CHAPTER: Clonal Haematopoiesis Assessment: Prevention, Treatment and Research

NCT ID: NCT07313059

Last Updated: 2025-12-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Total Enrollment

100 participants

Study Classification

OBSERVATIONAL

Study Start Date

2026-01-31

Study Completion Date

2033-11-30

Brief Summary

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People identified to have CH or thought to have possible CH due to unexplained low blood cell counts, including low red blood cells, white blood cells, or platelets will be asked to take part in the study.

Individuals who are confirmed to have CH and provide informed consent to participate in the study will have monitoring of their CH, assessment of the risk of heart diseases, blood cancers and personalised support. The researchers will also measure people's understanding of CH and how they feel after learning about CH.

Researchers will then record the relevant information from people with CH in a central database over time to track long-term health outcomes.

The information collected from the study will help create a blueprint for doctors to provide care for people with CH in the future, and guide further research into CH in Australia.

Participants will be asked to donate blood samples for the study for research purposes including CH monitoring and testing and also provide health information for the central database.

Detailed Description

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Clonal Haematopoiesis (CH) refers to the clonal outgrowth of a population of hematopoietic stem cells in the absence of haematologic neoplasms. The clonal cells share an acquired 'driver' mutation., and the presence of dysplasia and/or cytopenia.

CH is common in older populations, with a frequency of 10-30% in people above 70 years of age.

Most individuals with CH are asymptomatic; however, the condition increases the risk of multiple life-limiting complications, including myeloid neoplasms (MN), cardiovascular disease (CVD), cerebrovascular disease (CeVD) by 11.1, 1.4, and 2-fold, respectively. While CH is not yet treatable, identifying the condition may assist individuals in making informed decisions, including an individualised management plan for early detection of CH-associated complications. CH screening is not currently performed in routine clinical practice.

The optimal approach to surveillance for myeloid neoplasms in individuals with CH remains undefined. Some individuals undergo active surveillance with regular blood count monitoring to detect early progression to MN. Early detection of MN may also broaden the therapeutic window for individuals. Additionally, this allows individuals to connect with suitable clinical trials.

CVD is the most common CH-associated complication; hence CV (cardiovascular) risk modification is important both from an individual and public health standpoint.

CH screening may also refine the evaluation of individuals with unexplained cytopenia, as they may have underlying CH that has not been identified.

Given the uncertainties surrounding the identification and management, dedicated research clinics have been developed in the United States to evaluate the impact of screening of CH. These clinics provide a supportive environment to discuss the condition and engage multidisciplinary management. In addition to haematologists, individuals are supported by other specialities and allied health professionals. However, dedicated CH clinics are not yet available in Australia.

There is an urgent need for more research into CH as there are crucial knowledge gaps. This includes the role of CH screening and interventions to modify the biology and risks of CH-associated complications. Given the widespread use of next-generation sequencing (NGS) panels in Australia, it is likely that CH will be increasingly identified in routine clinical practice, creating a pool of individuals who may benefit from counselling and follow-up in a dedicated service.

This cohort study will establish a screening clinic and a prospectively characterised registry of individuals with CH to serve as the basis for clinical and translational studies in CH.

With consent, individuals with possible CH will undergo screening by molecular testing in the first dedicated multidisciplinary CH clinic in Australia whereby the health outcomes of participants with CH can be evaluated.

Conditions

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Clonal Hematopoiesis CCUS Clonal Cytopenia of Undetermined Significance Hematologic Disease and Disorders

Keywords

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Clonal Hematopoiesis CH CCUS

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

1. Aged 55 years and above
2. Confirmed CH or possible CH, with possible CH defined by:

a. Persistent, non-severe cytopenia, characterised by one or more of the following, present on at least 2 occasions, at least 4 months apart: i. Absolute neutrophil count (ANC) \< 1.8 x 109/L ii. Haemoglobin (Hb) \< 115 g/L in females, \< 135 g/L in males iii. Platelet count \< 150 x 109/L
3. Provision of written informed consent prior to any study-related assessments or procedures being carried out.

Exclusion Criteria

1. Severe cytopenia as defined by one or more of the following:

1. ANC \< 0.5 x109/L
2. Hb \< 80 g/L
3. Platelet count \< 50 x 109/L
2. Multilineage cytopenias:

a. Marked trilineage cytopenia with all the following present: i. ANC \< 1.0 x 109/L AND ii. Hb \< 110 g/L AND iii. Platelet count \< 100 x 109/L

b. Marked bilineage cytopenia, with two or more of the following present: i. ANC \< 1.0 x 109/L ii. Hb \< 110 g/L iii. Platelet count \< 100 x 109/L

Note: People with possible CH, with the above characteristics will be excluded from referral to the CH clinic and will instead have urgent investigation as an inpatient or in the haematology clinic. However, if no definite cause for cytopenia is identified, including CH, then individuals may be referred for CH screening at the CH clinic the discretion of the study PI
Minimum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Medical Research Future Fund

OTHER

Sponsor Role collaborator

Australian National University

OTHER

Sponsor Role collaborator

University of Auckland, New Zealand

OTHER

Sponsor Role collaborator

The Canberra Hospital

OTHER

Sponsor Role collaborator

Clinical Hub for Interventional Research (CHOIR)

OTHER_GOV

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Canberra Health Services

Canberra, Australian Capital Territory, Australia

Site Status

Countries

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Australia

Central Contacts

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Mark Polizzotto Professor

Role: CONTACT

Phone: +61000000000

Email: [email protected]

Facility Contacts

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Mark Polizzotto Professor

Role: primary

Other Identifiers

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24-CH-001

Identifier Type: -

Identifier Source: org_study_id