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

HLA-Compatible Related or Unrelated Donors With CD34+ Enriched, T-cell Depleted Peripheral Blood Stem Cells Isolated by the CliniMACS System in the Treatment of Patients With Hematologic Malignancies

Phase 2

Completed

Conditions: Multiple Myeloma
Acute Myelogenous Leukemia
Acute Lymphoblastic Leukemia
Myelodysplastic Syndrome

Interventions: Drug: Thiotepa
Radiation: total body irradiation
Drug: Busulfan
Drug: Melphalan
Drug: Cyclophosphamide
Drug: Fludarabine
Drug: Clofarabine
Procedure: (CliniMACS) T-cell depleted PBSC Transplant

First Posted Date: 2010-05-07

Last Posted Date: 2022-08-05

Lead Sponsor: Memorial Sloan Kettering Cancer Center

Target Recruit Count: 422

Registration Number: NCT01119066

Locations: 🇺🇸
Memorial Sloan Kettering Cancer Center, New York, New York, United States

Dasatinib in Combination With Melphalan and Prednisone to Treat Relapsed and Refractory Multiple Myeloma Patients

Phase 2

Terminated

Conditions: Myeloma

Interventions: Drug: dasatinib
Drug: melphalan
Drug: prednisone

First Posted Date: 2010-05-04

Last Posted Date: 2011-06-14

Lead Sponsor: Fondazione EMN Italy Onlus

Target Recruit Count: 8

Registration Number: NCT01116128

Locations: 🇮🇹
Mario Boccadoro, Torino, Italy

Cyclophosphamide, Lenalidomide and Dexamethasone (CRD) Versus Melphalan (200 mg/m2) Followed By Autologous Stem Cell Transplant (ASCT) In Newly Diagnosed Multiple Myeloma Subjects

Phase 3

Completed

Conditions: Multiple Myeloma

Interventions: Drug: Cyclophosphamide
Drug: Melphalan
Drug: Lenalidomide
Drug: Dexamethasone

First Posted Date: 2010-03-24

Last Posted Date: 2024-11-06

Lead Sponsor: Fondazione EMN Italy Onlus

Target Recruit Count: 389

Registration Number: NCT01091831

Locations: 🇮🇹
Division of Hematology, A.O.U. Città della Salute e della Scienza di Torino, Torino, Italy

Standard Chemotherapy With or Without Nelarabine or Rituximab in Treating Patients With Newly Diagnosed Acute Lymphoblastic Leukemia

Phase 3

Active, not recruiting

Conditions: Leukemia
Oral Complications
Mucositis

Interventions: Biological: rituximab
Biological: palifermin
Drug: cyclophosphamide
Procedure: allogeneic hematopoietic stem cell transplantation
Drug: cytarabine
Drug: daunorubicin hydrochloride
Radiation: total-body irradiation
Drug: etoposide
Drug: fludarabine phosphate
Drug: imatinib mesylate
Drug: melphalan
Drug: mercaptopurine
Drug: methotrexate
Drug: nelarabine
Drug: pegaspargase
Drug: vincristine sulfate

First Posted Date: 2010-03-12

Last Posted Date: 2024-05-10

Lead Sponsor: University College, London

Target Recruit Count: 1033

Registration Number: NCT01085617

Bortezomib and Dexamethasone Followed by High-Dose Melphalan and Stem Cell Transplantation for Primary (AL) Amyloidosis

Phase 2

Completed

Conditions: Amyloidosis

Interventions: Drug: Bortezomib
Drug: Dexamethasone
Drug: Melphalan

First Posted Date: 2010-03-09

Last Posted Date: 2020-09-25

Lead Sponsor: Boston Medical Center

Target Recruit Count: 35

Registration Number: NCT01083316

Locations: 🇺🇸
Boston Medical Center, Boston, Massachusetts, United States

Lenalidomide and High-Dose Melphalan

Phase 1

Completed

Conditions: Myeloma
Stem Cell Transplantation

Interventions: Drug: Lenalidomide
Drug: Melphalan
Procedure: Stem Cell Infusion

First Posted Date: 2010-03-03

Last Posted Date: 2016-06-06

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 61

Registration Number: NCT01079936

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

Melphalan and Dexamethasone With or Without Bortezomib in Treating Patients With Previously Untreated Systemic Light-Chain Amyloidosis

Phase 3

Completed

Conditions: Light Chain Deposition Disease
Primary Systemic Amyloidosis

Interventions: Drug: melphalan
Drug: dexamethasone
Drug: bortezomib

First Posted Date: 2010-03-02

Last Posted Date: 2022-02-01

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 11

Registration Number: NCT01078454

Locations: 🇺🇸
Illinois CancerCare-Canton, Canton, Illinois, United States
🇺🇸
Illinois CancerCare-Carthage, Carthage, Illinois, United States
🇺🇸
Emory University/Winship Cancer Institute, Atlanta, Georgia, United States

and more 206 locations

S0833, Bortezomib, Thalidomide, Lenalidomide, Combination Chemotherapy, and Autologous Stem Cell Transplant in Treating Patients With Newly Diagnosed Multiple Myeloma

Phase 2

Withdrawn

Conditions: Multiple Myeloma
Plasma Cell Myeloma

Interventions: Drug: bortezomib
Drug: cisplatin
Drug: cyclophosphamide
Drug: dexamethasone
Drug: doxorubicin hydrochloride
Drug: etoposide
Drug: lenalidomide
Drug: melphalan
Drug: thalidomide
Genetic: gene expression analysis
Genetic: microarray analysis
Other: laboratory biomarker analysis
Procedure: autologous-autologous tandem hematopoietic stem cell transplantation

First Posted Date: 2010-01-25

Last Posted Date: 2015-03-06

Lead Sponsor: SWOG Cancer Research Network

Registration Number: NCT01055301

Locations: 🇺🇸
Harborview Medical Center, Seattle, Washington, United States
🇺🇸
Fred Hutchinson Cancer Research Center, Seattle, Washington, United States
🇺🇸
Group Health Central Hospital, Seattle, Washington, United States

and more 18 locations