NCT00044122

Brief Summary

This study will examine growth factors that promote and inhibit mast cell proliferation resulting in mastocytosis, a disease of excessive mast cells in the body. These cells can release chemicals that cause itching, blisters, flushing, bone pain and abdominal pain. Patients up to 80 years of age with mastocytosis may be eligible for this 1-day study. Participants will have one visit at NIH lasting up to 8 hours, during which they will undergo the following tests and procedures:

  • Medical history and physical examination.
  • Laboratory studies, if medically indicated.
  • Blood tests to identify genetic changes important in the growth, development, and functioning of mast cells.
  • Bone marrow aspiration and biopsy. For the bone marrow procedure, the skin over the hipbone and the outer surface of the bone itself are numbed with local anesthesia. Then, a special needle is inserted into the hipbone and about 1 tablespoon of bone marrow is drawn into a syringe. Another needle is inserted into the same area to collect a small piece of the bone marrow. Additional procedures may include allergen testing, urinalysis, and 24-hour urine collection. Participants will receive an evaluation of their mastocytosis.

Trial Health

75
On Track

Trial Health Score

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

Enrollment
600

participants targeted

Target at P75+ for all trials

Geographic Reach
1 country

1 active site

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

First Submitted

Initial submission to the registry

August 17, 2002

Completed
2 days until next milestone

First Posted

Study publicly available on registry

August 19, 2002

Completed
1 month until next milestone

Study Start

First participant enrolled

September 18, 2002

Completed
Last Updated

April 29, 2026

Status Verified

August 29, 2025

First QC Date

August 17, 2002

Last Update Submit

April 28, 2026

Conditions

Keywords

MastocytosisNatural History

Outcome Measures

Primary Outcomes (1)

  • To obtain normal and neoplastic human mast cells from the bone marrow and peripheral blood of patients with mastocytosis in order to study the regulation of the proliferation and survival of these cells, and to assess the extent and classificati...

    continuing collection of cells from subjects with mastocytosis for ongoing experimentation in laboratory

    Patients return to NIH as necessary

Study Arms (4)

Adult Relatives

Relatives of patient with mastocytosis

Adults with Mastocytosis

Adults with documented mastocytosis

Pediatric Patients with Mastocytosis

Pediatric patients with documented mastocytosis

Pediatric Relatives

Pediatric relatives of patients with mastocytosis

Eligibility Criteria

Age1 Day - 80 Years
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

primary clinical

You may qualify if:

  • Participants with mastocytosis zero to 80 years of age may participate in telehealth visits, and two to 80 years of age may participate on-site at NIH Clinical Center.
  • Histologic evidence of increased mast cell number by bone marrow and/or skin biopsy or documentation of mastocytosis in the skin
  • supported with a photograph of diagnostic skin lesions
  • Must be under the care of a primary care physician to be enrolled.
  • Ability to provide informed consent.

You may not qualify if:

  • Anemia with hemoglobin less than 8 g/dL, hematocrit less than 24.
  • Any condition that in the opinion of the investigator contraindicates participation in this study.
  • Two to 80 years of age.
  • A biological relative without the diagnosis of mastocytosis by skin examination or histologic evidence in a skin or bone marrow biopsy
  • Participant has a primary medical care provider outside the NIH
  • Ability to provide informed consent.
  • Any condition that in the opinion of the investigator contraindicates participation in this study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Publications (5)

  • Carter MC, Metcalfe DD, Komarow HD. Mastocytosis. Immunol Allergy Clin North Am. 2014 Feb;34(1):181-96. doi: 10.1016/j.iac.2013.09.001. Epub 2013 Oct 7.

    PMID: 24262698BACKGROUND
  • Chan EC, Bai Y, Kirshenbaum AS, Fischer ER, Simakova O, Bandara G, Scott LM, Wisch LB, Cantave D, Carter MC, Lewis JC, Noel P, Maric I, Gilfillan AM, Metcalfe DD, Wilson TM. Mastocytosis associated with a rare germline KIT K509I mutation displays a well-differentiated mast cell phenotype. J Allergy Clin Immunol. 2014 Jul;134(1):178-87. doi: 10.1016/j.jaci.2013.12.1090. Epub 2014 Feb 28.

    PMID: 24582309BACKGROUND
  • Cruse G, Metcalfe DD, Olivera A. Functional deregulation of KIT: link to mast cell proliferative diseases and other neoplasms. Immunol Allergy Clin North Am. 2014 May;34(2):219-37. doi: 10.1016/j.iac.2014.01.002. Epub 2014 Mar 12.

    PMID: 24745671BACKGROUND
  • Krausfeldt LE, Cao V, Rodrigues R, Henderson WA, Eisch R, Scott LM, Metcalfe DD, Komarow HD. Evidence for dysbiosis in the gut microbiome of patients with systemic mastocytosis. J Allergy Clin Immunol Glob. 2025 Oct 9;5(1):100578. doi: 10.1016/j.jacig.2025.100578. eCollection 2026 Jan.

  • Wilson TM, Maric I, Simakova O, Bai Y, Chan EC, Olivares N, Carter M, Maric D, Robyn J, Metcalfe DD. Clonal analysis of NRAS activating mutations in KIT-D816V systemic mastocytosis. Haematologica. 2011 Mar;96(3):459-63. doi: 10.3324/haematol.2010.031690. Epub 2010 Dec 6.

Related Links

MeSH Terms

Conditions

Mastocytosis

Condition Hierarchy (Ancestors)

Neoplasms, Connective TissueNeoplasms, Connective and Soft TissueNeoplasms by Histologic TypeNeoplasmsMast Cell Activation DisordersImmune System Diseases

Study Officials

  • Hirsh D Komarow, M.D.

    National Institute of Allergy and Infectious Diseases (NIAID)

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Hye Jeong C Bolan, R.N.

CONTACT

Hirsh D Komarow, M.D.

CONTACT

Study Design

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

Study Record Dates

First Submitted

August 17, 2002

First Posted

August 19, 2002

Study Start

September 18, 2002

Last Updated

April 29, 2026

Record last verified: 2025-08-29

Data Sharing

IPD Sharing
Will not share

Locations