Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02670564




Registration number
NCT02670564
Ethics application status
Date submitted
3/07/2015
Date registered
2/02/2016
Date last updated
16/04/2019

Titles & IDs
Public title
ALL SCTped FORUM - Pharmacogenomic Study (add-on Study)
Scientific title
Allogeneic Stem Cell Transplant for Children and Adolescents With Acute Lymoblastic Leukemia FORUM - Pharmacogenomic Study (add-on Study)
Secondary ID [1] 0 0
NZ 2015-069
Secondary ID [2] 0 0
ZH 2014-0535
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acute Lymphoblastic Leukemia 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma
Cancer 0 0 0 0
Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
Cancer 0 0 0 0
Lymphoma (non Hodgkin's lymphoma) - Low grade lymphoma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - Pharmacogenomics
Other interventions - Busulfan plasma level measurements

Experimental: Total body irradiation (TBI) - The Pharmacogenomics add-on requires 2X 5ml blood EDTA for further DNA analyses.
Note this is an add-on study to NCT 01949129, THE ALL SCTped FORUM study. Please refer to the main study for further details.

Experimental: Busulfan - The Pharmacogenomics add-on requires 2X 5ml blood EDTA for further DNA analyses.
Note this is an add-on study to NCT 01949129, THE ALL SCTped FORUM study. Please refer to the main study for further details.

Experimental: Treosulfan - The Pharmacogenomics add-on requires 2X 5ml blood EDTA for further DNA analyses.
Note this is an add-on study to NCT 01949129, THE ALL SCTped FORUM study. Please refer to the main study for further details.


Other interventions: Pharmacogenomics
Blood samples (2x5ml EDTA tubes) should be collected just before the start of the conditioning regimen from every patient regardless of therapeutic arm by every centre and stored =-20°C Patient should be in remission (MRD negative) for this sampling, otherwise the sample should be taken using a mouth swab/saliva (not intravenously).
For second transplant patients, please provide DNA taken before first transplant or a fresh saliva samples.

Other interventions: Busulfan plasma level measurements
Bu PK analysis after the first dose of IV Bu (+potential subsequent ones).
Blood sampling:
->For Bu 4 X/d: Before the first Bu dose (Time 0), then straight after the end of infusion (Time 1), then at 15 min (Time 2), 30 min (Time 3), 1 hour (Time 4) and 4 hour (Time 5) after the end of infusion
->For Bu 1 X/d: Before the first Bu dose (Time 0), then straight after the end of infusion (Time 1), then at 1 hour (Time 2), 3 hour (Time 3), 5 hour (Time 4), 7 hour (Time 5) and 11 hour (Time 6) after the end of infusion.
For centers not performing BU TDM, perform Dried Blood Sampling (DBS) analysis:
-> 0.5ml blood sample should be collected and 5µl spotted onto DBS cards in duplicate. Dry them max 5 hours and then keep in a sealed envelope and store at -80°C, as below
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Genetic variants in participants as a marker of risk of Adverse events and/or Efficacy of the studied agents
Timepoint [1] 0 0
through study completion, an average of 2 years
Secondary outcome [1] 0 0
Number of participants with acute Graft-versus-host disease (aGvHD) according to the Glucksberg scale and Seattle criteria
Timepoint [1] 0 0
18 months after inclusion of first patient, afterwards, annually up to 10 years
Secondary outcome [2] 0 0
Number of participants with chronic Graft-versus-host disease (cGvHD) according to the Glucksberg scale and Seattle criteria
Timepoint [2] 0 0
18 months after inclusion of first patient, afterwards, annually up to 10 years
Secondary outcome [3] 0 0
Number of participants with VOD/SOS according to the Seattle criteria
Timepoint [3] 0 0
18 months after inclusion of first patient, afterwards, annually up to 10 years
Secondary outcome [4] 0 0
Number of participants with Neutrophil recovery as a measure of Safety and Tolerability
Timepoint [4] 0 0
18 months after inclusion of first patient, afterwards, annually up to 10 years
Secondary outcome [5] 0 0
Number of participants with Platelet recovery as a measure of Safety and Tolerability
Timepoint [5] 0 0
18 months after inclusion of first patient, afterwards, annually up to 10 years
Secondary outcome [6] 0 0
Number of participants with Primary graft failure or rejection as a measure of Safety and Tolerability
Timepoint [6] 0 0
18 months after inclusion of first patient, afterwards, annually up to 10 years
Secondary outcome [7] 0 0
Transplant related mortality (TRM)
Timepoint [7] 0 0
18 months after inclusion of first patient, afterwards, annually up to 10 years
Secondary outcome [8] 0 0
Event free survival (EFS)
Timepoint [8] 0 0
18 months after inclusion of first patient, afterwards, annually up to 10 years
Secondary outcome [9] 0 0
Overall survival (OS)
Timepoint [9] 0 0
18 months after inclusion of first patient, afterwards, annually up to 10 years
Secondary outcome [10] 0 0
Cumulative incidence of relapse
Timepoint [10] 0 0
18 months after inclusion of first patient, afterwards, annually up to 10 years

Eligibility
Key inclusion criteria
Note this is an add-on study to NCT 01949129, THE ALL SCTped FORUM study. Please refer to
the main study for further details.

Gender

- Both: both female and male participants are being studied

Age Limits

- Minimum Age: N/A

- Maximum Age: age at time of screening less than 18 years old

Accepts Healthy Volunteers: no

Eligibility Criteria



- Patients with ALL (except for patients with B-ALL)

- indication for allogeneic HSCT

- complete remission (CR) before HSCT

- written consent of the parents (legal guardian) and, if necessary, the minor patient
via "Informed Consent Form"

- no pregnancy

- no secondary malignancy

- no previous HSCT

- HSCT is performed in a study participating centre
Minimum age
No limit
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Non Hodgkin-Lymphoma

- ALL with extramedullary involvement with indication for TBI

- CNS involvement at the timepoint of screening

- Trisomy 21

- The whole protocol or essential parts are declined either by patient himself/herself
or the respective legal guardian

- No consent is given for saving and propagation of anonymous medical data for study
reasons

- Severe concomitant disease that does not allow treatment according to the protocol at
the investigator's discretion (e.g. malformation syndromes, cardiac malformations,
metabolic disorders)

- Karnofsky / Lansky score < 50%

- Subjects unwilling or unable to comply with the study procedures

Study design
Purpose of the study
Basic Science
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 4
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/04/2026
Actual
Sample size
Target
1000
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
Switzerland
State/province [1] 0 0
Cansearch Laboratory
Country [2] 0 0
Switzerland
State/province [2] 0 0
Basel
Country [3] 0 0
Switzerland
State/province [3] 0 0
Geneva
Country [4] 0 0
Switzerland
State/province [4] 0 0
Zurich

Funding & Sponsors
Primary sponsor type
Other
Name
Swiss Pediatric Oncology Group
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
ALL SCTped Forum
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Pharmacogenomics (PG) offers the opportunity to individualize treatment according to patient
genetic variations which influence activity of enzyme metabolizing or acting in the pathway
of prescribed chemotherapy drugs.

This add-on research aims to prospectively investigate variations in several candidate genes
related to all types of chemotherapeutic drugs and TBI used in the main related study NCT
01949129, THE ALL SCTped FORUM study for their potential role as predictive biomarkers of PK
variability and outcome of myeloablative therapy for pediatric patients receiving an
allogeneic hematopoietic stem cell transplantation in acute lymphoblastic leukemia.
Trial website
https://clinicaltrials.gov/ct2/show/NCT02670564
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Christina Peters, Prof. MD PhD
Address 0 0
St. Anna Kinderspital, Vienna, Austria
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Marc Ansari, MD, PD
Address 0 0
Country 0 0
Phone 0 0
+41 22 382 47 31
Fax 0 0
Email 0 0
Marc.Ansari@hcuge.ch
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT02670564