A Phase 2 interventional study of High-Dose Melphalan HCL for Injection (Propylene Glycol-Free) and Autologous Transplantation in Multiple Myeloma, sponsored by Acrotech Biopharma Inc.. Completed at 6 sites in United States. Open to participants aged Up to 70 Years. Per ClinicalTrials.gov, last updated 2020-04-21.
Sponsored by Acrotech Biopharma Inc. · Phase 2, Interventional, and Treatment
The purpose of this trial is to confirm the safety and efficacy of high-dose Melphalan HCL for Injection (Propylene Glycol-Free) as a myeloablative conditioning regimen in multiple myeloma patients (MM) undergoing autologous transplantation.
The sponsor of the current study is now Acrotech Biopharma, divested from Spectrum Pharmaceuticals Inc. (Spectrum), which licensed the Melphalan HC1 for Injection (Propylene Glycol-Free) product from Ligand Pharmaceuticals , formerly CyDex Pharmaceuticals, Inc. (CyDex). 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 Blood samples (5 timepoints post infusion) for population pharmacokinetic (PK) evaluation will be withdrawn through an indwelling i.v. cannula on the first day of administration of melphalan (Day -3) for all patients and then additional blood samples (2 timepoints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).
3,632 studies on the registry are indexed under Multiple Myeloma; 745 are open to participants now.
This study's enrollment of 61 is above the median of 41 across 2,907 interventional studies indexed under Multiple Myeloma.
Browse Multiple Myeloma studies →Acrotech Biopharma Inc. is the lead sponsor of 27 studies on the registry; 2 are open to participants now.
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Patients with adequate organ function as measured by:
Exclusion Criteria:
Subjects will receive only High-Dose Melphalan HCL for Injection (Propylene Glycol-free) at 200mg/m2 (100mg/m2/day for two days).
Drug: High-Dose Melphalan HCL for Injection (Propylene Glycol-Free) · Other: Autologous Transplantation
200 mg melphalan/m2 will be divided into two separate, consecutive doses of 100 mg/m2 administered on day -3 and day -2 prior to ASCT. The High-Dose Melphaln HCL for Injection (Propylene Glycol-Free) will be reconstituted to 5 mg/mL (also containing 270 mg/mL of Captisol®). The Melphalan HCL for Injection (Propylene Glycol Free) will be further diluted with normal saline to a concentration of no greater than 0.45 mg/mL and infused over 30 minutes ( + or - 3 minutes)via a central venous catheter.
Also known as: Evomela, Alkeran
Patients who are myeloablative conditioning in multiple myeloma undergoing autologous transplantation( patients own blood-forming stem cells are collected to replace diseased bone marrow or bone marrow damaged by cancer treatment)
Also known as: Hematopoietic stem cell transplantation
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
AE coding will be performed using the MedDRA Version 11.0 or greater. The severity of the toxicities will be graded according to the NCI CTCAE Version 4.0 whenever possible. AEs occurring when treatment starts on Day -3 through Day + 100 will be recorded in the AE section of the eCRF and causality will be assigned by the Principal Investigator.
Time frame: Up to Day +100
Mucositis Severity according to World Health Organization Scoring System
The baseline measurement will be the last assessment prior to receiving the first dose of study treatment. The on-treatment period will be defined as the day after the first dose of study treatment to 30 days after the last dose of study treatment. The incidence of severe mucositis (WHO Grade 3 or 4) will be summarized by frequencies and percentages. In addition, the incidence of oral mucositis will be summarized by WHO grade. Time from start of the first dose of study medication to peak oral mucositis score will also be calculated.
Time frame: Until Day +30
Mouth Pain Scores according to a Visual Analog Scale
Baseline VAS for mouth pain and dysphagia will be the VAS score collected prior to receiving the first dose of study medication. Analyses of changes and/or percent changes from baseline in the VAS scores will be analyzed for each time point collected during the 30 day on-treatment period. The minimum and maximum VAS scores during the 30 day on-treatment period will also be calculated for mouth pain and dysphagia, and the time to the minimum and maximum VAS scores will be summarized descriptively.
Time frame: Until Day +30
Treatment Related Mortality
TRM, which is defined as death not due to disease progression before Day +90/+100, will be calculated. This outcome measure will be summarized by the cumulative incidence estimated with 95% confidence intervals.
Time frame: Up to Day +100
MM response according to International Myeloma Working Group (IMWG) criteria.
Definition of active MM, clonal bone marrow plasma cells \>10% or biopsy-proven bony or extramedullary plasmacytoma and any one or more of the following CRAB features and myeloma-defining events: Evidence of end organ damage that can be attributed to the underlying plasma cell proliferative disorder and one or more biomarkers of malignancy (MDEs) defined in the criteria.
Time frame: At the Day +100 visit
Myeloablation
Absolute neutrophil count (ANC) \<0.5 × 109/L, absolute lymphocyte count (ALC) \<0.1 × 109/L, platelet count \<20,000/mm3, or bleeding requiring transfusion.
Time frame: Up to Day +30
Neutrophil engraftment
ANC \>0.5 × 109/L × first 3 consecutive daily assessments
Time frame: Up to Day +100
Platelet engraftment
Untransfused platelet measurement \>20,000/mm3 × first 3 consecutive daily assessments
Time frame: Up to Day +100
Non-engraftment
Failure to reach an ANC \>0.5 × 109/L × 3 consecutive daily assessments by Day +100.
Time frame: Up to Day +100
This study is completed, as verified in Apr 2020. You cannot join it, but the record below documents what was studied.
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Acrotech Biopharma Inc.