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Status unknownNCT04819607EMBRACEUpdated Aug 23, 2021

The Evaluating Multidisciplinary Bone Marrow Failure Care in Bone Marrow Failure and Related Disorders.

An observational study in Bone Marrow Failure Syndrome and Aplastic Anemia, sponsored by Peter MacCallum Cancer Centre, Australia. Status unknown at 1 site in Australia. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-08-23.

Primary conditionAnemia, AplasticRegistry: Bone Marrow Failure DisordersReclassifiedWhy we changed it
Registry
Bone Marrow Failure Disorders — the first condition term the registry derived for this study.
ClinicalTrials.gg
Anemia, Aplastic
Why
  1. The study's listed conditions name Anemia, Aplastic, a narrower term within Bone Marrow Failure Disorders.
Confidence
High: the new condition is named in the study's own listed conditions. Of 351 hand-checked studies reclassified this way, 68% were exactly right and 84% in the right disease family; the registry's first term was right for 5% (measured 2026-10-21). How we classify →
Wrong?
Tell us the study's main condition →

Sponsored by Peter MacCallum Cancer Centre, Australia · Observational

The sponsor has not verified this record recently (last verified Aug 2021), so the status shown — last known as Recruiting — may be out of date.
Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
100
Ages
18 Years and older
Sex
All
01

Study 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 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:

  1. Provide access to genetic testing with expert result interpretation
  2. 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.
  3. Provision of a suggested care plan to clinician
  4. Genetic counselling and provision of written disease specific information including details of suggested care plan to patient
  5. Genetic counselling and provision of written information and access to testing for at risk family members
  6. Dedicated team focused on many individually rare diseases with a demonstrated focus on provision of comprehensive care
  7. Opportunity to participate in local and international research

The EMBRACE study will focus on evaluating the model of care.

02

Conditions studied

  • Bone Marrow Failure Syndrome
  • Aplastic Anemia

Browse trials for

03

In context

Anemia, Aplastic Reclassified

371 studies on the registry are indexed under Anemia, Aplastic; 98 are open to participants now.

This study's planned enrollment of 100 is close to the median of 100 across 64 observational studies indexed under Anemia, Aplastic.

Browse Anemia, Aplastic studies →

Lead sponsor

Peter MacCallum Cancer Centre, Australia is the lead sponsor of 82 studies on the registry; 25 are open to participants now.

Compared within Anemia, Aplastic, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Bone Marrow Failure Disorders. The counts are of registry records indexed under each term, refreshed daily. How we classify →

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

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.

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

Patients must meet all the following criteria for study entry:

  1. Age > or = 18 years at enrolment
  2. Able to give (or responsible family member able to give informed consent)
  3. Patient is receiving care under the institutional guidelines/stream model of care

Exclusion criteria

Exclusion Criteria:

  1. Patient not eligible for management under the model of care
Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

05

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
100 participants (estimated)
Patient registry
No

Groups and cohorts

  • BMFS patients

    Patients with BMFS attending Peter MacCallum Cancer Centre haematology clinic.

    Other: model of care

Interventions

  • Othermodel of care

    Multidisciplinary model of care for BMFS patients.

06

What researchers measure

Primary outcomes

  1. 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

    Time frame: 12 months

Secondary outcomes

  1. 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

    Time frame: 12 months

  2. To quantitate healthcare usage of patients within the model

    Quantitate healthcare usage of patients within the model measured by questionnaire

    Time frame: 12 months

07

Study locations

1 of 1 sites recruiting
  • Peter MacCallum Cancer Centre
    Melbourne, Victoria 3000, Australia
    Recruiting
    • Lucy Fox · Sub investigator
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Jun 2021 (actual). Primary completion is estimated for Apr 2023, 22 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Mar 24, 2021
First posted
Mar 29, 2021
StartActual
Jun 9, 2021
Primary completionEst.
Apr 2023
CompletionEst.
Apr 2024
Last update
Aug 23, 2021
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Aug 23, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT04819607
Lead sponsor
Peter MacCallum Cancer Centre, Australia
Responsible party
Sponsor
First posted
Mar 29, 2021
Start date
Jun 9, 2021
Primary completion
Apr 2023 (estimated)
Completion
Apr 2024 (estimated)
Last update
Aug 23, 2021

Study contacts

Mary Panjari, PhD
Contact
mary.panjari@petermac.org
03 8559 5000
Piers Blombery, MBBS(Hons)
principal investigator · Peter MacCallum Cancer Centre, Australia

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in Aug 2021. You cannot join it, but the record below documents what was studied.

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Discussion

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