Use of Tamoxifen in Systemic Mastocytosis

Completed

• Conditions: Systemic Mastocytosis

• Registration Number: NCT01334996
• Lead Sponsor: Mayo Clinic

Brief Summary

In this study, the investigators will determine the utility of Tamoxifen, a non-cytotoxic agent, to improve quality of life, biochemical parameters, and bone marrow involvement in systemic mastocytosis patients having 1) up to 40% bone marrow infiltration by mast cells and/or 2) mediator-release symptoms which are not controlled by tolerated doses of standard "non-cytotoxic" medications regardless of the percentage bone marrow involvement by mastocytosis. The dose of Tamoxifen will be 40 mg/day and the duration of treatment will be for one year. Patients currently taking interferon alfa, imatinib mesylate, or cladribine will be excluded until these medications have been stopped.

Detailed Description

Not desired

Study Timeline

• Study Start: 2005-02
• Study First Submitted: 2007-12-25
• Study First Submitted QC: 2011-04-12
• Study First Posted: 2011-04-13
• Primary Completion: 2019-08-28
• Study Completion: 2019-08-28
• Status Verified: 2020-04
• Last Update Submitted: 2020-04-09
• Last Update Posted: 2020-04-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 1

Inclusion Criteria

• Systemic Mastocytosis

Read More

Exclusion Criteria

• Current treatment with Imatinib mesylate, cladribine or interferon alpha.

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Stability or reduction of the percent bone marrow involvement by mastocytosis, including stable mast cell morphology and phenotype.	1 year	up to 40% bone marrow infiltration by mast cells and/or 2) mediator-release symptoms which are not controlled by tolerated doses of standard "non-cytotoxic" medications regardless of the percentage bone marrow involvement by mastocytosis. The dose of Tamoxifen will be 40mg/day and the duration of treatment will be for one year.

Secondary Outcome Measures

Name	Time	Method
Stability or improvement in biochemical markers of systemic mastocytosis	1 year	Stability or improvement in biochemical markers of systemic mastocytosis (serum tryptase, calcitonin, urinary N-methyl histamine and prostaglandin F2 excretion/24 hours, liver function studies, lactic dehydrogenase, complete blood count with leukocyte differential)

Trial Locations

Locations (1)

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States