Study of Captisol Enabled Melphalan and Pharmacokinetics for Patients With Multiple Myeloma or Light Chain Amyloidosis That Are Receiving an Autologous Transplant.

NCT02909036 | Active Not Recruiting Phase 1

• 1 other identifier: 16-875
• Study Type: interventional
• Target: 46
• Locations: 1 country
• Sites: 1

Brief Summary

Captisol Enabled Melphalan, is a new formulation of the standard of care melphalan chemotherapy that in packaged in an inactive substance that is believed to help the chemotherapy be more stable (meaning that it doesn't lose its effect or need to be administered quickly after being mixed). It may also have fewer side effects such as problems with important levels of body electrolytes such as potassium, phosphorous and magnesium; and cause less kidney and heart damage\] than standard formulation melphalan. The purpose of this study is to determine if the investigators can achieve a certain level of Captisol Enabled Melphalan that would be best to use in treating Multiple Myeloma and AL Amyloidosis.

Conditions

Multiple Myeloma; Amyloidosis

Keywords

Melphalan; Autologous Hematopoietic Progenitor Cell Transplant; High Dose Therapy; 16-875

Outcome Measures

Primary Outcomes (1)

• Overall Response Rate (ORR)

  defined as stringent Complete Response (sCR), Complete Response (CR), Very Good Partial Response (VGPR), or Partial Response (PR) to high-dose CE melphalan in patients enrolled in the study between days 90-100 post-AHCT. Responses will be evaluated by the IMWG Response Criteria and Minor Response (MR) criteria. Responses will be evaluated by the standard AL Amyloid Organ Response Criteria.

  1 day

Study Arms (1)

Melphalan

EXPERIMENTAL

Test Dose CE Melphalan 10mg/m2 with PK studies Day -12 to Day-3, CE Melphalan Dose of Target AUC 13 mg/L/h infused with PK studies Day -2, 3-10 x 10\^6 CD 34+ cells/kg reinfused Day 0, Pegfilgrastim 6mg injection Day +1

Device: Melphalan; Drug: Pegfilgrastim; Procedure: Autologous Hematopoietic Progenitor Cell Transplant

Interventions

Melphalan DEVICE

Melphalan

Pegfilgrastim DRUG

Melphalan

Autologous Hematopoietic Progenitor Cell Transplant PROCEDURE

Melphalan

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥18 and ≤ 75
• Patients must have either:
• Symptomatic multiple myeloma who have responded to prior induction or salvage chemotherapy (i.e. chemosensitive disease): Patients who are receiving high-dose melphalan and AHCT as part of their initial therapy require at least a partial response (PR) as defined by the International Myeloma Working Group uniform response criteria for MM.
• Patients who are receiving high-dose melphalan and AHCT as part of salvage therapy require at least a minor response to their last line of therapy to document chemosensitive disease. There is no limit on the number of prior regimens received by the patient.
• Light chain (AL) amyloidosis who may be newly diagnosed or previously treated
• Histologic and serologic findings, reviewed at MSKCC, confirming the diagnosis of multiple myeloma or AL amyloidosis. Standard diagnostic criteria for multiple myeloma will be used, as per the International Myeloma Foundation consensus guidelines
• Patients must have at least 3 x 10\^6 CD34+ cells/kg frozen.
• Adequate organ function is required, defined as follows:
• Serum bilirubin ≤ 2.0 mg/dl
• AST, ALT and alkaline phosphatase \< 3 times the upper limit of laboratory normal
• Creatinine clearance ≥ 40 ml/min (24 hour urine collection)
• LVEF ≥ 45% by MUGA or rest ECHO
• Diffusing capacity ≥ 45% (adjusted for hemoglobin) predicted by pulmonary function testing
• Performance status (ECOG) ≤ 2
• A willingness to avoid pregnancy or fathering children in male and female subjects respectively

You may not qualify if:

• Unstable angina or myocardial infarction within 4 months of initiating therapy on trial, NYHA Class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, sick sinus syndrome, or electrocardiographic evidence of acute ischemia or Grade 3 conduction system abnormalities unless subject has a pacemaker
• Light chain (AL) amyloidosis patients with Mayo Cardiac Stage IIIB (defined as NT-proBNP\>8500 ng/L and Cardiac troponin (cTnT) \>0.035 μg/L)
• Pregnant or lactating females
• Nonhematologic malignancy within the past 3 years with the exception of a) adequately treated basal cell carcinoma, squamous cell skin cancer, or thyroid cancer; b) carcinoma in situ of the cervix or breast; c) prostate cancer of Gleason Grade 6 or less with stable prostate-specific antigen levels; or d) cancer considered cured by surgical resection or unlikely to impact survival during the duration of the study, such as localized transitional cell carcinoma of the bladder or benign tumors of the adrenal or pancreas
• Contraindication to CE melphalan or any of the required supportive treatments, including hypersensitivity to G-CSF or pegfilgrastim
• Any other medical condition or laboratory evaluation that, in the treating physician's or principal investigator's opinion, makes the patient unsuitable to participate in this clinical trial
• Any known allergy or allergic reactions to Captisol

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead
• Spectrum Pharmaceuticals, Inc: collaborator

Study Sites (1)

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Related Links

• Memorial Sloan Kettering Cancer Center

MeSH Terms

Conditions

Multiple Myeloma; Amyloidosis

Interventions

pegfilgrastim

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Heather Landau, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2016
• First Posted: September 21, 2016
• Study Start: September 1, 2016
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: October 10, 2025

Record last verified: 2025-10

Locations

US (1)