Multiple Myeloma (MM)
Conditions
Keywords
extramedullary multiple myeloma
Brief summary
This is a prospective, single-arm, exploratory Phase II clinical study evaluating the efficacy and safety of HP-001 capsules in combination with dexamethasone in patients with relapsed or refractory multiple myeloma (RRMM) with extramedullary disease (EMD).
Interventions
DRUGHP-001
HP-001 capsules will be administered orally at a dose of 0.6 mg once daily on Days 1-10 of each 28-day treatment cycle.
DRUGDexamethasone
Dexamethasone will be administered at a dose of 40 mg orally or intravenously on Days 1, 8, 15, and 22 of each 28-day treatment cycle.
Sponsors
Institute of Hematology & Blood Diseases Hospital, China
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Age ≥18 years at the time of signing informed consent; male or female. 2. ECOG performance status of 0-2. 3. Diagnosis of multiple myeloma according to IMWG criteria, with relapsed/refractory disease after at least 1 prior systemic treatment regimen; disease progression or failure to achieve a response after the most recent line of therapy; and extramedullary disease confirmed by imaging, defined as at least 1 soft-tissue extramedullary lesion ≥2 cm, at least 1 paramedullary lesion ≥5 cm, or \>2 lesions. 4. With or without measurable hematologic disease at screening. Measurable disease, if present, is defined as serum M-protein ≥1.0 g/dL, urine M-protein ≥200 mg/24 hours, or serum free light chain ≥10 mg/dL with an abnormal free light chain ratio. 5. Adequate organ function, including ANC ≥1.0 × 10⁹/L, hemoglobin ≥60 g/L, platelet count ≥50 × 10⁹/L, creatinine clearance ≥30 mL/min, total bilirubin ≤2 × ULN (≤3 × ULN for Gilbert syndrome), AST and ALT ≤2.5 × ULN, and INR or aPTT ≤1.5 × ULN. 6. Willing and able to comply with study procedures and follow-up.
Exclusion criteria
1. Smoldering multiple myeloma, monoclonal gammopathy of undetermined significance, Waldenström macroglobulinemia, POEMS syndrome, amyloidosis, or primary/secondary plasma cell leukemia. 2. Central nervous system involvement or clinical evidence of meningeal involvement. 3. Severe or uncontrolled cardiovascular disease, including unstable angina, symptomatic congestive heart failure, myocardial infarction within 6 months before enrollment, severe uncontrolled arrhythmia, or other cardiovascular/cerebrovascular conditions considered unsuitable by the investigator. 4. Major surgery within 4 weeks before the first dose or planned major surgery during the study. 5. Active infection, including HIV infection, active hepatitis B (HBV-DNA positive), active hepatitis C (HCV-RNA positive), active or latent syphilis, active tuberculosis, or other active infections considered unsuitable by the investigator. 6. Concurrent malignancy or other serious concomitant disease that may compromise participant safety or completion of the study. 7. Pregnant or breastfeeding women. 8. History of severe allergy or hypersensitivity to any component of the study treatment. 9. Any other condition that, in the investigator's judgment, makes the participant unsuitable for enrollment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall Response Rate (ORR) | From the first dose to the end of Cycle 24 (each cycle is 28 days). |
Secondary
| Measure | Time frame |
|---|---|
| Extramedullary Disease Objective Response Rate (EMD-ORR) | From the first dose to the end of Cycle 24 (each cycle is 28 days). |
| Very Good Partial Response or Better Rate (≥VGPR Rate) | From the first dose to the end of Cycle 24 (each cycle is 28 days). |
| Complete Response or Stringent Complete Response Rate (CR/sCR Rate) | From the first dose to the end of Cycle 24 (each cycle is 28 days). |
| Time to Response (TTR) | From the first dose to the date of the first documented overall response of PR or better, assessed through the end of Cycle 24 (each cycle is 28 days). |
| Duration of Response (DoR) | From the date of the first documented overall response of PR or better to the date of disease progression or death from any cause, whichever occurs first, with follow-up through 12 months after the last dose. |
| Progression-Free Survival (PFS) | From the date of the first dose to the date of disease progression or death from any cause, whichever occurs first, with follow-up through 12 months after the last dose. |
| Overall Survival (OS) | From the date of the first dose to the date of death from any cause, with follow-up through 12 months after the last dose. |
| Incidence and Severity of Adverse Events (AEs) | Up to 2 years. |
Countries
China
Contacts
CONTACTGang An, PhD&MD
Outcome results
None listed