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

Busulfan, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk Ewing's Tumors

Phase 2

Completed

Conditions: Sarcoma

Interventions: Biological: graft versus host disease prophylaxis/therapy
Drug: busulfan
Drug: melphalan
Drug: thiotepa
Procedure: allogeneic bone marrow transplantation
Procedure: allogeneic hematopoietic stem cell transplantation
Procedure: peripheral blood stem cell transplantation

First Posted Date: 2006-07-27

Last Posted Date: 2015-11-25

Lead Sponsor: Memorial Sloan Kettering Cancer Center

Target Recruit Count: 10

Registration Number: NCT00357396

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

Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia

Phase 2

Active, not recruiting

Conditions: Recurrent Childhood Acute Myeloid Leukemia
Refractory Anemia With Excess Blasts in Transformation
Refractory Anemia
De Novo Myelodysplastic Syndrome
Secondary Myelodysplastic Syndrome
Childhood Myelodysplastic Syndrome
Leukemia
Myelodysplastic Syndromes
Secondary Acute Myeloid Leukemia
Refractory Anemia With Excess Blasts

Interventions: Biological: filgrastim
Drug: busulfan
Drug: cyclosporine
Drug: fludarabine phosphate
Drug: melphalan
Drug: mycophenolate mofetil
Procedure: umbilical cord blood transplantation

First Posted Date: 2006-07-27

Last Posted Date: 2024-08-22

Lead Sponsor: Masonic Cancer Center, University of Minnesota

Target Recruit Count: 33

Registration Number: NCT00357565

Locations: 🇺🇸
Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States

High-Dose Sequential Therapy and Single Autologous Transplantation for Multiple Myeloma

Phase 2

Completed

Conditions: Multiple Myeloma

Interventions: Drug: Cyclophosphamide
Drug: Etoposide
Drug: Melphalan
Drug: Carmustine
Drug: Filgrastim

First Posted Date: 2006-07-10

Last Posted Date: 2017-12-12

Lead Sponsor: Stanford University

Target Recruit Count: 102

Registration Number: NCT00349778

Locations: 🇺🇸
Stanford University School of Medicine, Stanford, California, United States

Intensive Versus Conventional Treatment in Patients With Primary Amyloidosis

Phase 3

Completed

Conditions: Primary Systemic Amyloidosis (AL)

First Posted Date: 2006-06-26

Last Posted Date: 2007-06-28

Lead Sponsor: University Hospital, Limoges

Target Recruit Count: 100

Registration Number: NCT00344526

Locations: 🇫🇷
Service d'hématologie clinique, Nantes, France
🇫🇷
Hématologie Clinique, Tours, France
🇫🇷
Service d'immuno-hématologie, Hôpital Saint-Louis, Paris, France

and more 5 locations

Doxorubicin Hydrochloride Liposome, Melphalan, and Bortezomib in Treating Patients With Relapsed or Refractory Stage I, Stage II, or Stage III Multiple Myeloma

Phase 1

Conditions: Multiple Myeloma and Plasma Cell Neoplasm

First Posted Date: 2006-06-08

Last Posted Date: 2014-01-10

Lead Sponsor: Herbert Irving Comprehensive Cancer Center

Target Recruit Count: 32

Registration Number: NCT00334932

Locations: 🇺🇸
UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, California, United States
🇺🇸
Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center, New York, New York, United States

Study of Melphalan and Topotecan (MT) Followed by Autologous Stem Cell Rescue in Patients With Multiple Myeloma.

Phase 1

Completed

Conditions: Multiple Myeloma

Interventions: Drug: melphalan
Drug: topotecan (TPT)
Procedure: Autologous Stem Cell Rescue

First Posted Date: 2006-05-12

Last Posted Date: 2018-03-14

Lead Sponsor: H. Lee Moffitt Cancer Center and Research Institute

Target Recruit Count: 177

Registration Number: NCT00325416

Locations: 🇺🇸
H. Lee Moffitt Cancer Center & Research Institute, Tampa, Florida, United States

Hepatic Arterial Infusion With Melphalan Compared With Standard Therapy in Treating Patients With Unresectable Liver Metastases Due to Melanoma

Phase 3

Completed

Conditions: Intraocular Melanoma
Melanoma (Skin)
Metastatic Cancer

Interventions: Drug: regional chemotherapy
Drug: melphalan
Drug: systemic chemotherapy
Procedure: hepatic artery embolization

First Posted Date: 2006-05-11

Last Posted Date: 2021-06-21

Lead Sponsor: Delcath Systems Inc.

Target Recruit Count: 93

Registration Number: NCT00324727

Locations: 🇺🇸
UPMC Cancer Centers, Pittsburgh, Pennsylvania, United States
🇺🇸
H. Lee Moffitt Cancer Center and Research Institute at University of South Florida, Tampa, Florida, United States
🇺🇸
Providence Cancer Center at Providence Portland Medical Center, Portland, Oregon, United States

and more 9 locations

Bortezomib, Ascorbic Acid, and Melphalan in Treating Patients With Newly Diagnosed Multiple Myeloma

Phase 2

Completed

Conditions: Multiple Myeloma and Plasma Cell Neoplasm

First Posted Date: 2006-04-25

Last Posted Date: 2013-11-06

Lead Sponsor: Oncotherapeutics

Target Recruit Count: 35

Registration Number: NCT00317811

Locations: 🇺🇸
SUNY Downstate Medical Center, Brooklyn, New York, United States
🇺🇸
Florida Cancer Specialists - Bonita Springs, Bonita Springs, Florida, United States
🇺🇸
Oncotherapeutics, West Hollywood, California, United States

and more 5 locations

Combination Chemotherapy Followed By Stem Cell Transplant in Treating Young Patients With Progressive or Relapsed Anaplastic Large Cell Lymphoma

Not Applicable

Conditions: Lymphoma

First Posted Date: 2006-04-24

Last Posted Date: 2015-09-25

Lead Sponsor: European Inter-group Cooperation on Childhood and Adolescent Non Hodgkin Lymphoma

Target Recruit Count: 96

Registration Number: NCT00317408

Locations: 🇨🇿
University Hospital Brno, Brno, Czech Republic
🇧🇪
U.Z. Gasthuisberg, Leuven, Belgium
🇩🇪
Krankenhaus Muenchen Schwabing, Munich, Germany

and more 91 locations

Melphalan and Busulfan Followed By Donor Peripheral Stem Cell Transplant, Tacrolimus, and Methotrexate in Treating Patients With Multiple Myeloma

Phase 2

Completed

Conditions: Multiple Myeloma and Plasma Cell Neoplasm

First Posted Date: 2006-04-12

Last Posted Date: 2010-09-21

Lead Sponsor: Fred Hutchinson Cancer Center

Target Recruit Count: 20

Registration Number: NCT00313625

Locations: 🇺🇸
Fred Hutchinson Cancer Research Center, Seattle, Washington, United States

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