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Trial registered on ANZCTR
Registration number
ACTRN12620000141943
Ethics application status
Approved
Date submitted
17/12/2019
Date registered
12/02/2020
Date last updated
5/10/2022
Date data sharing statement initially provided
12/02/2020
Type of registration
Prospectively registered
Titles & IDs
Public title
Phase I trial of multi-virus-specific T cells for paediatric haploidentical -stem cell transplant recipients
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Scientific title
Phase I clinical trial of the safety of adoptive transfer of multi-virus-specific T cells into TCRaß+/CD19+-depleted haploidentical HSCT recipients
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Secondary ID [1]
300092
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Nil known
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Haplo-identical haematopoietic stem cell transplantation
315617
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Condition category
Condition code
Blood
313910
313910
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0
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Haematological diseases
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Infection
313911
313911
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0
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Other infectious diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
The investigational product (IP) is ‘multi-virus-specific T cells TI1’, and consists of T cells targeting cytomegalovirus (CMV), Epstein–Barr virus (EBV), BK polyomavirus (BKV) and adenovirus (AdV). In this trial, IP will be generated for each participant from the peripheral blood of the haploidentical haematopoietic stem cell transplant (haplo-HSCT) donor. The safety of the IP in a preventative (prophylactic) setting will be tested in 20 paediatric TCRaß+/CD19+-depleted haplo-HSCT recipients. Participants will be recruited from Queensland Children’s Hospital, The Royal Children’s Hospital Melbourne, The Children’s Hospital at Westmead, and Sydney Children’s Hospital.
Patients and donors will be recruited prior to donor stem cell mobilisation, and the IP will be generated from the peripheral blood of the donor, with manufacturing conducted at Q-Gen Cell Therapeutics.
IP infusions will commence following engraftment, once the criteria for infusion commencement are met, and each infusion will be given at the recruiting hospital. Participants will receive up to six fortnightly intravenous infusions (depending on the number of cells grown), at a dose of twenty million cells per metre squared of body surface area, suspended in 20 mL clinical grade normal saline. The IP will be administered intravenously over 5–10 min by a qualified person (e.g. registered nurse or clinician). This will be followed by a saline flush, which will take an additional 5–10 min.
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Intervention code [1]
316374
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Prevention
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Intervention code [2]
316375
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Treatment: Drugs
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Comparator / control treatment
No control group
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Control group
Uncontrolled
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Outcomes
Primary outcome [1]
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The incidence of adverse events, and clinically significant changes in laboratory tests and vital signs observations.
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Assessment method [1]
322308
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Timepoint [1]
322308
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Adverse events will be collected at each visit, commencing at the first infusion visit, and finishing approximately 10 weeks following the final infusion. Safety blood tests (FBC, ELFT, and viral load) will be collected at the first infusion visit until the final follow-up visit, approximately 7.5 months following the final infusion. Vital signs observations will be done during and immediately following each T cell infusion.
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Secondary outcome [1]
378029
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The change in the proportion of functional virus-specific T cells, from prior to the first infusion to the first follow-up visit and at final follow-up.
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Assessment method [1]
378029
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Timepoint [1]
378029
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Blood will be collected at each study visit (all infusions and follow-up visits) to allow the examination of peripheral blood mononuclear cells. These cells will be analysed to assess their phenotype and function over the course of the trial to determine any changes.
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Eligibility
Key inclusion criteria
1. Aged between 3 months and 18 years
2. Patient has a condition for which HSCT is indicated
3. Transplant physician determines that optimal donor is haploidentical*
4. Haplo-HSCT donor is aged 18 or above
5. Haplo-HSCT donor is available and willing to donate blood for therapy manufacture
* This decision is usually based upon lack of HLA matched sibling or fully matched unrelated donor, but may be influenced by other factors such as donor availability or urgency of HSCT.
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Minimum age
3
Months
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Maximum age
18
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
The treatment protocol in part or in its entirety is declined by either the patient or their legal guardian.
NB: Additional criteria are in place for the commencement of infusions
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Study design
Purpose of the study
Prevention
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 1
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Type of endpoint/s
Safety/efficacy
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Date of first participant enrolment
Anticipated
2/03/2020
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Actual
19/06/2020
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Date of last participant enrolment
Anticipated
1/12/2023
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Actual
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Date of last data collection
Anticipated
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Actual
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Sample size
Target
20
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Accrual to date
9
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
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Funding & Sponsors
Funding source category [1]
304538
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Charities/Societies/Foundations
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Name [1]
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Children's Hospital Foundation
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Address [1]
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Children’s Hospital Foundation, PO Box 8009, Woolloongabba Qld 4102
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Country [1]
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Australia
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Funding source category [2]
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Charities/Societies/Foundations
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Name [2]
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QIMR Berghofer Medical Research Institute
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Address [2]
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QIMR Berghofer, Locked Bag 2000 Royal Brisbane Hospital, Qld 4029
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Country [2]
304543
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Australia
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Primary sponsor type
Charities/Societies/Foundations
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Name
QIMR Berghofer Medical Research Institute
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Address
QIMR Berghofer, Locked Bag 2000 Royal Brisbane Hospital, Qld 4029
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Country
Australia
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Secondary sponsor category [1]
304816
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None
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Name [1]
304816
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Address [1]
304816
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Country [1]
304816
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Ethics approval
Ethics application status
Approved
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Ethics committee name [1]
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Children’s Health Queensland Hospital and Health Service HREC
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Ethics committee address [1]
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Level 7, Centre for Children’s Health Research, Queensland Children’s Hospital Precinct, 62 Graham St, South Brisbane QLD 4101
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Ethics committee country [1]
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Australia
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Date submitted for ethics approval [1]
304967
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Approval date [1]
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28/11/2019
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Ethics approval number [1]
304967
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Summary
Brief summary
Patients who receive a type of transplant called a haploidentical haematopoietic stem cell transplant (haplo-HSCT), which is not an ideal tissue match for the patient, are at a high risk of developing a viral infection. This is because the immune cells that can fight viruses (called T cells) are removed from the transplant before it is given. In this trial, we will test a new preventative T cell therapy for four common viruses in patients who have received a haplo-HSCT. The therapy will be grown from the blood of the transplant donor and given to patients after their transplant. One aim of this trial is to see if this T cell therapy is safe for transplant patients. In addition, we would like to see if the T cells that we grow in the laboratory from the transplant donors can help to restore the recipients’ immune systems. This may help prevent infectious complications from developing.
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Trial website
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Prof Rajiv Khanna
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Address
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QIMR Berghofer Centre for Immunotherapy and Vaccine Development, Tumour Immunology Laboratory
Level 10, CBCRC, QIMR Berghofer
300 Herston Rd
Herston QLD 4006
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Country
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Australia
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Phone
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+61 7 3362 0385
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Fax
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Email
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Rajiv.Khanna@qimrberghofer.edu.au
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Contact person for public queries
Name
98799
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Michelle Neller
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Address
98799
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QIMR Berghofer Medical Research Institute
300 Herston Rd
Herston QLD 4006
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Country
98799
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Australia
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Phone
98799
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+61 7 33620412
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Fax
98799
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+61738453510
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Email
98799
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immunotherapy@qimrberghofer.edu.au
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Contact person for scientific queries
Name
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Rajiv Khanna
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Address
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QIMR Berghofer Centre for Immunotherapy and Vaccine Development, Tumour Immunology Laboratory
Level 10, CBCRC, QIMR Berghofer
300 Herston Rd
Herston QLD 4006
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Country
98800
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Australia
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Phone
98800
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+61 7 3362 0385
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Fax
98800
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Email
98800
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Rajiv.Khanna@qimrberghofer.edu.au
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Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
Sponsor discretion to not share.
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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.
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