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

NCT04189432 | Unknown Phase 2

• 1 other identifier: SCM-CGH2001
• Study Type: interventional
• Target: 84
• Locations: 1 country
• Sites: 11

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 \[2014\]).

Conditions

Chronic Graft-versus-host-disease

Outcome Measures

Primary Outcomes (1)

• 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.

  Week 12

Secondary Outcomes (5)

• Percentage of Participants who Achieve Complete Response (CR) or Partial Response (PR) (i.e. Overall Response Rate [ORR]).

  Week 0, 2, 4, 6, 8, 16, 20, 24 and 48

• Organ-specific Assessments

  Week 0, 2, 4, 6, 8, 16, 20, 24 and 48

• Patient-Reported Outcomes

  Week 0, 2, 4, 6, 8, 16, 20, 24 and 48

• Clinician-Assessed Global Rating/Scale

  Week 0, 2, 4, 6, 8, 16, 20, 24 and 48

• Failure-free Survival

  Week 24 and 48

Study Arms (2)

SCM-CGH

EXPERIMENTAL

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

Biological: SCM-CGH

Placebo

PLACEBO COMPARATOR

3 times with 2-week intervals by IV infusion.

Other: Placebo

Interventions

SCM-CGH BIOLOGICAL

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).

SCM-CGH

Placebo OTHER

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).

Placebo

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• SCM Lifescience Co., LTD.: lead

Study Sites (11)

National Cancer Center

Goyang-si, Gyeonggi-do, 10408, South Korea

Location

Chonnam National University Hwasun Hospital

Hwasun, Jeollanam-do, 58128, South Korea

Location

Pusan National University Hospital

Busan, 49241, South Korea

Location

Kosin University Gospel Hospital

Busan, 49267, South Korea

Location

Kyungpook National University Hospital

Daegu, South Korea

Location

Inha University Hospital

Incheon, 22332, South Korea

Location

Seoul National University Seoul

Seoul, 03080, South Korea

Location

Severance Hospital

Seoul, 03722, South Korea

Location

Asan Medical Center

Seoul, 05505, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

The Catholic University of Korea Seoul St. Mary's Hospital

Seoul, 06591, South Korea

Location

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: A Multicenter, Randomized, Parallel Group, Double-blind

Study Record Dates

• First Submitted: December 4, 2019
• First Posted: December 6, 2019
• Study Start: September 28, 2016
• Primary Completion: March 31, 2024
• Study Completion: September 30, 2024
• Last Updated: June 8, 2023

Record last verified: 2023-06

Data Sharing

• IPD Sharing: Will not share

Locations

KR (11)