A Phase IIb, Multicenter, Open-Label, Safety and Efficacy Study of High Dose Melphalan HCL for Injection (Propylene Glycol-Free)for Myeloablative Conditioning in Multiple Myeloma Patients Undergoing Autologous Transplantation
The sponsor of the current study, Ligand Pharmaceuticals Inc. (Ligand), previously CyDex
Pharmaceuticals, Inc. (CyDex), is developing Melphalan HCL for Injection (Propylene
Glycol-Free) as an orphan drug product for use as a high dose conditioning treatment prior
to hematopoietic progenitor (stem) cell transplantation. This new injectable form of
melphalan HCL incorporates Captisol®, β cyclodextrin sulfobutyl ether sodium salts (also
known as [SBE]7m-β-CD), into the product. Captisol is present to facilitate the use of an
all aqueous diluent (normal saline) for reconstitution and administration of the
freeze-dried product in place of the propylene glycol-ethanol diluent necessary for the
currently used melphalan intravenous product. Captisol provides for solubilization and
improved stability of the all aqueous reconstituted and diluted infusion solution.
This is the second of two studies supporting product registration. This study will be a
multicenter study of high-dose Melphalan HCL for Injection (Propylene Glycol-Free) conducted
in 60 patients who have symptomatic MM and qualify for autologous stem cell transplantation
(ASCT).
During the Study Period, patients will receive 100mg/m2 of either Melphalan HCL for
Injection (Propylene Glycol-Free) on Day -3 and on Day -2 for a total dose of 200mg/m2.
Blood samples (5 timepoints post infusion) for population pharmacokinetic (PK) evaluation
will be withdrawn through an indwelling i.v. cannula on the first day of administration of
melphalan (Day -3) for all patients and then additional blood samples (2 timepoints post
infusion) drawn in a subset of patients on the second day of melphalan administration (Day
-2).
Following one day of rest after the high dose myeloablative conditioning (Day -1), patients
will receive an autologous graft (Day 0).
Interventional
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Up to Day +100
Yes
Tim Freeman
Study Director
Clinipace Worldwide
United States: Food and Drug Administration
CDX-353-002
NCT01660633
December 2012
March 2014
Name | Location |
---|---|
Washington University School of Medicine | Saint Louis, Missouri 63110 |
University of Kansas Medical Center | Kansas City, Kansas 66160-7353 |
University of Massachusetts | Worcester, Massachusetts 01655 |
Medical College of Wisconsin/Froedtert Hospital | Milwaukee, Wisconsin 53226 |