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

Pooled Unrelated Donor Umbilical Cord Blood Transplant For Hematologic Malignancy Needing Allogeneic Stem Cell Transplant Without Related HLA-Match

Phase 2

Terminated

Conditions: Acute Myelogenous Leukemia
Chronic Myelogenous Leukemia
Aplastic Anemia
Acute Lymphocytic Leukemia
Myelodysplastic Syndrome
Chronic Lymphocytic Leukemia
Multiple Myeloma
Hodgkins Disease
Non-Hodgkins Lymphoma

Interventions: Drug: Busulfan
Drug: Clofarabine
Drug: Fludarabine
Drug: Melphalan
Drug: Carmustine
Drug: Etoposide
Drug: Cytarabine

First Posted Date: 2011-12-28

Last Posted Date: 2013-11-25

Lead Sponsor: Texas Oncology Cancer Center

Target Recruit Count: 1

Registration Number: NCT01500161

Locations: 🇺🇸
Texas Oncology, Amarillo, Texas, United States

Radiolabeled Monoclonal Antibody Therapy and Combination Chemotherapy Before Stem Cell Transplant in Treating Patients With Primary Refractory or Relapsed Hodgkin Lymphoma

Phase 1

Active, not recruiting

Conditions: Recurrent Adult Hodgkin Lymphoma

Interventions: Biological: basiliximab
Drug: carmustine
Drug: etoposide
Drug: cytarabine
Drug: melphalan
Other: pharmacological study
Other: laboratory biomarker analysis
Procedure: autologous hematopoietic stem cell transplantation
Biological: yttrium Y 90-labeled basiliximab

First Posted Date: 2011-11-22

Last Posted Date: 2024-03-19

Lead Sponsor: City of Hope Medical Center

Target Recruit Count: 25

Registration Number: NCT01476839

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

Cord Blood Fucosylation to Enhance Homing and Engraftment in Patients With Hematologic Malignancies

Phase 1

Completed

Conditions: Lymphoma
Leukemia
Transplantation, Bone Marrow
Transplantation Infection
Blood And Marrow Transplantation

Interventions: Drug: Fludarabine
Drug: Melphalan
Drug: Mycophenolate mofetil
Drug: Tacrolimus
Procedure: Cord Blood Infusion
Drug: Rituximab
Drug: ATG
Drug: Busulfan
Drug: Clofarabine
Radiation: Total Body Irradiation (TBI)

First Posted Date: 2011-11-11

Last Posted Date: 2017-04-28

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 33

Registration Number: NCT01471067

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

Auto Transplant High Dose Melphalan vs High Dose Melphalan+Bortezomib in Pts With Multiple Myeloma Age 65 Years or Older

Phase 2

Terminated

Conditions: Multiple Myeloma

Interventions: Drug: Melphalan
Drug: Bortezomib

First Posted Date: 2011-10-17

Last Posted Date: 2023-11-28

Lead Sponsor: Hackensack Meridian Health

Target Recruit Count: 63

Registration Number: NCT01453088

Locations: 🇺🇸
Hackensack University Medical Center, Hackensack, New Jersey, United States
🇺🇸
Lombardi Comprehensive Cancer Center at Georgetown University Medical Center, Washington, District of Columbia, United States

Vorinostat With Gemcitabine, Busulfan, and Melphalan With Stem Cell Transplant (SCT) in Relapsed or Refractory Lymphoid Malignancies

Phase 1

Completed

Conditions: Lymphoma

Interventions: Drug: Vorinostat
Drug: Gemcitabine
Drug: Busulfan
Drug: Melphalan
Procedure: Stem Cell Infusion
Drug: Rituximab
Drug: G-CSF
Drug: Palifermin
Drug: Dexamethasone acetate

First Posted Date: 2011-08-22

Last Posted Date: 2015-11-18

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 78

Registration Number: NCT01421173

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

A Randomized Trial to Compare Busulfan + Melphalan 140 mg/m2 With Melphalan 200 mg/m2 as Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation for Multiple Myeloma

Phase 3

Completed

Conditions: Myeloma

Interventions: Drug: Busulfan
Drug: Melphalan
Other: Questionnaire
Drug: High Dose Melphalan
Drug: G-CSF
Procedure: Stem cell transplant

First Posted Date: 2011-08-10

Last Posted Date: 2020-04-21

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 205

Registration Number: NCT01413178

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

Reduced Intensity Double Umbilical Cord Blood Transplantation

Phase 2

Completed

Conditions: Hodgkin's Lymphoma
Chronic Lymphocytic Leukemia
Non-Hodgkin's Lymphoma
Multiple Myeloma
Acute Myelogenous Leukemia

Interventions: Drug: Fludarabine
Drug: Melphalan
Radiation: Total Body Radiation
Biological: Cord Blood

First Posted Date: 2011-08-03

Last Posted Date: 2019-01-23

Lead Sponsor: Massachusetts General Hospital

Target Recruit Count: 33

Registration Number: NCT01408563

Locations: 🇺🇸
Beth Israel Deaconess Medical Center, Boston, Massachusetts, United States
🇺🇸
Massachusetts General Hospital, Boston, Massachusetts, United States
🇺🇸
Dana-Farber Cancer Institute, Boston, Massachusetts, United States