AzTrials
AzTrials

MastocytosisNCT00001756

Study of Mast Cell Precursors

An observational study of Mastocytosis and Healthy Volunteer, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This study will investigate mast cell precursors that circulate in the blood. In a group of diseases collectively known as mastocytosis, mast cells accumulate in abnormal amounts in the skin, lymphoid tissues, bone marrow, liver and spleen. Read more about this study

Who is it for?
Ages 18 to 70 yearsHealthy volunteers can take part
Conditions the study is for
Mastocytosis, Healthy Volunteer
How many people
202 participants
Where
1 siteUnited States
Study dates
3 Dec 1997to 3 Nov 2022
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 to 70 years
  • All sexes
  • Healthy volunteers accepted
  • Adult, Older adult

Study population: Primary Clinical

  • SUBJECT INCLUSION CRITERIA:

Healthy Volunteers must:

  1. Be 18-70 years of age
  2. Be healthy
  3. Have adequate peripheral venous access
  4. Have normal renal function (creatinine less than or equal to 1.5mg/dL; less than or equal to 1 plus proteinuria)
  5. Have normal hepatic function (bilirubin less than or equal to 1.5 mg/dL)
  6. Have normal hematologic function (WBC greater than or equal to 3000/mm(3); granulocytes greater than or equal to 1500/mm(3) ; platelets greater than or equal to 175,000; hemoglobin greater than or equal to 12.5 g/dL)

Patients must:

  1. Be 18-70 years of age
  2. Have mast cell hyperplasia compatible with a diagnosis of systemic mastocytosis (applicable to systemic mastocytosis patients only) or other allergic, hematologic, or immunologic condition
  3. Have adequate peripheral venous access or be willing to have a central line placed.
  4. First be admitted as inpatients under an existing NIH protocol
  5. Have preserved renal function (creatinine less than or equal to 2 mg/dL; less than or equal to 2 plus proteinuria)
  6. Have preserved hepatic function (bilirubin less than or equal to 1.5 mg/dL)
  7. Have preserved hematologic function (WBC greater than or equal to 3000/mm(3); granulocytes greater than or equal to 1500/mm(3) ; platelets greater than or equal to 175,000; hemoglobin greater than or equal to 12.5 g/dL)

All female subjects of childbearing potential:

  1. May be enrolled if using effective contraception
  2. Have a negative serum or urine pregnancy test determined within 72 hours before beginning Plerixafor or G-CSF administration

SUBJECT EXCLUSION CRITERIA:

All subjects must not meet any of the following criteria:

Healthy Volunteers and patients must not:

  1. Have active bacterial, fungal or viral infections
  2. Have viral screens positive for HIV or hepatitis B or C
  3. Be pregnant or lactating
  4. Have a history of autoimmune disease such as rheumatoid arthritis, vasculitis, pyoderma gangrenosum or similar disorder
  5. Have any condition, which in the judgment of the investigator, might place the subject at undue risk

Healthy Volunteers with any of the following will be excluded:

  1. Splenomegaly, pulmonary fibrosis and other related conditions
  2. Use of any investigative drugs within the past 12 months
  3. Have a significant coagulation disorder

Systemic Mastocytosis and Mast Cell Related Condition Patients with any of the following will be excluded:

  1. Patients taking any other growth factors, cytokines or investigative drugs
  2. Patients who are hemodynamically unstable (blood pressure systolic of lower than 105 or diastolic lower than 65)

Trial locations and contacts

1 site in 1 country.

United States1 site

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892

Other trials in United States

About this study

Official title: The Characterization of CD34+ Derived Mast Cell Precursors

This study will investigate mast cell precursors that circulate in the blood. In a group of diseases collectively known as mastocytosis, mast cells accumulate in abnormal amounts in the skin, lymphoid tissues, bone marrow, liver and spleen. Some forms of mastocytosis have a generally good prognosis; for others, the prognosis is poorer. There is no known cure for any form of the disease. A better understanding of mast cells and how they respond to certain substances may provide insights that will lead to effective treatments for mastocytosis.

Patients with systemic mastocytosis and normal healthy volunteers between the ages of 20 and 60 may be eligible for this 8-day study. Participants will undergo the following procedures:

  • Day 1 Medical history, physical examination, and blood tests to assess general health status
  • Days 2 through 6 Daily injections under the skin of G-CSF a hormone that stimulates white blood cell production
  • Day 7 Leukapheresis a procedure for collecting large numbers of white blood cells. In leukapheresis, blood is drawn through a needle placed in an arm and channeled into a cell separator machine. The white cells are collected and the rest of the blood is returned to the body through a needle in the other arm. The procedure takes up to 3 hours.
  • Days 7 and 8 Blood draw (about 1 teaspoon) to monitor white blood cell counts.
Read the detailed description

The purpose of this protocol is to obtain large numbers of CD34+ cells from the peripheral blood of healthy volunteers and patients with systemic mastocytosis or other related allergic, hematological, and immunological conditions by leukapheresis for culture and characterization of mast cell progenitor cells and their response to various cytokines and anti-mitotic agents. Healthy volunteers and patients will be adults of both sexes from 18 to 70 years of age. Granulocyte colony stimulating factor (G-CSF) will be administered to healthy volunteers at dose of 10 mcg/kg/day as a subcutaneous dose daily for 5 days not to exceed 960mcg. Patients will receive Plerixafor at a dose of 0.24 mg/kg as a single subcutaneous dose not to exceed 24mg the night before leukapheresis as a mobilizing agent for CD34+ cells. In identified patients where leukapheresis yield is considered adequate without stimulation by the principal investigator, leukapheresis may proceed without stimulation. Healthy volunteers will undergo a single leukapheresis at day 7, and patients will undergo a single leukapheresis at day 3 or 4. This is not a therapeutic protocol and does not involve reinfusion of any manipulated cells, viruses or DNA constructs back into human subjects.

Keywords
Leukapheresis, Mastocytosis, Mast Cells, Normal Volunteer, Natural History, Systemic Mastocytosis
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.

Study groups in NCT00001756
Study groupRoleWhat they receive
Healthy VolunteersHealthy Volunteers
PatientsPatients have mast cell hyperplasia compatible with a diagnosis of systemic mastocytosis (applicable to systemic mastocytosis patients only) or other allergic, hematologic, orimmunologic condition.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Use of G-CSF administration healthy volunteers to mobilize and enhance CD34+ hematopoietic progenitor cell numbers into the peripheral blood in order to culture and characterize human mast cells, study and characterize CD34+ -derived human ma...

    Use of G-CSF administration healthy volunteers to mobilize and enhance CD34+ hematopoietic progenitor cell numbers into the peripheral blood in order to culture and characterize human mast cells, study and characterize CD34+ -derived human mast cells.

    Measured: 12/31/2029

  2. Collection of CD34+ cells with or without use of Plerixafor administration in patients with systemic mastocytosis and other related allergic, hematological and immunological conditions to mobilize and enhance CD34+ cells into the peripheral b...

    Collection of CD34+ cells with or without use of Plerixafor administration in patients with systemic mastocytosis and other related allergic, hematological and immunological conditions to mobilize and enhance CD34+ cells into the peripheral blood to culture and learn how mast cells contribute to these disease states.

    Measured: 12/31/2029

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Nov 1999 (estimated)
  2. Study start3 Dec 1997
  3. Primary completion1 Nov 2021
  4. Study completion3 Nov 2022

Resources

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
Case-control
Time perspective
Prospective
Enrolment
202 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00001756
Sponsor's protocol ID
980027
Other IDs
98-I-0027
Status last verified
Nov 2022
First submitted
3 Nov 1999
Last updated
7 Nov 2022
Results posted
No

Publications

  • Schwinger W, Mache C, Urban C, Beaufort F, Toglhofer W. Single dose of filgrastim (rhG-CSF) increases the number of hematopoietic progenitors in the peripheral blood of adult volunteers. Bone Marrow Transplant. 1993 Jun;11(6):489-92.

    PubMed 8334430

  • Metcalfe DD. Classification and diagnosis of mastocytosis: current status. J Invest Dermatol. 1991 Mar;96(3 Suppl):2S-4S; discussion 4S, 60S-65S. doi: 10.1111/1523-1747.ep12468882.

    PubMed 16799601

  • Falduto GH, Pfeiffer A, Zhang Q, Yin Y, Metcalfe DD, Olivera A. A Critical Function for the Transcription Factors GLI1 and GLI2 in the Proliferation and Survival of Human Mast Cells. Front Immunol. 2022 Feb 16;13:841045. doi: 10.3389/fimmu.2022.841045. eCollection 2022.

    PubMed 35251038

  • 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.

    PubMed 21134978

  • Prussin C, Lee J, Foster B. Eosinophilic gastrointestinal disease and peanut allergy are alternatively associated with IL-5+ and IL-5(-) T(H)2 responses. J Allergy Clin Immunol. 2009 Dec;124(6):1326-32.e6. doi: 10.1016/j.jaci.2009.09.048.

    PubMed 20004787

Questions about this trial

What is this clinical trial studying?

An observational study of Mastocytosis and Healthy Volunteer, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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 7 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. 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?

Use of G-CSF administration healthy volunteers to mobilize and enhance CD34+ hematopoietic progenitor cell numbers into the peripheral blood in order to culture and characterize human mast cells, study and characterize CD34+ -derived human ma... (time frame: 12/31/2029). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00001756, which also lists 5 linked publications.

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 7 Nov 2022.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in