Bone Marrow Failure SyndromeEMBRACENCT04819607
The Evaluating Multidisciplinary Bone Marrow Failure Care in Bone Marrow Failure and Related Disorders.
An observational study of Bone Marrow Failure Syndrome and Aplastic Anemia, run by Peter MacCallum Cancer Centre, Australia at 1 site in Australia.
Trial summary
This is a prospective hybrid implementation-effectiveness study of a model of care for patients with bone marrow failure syndromes and inherited predisposition to haematological malignancy that includes comprehensive diagnostic genomic evaluation, multidisciplinary case review, provision of clinical care including… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Bone Marrow Failure Syndrome, Aplastic Anemia
- What is being tested?
- model of care
- How many people
- 100 participants
- Where
- 1 siteAustralia
- Study dates
- 9 Jun 2021to Apr 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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
- No healthy volunteers
- Adult, Older adult
Study population: Patients with a diagnosis of inherited bone marrow disorder (IBMD) including traditional inherited bone marrow failure syndromes (BMFS) and germline predisposition to haematological malignancy who meet all the inclusion criteria will be eligible for participation in this study.
Who may be able to join
- Patients must meet all the following criteria for study entry:
- Age > or = 18 years at enrolment
- Able to give informed consent (or responsible family member able to give informed consent)
- Patient is receiving care under the institutional guidelines/stream model of care
Who cannot join
- 1. Patient not eligible for management under the model of care
Trial locations and contacts
1 site in 1 country.
Australia1 site, 1 recruiting
- Recruiting
Peter MacCallum Cancer Centre
Melbourne, Victoria, 3000
Lucy Fox
About this study
Official title: The Evaluating Multidisciplinary Bone Marrow Failure Care in Bone Marrow Failure and Related Disorders.(The EMBRACE Study)
This is a prospective hybrid implementation-effectiveness study of a model of care for patients with bone marrow failure syndromes and inherited predisposition to haematological malignancy that includes comprehensive diagnostic genomic evaluation, multidisciplinary case review, provision of clinical care including from clinical haematologists, medical geneticists and genetic counsellors.
Read the detailed description
The inherited bone marrow disorders (IBMD) comprise both the traditional inherited bone marrow failure syndromes (BMFS) and the more recently described entity of germline predisposition to haematological malignancy. Patients with IBMD often present highly complex health issues that are unique and not commonly encountered. As a consequence, clinicians may feel less confident in managing these unique issues given their lack of frequent exposure to this patient group. Recent advances in genomics have resulted in more accurate diagnosis of IBMD and their subtyping and have resulted in a rapidly evolving field with multiple opportunities to improve patient outcomes.
Stage 1 of this study evaluated and quantified the current challenges faced by 3 stakeholder groups, patients with IBMD, patient advocates and nationally, clinicians who care for IBMD patients. The results demonstrated that the challenges described in Stage 1 may be overcome by instigating a multidisciplinary approach to care for BMF patients. Interventions designed to address the challenges identified in stage 1 form the model of multidisciplinary care to be implemented in the Peter MacCallum Cancer Centre/ Royal Melbourne Hospital clinic. The model of care involves:
- Provide access to genetic testing with expert result interpretation
- Presentation of clinical information and relevant investigations (including genetic) at a multidisciplinary team meeting involving haematologists, molecular haematopathologists, clinical genetics, genetic counsellors, scientists and specialised nurses.
- Provision of a suggested care plan to clinician
- Genetic counselling and provision of written disease specific information including details of suggested care plan to patient
- Genetic counselling and provision of written information and access to testing for at risk family members
- Dedicated team focused on many individually rare diseases with a demonstrated focus on provision of comprehensive care
- Opportunity to participate in local and international research
The EMBRACE study will focus on evaluating the model of care.
- Conditions
- Bone Marrow Failure Syndrome, Aplastic Anemia
- Being tested
- model of care
- Medical subject headings
- Bone Marrow Failure Disorders, Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
model of careOther
Multidisciplinary model of care for BMFS patients.
| Study group | Role | What they receive |
|---|---|---|
| BMFS patients | Patients with BMFS attending Peter MacCallum Cancer Centre haematology clinic. Receives: Other: model of care |
What is being measured
The primary outcome is the main question the study is designed to answer.
to assess the effectiveness and implementation of the model of care through a mixture of objective metrics and patient /physician reported outcomes
Effectiveness - patient/physician reported outcomes of satisfaction with model of care as measured by questionnaire Implementation - compliance with of model as measured by audit against pre-specified key performance indicators
Measured: 12 months
Secondary outcomes (2)
To assess longitudinal quality of life assessment in patients being treated with the model
Longitudinal quality of life assessment in patients being treated with the model measured by the EORTC QLQ-C30
Measured: 12 months
To quantitate healthcare usage of patients within the model
Quantitate healthcare usage of patients within the model measured by questionnaire
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2021
- Study start9 Jun 2021
- Primary completionApr 2023 (estimated)
- Study completionApr 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bone Marrow Failure SyndromeStudies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peter MacCallum Cancer Centre, Australia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Piers Blombery, MBBS(Hons), Principal investigator, Peter MacCallum Cancer Centre, Australia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04819607
- Sponsor's protocol ID
- 21/44
- Acronym
- EMBRACE
- Status last verified
- Aug 2021
- First submitted
- 24 Mar 2021
- Last updated
- 23 Aug 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Evaluating Multidisciplinary Bone Marrow Failure Care in Bone Marrow Failure and Related Disorders.(The EMBRACE Study)
- ClinicalTrials.gov ID
- NCT04819607
- Acronym / sponsor ID
- EMBRACE / 21/44
- Conditions
- Bone Marrow Failure Syndrome, Aplastic Anemia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Peter MacCallum Cancer Centre, AustraliaUniversity, hospital or other
- Study start
- 9 Jun 2021 (actual)
- Primary completion
- Apr 2023 (estimated)
- Study completion
- Apr 2024 (estimated)
- First posted
- 29 Mar 2021 (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.
This is a prospective hybrid implementation-effectiveness study of a model of care for patients with bone marrow failure syndromes and inherited predisposition to haematological malignancy that includes comprehensive diagnostic genomic evaluation, multidisciplinary case review, provision of clinical care including from clinical haematologists, medical geneticists and genetic counsellors.
Detailed description
The inherited bone marrow disorders (IBMD) comprise both the traditional inherited bone marrow failure syndromes (BMFS) and the more recently described entity of germline predisposition to haematological malignancy. Patients with IBMD often present highly complex health issues that are unique and not commonly encountered. As a consequence, clinicians may feel less confident in managing these unique issues given their lack of frequent exposure to this patient group. Recent advances in genomics have resulted in more accurate diagnosis of IBMD and their subtyping and have resulted in a rapidly evolving field with multiple opportunities to improve patient outcomes.
Stage 1 of this study evaluated and quantified the current challenges faced by 3 stakeholder groups, patients with IBMD, patient advocates and nationally, clinicians who care for IBMD patients. The results demonstrated that the challenges described in Stage 1 may be overcome by instigating a multidisciplinary approach to care for BMF patients. Interventions designed to address the challenges identified in stage 1 form the model of multidisciplinary care to be implemented in the Peter MacCallum Cancer Centre/ Royal Melbourne Hospital clinic. The model of care involves:
- Provide access to genetic testing with expert result interpretation
- Presentation of clinical information and relevant investigations (including genetic) at a multidisciplinary team meeting involving haematologists, molecular haematopathologists, clinical genetics, genetic counsellors, scientists and specialised nurses.
- Provision of a suggested care plan to clinician
- Genetic counselling and provision of written disease specific information including details of suggested care plan to patient
- Genetic counselling and provision of written information and access to testing for at risk family members
- Dedicated team focused on many individually rare diseases with a demonstrated focus on provision of comprehensive care
- Opportunity to participate in local and international research
The EMBRACE study will focus on evaluating the model of care.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BMFS patients | – | Other: model of carePatients with BMFS attending Peter MacCallum Cancer Centre haematology clinic. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with a diagnosis of inherited bone marrow disorder (IBMD) including traditional inherited bone marrow failure syndromes (BMFS) and germline predisposition to haematological malignancy who meet all the inclusion criteria will be eligible for participation in this study.
Key inclusion criteria (4 listed)
- Age > or = 18 years at enrolment
- Able to give informed consent (or responsible family member able to give informed consent)
- Patient is receiving care under the institutional guidelines/stream model of care
Key exclusion criteria (1 listed)
- 1. Patient not eligible for management under the model of care
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 |
|---|---|---|
| model of careMultidisciplinary model of care for BMFS patients. | Other | BMFS patients |
Primary outcome
to assess the effectiveness and implementation of the model of care through a mixture of objective metrics and patient /physician reported outcomes
Time frame: 12 months
Effectiveness - patient/physician reported outcomes of satisfaction with model of care as measured by questionnaire Implementation - compliance with of model as measured by audit against pre-specified key performance indicators
2 secondary outcomes
To assess longitudinal quality of life assessment in patients being treated with the model
Time frame: 12 months
To quantitate healthcare usage of patients within the model
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2021.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Piers Blombery, MBBS(Hons) principal investigator
Peter MacCallum Cancer Centre, Australia
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 Bone Marrow Failure Syndrome and Aplastic Anemia, run by Peter MacCallum Cancer Centre, Australia at 1 site in Australia.
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 4 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 23 Aug 2021. 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 Australia. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Mary Panjari, PhD (03 8559 5000, mary.panjari@petermac.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
to assess the effectiveness and implementation of the model of care through a mixture of objective metrics and patient /physician reported outcomes (time frame: 12 months). 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 23 Aug 2021.