NCT00001356

Brief Summary

This study will determine what growth factors are involved in promoting and inhibiting mastocytosis-an abnormal increase of mast cells in one or more organ systems. Mast cells release chemicals that can cause itching, blisters, flushing, bone pain, and abdominal pain. Little is known about the disease and there is no cure. Steroids and antihistamines can help reduce some symptoms. Patients from birth to 80 years of age with increased mast cells in at least one organ system may be eligible for this 3-year study. Family members may also be enrolled for genetic testing. Patients will be evaluated yearly at the NIH Clinical Center with the following tests and procedures:

  • Medical history and physical examination.
  • Blood samples.
  • Laboratory blood tests, as medically indicated.
  • Bone marrow aspiration and biopsy - For the bone marrow aspiration and biopsy, the back hipbone is punctured with a sterile needle. Five milliliters (1 teaspoon) of marrow is withdrawn through a syringe and a 1/2-inch piece of tissue is extracted with a special needle. The blood and bone marrow samples will be used for clinical care and for research to determine if mastocytosis is due to mast cell growth factors or genetic changes. Patients who require further evaluation and tests will have recommendations made to their primary physician. Any patient who requires immediate treatment will be admitted to the hospital. Standard medical treatment may include antihistamines for itching; steroids for severe abdominal symptoms such as cramping, diarrhea, and evidence of increased mast cells determined by an upper GI study; and adrenaline for anaphylactic shock. Patients who do not respond to conventional treatment may be offered participation in an experimental therapy study. Participating family members will have a medical history and a blood sample drawn to look for genetic abnormalities.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
300

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started May 1993

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
completed

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 Start

First participant enrolled

May 1, 1993

Completed
6.5 years until next milestone

First Submitted

Initial submission to the registry

November 3, 1999

Completed
1 day until next milestone

First Posted

Study publicly available on registry

November 4, 1999

Completed
2.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2002

Completed
Last Updated

March 4, 2008

Status Verified

May 1, 2002

First QC Date

November 3, 1999

Last Update Submit

March 3, 2008

Conditions

Mastocytosis

Keywords

IL-3Stem Cell FactorCytokinesCell DifferentiationReceptorsBone MarrowMastocytosis

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Subjects include children and adults from birth to 80 years of age. Participants must have histologic evidence of an increased mast cell number in at least one organ system. Must be willing to be seen at the NIH according to protocol guidelines. Routine medical care must be available through their referral physician. Patients with mastocytosis now followed at the NIH on protocol 88-I-0190 will be continued on this study.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (1)

National Institute of Allergy and Infectious Diseases (NIAID)

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

  • Kirshenbaum AS, Kessler SW, Goff JP, Metcalfe DD. Demonstration of the origin of human mast cells from CD34+ bone marrow progenitor cells. J Immunol. 1991 Mar 1;146(5):1410-5.

    PMID: 1704394BACKGROUND

  • Kirshenbaum AS, Goff JP, Kessler SW, Mican JM, Zsebo KM, Metcalfe DD. Effect of IL-3 and stem cell factor on the appearance of human basophils and mast cells from CD34+ pluripotent progenitor cells. J Immunol. 1992 Feb 1;148(3):772-7.

    PMID: 1370517BACKGROUND

  • Kettelhut BV, Metcalfe DD. Pediatric mastocytosis. J Invest Dermatol. 1991 Mar;96(3):15S-18S.

    PMID: 1705949BACKGROUND

MeSH Terms

Conditions

Mastocytosis

Condition Hierarchy (Ancestors)

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

Study Design

Study Type
observational
Sponsor Type
NIH

Study Record Dates

First Submitted

November 3, 1999

First Posted

November 4, 1999

Study Start

May 1, 1993

Study Completion

May 1, 2002

Last Updated

March 4, 2008

Record last verified: 2002-05

Locations

US(1)