Clinical trial NCT04189432: 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
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Chronic Graft-versus-host-disease
Lead sponsor: SCM Lifescience Co., LTD.
Sponsor class: INDUSTRY
First posted: 2019-12-06
Enrollment: 84
Sex: ALL
Minimum age (years): 19
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: A Multicenter, Randomized, Parallel Group, Double-blind
Primary purpose: TREATMENT

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 \[2014\]).


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

Primary outcomes:
measure: Percentage of Participants who Achieve Complete Response (CR) or Partial Response (PR) (i.e. Overall Response Rate [ORR])
timeFrame: Week 12
Secondary outcomes:
measure: Percentage of Participants who Achieve Complete Response (CR) or Partial Response (PR) (i.e. Overall Response Rate [ORR]).
timeFrame: Week 0, 2, 4, 6, 8, 16, 20, 24 and 48
measure: Organ-specific Assessments
timeFrame: Week 0, 2, 4, 6, 8, 16, 20, 24 and 48
measure: Patient-Reported Outcomes
timeFrame: Week 0, 2, 4, 6, 8, 16, 20, 24 and 48
measure: Clinician-Assessed Global Rating/Scale
timeFrame: Week 0, 2, 4, 6, 8, 16, 20, 24 and 48
measure: Failure-free Survival
timeFrame: Week 24 and 48
Other outcomes:
Locations:
city: Goyang-si
country: Korea, Republic of
facility: National Cancer Center
state: Gyeonggi-do
city: Hwasun
country: Korea, Republic of
facility: Chonnam National University Hwasun Hospital
state: Jeollanam-do
city: Busan
country: Korea, Republic of
facility: Pusan National University Hospital
city: Busan
country: Korea, Republic of
facility: Kosin University Gospel Hospital
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Hospital
city: Incheon
country: Korea, Republic of
facility: Inha University Hospital
city: Seoul
country: Korea, Republic of
facility: Seoul National University Seoul
city: Seoul
country: Korea, Republic of
facility: Severance Hospital
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: The Catholic University of Korea Seoul St. Mary's Hospital
Study officials:
References: