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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02923375




Registration number
NCT02923375
Ethics application status
Date submitted
3/10/2016
Date registered
4/10/2016
Date last updated
11/08/2020

Titles & IDs
Public title
A Study of CYP-001 for the Treatment of Steroid-Resistant Acute Graft Versus Host Disease
Scientific title
An Open-Label Phase 1 Study to Investigate the Safety and Efficacy of CYP-001 for the Treatment of Adults With Steroid-Resistant Acute Graft Versus Host Disease
Secondary ID [1] 0 0
2016-000070-38
Secondary ID [2] 0 0
CYP-GvHD-P1-01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Graft vs Host Disease 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Mesenchymoangioblast-derived mesenchymal stem cells

Experimental: Cohort A - Mesenchymoangioblast-derived mesenchymal stem cells (CYP-001) at a dose of 1 million cells/kg (up to a maximum of 100 million cells) by IV infusion on two occasions (Day 0 and Day 7)

Experimental: Cohort B - Mesenchymoangioblast-derived mesenchymal stem cells (CYP-001) at a dose of 2 million cells/kg (up to a maximum of 200 million cells) by IV infusion on two occasions (Day 0 and Day 7)


Treatment: Other: Mesenchymoangioblast-derived mesenchymal stem cells
The active agent in CYP-001 is allogeneic mesenchymoangioblast-derived mesenchymal stem cells (MCA-derived MSCs), which are produced using the proprietary Cymerus™ platform technology. Cymerus™ refers to the process of generating cell-based products from intermediate cells, MCAs, which in turn are derived from induced pluripotent stem cells or iPSCs. The iPSCs used in the Cymerus™ process were derived from blood donated by a fully-consented healthy adult donor, and were reprogrammed using a transgene-free, viral-free and feeder-free technique.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence and severity of treatment emergent adverse events [safety and tolerability]
Timepoint [1] 0 0
28 days
Primary outcome [2] 0 0
Incidence and severity of serious adverse events deemed possibly related to CYP-001 [safety and tolerability]
Timepoint [2] 0 0
100 days
Secondary outcome [1] 0 0
Complete Response by Day 28
Timepoint [1] 0 0
28 days
Secondary outcome [2] 0 0
Partial Response by Day 28
Timepoint [2] 0 0
28 days
Secondary outcome [3] 0 0
Overall Survival at Day 28
Timepoint [3] 0 0
28 days
Secondary outcome [4] 0 0
Complete Response by Day 100
Timepoint [4] 0 0
100 days
Secondary outcome [5] 0 0
Partial Response by Day 100
Timepoint [5] 0 0
100 days
Secondary outcome [6] 0 0
Overall Survival at Day 100
Timepoint [6] 0 0
100 days

Eligibility
Key inclusion criteria
* Diagnosis using consensus grading with steroid-resistant Grade II-IV acute GvHD, after a haematopoietic stem cell transplant for a haematological disorder.
* Life expectancy of at least one month.
* Agree to have follow-up data collected for two years after their initial dose of CYP-001 (under a separate protocol).
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Pregnant or breastfeeding or plan to become pregnant within three months of receiving their last dose of CYP-001.
* Have received any investigational research agent within 30 days or five half-lives (whichever is longer) prior to the first dose of IMP.
* Known or suspected current alcohol or substance abuse problem.
* Progressive or relapsing haematological malignancy, a current solid tumour, or previous malignant solid tumour that is likely to recur during the period of the study (with the exception of a past history of basal or squamous cell carcinomas).
* Heart failure (NYHA Functional Class II-IV) and/or pulmonary failure.
* Haemodynamically unstable and/or at high risk of cardiovascular events.
* Terminal organ failure.
* Meningitis, pneumonia with hypoxemia, HIV or another severe or uncontrolled systemic infection, which in the opinion of the investigator is likely to impact on the ability of the patient to participate in the trial.

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
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/03/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/06/2020
Sample size
Target
Accrual to date
Final
16
Recruitment in Australia
Recruitment state(s)
NSW,SA
Recruitment hospital [1] 0 0
Sydney Local Health District - Sydney
Recruitment hospital [2] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment postcode(s) [1] 0 0
- Sydney
Recruitment postcode(s) [2] 0 0
- Adelaide
Recruitment outside Australia
Country [1] 0 0
United Kingdom
State/province [1] 0 0
Bristol
Country [2] 0 0
United Kingdom
State/province [2] 0 0
Leeds
Country [3] 0 0
United Kingdom
State/province [3] 0 0
Liverpool
Country [4] 0 0
United Kingdom
State/province [4] 0 0
Manchester
Country [5] 0 0
United Kingdom
State/province [5] 0 0
Nottingham

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Cynata Therapeutics Limited
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to assess the safety, tolerability and efficacy of two infusions of CYP-001 in adults with steroid-resistant GvHD.
Trial website
https://clinicaltrials.gov/study/NCT02923375
Trial related presentations / publications
Bloor AJC, Patel A, Griffin JE, Gilleece MH, Radia R, Yeung DT, Drier D, Larson LS, Uenishi GI, Hei D, Kelly K, Slukvin I, Rasko JEJ. Production, safety and efficacy of iPSC-derived mesenchymal stromal cells in acute steroid-resistant graft versus host disease: a phase I, multicenter, open-label, dose-escalation study. Nat Med. 2020 Nov;26(11):1720-1725. doi: 10.1038/s41591-020-1050-x. Epub 2020 Sep 14.
Public notes

Contacts
Principal investigator
Name 0 0
Kilian Kelly, PhD
Address 0 0
Cynata Therapeutics Limited
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02923375