HP-001 Plus Dexamethasone for Relapsed/Refractory Multiple Myeloma With Extramedullary Disease (BEYOND-MM 001)

September 4, 2026 updated by: Institute of Hematology & Blood Diseases Hospital, China

A Prospective, Single-Arm, Exploratory Phase II Clinical Trial of HP-001 in Combination With Dexamethasone for Relapsed/Refractory Multiple Myeloma With Extramedullary Disease

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).

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

20

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
      • Tianjin, China
        • Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences, Tianjin, 300000
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: HP-001 Plus Dexamethasone
Participants will receive HP-001 capsules at 0.6 mg orally once daily on Days 1-10 of each 28-day treatment cycle, in combination with dexamethasone 40 mg administered orally or intravenously on Days 1, 8, 15, and 22 of each cycle. Treatment will continue until disease progression, unacceptable toxicity, withdrawal of consent, the investigator determines that continued treatment is no longer appropriate, or completion of 24 treatment cycles, whichever occurs first.
Drug: HP-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.
Drug: Dexamethasone
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Overall Response Rate (ORR)
Time Frame: From the first dose to the end of Cycle 24 (each cycle is 28 days).
From the first dose to the end of Cycle 24 (each cycle is 28 days).

Secondary Outcome Measures

Outcome Measure
Time Frame
Extramedullary Disease Objective Response Rate (EMD-ORR)
Time Frame: From the first dose to the end of Cycle 24 (each cycle is 28 days).
From the first dose to the end of Cycle 24 (each cycle is 28 days).
Very Good Partial Response or Better Rate (≥VGPR Rate)
Time Frame: From the first dose to the end of Cycle 24 (each cycle is 28 days).
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)
Time Frame: From the first dose to the end of Cycle 24 (each cycle is 28 days).
From the first dose to the end of Cycle 24 (each cycle is 28 days).
Time to Response (TTR)
Time Frame: 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).
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)
Time Frame: 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.
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)
Time Frame: 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.
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)
Time Frame: 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.
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)
Time Frame: Up to 2 years.
Up to 2 years.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

September 1, 2026

Primary Completion (Estimated)

December 31, 2027

Study Completion (Estimated)

December 31, 2028

Study Registration Dates

First Submitted

August 24, 2026

First Submitted That Met QC Criteria

September 4, 2026

First Posted (Actual)

September 10, 2026

Study Record Updates

Last Update Posted (Actual)

September 10, 2026

Last Update Submitted That Met QC Criteria

September 4, 2026

Last Verified

September 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IIT2026091

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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