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

Bortezomib-Melphalan Conditioning Regimen vs Melphalan for Frontline Transplant Eligible Patients With Multiple Myeloma

Phase 3

Completed

Conditions: Multiple Myeloma

Interventions: Drug: Bortezomib-Melphalan
Drug: Melphalan

First Posted Date: 2014-07-22

Last Posted Date: 2022-05-23

Lead Sponsor: University Hospital, Toulouse

Target Recruit Count: 300

Registration Number: NCT02197221

Locations: 🇫🇷
Chu Nancy, Nancy, France
🇫🇷
Hôtel Dieu, Nantes, France
🇧🇪
Chu Liege, Liege, Belgium

and more 43 locations

A Study of Combination of Daratumumab and Velcade (Bortezomib) Melphalan-Prednisone (DVMP) Compared to Velcade Melphalan-Prednisone (VMP) in Participants With Previously Untreated Multiple Myeloma

Phase 3

Completed

Conditions: Multiple Myeloma

Interventions: Drug: Velcade
Drug: Melphalan
Drug: Prednisone
Drug: Daratumumab IV
Drug: Dexamethasone
Drug: Daratumumab SC

First Posted Date: 2014-07-21

Last Posted Date: 2024-12-09

Lead Sponsor: Janssen Research & Development, LLC

Target Recruit Count: 706

Registration Number: NCT02195479

A Pilot Study of Immunotherapy Including Haploidentical NK Cell Infusion Following CD133+ Positively-Selected Autologous Hematopoietic Stem Cells in Children With High Risk Solid Tumors or Lymphomas

Phase 1

Completed

Conditions: Neuroblastoma
Lymphoma
High-risk Tumor

Interventions: Biological: CD133+ selected autologous stem cell infusion
Biological: IL-2
Biological: hu14.18K322A
Drug: Melphalan
Drug: Busulfan
Drug: Bendamustine
Biological: GM-CSF
Drug: Etoposide
Drug: Carboplatin
Device: Haploidentical natural killer cell infusion
Drug: Cytarabine
Biological: G-CSF
Device: CliniMACS
Drug: Etoposide phosphate

First Posted Date: 2014-05-06

Last Posted Date: 2017-12-22

Lead Sponsor: St. Jude Children's Research Hospital

Target Recruit Count: 8

Registration Number: NCT02130869

Locations: 🇺🇸
St. Jude Children's Research Hospital, Memphis, Tennessee, United States

Alternating Systemic Chemotherapy and Intra-Arterial Melphalan (IAM) Chemotherapy in Children With Intra-Ocular Retinoblastoma

Phase 1

Terminated

Conditions: Retinoblastoma
Advanced Intra-Ocular Retinoblastoma

Interventions: Drug: Melphalan
Drug: Carboplatin
Drug: Etoposide
Drug: Vincristine

First Posted Date: 2014-04-17

Last Posted Date: 2020-04-16

Lead Sponsor: University of California, San Francisco

Target Recruit Count: 6

Registration Number: NCT02116959

Locations: 🇺🇸
University of California, San Francisco, San Francisco, California, United States

Carfilzomib/SAHA Combined With High-Dose Gemcitabine/Busulfan/Melphalan With Autologous Stem Cell Transplant in Myeloma

Phase 2

Withdrawn

Conditions: Myeloma

Interventions: Drug: Carfilzomib
Drug: SAHA
Drug: Gemcitabine
Drug: Busulfan
Drug: Melphalan
Procedure: Stem Cell Transplant (SCT)
Drug: Palifermin
Drug: Dexamethasone
Drug: Caphosol
Drug: Glutamine
Drug: Pyridoxine

First Posted Date: 2014-04-15

Last Posted Date: 2014-07-11

Lead Sponsor: M.D. Anderson Cancer Center

Registration Number: NCT02114502

Microdose Study of Melphalan, Bortezomib and Dexamethasone

Early Phase 1

Conditions: Multiple Myeloma

Interventions: Drug: Melphalan
Drug: Bortezomib
Drug: Dexamethasone

First Posted Date: 2014-04-10

Last Posted Date: 2015-12-08

Lead Sponsor: Henrik Gregersen

Target Recruit Count: 6

Registration Number: NCT02109861

Locations: 🇩🇰
Department of Haematology, Aalborg University Hospital, Aalborg, Denmark

Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma

Phase 1

Completed

Conditions: Unilateral Retinoblastoma

Interventions: Drug: Melphalan

First Posted Date: 2014-03-26

Last Posted Date: 2023-10-13

Lead Sponsor: Children's Oncology Group

Target Recruit Count: 14

Registration Number: NCT02097134

Locations: 🇺🇸
UCSF Medical Center-Mission Bay, San Francisco, California, United States
🇺🇸
M D Anderson Cancer Center, Houston, Texas, United States
🇺🇸
UCSF Medical Center-Parnassus, San Francisco, California, United States

and more 9 locations

A Study of Oprozomib, Melphalan, and Prednisone in Transplant Ineligible Patients With Newly Diagnosed Multiple Myeloma

Phase 1

Completed

Conditions: Multiple Myeloma

Interventions: Drug: Oprozomib
Drug: Melphalan
Drug: Prednisone

First Posted Date: 2014-02-27

Last Posted Date: 2017-05-02

Lead Sponsor: Amgen

Target Recruit Count: 9

Registration Number: NCT02072863

Locations: 🇮🇹
Hospital City of Health and Science of Turin, Hematology 1 Division, Turin, Italy
🇮🇹
Ospedale Oncologico Regionale, Rionero in Vulture, Potenza, Italy
🇳🇱
Erasmus MC, Department of Hematology, Rotterdam, Netherlands

and more 5 locations