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Trial registered on ANZCTR


Registration number
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
Sex
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
4/05/2009
Actual
4/05/2009
Date of last participant enrolment
Anticipated
28/02/2014
Actual
10/06/2013
Date of last data collection
Anticipated
Actual
Sample size
Target
5
Accrual to date
Final
4
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
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
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
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
[email protected]
Contact person for public queries
Name 38031 0
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
[email protected]
Contact person for scientific queries
Name 38032 0
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
[email protected]

No information has been provided regarding IPD availability


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.