Trial Review

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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
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
Secondary ID [1] 300092 0
Nil known
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Haplo-identical haematopoietic stem cell transplantation 315617 0
Condition category
Condition code
Blood 313910 313910 0 0
Haematological diseases
Infection 313911 313911 0 0
Other infectious diseases

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 316374 0
Prevention
Intervention code [2] 316375 0
Treatment: Drugs
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 322308 0
The incidence of adverse events, and clinically significant changes in laboratory tests and vital signs observations.
Timepoint [1] 322308 0
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.
Secondary outcome [1] 378029 0
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.
Timepoint [1] 378029 0
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.

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.
Minimum age
3 Months
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Prevention
Allocation to intervention
Non-randomised 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
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
2/03/2020
Actual
19/06/2020
Date of last participant enrolment
Anticipated
1/12/2023
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
20
Accrual to date
9
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC

Funding & Sponsors
Funding source category [1] 304538 0
Charities/Societies/Foundations
Name [1] 304538 0
Children's Hospital Foundation
Country [1] 304538 0
Australia
Funding source category [2] 304543 0
Charities/Societies/Foundations
Name [2] 304543 0
QIMR Berghofer Medical Research Institute
Country [2] 304543 0
Australia
Primary sponsor type
Charities/Societies/Foundations
Name
QIMR Berghofer Medical Research Institute
Address
QIMR Berghofer, Locked Bag 2000 Royal Brisbane Hospital, Qld 4029
Country
Australia
Secondary sponsor category [1] 304816 0
None
Name [1] 304816 0
Address [1] 304816 0
Country [1] 304816 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 304967 0
Children’s Health Queensland Hospital and Health Service HREC
Ethics committee address [1] 304967 0
Ethics committee country [1] 304967 0
Australia
Date submitted for ethics approval [1] 304967 0
Approval date [1] 304967 0
28/11/2019
Ethics approval number [1] 304967 0

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

Contacts
Principal investigator
Name 98798 0
Prof Rajiv Khanna
Address 98798 0
QIMR Berghofer Centre for Immunotherapy and Vaccine Development, Tumour Immunology Laboratory
Level 10, CBCRC, QIMR Berghofer
300 Herston Rd
Herston QLD 4006
Country 98798 0
Australia
Phone 98798 0
+61 7 3362 0385
Fax 98798 0
Email 98798 0
[email protected]
Contact person for public queries
Name 98799 0
Michelle Neller
Address 98799 0
QIMR Berghofer Medical Research Institute
300 Herston Rd
Herston QLD 4006
Country 98799 0
Australia
Phone 98799 0
+61 7 33620412
Fax 98799 0
+61738453510
Email 98799 0
[email protected]
Contact person for scientific queries
Name 98800 0
Rajiv Khanna
Address 98800 0
QIMR Berghofer Centre for Immunotherapy and Vaccine Development, Tumour Immunology Laboratory
Level 10, CBCRC, QIMR Berghofer
300 Herston Rd
Herston QLD 4006
Country 98800 0
Australia
Phone 98800 0
+61 7 3362 0385
Fax 98800 0
Email 98800 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Sponsor discretion to not share.


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.