search
Back to results

Efficacy and Safety of SCM-CGH in Patients With Steroid-Refractory or Dependent Chronic Graft-Versus-Host Disease

Primary Purpose

Chronic Graft-versus-host-disease

Status
Active
Phase
Phase 2
Locations
Korea, Republic of
Study Type
Interventional
Intervention
SCM-CGH
Placebo
Sponsored by
SCM Lifescience Co., LTD.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Chronic Graft-versus-host-disease

Eligibility Criteria

19 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Subjects who are males or females aged >= 19 years, 40kg to 80kg in weight
  • Steroid dependent/refractory chronic graft versus host disease (cGVHD) defined as the National Institutes of Health (NIH) criteria (2014) below at any time post-hematopoietic cell transplant (post-HCT):

Refractory disease, defined as, 1) when cGVHD manifestations progress despite the use of a regimen containing glucocorticoid (prednisolone at >=1 mg/kg/day for at least 2 weeks) or 2), 3) Persist without improvement despite continued treatment with glucocorticoid (prednisolone at >=0.5 mg/kg/day or 1 mg/kg every other day) for at least 4 weeks Dependent disease, defined as, 4), 5) when glucocorticoid (prednisolone doses greater than or equal to [>=] 0.25 milligram per kilogram per day (mg/kg/day)or >=0.5 milligram per kilogram (mg/kg) every other day) are needed to prevent recurrence or progression of manifestations as demonstrated by unsuccessful attempts to taper the dose to lower levels on at least 2 occasions, separated by at least 8 weeks.

  • Participants must be receiving less than 3 systemic glucocorticoid therapies or other immunosuppressive therapies in addition to glucocorticoids for cGVHD for at least 4 weeks before Screening visit. The dose of steroids or Immunosuppressant must be stable for 14 days(2 weeks) prior to starting SCM-CGH or Placebo.
  • Laboratory test sufficiency as follows; Absolute neutrophil count (ANC) ≥ 1,000 cells/mm3 Serum creatinine < 2 x upper limit of normal (ULN)

Exclusion Criteria:

  • Active acute graft versus host disease (GVHD)
  • Active infection with human immunodeficiency virus (HIV), hepatitis B virus, or hepatitis C virus (HCV)
  • Uncontrolled underlying disease such as moderate or severe infections and hemorrhage
  • Severe Heart failure (NYHA class III/IV), congestive heart failure or arrhythmia requiring treatment
  • History of allogenic hematopoietic stem cell more than once
  • Positive reaction of a Penicillin test at screening
  • History of relapse of causative diseases (ALL, CML, CLL, AML, NHL, multiple myeloma e.t.c.) with hematopoietic stem cell transplantation or diagnosed with secondary malignant diseases after hematopoietic stem cell transplantation
  • History of Anti-thymocyte globulin(ATG) for 2 weeks before Screening visit
  • History of pulmonary embolism or deep venous thrombosis for 24 weeks before Screening visit

Sites / Locations

  • National Cancer Center
  • Chonnam National University Hwasun Hospital
  • Pusan National University Hospital
  • Kosin University Gospel Hospital
  • Kyungpook National University Hospital
  • Inha University Hospital
  • Seoul National University Seoul
  • Severance Hospital
  • Asan Medical Center
  • Samsung Medical Center
  • The Catholic University of Korea Seoul St. Mary's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

SCM-CGH

Placebo

Arm Description

Ingredient: Allogeneic human bone marrow-derived mesenchymal stem cells Dose: 1x10^6 cells/Kg

3 times with 2-week intervals by IV infusion.

Outcomes

Primary Outcome Measures

Percentage of Participants who Achieve Complete Response (CR) or Partial Response (PR) (i.e. Overall Response Rate [ORR])
ORR is defined as the percentage of participants who achieve complete response (CR) or partial response (PR). Response is defined by the National Institutes of Health (NIH) Consensus Development Project Criteria (2014) and must occur, in the absence of new therapy for chronic graft versus host disease (cGVHD) and absence of progression of underlying disease or death. CR: Resolution of all manifestations in each organ or site. PR: Improvement in at least 1 organ or site without progression in any other organ or site. cGVHD Progression: Clinically meaningful worsening in 1 or more organs regardless of improvement in other organs.

Secondary Outcome Measures

Percentage of Participants who Achieve Complete Response (CR) or Partial Response (PR) (i.e. Overall Response Rate [ORR]).
ORR is defined as the percentage of participants who achieve complete response (CR) or partial response (PR). Response is defined by the National Institutes of Health (NIH) Consensus Development Project Criteria (2014) and must occur, in the absence of new therapy for chronic graft versus host disease (cGVHD) and absence of progression of underlying disease or death. CR: Resolution of all manifestations in each organ or site. PR: Improvement in at least 1 organ or site without progression in any other organ or site. cGVHD Progression: Clinically meaningful worsening in 1 or more organs regardless of improvement in other organs.
Organ-specific Assessments
Percentage of participants with organ-specific assessments is defined as rate of NIH defined CR or PR.
Patient-Reported Outcomes
Patient-reported Chronic GVHD-specific Measures are defined by the National Institutes of Health (NIH) Consensus Development Project Criteria (2014).
Clinician-Assessed Global Rating/Scale
Global Rating(0~3)/Scale(0~10) of clinician-assessed chronic GVHD-specific measures are defined by the National Institutes of Health (NIH) Consensus Development Project Criteria (2014).
Failure-free Survival
Failure-free survival, defined as absence of relapse, death and addition of new treatment, survival free of impairment, or reduction in steroid dosing that do not rely on direct assessment of organ responses.

Full Information

First Posted
December 4, 2019
Last Updated
June 7, 2023
Sponsor
SCM Lifescience Co., LTD.
search

1. Study Identification

Unique Protocol Identification Number
NCT04189432
Brief Title
Efficacy and Safety of SCM-CGH in Patients With Steroid-Refractory or Dependent Chronic Graft-Versus-Host Disease
Official Title
A Multicenter, Randomized, Parallel Group, Double-blind, Phase 2 Trial to Evaluate Efficacy and Safety of SCM-CGH in Patients With Steroid-Refractory or Dependent Chronic Graft-Versus-Host Disease
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 28, 2016 (Actual)
Primary Completion Date
March 31, 2024 (Anticipated)
Study Completion Date
September 30, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
SCM Lifescience Co., LTD.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief Summary
The purpose of this study is to evaluate efficacy of SCM-CGH in participants with steroid dependent/refractory chronic graft versus host disease (cGVHD) by measuring overall cGVHD response (complete response [CR] and partial response [PR] defined by National Institutes of Health [NIH] consensus development project criteria [2014]).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chronic Graft-versus-host-disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
A Multicenter, Randomized, Parallel Group, Double-blind
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
84 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
SCM-CGH
Arm Type
Experimental
Arm Description
Ingredient: Allogeneic human bone marrow-derived mesenchymal stem cells Dose: 1x10^6 cells/Kg
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
3 times with 2-week intervals by IV infusion.
Intervention Type
Biological
Intervention Name(s)
SCM-CGH
Intervention Description
SCM-CGH will be administrated 3 times with 2-week intervals by IV infusion to subjects at Weeks 0, 2 and 4 (Visits 2, 3 and 4).
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Placebo will be administrated 3 times with 2-week intervals by IV infusion to subjects at Weeks 0, 2 and 4 (Visits 2, 3 and 4).
Primary Outcome Measure Information:
Title
Percentage of Participants who Achieve Complete Response (CR) or Partial Response (PR) (i.e. Overall Response Rate [ORR])
Description
ORR is defined as the percentage of participants who achieve complete response (CR) or partial response (PR). Response is defined by the National Institutes of Health (NIH) Consensus Development Project Criteria (2014) and must occur, in the absence of new therapy for chronic graft versus host disease (cGVHD) and absence of progression of underlying disease or death. CR: Resolution of all manifestations in each organ or site. PR: Improvement in at least 1 organ or site without progression in any other organ or site. cGVHD Progression: Clinically meaningful worsening in 1 or more organs regardless of improvement in other organs.
Time Frame
Week 12
Secondary Outcome Measure Information:
Title
Percentage of Participants who Achieve Complete Response (CR) or Partial Response (PR) (i.e. Overall Response Rate [ORR]).
Description
ORR is defined as the percentage of participants who achieve complete response (CR) or partial response (PR). Response is defined by the National Institutes of Health (NIH) Consensus Development Project Criteria (2014) and must occur, in the absence of new therapy for chronic graft versus host disease (cGVHD) and absence of progression of underlying disease or death. CR: Resolution of all manifestations in each organ or site. PR: Improvement in at least 1 organ or site without progression in any other organ or site. cGVHD Progression: Clinically meaningful worsening in 1 or more organs regardless of improvement in other organs.
Time Frame
Week 0, 2, 4, 6, 8, 16, 20, 24 and 48
Title
Organ-specific Assessments
Description
Percentage of participants with organ-specific assessments is defined as rate of NIH defined CR or PR.
Time Frame
Week 0, 2, 4, 6, 8, 16, 20, 24 and 48
Title
Patient-Reported Outcomes
Description
Patient-reported Chronic GVHD-specific Measures are defined by the National Institutes of Health (NIH) Consensus Development Project Criteria (2014).
Time Frame
Week 0, 2, 4, 6, 8, 16, 20, 24 and 48
Title
Clinician-Assessed Global Rating/Scale
Description
Global Rating(0~3)/Scale(0~10) of clinician-assessed chronic GVHD-specific measures are defined by the National Institutes of Health (NIH) Consensus Development Project Criteria (2014).
Time Frame
Week 0, 2, 4, 6, 8, 16, 20, 24 and 48
Title
Failure-free Survival
Description
Failure-free survival, defined as absence of relapse, death and addition of new treatment, survival free of impairment, or reduction in steroid dosing that do not rely on direct assessment of organ responses.
Time Frame
Week 24 and 48

10. Eligibility

Sex
All
Minimum Age & Unit of Time
19 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Subjects who are males or females aged >= 19 years, 40kg to 80kg in weight Steroid dependent/refractory chronic graft versus host disease (cGVHD) defined as the National Institutes of Health (NIH) criteria (2014) below at any time post-hematopoietic cell transplant (post-HCT): Refractory disease, defined as, 1) when cGVHD manifestations progress despite the use of a regimen containing glucocorticoid (prednisolone at >=1 mg/kg/day for at least 2 weeks) or 2), 3) Persist without improvement despite continued treatment with glucocorticoid (prednisolone at >=0.5 mg/kg/day or 1 mg/kg every other day) for at least 4 weeks Dependent disease, defined as, 4), 5) when glucocorticoid (prednisolone doses greater than or equal to [>=] 0.25 milligram per kilogram per day (mg/kg/day)or >=0.5 milligram per kilogram (mg/kg) every other day) are needed to prevent recurrence or progression of manifestations as demonstrated by unsuccessful attempts to taper the dose to lower levels on at least 2 occasions, separated by at least 8 weeks. Participants must be receiving less than 3 systemic glucocorticoid therapies or other immunosuppressive therapies in addition to glucocorticoids for cGVHD for at least 4 weeks before Screening visit. The dose of steroids or Immunosuppressant must be stable for 14 days(2 weeks) prior to starting SCM-CGH or Placebo. Laboratory test sufficiency as follows; Absolute neutrophil count (ANC) ≥ 1,000 cells/mm3 Serum creatinine < 2 x upper limit of normal (ULN) Exclusion Criteria: Active acute graft versus host disease (GVHD) Active infection with human immunodeficiency virus (HIV), hepatitis B virus, or hepatitis C virus (HCV) Uncontrolled underlying disease such as moderate or severe infections and hemorrhage Severe Heart failure (NYHA class III/IV), congestive heart failure or arrhythmia requiring treatment History of allogenic hematopoietic stem cell more than once Positive reaction of a Penicillin test at screening History of relapse of causative diseases (ALL, CML, CLL, AML, NHL, multiple myeloma e.t.c.) with hematopoietic stem cell transplantation or diagnosed with secondary malignant diseases after hematopoietic stem cell transplantation History of Anti-thymocyte globulin(ATG) for 2 weeks before Screening visit History of pulmonary embolism or deep venous thrombosis for 24 weeks before Screening visit
Facility Information:
Facility Name
National Cancer Center
City
Goyang-si
State/Province
Gyeonggi-do
ZIP/Postal Code
10408
Country
Korea, Republic of
Facility Name
Chonnam National University Hwasun Hospital
City
Hwasun
State/Province
Jeollanam-do
ZIP/Postal Code
58128
Country
Korea, Republic of
Facility Name
Pusan National University Hospital
City
Busan
ZIP/Postal Code
49241
Country
Korea, Republic of
Facility Name
Kosin University Gospel Hospital
City
Busan
ZIP/Postal Code
49267
Country
Korea, Republic of
Facility Name
Kyungpook National University Hospital
City
Daegu
Country
Korea, Republic of
Facility Name
Inha University Hospital
City
Incheon
ZIP/Postal Code
22332
Country
Korea, Republic of
Facility Name
Seoul National University Seoul
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Facility Name
Severance Hospital
City
Seoul
ZIP/Postal Code
03722
Country
Korea, Republic of
Facility Name
Asan Medical Center
City
Seoul
ZIP/Postal Code
05505
Country
Korea, Republic of
Facility Name
Samsung Medical Center
City
Seoul
ZIP/Postal Code
06351
Country
Korea, Republic of
Facility Name
The Catholic University of Korea Seoul St. Mary's Hospital
City
Seoul
ZIP/Postal Code
06591
Country
Korea, Republic of

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Efficacy and Safety of SCM-CGH in Patients With Steroid-Refractory or Dependent Chronic Graft-Versus-Host Disease

We'll reach out to this number within 24 hrs