Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12624001318572
Ethics application status
Approved
Date submitted
7/10/2024
Date registered
31/10/2024
Date last updated
31/10/2024
Date data sharing statement initially provided
31/10/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
New Frontiers 2 (NF2): Clinical Registry and Biobank for Patients with Neurofibromatosis Type 2-Related Schwannomatosis and Other Rare Forms of Schwannomatosis (SWN)
Scientific title
New Frontiers 2 (NF2): Clinical Registry and Biobank for Patients with Neurofibromatosis Type 2-Related Schwannomatosis and Other Rare Forms of Schwannomatosis (SWN)
Secondary ID [1] 313068 0
None
Universal Trial Number (UTN)
Trial acronym
New Frontiers 2
Linked study record

Health condition
Health condition(s) or problem(s) studied:
NF2-related schwannomatosis 335298 0
Neurofibromatosis type 2 335299 0
Condition category
Condition code
Human Genetics and Inherited Disorders 331872 331872 0 0
Other human genetics and inherited disorders
Cancer 331873 331873 0 0
Brain
Neurological 332052 332052 0 0
Other neurological disorders

Intervention/exposure
Study type
Observational
Patient registry
True
Target follow-up duration
5
Target follow-up type
Years
Description of intervention(s) / exposure
Neurofibromatosis Type 2 (NF2) is a rare genetic disorder caused by mutations in the NF2 gene, leading to the development of benign tumors on nerves, including vestibular schwannomas, meningiomas, and ependymomas. Typically presenting in early adulthood, NF2 is associated with significant morbidity, including hearing loss, vision impairment, and neurological deficits, greatly affecting quality of life. Due to its rarity, treatment options are limited to symptomatic management, and there is a pressing need for real-world data through clinical registries to better understand the disease and inform future research efforts.

New Frontiers 2 is a non-interventional clinical registry and virtual biobank designed to understand the treatment and outcomes of patients with Neurofibromatosis Type 2 related schwannomatosis (NF2) and other rare forms of schwannomatosis (SWN) in routine clinical practice at participating hospitals. The clinical registry will help identify the characteristics and clinical outcomes of patients with SWN and enable comparisons across multiple centres. In addition, the virtual biobank will support a wide range of research projects, including quality assurance audits, registry-based clinical trials, and translational research.

Data captured in the clinical registry will include patient presentation, disease course, surgical interventions, systemic therapies, and details of multi-disciplinary management. Biospecimens such as archival tumour tissue, cerebrospinal fluid (CSF), and blood may be collected from tissue banks or anatomical pathology departments. Only leftover biomaterial from standard-of-care procedures will be used in this study, no additional procedures are performed. This is a non-interventional study.

Authorised study personnel will handle the collection of biospecimens and clinical data. No input beyond standard care will be required from participants. Requests for tissue will be made to relevant tissue custodians, such as biobanks or anatomical pathology departments where the biospecimens are held, and clinical data will be sourced from patient medical records by site personnel.

The target follow-up duration of the study is annually over 5 years.
Intervention code [1] 329619 0
Not applicable
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 339485 0
Number of patients registered on the New Frontiers 2 clinical registry with associated clinical data collected.
Timepoint [1] 339485 0
Assessed once per year for five years from first enrolment.
Secondary outcome [1] 440249 0
Tumour types identified, including sub-types based on genetic profiling, identified among patients with single or multiple tumours.
Timepoint [1] 440249 0
Assessed once per year for five years from first enrolment.
Secondary outcome [2] 440250 0
Identification of genetic alterations contributing to tumour formation and progression in the patient population.
Timepoint [2] 440250 0
Ongoing, with final analysis at study completion.
Secondary outcome [3] 440251 0
Number of registry-based translational projects and clinical trials facilitated by the New Frontiers 2 registry.
Timepoint [3] 440251 0
Assessed once per year for five years from first enrolment.
Secondary outcome [4] 440765 0
Types of treatment strategies used; including surgical interventions, systemic therapies, radiotherapy, and audiology results. This will be assessed as a composite outcome.
Timepoint [4] 440765 0
Assessed once per year for five years from first enrolment.
Secondary outcome [5] 440959 0
Prevalence of family history of cancer or NF-2 related schwannomatosis in the participant population.
Timepoint [5] 440959 0
Assessed once per year for five years from first enrolment.
Secondary outcome [6] 440960 0
Composite measure of clinical outcomes by progression-free survival (PFS) and overall survival (OS) in patients with NF2-related schwannomatosis.
Timepoint [6] 440960 0
Ongoing, with final analysis at study completion.

Eligibility
Key inclusion criteria
1) Fulfil the diagnosis of NF2-associated Schwannomatosis*
2) The patient and/or their parent/guardian (if the patient is under 18 years of age) must be able to provide written consent for participation in study activities.

*Diagnostic criteria for NF2-associated Schwannomatosis (SWN):
Presence of bilateral vestibular schwannoma, or an identical pathogenic variant of NF2 in at least 2 anatomically distinct NF2-associated tumors (schwannoma, meningioma, ependymoma)
OR
Two of the following major criteria
OR
One major AND two minor criteria
Major criteria:

Unilateral vestibular schwannoma
Non-sibling first-degree relative with NF2
Two or more meningiomas
NF2 pathogenic variant in unaffected tissue
Minor criteria:

Ependymoma, meningioma, or schwannoma (can count >1 of a type)
Juvenile subcapsular or cortical cataract, retinal hamartoma, epiretinal membrane in a person <40 years (can count only once)
Diagnostic criteria for SMARCB1- or LZTR1-related SWN:

At least 1 pathologically confirmed schwannoma or hybrid nerve sheath tumor and a SMARCB1 (or LZTR1) pathogenic variant in unaffected tissue
OR
Shared SMARCB1 or LZTR1 pathogenic variant in 2 schwannomas or hybrid nerve sheath tumors
Diagnostic criteria for SWN in the absence of pathogenic variants in blood, but presence of loss of chromosome 22q in multiple schwannomas:

LOH of the same chromosome 22q markers in 2 anatomically distinct schwannomas or hybrid nerve sheath tumors
AND
A different NF2 pathogenic variant in each tumor that cannot be detected in unaffected tissue
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
None.

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Defined population
Timing
Both
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
1/12/2024
Actual
Date of last participant enrolment
Anticipated
1/12/2029
Actual
Date of last data collection
Anticipated
1/12/2030
Actual
Sample size
Target
300
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 27195 0
The Royal Childrens Hospital - Parkville
Recruitment hospital [2] 27196 0
Royal Melbourne Hospital - City campus - Parkville
Recruitment hospital [3] 27197 0
Royal North Shore Hospital - St Leonards
Recruitment hospital [4] 27198 0
Peter MacCallum Cancer Centre - Melbourne
Recruitment postcode(s) [1] 43278 0
3052 - Parkville
Recruitment postcode(s) [2] 43279 0
2065 - St Leonards
Recruitment postcode(s) [3] 43280 0
3000 - Melbourne

Funding & Sponsors
Funding source category [1] 317508 0
Charities/Societies/Foundations
Name [1] 317508 0
Flicker of Hope
Country [1] 317508 0
Australia
Primary sponsor type
Other Collaborative groups
Name
Walter and Eliza Hall Institute of Medical Research
Address
Country
Australia
Secondary sponsor category [1] 319885 0
None
Name [1] 319885 0
Address [1] 319885 0
Country [1] 319885 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 316220 0
The Royal Melbourne Hospital Human Research Ethics Committee
Ethics committee address [1] 316220 0
Ethics committee country [1] 316220 0
Australia
Date submitted for ethics approval [1] 316220 0
25/10/2023
Approval date [1] 316220 0
20/08/2024
Ethics approval number [1] 316220 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 137194 0
Dr Jim Whittle
Address 137194 0
Peter MacCallum Cancer Centre, 305 Grattan Street, Melbourne VIC 3000 Australia
Country 137194 0
Australia
Phone 137194 0
+61 3 8559 5000
Fax 137194 0
Email 137194 0
jim.whittle@petermac.org
Contact person for public queries
Name 137195 0
Lucy Riley
Address 137195 0
Walter and Eliza Hall Institute, 1G Royal Parade, Parkville VIC 3052
Country 137195 0
Australia
Phone 137195 0
+61 3 9345 2767
Fax 137195 0
Email 137195 0
nf2registry@wehi.edu.au
Contact person for scientific queries
Name 137196 0
Lucy Riley
Address 137196 0
Walter and Eliza Hall Institute, 1G Royal Parade, Parkville VIC 3052
Country 137196 0
Australia
Phone 137196 0
+61 3 9345 2767
Fax 137196 0
Email 137196 0
nf2registry@wehi.edu.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Registry data, not to be shared at an individual level


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.