MastocytosisNCT06440148
Relationship Between Circulating Sclerostin and Bone Lesions in Patients With Mastocytosis
An observational study of Mastocytosis and Bones, run by Medical University of Lublin at 1 site in Poland.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Mastocytosis, Bones
- What is being tested?
- SCLEROSTIN
- How many people
- 50 participants
- Where
- 1 sitePoland
- Study dates
- 1 Sep 2019to 31 Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
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.
Who may be able to join
- Age > 18 years
- Mastocytosis defined according to WHO criteria
- Known KIT mutation status
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Poland1 site, 1 recruiting
- Recruiting
Department of Hematooncology and Bone Marrow Transplantation
Lublin, Lublin Voivodeship, 20-081
Aneta Szudy-Szczyrek, MD., PhD., aneta.szudy-szczyrek@umlub.pl
About this study
Official title: 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.
Read the 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.
- Conditions
- Mastocytosis, Bones
- Being tested
- SCLEROSTIN
- Keywords
- Bones, Mastocytosis, Osteolysis, Osteosclerosis, Sclerostin
- Medical subject headings
- Mastocytosis, Osteolysis, Osteosclerosis, Sclerosteosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SCLEROSTINBiological
Pathogenesis of mastocytosis bone disease
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
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
Measured: 1 year
dimensions of osteolytic lesions (in mm)
The Primary Outcome Measures concern:
- the measurements of the dimensions of osteolytic bone lesions on low-dose computed tomography in patients with mastocytosis
Measured: 1 year
dimensions of osteosclerotic lesions (in mm)
The Primary Outcome Measures concern:
- the measurements of the dimensions of osteosclerotic bone lesions on low-dose computed tomography in patients with mastocytosis
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jun 2024
- Study start1 Sep 2019
- Primary completion1 May 2024
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MastocytosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Retrospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Medical University of Lublin
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Aneta Szudy Szczyrek, Medical University of Lublin
- Investigators
- Aneta A Szudy-Szczyrek, MD., PhD., Principal investigator, Medical University of Lublin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06440148
- Sponsor's protocol ID
- SCLERMAST01
- Status last verified
- May 2024
- First submitted
- 6 May 2024
- Last updated
- 3 Jun 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Role of Sclerostin in Mastocytosis Bone Disease
- ClinicalTrials.gov ID
- NCT06440148
- Acronym / sponsor ID
- SCLERMAST01
- Conditions
- Mastocytosis, Bones
- Keywords
- Bones, Mastocytosis, Osteolysis, Osteosclerosis, Sclerostin
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Medical University of LublinUniversity, hospital or other
- Study start
- 1 Sep 2019 (actual)
- Primary completion
- 1 May 2024 (actual)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 3 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Case-control
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (3 listed)
- Age > 18 years
- Mastocytosis defined according to WHO criteria
- Known KIT mutation status
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SCLEROSTINPathogenesis of mastocytosis bone disease | Biological | – |
Primary outcome
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
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
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2024.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Aneta A Szudy-Szczyrek, MD., PhD. principal investigator
Medical University of Lublin
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mastocytosis and Bones, run by Medical University of Lublin at 1 site in Poland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. 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: looking for participants now, as of the sponsor's last update on 3 Jun 2024. 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 Poland. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Aneta Szudy-Szczyrek, MD., PhD. (+48815345468, aneta.szudy-szczyrek@umlub.pl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Jun 2024.