Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12607000279426
Ethics application status
Approved
Date submitted
9/05/2007
Date registered
25/05/2007
Date last updated
8/03/2011
Type of registration
Retrospectively registered

Titles & IDs
Public title
A phase I multicentre open label dose-escalation study of unrelated, Major Histocompatibility (MHC)-unmatched placenta-derived mesenchymal stem cells (MSC) in recipients of unrelated umbilical cord blood haematopoietic stem cell (HSC) transplants.
MMRI CT4-MSC-UCB-001 Mater 954A
Scientific title
A phase I multicentre open label dose-escalation study to investigate the safety and feasibility of escalating doses of unrelated, Major Histocompatibility (MHC)-unmatched placenta-derived mesenchymal stem cells (MSC) in recipients of unrelated umbilical cord blood haematopoietic stem cell (HSC) transplants.
MMRI CT4-MSC-UCB-001 Mater 954A
Secondary ID [1] 259753 0
MMRI CT4-MSC-UCB-001
Universal Trial Number (UTN)
Trial acronym
MMRI CT4 MSC UCB 001
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Patients undergoing unrelated unmatched umbilical cord blood stem cell transplants 1819 0
Condition category
Condition code
Cancer 1910 1910 0 0
Children's - Leukaemia & Lymphoma
Cancer 3055 3055 0 0
Leukaemia - Acute leukaemia
Cancer 3056 3056 0 0
Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
Cancer 3057 3057 0 0
Myeloma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
There are 3 groups of patients. In each group there are 3 patients. All umbilical cord blood recipients (patients) will receive only one dose of MSCs intravenously
Group 1 will receive 1 x 10E6 MSC/kg
Group 2 will receive 3.3 x 10E6 MSC/kg
Group 3 will receive 10 x 10E6 MSC/kg
Intervention code [1] 1746 0
Treatment: Drugs
Comparator / control treatment
No comparator group
Control group
Dose comparison

Outcomes
Primary outcome [1] 2717 0
Infusional toxicity
Timepoint [1] 2717 0
15minutes, 30minutes, 1 hour, 2 hours and 4 hours post infusion of MSCs
Primary outcome [2] 2718 0
Adverse events including infections and recurrence of the underlying malignancy
Timepoint [2] 2718 0
Day 1 through to Day 27 on a daily basis, Day 28, Day 42 (+/- 3 days), Day 100 (+/-14 days) 12 months (+/0 14 days) and 24 months (+/-14 days)
Primary outcome [3] 2719 0
Formation of Ectopic Tissue Foci.
Timepoint [3] 2719 0
Day 100, 12 and 24 months ( +/- 14 days)
Primary outcome [4] 2720 0
Determination Of relapse
Timepoint [4] 2720 0
On recurrence of original malignant disease after transplantation
Primary outcome [5] 2721 0
Human Mesenchymal Stem Cells and Umbilical Cord Blood Chimersim in Bone Marrow:
Timepoint [5] 2721 0
Once during days 21-28, and on days 42,100 and 1 year
Secondary outcome [1] 4584 0
Engraftment of platelets
Timepoint [1] 4584 0
Is defined as the day post transplant which is the first of 3 consecutive measurements when the platelet count is equal to, or greater than, 20 x 109/ul without transfusion support in the previous 7 days.
Secondary outcome [2] 4585 0
Engraftment of neutrophils
Timepoint [2] 4585 0
Is defined as the day post transplant which is the first of 3 consecutive measurements when the neutrophil count is equal to, or greater than, 0.5 x 109/ul.
Secondary outcome [3] 4586 0
Incidence and severity of acute Graft Versus Host Disease
Timepoint [3] 4586 0
Day 28, day 42 and day 100 post transplantation.

Eligibility
Key inclusion criteria
Patient is willing and has a 4/6, 5/6 or 6/6 Human Leucocyte Antigen matched unrelated donor Umbilical Cord blood graft with a cell dose >2.0 x 107 nucleated cells/kg recipient actual body weight.Patient or guardian must furnish written informed consent. Patients must have a life-threatening disease requiring treatment by unrelated cord blood transplantation Adequate cardiac function with a left ventricular ejection fraction > 45% of predicted.Adequate pulmonary function as defined as no severe or symptomatic restrictive or obstructive lung disease, and pulmonary function testing showing an Forced Expiratory Volume in 1 second >50% of predicted and a Diffuse Lung capacity for Carbon monoxide >50% of predicted. (Children less than 6 years of age must have normal oxygen saturation, in the opinion of the Investigator)Adequate renal function as defined by a creatinine clearance >40% of normal.Adequate hepatic function as defined by a total bilirubin < 2x normal or absence of hepatic fibrosis/cirrhosis.Adequate neurologic function as defined by no evidence of a severe central or peripheral neurological abnormality. Adequate immunologic function as defined by no evidence of active infection at the time of the transplant preparative regimen.Female patients are not pregnant, not breast-feeding and are using adequate birth control techniquePatient must be Human Immunodeficiency Virus (HIV)-1 & 2 antibody, Human Immunodeficiency Virus (HIV)-1 antigen, and Human lymphotropic Virus (HTLV) I & II antibody sero-negative Patient has an Eastern Cooperative Group (ECOG) performance status of 0, 1, or a Karnofsky/ Lansky score of 70.(Use Lansky Play Scale for patients less than 16 y.o.)Patient must demonstrate ability to be compliant with medical regimen.
Minimum age
0 Years
Maximum age
40 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Patient has a consenting Human Luekocyte Antigens (HLA)-A-B-DRB1 identical or 5/6 HLA antigen matched related hematopoietic stem cell donor.Patient does not have a minimum Umbilical Cord Blood dose of at least 2.0 x 107 total nucleated cells/kg actual body weight (based on cryopreserved cell count of umbilical cord blood product) available from the unrelated Umbilical Cord Blood donor who is a 4/6, 5/6, or 6/6 HLA-matched.Patient has an active infection at time of transplantation.Patient has active alcohol or substance abuse within 6 months of study entry.Patient is enrolled on another investigational agent concurrently.Patient has any medical condition, which, in the opinion of the clinical investigator, would interfere with the evaluation of the patient.Patient has had a prior hematopoietic stem cell transplant or solid organ transplant

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)
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
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Date of first participant enrolment
Anticipated
9/05/2007
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
9
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment postcode(s) [1] 600 0
2031
Recruitment postcode(s) [2] 601 0
2145

Funding & Sponsors
Funding source category [1] 2056 0
Charities/Societies/Foundations
Name [1] 2056 0
Inner Wheel of Australia
Country [1] 2056 0
Australia
Primary sponsor type
Individual
Name
Mater Medical Research Institute
Address
Level 3
Aubigny Place
Raymond Terrace
South Brisbane Queensland 4101
Country
Australia
Secondary sponsor category [1] 1863 0
Hospital
Name [1] 1863 0
Mater Health Services
Address [1] 1863 0
Raymond Terrace South Brisbane Q 4101
Country [1] 1863 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 3819 0
Mater Health Services (incorporating Mater Medical Research Institute and Mater Mother's Hospital
Ethics committee address [1] 3819 0
Raymond Terrace South Brisbane Q 4101
Ethics committee country [1] 3819 0
Australia
Date submitted for ethics approval [1] 3819 0
Approval date [1] 3819 0
23/02/2007
Ethics approval number [1] 3819 0
954A
Ethics committee name [2] 3820 0
Westmead Hospital
Ethics committee address [2] 3820 0
Cnr Hawkesbury and Darcy Roads Westmead NSW 2145
Ethics committee country [2] 3820 0
Australia
Date submitted for ethics approval [2] 3820 0
Approval date [2] 3820 0
25/10/2006
Ethics approval number [2] 3820 0
2006/7/4.8(2386)
Ethics committee name [3] 3821 0
Sydney Children's Hospital
Ethics committee address [3] 3821 0
Hight Street Randwick NSW 2031
Ethics committee country [3] 3821 0
Australia
Date submitted for ethics approval [3] 3821 0
Approval date [3] 3821 0
24/04/2007
Ethics approval number [3] 3821 0
07/016

Summary
Brief summary
Phase 1

This trial investigates the safety and feasibility of escalating doses of mesenchymal stem cells (multipotent stem cells that can differentiate into a variety of cell types) in people with life-threatening disease requiring umbilical cord blood transfusion.

Who is it for?
You can join this study if you have a life threatening disease – either leukaemia, lymphoma or myeloma – requiring umbilical cord blood transfusion. There are only nine participants in this trial.

Trial details
Participants will receive an infusion of mesenchymal stem cells. There will be three groups of three people only, given different doses of mesenchymal stem cells. Infusion toxicity will be measured up to 4 hours after transfusion. Adverse events including infection and recurrence of cancer up to 2 years after transfusion will also be measured. This trial aims to test the safety and feasibility of treatment with mesenchymal stem cells.

The study will test the safety and feasibility of manufacturing mesenchymal stem cells from the placentas of women undergoing elective Caesarean section and transplanting the cells into people with life threatening blood cancers. The placentas would otherwise be discarded.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 27849 0
Address 27849 0
Country 27849 0
Phone 27849 0
Fax 27849 0
Email 27849 0
Contact person for public queries
Name 10935 0
Sonia Hancock
Address 10935 0
Mater Medical Research Institute
Level 3
Aubigny Place
Raymond Terrace
South Brisbane QLD 4101
Country 10935 0
Australia
Phone 10935 0
+61 7 38401558
Fax 10935 0
+61 7 38402134
Email 10935 0
shancock@mmri.mater.org.au
Contact person for scientific queries
Name 1863 0
Professor Kerry Atkinson
Address 1863 0
Division of Cancer Services
Mater Health Services
Level 3
MAH
Raymond Terrace
South Brisbane QLD 4101
Country 1863 0
Australia
Phone 1863 0
+61 7 3163 3429
Fax 1863 0
Email 1863 0
kerry.atkinson@mater.org.au

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.