Relationship Between Circulating Sclerostin and Bone Lesions in Patients With Mastocytosis

May 28, 2024 updated by: Aneta Szudy Szczyrek, Medical University of Lublin

Role of Sclerostin in Mastocytosis Bone Disease

Mastocytosis is very rare and highly heterogeneous group of disorders, characterized by the accumulation of clonal mast cells which can infiltrate several organs and tissues.

Bones are the most frequent localization of systemic mastocytosis. The aim of our research was to explain the potential role of sclerostin in the pathogenesis of bone disease in mastocytosis.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Mastocytosis is a heterogeneous group of disorders, characterized by the accumulation of clonal mast cells which can infiltrate several organs, such as the skin, bone marrow or liver. The skeleton is the most frequent localization of systemic mastocytosis (SM). Bone involvement occurs in approximately 70% of SM patients. Pathogenesis of mastocytosis bone disease is poorly understood.

The aim of our research is to explain the potential role of sclerostin, a recently discovered bone tissue protein, in the pathogenesis of bone changes in patients with mastocytosis.

The study group consists of adult patients with mastocytosis divided according to their clinical variants of disease (aggressive systemic mastocytosis - ASM, systemic mastocytosis with an associated hematological neoplasms SM-AHN, smouldering systemic mastocytosis - SSM, indolent systemic mastocytosis - ISM and cutaneous mastocytosis - CM; and group of healthy volunteers.

The concentration of sclerostin, bioactive sclerostin and expression of the SOST gene in human plasma and HMC-1.2 human mast cell culture supernatants is assessed. The Real-Time PCR method is used to evaluate the expression of sclerostin at the mRNA level, while the concentration of the sclerostin protein and its bioactive form is assessed using the enzyme immunoassay ELISA method. The obtained results are correlated with selected demographic, clinical, laboratory and radiological findings. Low-dose CT scan is used to assess bone changes.

These preliminary results could serve that sclerostin may be a new therapeutic target in patients with mastocytosis.

Study Type

Observational

Enrollment (Estimated)

50

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Poland
    • Lubelskie
      • Lublin, Lubelskie, Poland, 20-081
        • Recruiting
        • Department of Hematooncology and Bone Marrow Transplantation
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

Yes

Sampling Method

Probability Sample

Study Population

The study group consists of adult patients diagnosed with mastocytosis, divides according to the clinical variants: aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), indolent systemic mastocytosis (ISM), smoldering systemic mastocytosis (SSM) and cutaneous mastocytosis (CM).

The diagnosis of mastocytosis should be established based on biopsy of the affected organ (bone marrow trephine biopsy, skin affected by the disease), following the 2022 WHO classification. The control group comprised 30 healthy volunteers matched with the study group in terms of age and gender.

Description

Inclusion Criteria:

  • Age > 18 years
  • Mastocytosis defined according to WHO criteria
  • Known KIT mutation status

Exclusion Criteria:

  • History of organ transplant
  • Inability to give informed consent
  • Pregnancy, Breastfeeding
  • Vulnerable Patient, defined as: patient with another uncontrolled severe disease; patient under juridical protection

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
plasma sclerostin measurements (in pmol/l) SOST gene expression by Real-Time PCR dimensions of osteolytic lesions on low-dose computed tomography (in mm) dimensions of osteosclerotic lesions on low-dose computed tomography (in mm)
Time Frame: 1 year

The Primary Outcome Measures concern:

  • the measurements of plasma levels of sclerostin and its bioactive form in patients with mastocytosis and healthy volunteers
  • the measurements of levels of sclerostin and its bioactive form in HMC-1.2 human mast cells unstimulated and stimutaled with Il-6
1 year
dimensions of osteolytic lesions (in mm)
Time Frame: 1 year

The Primary Outcome Measures concern:

- the measurements of the dimensions of osteolytic bone lesions on low-dose computed tomography in patients with mastocytosis
1 year
dimensions of osteosclerotic lesions (in mm)
Time Frame: 1 year

The Primary Outcome Measures concern:

- the measurements of the dimensions of osteosclerotic bone lesions on low-dose computed tomography in patients with mastocytosis
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Medical University of Lublin

Investigators

  • Principal Investigator: Aneta A Szudy-Szczyrek, MD., PhD., Medical University of Lublin

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 1, 2019

Primary Completion (Actual)

May 1, 2024

Study Completion (Estimated)

December 31, 2026

Study Registration Dates

First Submitted

May 6, 2024

First Submitted That Met QC Criteria

May 28, 2024

First Posted (Actual)

June 3, 2024

Study Record Updates

Last Update Posted (Actual)

June 3, 2024

Last Update Submitted That Met QC Criteria

May 28, 2024

Last Verified

May 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SCLERMAST01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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