A Study of CYP-001 for the Treatment of Steroid-Resistant Acute Graft Versus Host Disease
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
2 other identifiers
interventional
16
2 countries
7
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 Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Mar 2017
Typical duration for phase_1
7 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 3, 2016
CompletedFirst Posted
Study publicly available on registry
October 4, 2016
CompletedStudy Start
First participant enrolled
March 1, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 28, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
June 30, 2020
CompletedAugust 11, 2020
August 1, 2020
1.5 years
October 3, 2016
August 8, 2020
Conditions
Outcome Measures
Primary Outcomes (2)
Incidence and severity of treatment emergent adverse events [safety and tolerability]
Safety
28 days
Incidence and severity of serious adverse events deemed possibly related to CYP-001 [safety and tolerability]
Safety
100 days
Secondary Outcomes (6)
Complete Response by Day 28
28 days
Partial Response by Day 28
28 days
Overall Survival at Day 28
28 days
Complete Response by Day 100
100 days
Partial Response by Day 100
100 days
- +1 more secondary outcomes
Study Arms (2)
Cohort A
EXPERIMENTALMesenchymoangioblast-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)
Cohort B
EXPERIMENTALMesenchymoangioblast-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)
Interventions
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.
Eligibility Criteria
You may qualify if:
- 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).
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (7)
Sydney Local Health District
Sydney, New South Wales, Australia
Royal Adelaide Hospital
Adelaide, South Australia, Australia
NHS Foundation Trust
Bristol, United Kingdom
NHS Trust
Leeds, United Kingdom
NHS Foundation Trust
Liverpool, United Kingdom
NHS Foundation Trust
Manchester, United Kingdom
NHS Foundation Trust
Nottingham, United Kingdom
Related Publications (2)
Kelly K, Bloor AJC, Griffin JE, Radia R, Yeung DT, Rasko JEJ. Two-year safety outcomes of iPS cell-derived mesenchymal stromal cells in acute steroid-resistant graft-versus-host disease. Nat Med. 2024 Jun;30(6):1556-1558. doi: 10.1038/s41591-024-02990-z. Epub 2024 May 22.
PMID: 38778211DERIVEDBloor 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.
PMID: 32929265DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Kilian Kelly, PhD
Cynata Therapeutics Limited
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 3, 2016
First Posted
October 4, 2016
Study Start
March 1, 2017
Primary Completion
August 28, 2018
Study Completion
June 30, 2020
Last Updated
August 11, 2020
Record last verified: 2020-08