Trial Review

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Trial registered on ANZCTR


Registration number
Help: Registration number
ACTRN12626001189314
Ethics application status
Help: Ethics application status
Approved
Date submitted
Help: Date submitted
24/08/2026
Date registered
Help: Date registered
24/09/2026
Date last updated
Help: Date last updated
24/09/2026
Date data sharing statement initially provided
Help: Data sharing statement date
24/09/2026
Type of registration
Help: Type of registration
Prospectively registered

Titles & IDs
Public title
Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults (SACHA)
Scientific title
Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults in Therapeutic Failure or Relapse and Not Eligible for a Clinical Trial
Additional trial identifiers [1] 318008 0
Nil known
Universal Trial Number (UTN)
Trial acronym
SACHA– International
Related trial records

Health condition
Health condition(s) studied:
Paediatric Cancer 343114 0
Cancer 343454 0
Condition category
Condition code
Cancer 339202 339202 0 0
Children's - Other
Cancer 339552 339552 0 0
Any cancer

Intervention/exposure
Study type
Observational
Patient registry
True
Target follow-up duration
2
Target follow-up type
Years
Description of the intervention or exposure
This prospective observational, international registry includes children, adolescents and young adults aged 0–25 years with a paediatric malignancy (solid tumour or leukaemia), or a related condition, who are not eligible for a clinical trial or have no therapeutic option available in an ongoing clinical trial. The exposure is treatment with an innovative anticancer medicine prescribed as part of routine clinical care, either through compassionate/early access or off-label use. Treatment is selected following review by a Paediatric Multidisciplinary Tumour Board. SACHA does not assign or modify treatment. There is no additional study-specific treatment or intervention. Data are collected from the medical record and include disease characteristics, treatment details, treatment activity/response and treatment-related adverse events. Disease response is assessed by the treating physician using applicable disease-specific criteria, and relevant adverse events attributable to treatment are collected and graded. Participants are observed from treatment commencement, with data updated every 3 months until end of treatment or participant death for a maximum of 2 years, whichever occurs first, within the study’s 5-year overall duration and 4-year inclusion period. No study-specific questionnaires or additional physical assessments are required; clinical assessments are those undertaken as part of routine care.
Intervention code [1] 334970 0
Not applicable
Description of the comparator or control
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 346628 0
Access to innovative anticancer therapies outside clinical trials. This is assessed as a composite outcome comprising the innovative treatment, the indication for treatment and the access pathway (e.g.. compassionate/early access or off-label use
Timepoint [1] 346628 0
At treatment commencement
Secondary outcome [1] 466781 0
Incidence and severity of adverse drug reactions associated with innovative anticancer therapies
Timepoint [1] 466781 0
Month 3 and every 3 months thereafter, until end of treatment or participant death, for up to 2 years from treatment commencement.
Secondary outcome [2] 466782 0
Best disease response to innovative anticancer therapy. This is assessed as a composite secondary outcome across eligible disease types and recorded as complete response, partial response, stable disease or progressive disease, as applicable.
Timepoint [2] 466782 0
Month 3, every 3 months thereafter until end of treatment or participant death, for up 2 years from treatment commencement.

Eligibility
Key inclusion criteria
• Age less than or equal to 25 at the time of inclusion in the study
• Patient with a paediatric malignancy (solid tumour or leukemia), or other related condition
• Patient not currently enrolled on any other trial for ongoing therapeutic purposes
• Patient treated with compassionate use of experimental drugs or off-label administration use of anti-cancer medicines first approved in adults after 2007 in Europe
• Patient discussed in a Paediatric multidisciplinary tumor boards (MDTB) to validate the inclusion in the SACHA study
• Consent to participate by the patient or parent/legal guardian in the study
Minimum age
0 Years
Maximum age
25 Years
Sex
Both males and females
Healthy volunteers
No
Key exclusion criteria
• Patient receiving the drug within a clinical trial.
• Oral refusal to participate to the study, by the patient or his/her parents/legal representatives.

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

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
30/09/2026
Actual
Date of last participant enrolment
Anticipated
30/06/2027
Actual
Date of last data collection
Anticipated
30/06/2028
Actual
Sample size
Target
50
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,SA,TAS,WA,VIC
Recruitment hospital [1] 29974 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 29975 0
John Hunter Children's Hospital - New Lambton
Recruitment hospital [3] 29976 0
Monash Children’s Hospital - Clayton
Recruitment hospital [4] 29977 0
The Royal Childrens Hospital - Parkville
Recruitment hospital [5] 29978 0
Perth Children's Hospital - Nedlands
Recruitment hospital [6] 29979 0
Royal Hobart Hospital - Hobart
Recruitment hospital [7] 29980 0
Womens and Childrens Hospital - North Adelaide
Recruitment postcode(s) [1] 46365 0
2145 - Westmead
Recruitment postcode(s) [2] 46366 0
2305 - New Lambton
Recruitment postcode(s) [3] 46367 0
3168 - Clayton
Recruitment postcode(s) [4] 46368 0
3052 - Parkville
Recruitment postcode(s) [5] 46369 0
6009 - Nedlands
Recruitment postcode(s) [6] 46370 0
7000 - Hobart
Recruitment postcode(s) [7] 46371 0
5006 - North Adelaide

Funding & Sponsors
Funding source category [1] 322555 0
Charities/Societies/Foundations
Name [1] 322555 0
My Room Children’s Cancer Charity
Country [1] 322555 0
Australia
Funding source category [2] 322696 0
Government body
Name [2] 322696 0
Department of Health, Disability and Ageing - Medical Research Future Fund (MRFF)
Country [2] 322696 0
Australia
Funding source category [3] 322697 0
Hospital
Name [3] 322697 0
Institut Gustave Roussy
Country [3] 322697 0
France
Primary sponsor type
Other Collaborative groups
Name
Australian and New Zealand Children's Haematology/ Oncology Group
Address
Country
Australia
Secondary sponsor category [1] 325476 0
Hospital
Name [1] 325476 0
Institut Gustave Roussy
Address [1] 325476 0
Country [1] 325476 0
France

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 321033 0
Women's and Children's Health Network Human Research Ethics Committee
Ethics committee address [1] 321033 0
Ethics committee country [1] 321033 0
Australia
Date submitted for ethics approval [1] 321033 0
19/11/2024
Approval date [1] 321033 0
10/02/2025
Ethics approval number [1] 321033 0
2024/HRE00258

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

Contacts
Principal investigator
Name 152438 0
Dr Vickyanne Carruthers
Address 152438 0
Women’s and Children’s Hospital, 72 King William Road, North Adelaide SA 5006
Country 152438 0
Australia
Phone 152438 0
+61 8 8161 7411
Fax 152438 0
Email 152438 0
[email protected]
Contact person for public queries
Name 152439 0
Vickyanne Carruthers
Address 152439 0
Women’s and Children’s Hospital, 72 King William Road, North Adelaide SA 5006
Country 152439 0
Australia
Phone 152439 0
+61 8 8161 7411
Fax 152439 0
Email 152439 0
[email protected]
Contact person for scientific queries
Name 152440 0
Vickyanne Carruthers
Address 152440 0
Women’s and Children’s Hospital, 72 King William Road, North Adelaide SA 5006
Country 152440 0
Australia
Phone 152440 0
+61 8 8161 7411
Fax 152440 0
Email 152440 0
[email protected]

Data sharing statement
Will the study consider sharing individual participant data?
No


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.