Clinical Study of Safety and Tolerability of Melphalan Hydrochloride for R/R MM

NCT05438394 | Unknown Phase 1 FDA Drug

• 1 other identifier: 2021PHD013-001
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

This study is an investigator-initiated clinical study to evaluate the safety and tolerability of melphalan hydrochloride for injection in patients with relapsed or relapsed refractory multiple myeloma. Using the "3 + 3" dose escalation principle, 3-6 subjects per dose were enrolled, depending on the dose limiting toxicity (DLT) observed in the first cycle of chemotherapy for each subject. After completing the 21-day assessment of the first cycle of chemotherapy, if there was no DLT, the study started for the next dose group.

Conditions

Multiple Myeloma

Outcome Measures

Primary Outcomes (1)

• Dose limiting toxicity (DLT)

  Hematological toxicity: Grade 4 thrombocytopenia OR Grade 4 neutropenia more than 7 days; Any non - hematological toxicity of grade 3 or above, except: 1) Grade 3 nausea / vomiting or diarrhea with duration less than 72 hours; 2) Grade 3 fatigue with duration less than one week; Grade 3 fever; Grade 4 infusion reaction;

  one month

Secondary Outcomes (4)

• The overall response rate

  one month

• TTP

  12 months

• Progression-Free Survival (PFS)

  12 months

• Overall Survival (OS)

  12 months

Study Arms (1)

melphalan hydrochloride for injection

EXPERIMENTAL

Drug Name: melphalan hydrochloride The dosage of different dosage groups were: 9 mg/m2; 18 mg/m2; 27 mg/m2; 40 mg/m2 Administration frequency: once for each subject

Drug: melphalan hydrochloride for injection

Interventions

melphalan hydrochloride for injection DRUG

Intravenous (iv) infusion for 20 minutes on Day 1 of a 21-day chemotherapy cycle

melphalan hydrochloride for injection

Eligibility Criteria

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

You may qualify if:

• Age ≥ 18 years old, male or female;
• Relapsed or refractory multiple myeloma according to IMWG. There is no requirement for the number of previous treatment lines in this study
• With any one of the following measurable indicators: serum protein electrophoresis (SPEP) ≥ 1 g/dL (10 g/L), if IgA, IgD, IgE or IgM multiple myeloma subjects, ≥ 0.5 g/dL (≥ 5 g/L); urine M protein ≥ 200 mg/24h; serum free light chain (FLC) ≥ 10 mg/dL and abnormal serum free light chain kappa/lambda ratio;
• Life expectancy ≥ 6 months;
• ECOG score ≤ 2;
• ECG QT interval ≤ 470 ms;
• Neutrophil ≥ 1.0\*10\^9/L, platelet ≥ 75\*10\^9/L (or 50 \*10\^9/L if plasma cell infiltration in bone marrow more than 50%);
• Tbil ≤1.5 ULN，AST and ALT≤3.0 ULN （Gilbert syndrome except);
• eGFR ≥ 45 ml/min or creatinine ≤ 2mg/dl
• Understand the contents of this study and have signed the informed consent form.

You may not qualify if:

• primary amyloidosis, MGUS, or smoldering multiple myeloma or plasma cell leukemia (according to the World Health Organization criteria: peripheral blood ≥ 5%) or POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin) ;
• primary refractory disease (i.e., no at least MR response to any prior therapy);
• combined clinically significant comorbidities (investigator judged);
• the presence of active infection;
• history of other malignant tumors (except multiple myeloma) within 3 years before signing the informed consent;
• Pregnant or lactating women;
• radiotherapy or other anti-myeloma treatment within 14 days before signing the informed consent;

Sponsors & Collaborators

• Peking University People's Hospital: lead
• CASI Pharmaceuticals, Inc.: collaborator

Study Sites (1)

Peking University People's Hospital

Beijing, Beijing Municipality, 100044, China

RECRUITING

MeSH Terms

Conditions

Multiple Myeloma

Interventions

Melphalan; Injections

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Drug Administration Routes; Drug Therapy; Therapeutics

Study Officials

• Jin Lu

  Peking University People's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yang Liu

CONTACT

+86-13716926210; pkuphliuyang@bjmu.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: Four dose groups. Using the "3 + 3" dose escalation principle, 3-6 subjects per dose group, according to the dose limiting toxicity (DLT) observed in the first cycle of chemotherapy for each subject. After completing the 21-day assessment of the first cycle of chemotherapy, if there was no DLT, the study started for the next dose group.

Study Record Dates

• First Submitted: June 26, 2022
• First Posted: June 30, 2022
• Study Start: October 13, 2022
• Primary Completion: December 31, 2023
• Study Completion: December 31, 2023
• Last Updated: April 14, 2023

Record last verified: 2023-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)