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Trial registered on ANZCTR


Trial ID
ACTRN12613000338763
Ethics application status
Approved
Date submitted
22/02/2013
Date registered
27/03/2013
Date last updated
20/10/2015
Type of registration
Retrospectively registered

Titles & IDs
Public title
Haploidentical donor stem cell transplantation for haematological malignancies - a Phase 1/2 pilot study in an Australian population
Scientific title
A comparison of leukaemia free and overall survival following Haploidentical donor stem cell transplantation compared to matched unrelated donor stem cell transplantation for haematological malignancies
Secondary ID [1] 281997 0
nil
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Haematological malignancies 288433 0
Condition category
Condition code
Blood 288782 288782 0 0
Haematological diseases
Cancer 289034 289034 0 0
Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
Cancer 289035 289035 0 0
Leukaemia - Acute leukaemia

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Patients are given Peripheral blood stem cells (PBSCs) collected from a haploidentical family member after completing myeloablative chemotherapy.

The myeloablative chemotherapy consists of the following:

* Cytosine arabinoside 2g/m^2 twice daily by intravenous infusion on Days -10 & -9

* Busulfan 3.2mg/kg/day by intravenous infusion on Days -8 to -6 (Pharmacokinetically monitored and adjusted to target AUC 4500micrometre/min)

* Cyclophosphamide 1.8g/m^2/day by intravenous infusion on days -5 & -4

* CCNU (Lomustine) 200mg/m^2 by oral administration on Day -3 only

* ATG (thymoglobuline) 2.5mg/kg/day by intravenous infusion on Days -5 to -2

On day 0 participants then undergo infusion of unmanipulated GCSF primed bone marrow harvest +/PBSC supplementation (on second day 0) to achieve MNC & CD34

minimum thresholds (>4.0x10^8/kg & >2.0x10^6/kg respectively).

All participants will also undergo a GVHD prophylaxis regimen, which consists of:

* Cyclosporin 2.5mg/kg/day initially by intravenous infusion thence orally once tolerated from Day -9; At day +45 cyclosporin taper should be commenced (unless active GVHD) ~10% per month

* Mycophenolate 0.5g orally twice daily from Day -9; to be ceased on day +60 (unless active GVHD)

* Methotrexate 15mg/m^2 by intravenous infusion on Day +1

* Methotrexate 10mg/m^2 by intravenous infusion on Days +3,+6,+11

* GCSF 5mcg/kg subcutaneously daily until neutrophil recovery of >2.0x10^9/L for at least 2 days.

Intervention code [1] 286568 0
Treatment: Other
Intervention code [2] 286588 0
Treatment: Drugs
Comparator / control treatment
Compare the results with historical results of St Vincent's Hospital patients who have undergone a standard of care allogeneic stem cell transplant over 12 month period from Feb 2011 to Feb 2012.
Control group
Historical

Outcomes
Primary outcome [1] 288940 0
Leukaemia free survival determined by clinical assessment, full blood count and bone marrow examination.
Timepoint [1] 288940 0
3, 6, 9, 12 months
Primary outcome [2] 289147 0
Overall Survival is a clinical endpoint defined as time to death
Timepoint [2] 289147 0
3, 6, 9, 12 months
Secondary outcome [1] 301395 0
Rates of acute Graft vs Host Disease (GVHD), assessed clinically and with laboratory investigations, according to standard definitions.
Timepoint [1] 301395 0
Day 100
Secondary outcome [2] 301396 0
Rates of chronic Graft vs Host Disease (GVHD) determined clinically.
Timepoint [2] 301396 0
12 months
Secondary outcome [3] 301397 0
Time to neutrophil and platelet engraftment, determined by full blood counts.
Timepoint [3] 301397 0
Day 30

Eligibility
Key inclusion criteria
ongoing complete remission of underlying haematological malignancy
Absence of a 100% matched or single-mismatch related donor or single umbilical cord blood with 4/6 HLA match.
Presence of 1 haploidentical famiy donor
Absence of active fungalor viral (inc CMV) infection.
ECOG<2
Preserved cardiac/renal/liver function
Minimum age
16 Years
Maximum age
50 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Life expectance <3 months.
Psychiatric condition interfering with informed consent.
History of active malignant disease within 5 yrs excl SCC or BCC of skin.
Previuos allogeneic stem cell transplant
Taking other 'investigational' drugs

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
open label
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
Single group
Other design features
Phase
Phase 1 / Phase 2
Type of endpoint(s)
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Current
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 636 0
St Vincent's Hospital (Darlinghurst) - Darlinghurst
Recruitment postcode(s) [1] 6369 0
2010 - Darlinghurst

Funding & Sponsors
Funding source category [1] 286775 0
Hospital
Name [1] 286775 0
St Vincent's Hospital,Sydney
Address [1] 286775 0
390 Victoria St
Darlinghurst NSW 2010
Country [1] 286775 0
Australia
Primary sponsor type
Hospital
Name
St Vincent's Hospital,Sydney
Address
390 Victoria St
Darlinghurst NSW 2010
Country
Australia
Secondary sponsor category [1] 285558 0
None
Name [1] 285558 0
Address [1] 285558 0
Country [1] 285558 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 288840 0
St Vincents Hospital Human Research Ethics Committee
Ethics committee address [1] 288840 0
St Vincents Hospital
390 Victoria St
Darlinghurst NSW 2010
Ethics committee country [1] 288840 0
Australia
Date submitted for ethics approval [1] 288840 0
09/12/2008
Approval date [1] 288840 0
16/03/2009
Ethics approval number [1] 288840 0
HREC/08/SVH/183

Summary
Brief summary
This study aims to evaluate the effect of haploidentical donor stem cell transplantation in patients with haematological malignancies.
Who is it for? You may be eligible to join this study if you are aged between 16-50 years and have ongoing complete remission of underlying haematological malignancy. You should have available a haploidentical family donor. This is a donor who is a 50% match, and can be a parent, sibling or child.
Trial details All participants in this trial will undergo ten days of chemotherapy treatment, followed by infusion of peripheral blood stem cells (PBSCs) from the donor into the vein (i.e. intravenously). Participants will also be administered a number of medications to prevent graft versus host disease (GVHD), which is a common complication following stem cell transplantation. Patients will be regularly assessed for up to 12 months in order to evaluate clinical efficacy and safety of treatment.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 38030 0
A/Prof Anthony Dodds
Address 38030 0
Kinghorn Cancer Centre
St Vincents Hospital
370 Victoria St
Darlinghurst NSW 2010
Country 38030 0
Australia
Phone 38030 0
61 2 9355 5656
Fax 38030 0
61 2 9355 5735
Email 38030 0
svhcancer_research@stvincents.com.au
Contact person for public queries
Name 38031 0
Ms Patricia Plenge
Address 38031 0
Kinghorn Cancer Centre
St Vincents Hospital
370 Victoria St
Darlinghurst NSW 2010
Country 38031 0
Australia
Phone 38031 0
61 2 9355 5656
Fax 38031 0
61 2 9355 5735
Email 38031 0
svhcancer_research@stvincents.com.au
Contact person for scientific queries
Name 38032 0
A/Prof Anthony Dodds
Address 38032 0
Kinghorn Cancer Centre
St Vincents Hospital
370 Victoria St
Darlinghurst NSW 2010
Country 38032 0
Australia
Phone 38032 0
61 2 9355 5656
Fax 38032 0
612 9355 5735
Email 38032 0
svhcancer_research@stvincents.com.au