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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02923375
Recruitment Status : Active, not recruiting
First Posted : October 4, 2016
Last Update Posted : January 30, 2019
Sponsor:
Information provided by (Responsible Party):
Cynata Therapeutics Limited

Tracking Information
First Submitted Date  ICMJE October 3, 2016
First Posted Date  ICMJE October 4, 2016
Last Update Posted Date January 30, 2019
Actual Study Start Date  ICMJE March 1, 2017
Actual Primary Completion Date August 28, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 2, 2017)
  • Incidence and severity of treatment emergent adverse events [safety and tolerability] [ Time Frame: 28 days ]
    Safety
  • Incidence and severity of serious adverse events deemed possibly related to CYP-001 [safety and tolerability] [ Time Frame: 100 days ]
    Safety
Original Primary Outcome Measures  ICMJE
 (submitted: October 3, 2016)
Safety and tolerability [ Time Frame: 100 days ]
Physical examination evaluations, treatment emergent adverse events, safety laboratory evaluations, vital signs, pulse oximtery
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 3, 2016)
  • 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
  • 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
  • Overall Survival at Day 28 [ Time Frame: 28 days ]
    Proportion of participants who survive until Day 28
  • Complete Response by Day 100 [ Time Frame: 100 days ]
    Proportion of participants who show a Complete Response by Day 100
  • Partial Response by Day 100 [ Time Frame: 100 days ]
    Proportion of participants who show a Partial Response by Day 100
  • Overall Survival at Day 100 [ Time Frame: 100 days ]
    Proportion of participants who survive until Day 100
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of CYP-001 for the Treatment of Steroid-Resistant Acute Graft Versus Host Disease
Official Title  ICMJE 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
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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Graft vs Host Disease
Intervention  ICMJE 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
Study Arms  ICMJE
  • 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)
    Intervention: Biological: Mesenchymoangioblast-derived mesenchymal stem cells
  • 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)
    Intervention: Biological: Mesenchymoangioblast-derived mesenchymal stem cells
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: October 3, 2016)
16
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 2020
Actual Primary Completion Date August 28, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02923375
Other Study ID Numbers  ICMJE CYP-GvHD-P1-01
2016-000070-38 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Cynata Therapeutics Limited
Study Sponsor  ICMJE Cynata Therapeutics Limited
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Kilian Kelly, PhD Cynata Therapeutics Limited
PRS Account Cynata Therapeutics Limited
Verification Date January 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP