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A Study of CYP-001 for the Treatment of Steroid-Resistant Acute Graft Versus Host Disease

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ClinicalTrials.gov Identifier: NCT02923375
Recruitment Status : Active, not recruiting
First Posted : October 4, 2016
Last Update Posted : May 30, 2018
Sponsor:
Information provided by (Responsible Party):
Cynata Therapeutics Limited

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.

Condition or disease Intervention/treatment Phase
Graft vs Host Disease Biological: Mesenchymoangioblast-derived mesenchymal stem cells Phase 1

Detailed Description:

This is a multi-centre, open label, dose escalation study to assess the safety, tolerability and efficacy of two infusions of CYP-001, in adults who have steroid-resistant GvHD.

Participants will receive standard of care treatment throughout the study, according to local procedures. The first eight participants will be enrolled in Cohort A and receive a CYP-001 dose of 1 million cells per kg, up to a maximum dose of 100 million cells, on Day 0 and Day 7. Subject to a safety review of data from Cohort A, an additional eight participants will be enrolled into Cohort B and receive a CYP-001 dose of 2 million cells/kg, up to a maximum dose of 200 million cells, on Day 0 and Day 7. The primary evaluation period concludes for each participant 100 days after the first dose of CYP-001. Participants will have study visits on Days 0, 3, 7, 14, 21, 28, 60 and 100. Subsequently, participants will enter a long term follow-up period, which concludes 2 years after the first dose of CYP-001.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : March 1, 2017
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : May 2020


Arm Intervention/treatment
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)
Biological: 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.
Other Name: CYP-001

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)
Biological: 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.
Other Name: CYP-001




Primary Outcome Measures :
  1. Incidence and severity of treatment emergent adverse events [safety and tolerability] [ Time Frame: 28 days ]
    Safety

  2. Incidence and severity of serious adverse events deemed possibly related to CYP-001 [safety and tolerability] [ Time Frame: 100 days ]
    Safety


Secondary Outcome Measures :
  1. Complete Response by Day 28 [ Time Frame: 28 days ]
    Proportion of participants who show a Complete Response (absence of any signs or symptoms of GvHD) by Day 28

  2. Partial Response by Day 28 [ Time Frame: 28 days ]
    Proportion of participants who show a Partial Response (improvement in the severity of GvHD by at least one grade compared to baseline) by Day 28

  3. Overall Survival at Day 28 [ Time Frame: 28 days ]
    Proportion of participants who survive until Day 28

  4. Complete Response by Day 100 [ Time Frame: 100 days ]
    Proportion of participants who show a Complete Response by Day 100

  5. Partial Response by Day 100 [ Time Frame: 100 days ]
    Proportion of participants who show a Partial Response by Day 100

  6. Overall Survival at Day 100 [ Time Frame: 100 days ]
    Proportion of participants who survive until Day 100



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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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).

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02923375


Locations
Australia, New South Wales
Sydney Local Health District
Sydney, New South Wales, Australia
Australia, South Australia
Royal Adelaide Hospital
Adelaide, South Australia, Australia
United Kingdom
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
Sponsors and Collaborators
Cynata Therapeutics Limited
Investigators
Study Director: Kilian Kelly, PhD Cynata Therapeutics Limited

Responsible Party: Cynata Therapeutics Limited
ClinicalTrials.gov Identifier: NCT02923375     History of Changes
Other Study ID Numbers: CYP-GvHD-P1-01
2016-000070-38 ( EudraCT Number )
First Posted: October 4, 2016    Key Record Dates
Last Update Posted: May 30, 2018
Last Verified: May 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes

Additional relevant MeSH terms:
Graft vs Host Disease
Immune System Diseases