Amyloidosis
Conditions
Brief summary
The purpose of this study is to evaluate the efficacy and safety of SCTC21C plus cyclophosphamide, bortezomib and dexamethasone (VCd) compared with VCd alone in treatment of newly diagnosed amyloid light chain (AL) amyloidosis participants.
Detailed description
This study comprises two phases: Part 1 is the safety run in, while Part 2 is a randomized, controlled, open-label, multicenter study. Both parts are divided into three stages: the screening period (up to 28 days before first dose/randomization), the treatment period (from Cycle 1 \[28 days\] Day 1 and continues until disease progression or unacceptable toxicity), and the follow-up period (Postintervention). Safety endpoints include treatment-emergent adverse events , treatment-related adverse events, serious adverse events, clinical laboratory tests, vital signs, physical examinations, electrocardiograms , etc. Efficacy endpoints include Overall Complete Hematologic Response (CHR)，objective response rate (ORR), Hematologic Very Good Partial Response (VGPR) or Better Rate, Overall Survival (OS).
Interventions
DRUGSCTC21C
Pharmaceutical form: Solution for infusion； Route of administration: Subcutaneous
DRUGBortezomib
Pharmaceutical form: Lyophilized powder for injection； Route of administration: Subcutaneous
DRUGDexamethasone
Pharmaceutical form: Tablets, ampoules or vials for injection； Route of administration: Oral/Intravenous
DRUGCyclophosphamide
Pharmaceutical form: Tablets, ampoules or vials for injection； Route of administration: Oral/Intravenous
Sponsors
Sinocelltech Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Histopathological diagnosis of amyloidosis based on detection by immunohistochemistry and polarizing light microscopy of green bi-refringent material in congo red stained tissue specimens or characteristic electron microscopy appearance; * Measurable disease of amyloid light-chain (AL) amyloidosis; * One or more organs impacted by AL amyloidosis according to consensus guidelines * Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) 0, 1 or 2
Exclusion criteria
* Prior therapy for AL amyloidosis; * Other amyloidosis; * Uncontrolled infection. * Subjects with conditions that may affect safety or efficacy assessments include, but are not limited to, cardiovascular, respiratory, endocrine/metabolic, immune system, hepatic, gastrointestinal (such as gastrointestinal bleeding, perforation, ulcers, etc.), and malignant neoplasms, and are deemed clinically significant by the investigator. * Subjects who have undergone major surgery or experienced significant trauma within 4 weeks prior to the first use of the investigational drug, or who require elective surgery during the trial period. * Received a live or attenuated vaccine within 30 days prior to the first dose; Female subjects who are currently breastfeeding. * Subjects with mental disorders or poor compliance, or other circumstances deemed unsuitable for participation in this study by other investigators.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Overall Complete Hematologic Response (CHR) | Up to approximately 50 months after the First Participant In (FPI) | Overall CHR rate was defined as percentage of participants who achieved CHR, according to the International Amyloidosis Consensus Criteria. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Major Organ Deterioration Progression-Free Survival (MOD-PFS) | Up to approximately 50 months after the FPI | MOD-PFS was defined as duration from the date of randomization to either hematologic progression, or major organ deterioration (clinical manifestation of cardiac failure or renal failure), or death, whichever occurred first. |
| Percentage of Participants Who Achieved Complete Hematologic Response (CHR) at 6 Months | Month 6 | CHR rate was defined as percentage of participants who achieved CHR, according to the International Amyloidosis Consensus Criteria. |
| Duration of Complete Hematologic Response (CHR) | Up to approximately 50 months after the FPI | Duration of CHR was defined as the time between the date of initial documentation of CHR to the date of first documented evidence of hematologic progressive diseased. |
| Hematologic Very Good Partial Response (VGPR) or Better Rate | Up to approximately 50 months after the FPI | Hematologic VGPR or Better Rate was defined as percentage of participants who achieved hematologic Complete response (CR) or VGPR. |
| Overall Survival (OS) | Up to approximately 50 months after the FPI | Overall survival (OS) was measured from the date of randomization to the date of the participant's death. |
| Adverse Events | Up to approximately 50 months after the FPI | Treatment-emergent adverse events/serious adverse events |
Countries
China
Outcome results
None listed