Melphalan

Generic Name: Melphalan

Brand Names: Alkeran, Evomela, Phelinun, Pepaxti

Drug Type: Small Molecule

Chemical Formula: C_₁₃H_₁₈Cl_₂N_₂O_₂

CAS Number: 148-82-3

Unique Ingredient Identifier: Q41OR9510P

Background: Melphalan is a nitrogen mustard or bischloroethylamine type alkylating agent. It was first synthesized in the early 1950s by substituting L-phenylalanine for the methyl group on nitrogen mustard. Melphalan is used in the treatment of multiple myeloma and ovarian carcinoma. It is also used for high-conditioning before hematopoietic stem cell transplant. It is also used to treat uveal melanoma with unresectable hepatic metastases.

Indication: Melphalan is indicated for use as a high-dose conditioning treatment prior to hematopoietic stem cell transplantation in patients with multiple myeloma. It is also indicated for the palliative treatment of multiple myeloma and for the palliation of non-resectable epithelial carcinoma of the ovary.

Melphalan is a component of HEPZATO KIT, a liver-directed therapy indicated for the treatment of adults with uveal melanoma with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation.

Associated Conditions: Multiple Myeloma (MM), Unresectable Liver Metastasis, Unresectable Ovarian Cancer (Epithelial)

Associated Therapies: Conditioning regimens for allogeneic stem cell transplantation therapy

Clinical Trials

Lenalidomide Combined With Vorinostat/Gemcitabine/Busulfan/Melphalan With Autologous Stem-Cell Transplantation in Diffuse Large B-Cell Lymphoma of the ABC Subtype

Phase 1

Terminated

Conditions: Lymphoma

Interventions: Drug: Lenalidomide
Drug: Vorinostat
Drug: Gemcitabine
Drug: Busulfan
Drug: Melphalan
Drug: Rituximab
Drug: Dexamethasone
Drug: Caphosol
Drug: Glutamine
Drug: Pyridoxine
Drug: Enoxaparin
Procedure: Stem Cell Transplant
Drug: Palifermin

First Posted Date: 2015-10-28

Last Posted Date: 2019-12-13

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 8

Registration Number: NCT02589145

Locations: 🇺🇸
University of Texas MD Anderson Cancer Center, Houston, Texas, United States

Reduced Intensity Conditioning Transplant Using Haploidentical Donors

Phase 2

Completed

Conditions: Myelofibrosis
Multiple Myeloma
Myelodysplastic Syndrome
Non-Hodgkin's Lymphoma
Acute Lymphocytic Leukemia
Chronic Lymphocytic Leukemia
CMML
Chronic Myelogenous Leukemia
Hodgkin's Lymphoma
Acute Myelogenous Leukemia

Interventions: Drug: Fludarabine
Drug: Melphalan
Drug: Cyclophosphamide
Procedure: peripheral blood stem cell transplant

First Posted Date: 2015-10-20

Last Posted Date: 2023-04-27

Lead Sponsor: Northside Hospital, Inc.

Target Recruit Count: 25

Registration Number: NCT02581007

Locations: 🇺🇸
Northside Hospital, Atlanta, Georgia, United States

VP-16, Ifosfamide, Dexamethasone, L-asparaginase Chemotherapy in Patients With Extranodal Natural Killer T Cell Lymphoma (VIDL+ASCT)

Phase 2

Conditions: Extranodal NK-T-Cell Lymphoma

Interventions: Drug: Etoposide
Drug: Ifosfamide
Drug: Dexamethasone
Drug: L-asparaginase
Drug: Busulfan
Drug: Melphalan

First Posted Date: 2015-09-09

Last Posted Date: 2020-10-22

Lead Sponsor: Samsung Medical Center

Target Recruit Count: 27

Registration Number: NCT02544425

Locations: 🇰🇷
Samsung Medical Center, Seoul, Korea, Republic of

Ruxolitinib Phosphate, Tacrolimus and Sirolimus in Preventing Acute Graft-versus-Host Disease During Reduced Intensity Donor Hematopoietic Cell Transplant in Patients With Myelofibrosis

Phase 1

Withdrawn

Conditions: Primary Myelofibrosis, Prefibrotic Stage
Secondary Acute Myeloid Leukemia
Acute Myeloid Leukemia in Remission
Primary Myelofibrosis
Secondary Myelofibrosis

Interventions: Procedure: Allogeneic Bone Marrow Transplantation
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Drug: Fludarabine Phosphate
Other: Laboratory Biomarker Analysis
Drug: Melphalan
Procedure: Peripheral Blood Stem Cell Transplantation
Other: Pharmacological Study
Drug: Ruxolitinib Phosphate
Drug: Sirolimus
Drug: Tacrolimus

First Posted Date: 2015-08-19

Last Posted Date: 2016-09-14

Lead Sponsor: City of Hope Medical Center

Registration Number: NCT02528877

Locations: 🇺🇸
City of Hope Medical Center, Duarte, California, United States

Isolated Limb Perfusion With Melphalan in Treating Patients With Stage IIIB-IV Melanoma or Sarcoma

Not Applicable

Withdrawn

Conditions: Eccrine Carcinoma of the Skin
Stage IV Melanoma
Recurrent Adult Soft Tissue Sarcoma
Squamous Cell Carcinoma of the Skin
Stage IIIB Melanoma
Stage IV Adult Soft Tissue Sarcoma
Recurrent Melanoma
Recurrent Skin Cancer
Stage IIIC Melanoma
Basal Cell Carcinoma of the Skin

Interventions: Drug: isolated limb perfusion
Drug: melphalan
Procedure: quality-of-life assessment

First Posted Date: 2015-07-23

Last Posted Date: 2015-07-23

Lead Sponsor: Albert Einstein College of Medicine

Registration Number: NCT02507076

Locations: 🇺🇸
Albert Einstein College of Medicine, Bronx, New York, United States

Panobinostat, Gemcitabine Hydrochloride, Busulfan, and Melphalan Before Stem Cell Transplant in Treating Patients With Refractory or Relapsed Multiple Myeloma

Phase 2

Completed

Conditions: Recurrent Plasma Cell Myeloma
Plasma Cell Leukemia
Plasmacytoma
Refractory Plasma Cell Myeloma

Interventions: Procedure: Autologous Hematopoietic Stem Cell Transplantation
Drug: Busulfan
Drug: Gemcitabine Hydrochloride
Other: Laboratory Biomarker Analysis
Drug: Melphalan
Drug: Panobinostat
Procedure: Peripheral Blood Stem Cell Transplantation
Other: Pharmacological Study

First Posted Date: 2015-07-23

Last Posted Date: 2024-06-05

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 83

Registration Number: NCT02506959

Locations: 🇺🇸
M D Anderson Cancer Center, Houston, Texas, United States

Combination Chemotherapy and Donor Stem Cell Transplant Followed by Ixazomib Citrate Maintenance Therapy in Treating Patients With Relapsed High-Risk Multiple Myeloma

Phase 1

Completed

Conditions: Recurrent Plasma Cell Myeloma
Plasma Cell Leukemia

Interventions: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Drug: Carmustine
Drug: Cytarabine
Drug: Etoposide
Drug: Ixazomib Citrate
Drug: Melphalan
Drug: Methotrexate
Procedure: Peripheral Blood Stem Cell Transplantation
Other: Quality-of-Life Assessment
Drug: Tacrolimus

First Posted Date: 2015-07-21

Last Posted Date: 2020-11-23

Lead Sponsor: OHSU Knight Cancer Institute

Target Recruit Count: 11

Registration Number: NCT02504359

Locations: 🇺🇸
Duke University Medical Center, Durham, North Carolina, United States
🇺🇸
OHSU Knight Cancer Institute, Portland, Oregon, United States