane University School of Medicine

New Orleans, Louisiana, 70112, United States

RECRUITING

AllerVie Health

Glenn Dale, Maryland, 20769, United States

RECRUITING

Barnes-Jewish West County Hospital

St Louis, Missouri, 63141, United States

RECRUITING

Somnos Clinical Research

Lincoln, Nebraska, 68510, United States

RECRUITING

Mount Sinai Hospital

New York, New York, 10029, United States

RECRUITING

The University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

RECRUITING

Allergy, Asthma & Clinical Research Center

Oklahoma City, Oklahoma, 73120, United States

RECRUITING

Allergy & Clinical Immunology Associates

Pittsburgh, Pennsylvania, 15241, United States

RECRUITING

Care Access Research

Warwick, Rhode Island, 02886, United States

RECRUITING

AIR Care

Dallas, Texas, 75231, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Blood, Serum, Tissue

MeSH Terms

Conditions

MastocytosisLeukemia, Myelomonocytic, ChronicPostural Orthostatic Tachycardia Syndrome

Interventions

Mass Screening

Condition Hierarchy (Ancestors)

Neoplasms, Connective TissueNeoplasms, Connective and Soft TissueNeoplasms by Histologic TypeNeoplasmsMast Cell Activation DisordersImmune System DiseasesLeukemia, MyeloidLeukemiaMyelodysplastic-Myeloproliferative DiseasesBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsOrthostatic IntolerancePrimary DysautonomiasAutonomic Nervous System DiseasesNervous System Diseases

Intervention Hierarchy (Ancestors)

Diagnostic Techniques and ProceduresDiagnosisHealth SurveysSurveys and QuestionnairesData CollectionEpidemiologic MethodsInvestigative TechniquesDiagnostic ServicesPreventive Health ServicesHealth ServicesHealth Care Facilities Workforce and ServicesHealth Care Evaluation MechanismsQuality of Health CareHealth Care Quality, Access, and EvaluationPublic HealthEnvironment and Public HealthPublic Health Practice

Central Study Contacts

Blueprint Medicines, EU Contact

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 20, 2025

First Posted

August 27, 2025

Study Start

October 17, 2025

Primary Completion (Estimated)

July 31, 2028

Study Completion (Estimated)

October 31, 2028

Last Updated

April 20, 2026

Record last verified: 2026-04

Locations