Melphalan Hydrochloride in Treating Participants With Newly-Diagnosed Multiple Myeloma Undergoing Donor Stem Cell Transplantation

NCT03417284 | Completed Phase 1 Results FDA Drug

• 2 other identifiers: 2017-0399NCI-2018-00906
• Study Type: interventional
• Target: 62
• Locations: 1 country
• Sites: 1

Brief Summary

This phase I/II trial studies the side effects and best dose of melphalan hydrochloride in treating participants with newly-diagnosed multiple myeloma who are undergoing a donor stem cell transplantation. Giving chemotherapy before a donor stem cell transplantation helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the participant, they may help the participant's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Giving melphalan hydrochloride before a donor stem cell transplantation may work better than standard chemotherapy in helping to prevent multiple myeloma from coming back.

Conditions

Plasma Cell Myeloma

Outcome Measures

Primary Outcomes (1)

• Optimal Dose and Schedule of Evomela Prior to Autologous Transplant.

  Dose limiting toxicity will be defined as grade 4 mucositis or any grade 4 or 5 non-hematologic or non-infectious toxicity occurring within 30 days from the start of infusion.

  Within 30 days after the start of infusion

Secondary Outcomes (3)

• Progression-free Survival (PFS)

  From the date of Evomela injection up to 1 year

• Treatment Related Mortality (TRM).

  At day 90 post-transplant

• Number of Participants That Have Achieved Complete Response (CR).

  At 90 days post-transplant

Study Arms (2)

Group 1 (melphalan hydrochloride, HSCT, filgrastim)

EXPERIMENTAL

PREPARATIVE REGIMEN: Participants receive melphalan hydrochloride IV over 30-60 minutes on day -2. TRANSPLANT: Participants in both groups undergo donor stem cell transplantation IV on day 0 over 30-60 minutes. POST-TRANSPLANT: Participants in both groups receive filgrastim-sndz SC QD starting on day 5 and continuing in the absence of disease progression, unacceptable toxicity, or until evidence of an ANC of 0.5 x 10\^9/L.

Procedure: Autologous Hematopoietic Stem Cell Transplantation; Biological: Filgrastim-sndz; Drug: Melphalan Hydrochloride

Group 2 (melphalan hydrochloride, HSCT, filgrastim)

EXPERIMENTAL

PREPARATIVE REGIMEN: Participants receive melphalan hydrochloride IV over 8-9 hours on day -2. TRANSPLANT: Participants in both groups undergo donor stem cell transplantation IV on day 0 over 30-60 minutes. POST-TRANSPLANT: Participants in both groups receive filgrastim-sndz SC QD starting on day 5 and continuing in the absence of disease progression, unacceptable toxicity, or until evidence of an ANC of 0.5 x 10\^9/L.

Procedure: Autologous Hematopoietic Stem Cell Transplantation; Biological: Filgrastim-sndz; Drug: Melphalan Hydrochloride

Interventions

Autologous Hematopoietic Stem Cell Transplantation PROCEDURE

Undergo HSCT

Also known as: AHSCT, Autologous Hematopoietic Cell Transplantation, Autologous Stem Cell Transplantation, Stem Cell Transplantation, Autologous

Group 1 (melphalan hydrochloride, HSCT, filgrastim); Group 2 (melphalan hydrochloride, HSCT, filgrastim)

Filgrastim-sndz BIOLOGICAL

Given SC

Also known as: Filgrastim Biosimilar Filgrastim-sndz, Zarxio

Group 1 (melphalan hydrochloride, HSCT, filgrastim); Group 2 (melphalan hydrochloride, HSCT, filgrastim)

Melphalan Hydrochloride DRUG

Given IV

Also known as: Alkeran, Alkerana, Evomela

Group 1 (melphalan hydrochloride, HSCT, filgrastim); Group 2 (melphalan hydrochloride, HSCT, filgrastim)

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with non-relapsed multiple myeloma in complete response (CR), partial remission (PR), very good partial remission (VGPR), or symptomatic stable disease (no evidence of progression) including patients with light chain multiple myeloma (MM) detected in the serum by free light chain assay OR
• Patients with non-secretory multiple myeloma (absence of a monoclonal protein \[M protein\] in serum as measured by electrophoresis \[serum protein electrophoresis (SPEP)\] and immunofixation (serum immunofixation electrophoresis \[SIFE\]) and the absence of Bence Jones protein in the urine \[urine protein electrophoresis (UPEP)\] defined by use of conventional electrophoresis and immunofixation \[urine immunofixation electrophoresis (UIFE) techniques\]) but with measurable disease on imaging studies like magnetic resonance imaging (MRI), computed tomography (CT) scan or positron emission tomography (PET) scan.
• Patients who have received at least two cycles of initial systemic therapy and are within 2 to 12 months of the first dose. Mobilization therapy is not considered initial therapy.
• Karnofsky performance score 70% or higher.
• Left ventricular ejection fraction at rest \> 40% within 3 months of registration.
• Bilirubin \< 2 x the upper limit of normal (except patients with Gilbert syndrome in whom bilirubin level of \> 2 x upper normal limit will be allowed)
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \< 2.5 x the upper limit of normal.
• Creatinine clearance of \>= 40 mL/min, estimated or calculated using the Cockcroft-Gault equation.
• Pulmonary function: diffusing capacity of the lungs for carbon monoxide (DLCO), forced expiratory volume in one second (FEV1), forced vital capacity (FVC) \> 50% of predicted value (corrected for hemoglobin) within 3 months of registration.
• All female and male subjects of reproductive potential must consent to the use of effective contraceptive methods as advised by the study doctor during treatment.
• Signed informed consent form.

You may not qualify if:

• Patients with uncontrolled bacterial, viral or fungal infections (currently taking medication and progression of clinical symptoms).
• Patients seropositive for the human immunodeficiency virus (HIV).
• Patients with history of myocardial infarction within 6 months prior to enrollment or has New York Heart Association (NYHA) class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities.
• Patients participating in an investigational new drug protocol within 14 days before enrollment.
• Female patients who are pregnant (positive beta-human chorionic gonadotropin \[b-HCG\]) or breast feeding.
• Prior hematopoietic cell transplantation allogeneic or autologous (A prior autologous HCT will be allowed as long as it was part of tandem transplantation).
• Prior organ transplant requiring immunosuppressive therapy

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• University of Texas MD Anderson Cancer Center Website

MeSH Terms

Conditions

Multiple Myeloma

Interventions

Stem Cell Transplantation; Filgrastim; Granulocyte Colony-Stimulating Factor; Melphalan

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)

Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids

Results Point of Contact

• Title: Qaiser Bashir, MD / Stem Cell Transplantation
• Organization: The University of Texas MD Anderson Cancer Center

QBashir@mdanderson.org

713-794-4422

Study Officials

• Qaiser Bashir

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 24, 2018
• First Posted: January 31, 2018
• Study Start: October 9, 2019
• Primary Completion: July 18, 2024
• Study Completion: July 18, 2024
• Last Updated: May 1, 2025
• Results First Posted: May 1, 2025

Record last verified: 2024-07

Locations

US (1)