Skip to main content
OpenTrials
Recruiting

NCT Number: NCT06440148

Relationship Between Circulating Sclerostin and Bone Lesions in Patients With Mastocytosis

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Mastocytosis Bone Diseases Bone Diseases, Developmental Bone Resorption Bones Immune System Diseases Mast Cell Activation Disorders Musculoskeletal Diseases Neoplasms Neoplasms by Histologic Type Neoplasms, Connective Tissue Neoplasms, Connective and Soft Tissue Osteochondrodysplasias Osteolysis Osteosclerosis Sclerosteosis

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Department of Hematooncology and Bone Marrow Transplantation

Lublin, Lublin Voivodeship, 20-081, Poland

Location status: Recruiting

Location contact

Aneta Szudy-Szczyrek, MD., PhD.

CONTACT

[email protected]

About this study

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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

SCLEROSTIN

Biological

Pathogenesis of mastocytosis bone disease

Primary outcomes

  1. 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

  2. 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

  3. 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

Study contacts

Contact information is provided by the study sponsor or research team.

Aneta Szudy-Szczyrek, MD., PhD.

CONTACT

[email protected]

+48815345468

Sponsors and collaborators

Lead sponsor

Medical University of Lublin

Other

Registry information

Official study title

Role of Sclerostin in Mastocytosis Bone Disease

Important dates

Study start
2019
Primary completion
2024
Study completion
2026
First posted
Jun 3, 2024
Registry last updated
Jun 3, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.