NCT04819607

Brief 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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
100

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Jun 2021

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 24, 2021

Completed
5 days until next milestone

First Posted

Study publicly available on registry

March 29, 2021

Completed
2 months until next milestone

Study Start

First participant enrolled

June 9, 2021

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2023

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2024

Completed
Last Updated

August 23, 2021

Status Verified

August 1, 2021

Enrollment Period

1.8 years

First QC Date

March 24, 2021

Last Update Submit

August 16, 2021

Conditions

Outcome Measures

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

    12 months

Secondary Outcomes (2)

  • To assess longitudinal quality of life assessment in patients being treated with the model

    12 months

  • To quantitate healthcare usage of patients within the model

    12 months

Study Arms (1)

BMFS patients

Patients with BMFS attending Peter MacCallum Cancer Centre haematology clinic.

Other: model of care

Interventions

Multidisciplinary model of care for BMFS patients.

BMFS patients

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • 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

You may not qualify if:

  • \. Patient not eligible for management under the model of care

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Peter MacCallum Cancer Centre

Melbourne, Victoria, 3000, Australia

RECRUITING

MeSH Terms

Conditions

Bone Marrow Failure DisordersAnemia, Aplastic

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesAnemia

Study Officials

  • Piers Blombery, MBBS(Hons)

    Peter MacCallum Cancer Centre, Australia

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 24, 2021

First Posted

March 29, 2021

Study Start

June 9, 2021

Primary Completion

April 1, 2023

Study Completion

April 1, 2024

Last Updated

August 23, 2021

Record last verified: 2021-08

Data Sharing

IPD Sharing
Will not share

Locations