Multiple MyelomaPhase 1NCT02909036
Study of Captisol Enabled Melphalan and Pharmacokinetics for Patients With Multiple Myeloma or Light Chain Amyloidosis That Are Receiving an Autologous Transplant.
A Phase 1 study testing Melphalan, Pegfilgrastim and Autologous Hematopoietic Progenitor Cell Transplant in people with Multiple Myeloma and Amyloidosis, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Multiple Myeloma, Amyloidosis
- What is being tested?
- Melphalan, Pegfilgrastim and Autologous Hematopoietic Progenitor Cell Transplant
- Type of treatment
- Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 46 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2016to Sep 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- OR
- Light chain (AL) amyloidosis who may be newly diagnosed or previously treated
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- 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
- A woman of childbearing potential must have a negative serum pregnancy test (β-human chorionic gonadotropin [β-hCG]) at screening and must be willing to avoid pregnancy during the study treatment period and for a specified duration (1 year post HCT) after the end of treatment.
- Women of childbearing potential who have a negative serum pregnancy test at screening must practice a highly effective method of birth control (with at least 99% certainty) from screening through safety follow-up. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the subject and their understanding confirmed.
- Men who are enrolled must agree to take appropriate precautions to avoid fathering children (with at least 99% certainty) from screening through safety follow-up. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the subject and their understanding confirmed
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
About this study
Official title: Pharmacokinetic (PK)-Directed Dosing of Captisol Enabled Melphalan for Patients With Multiple Myeloma or Light Chain (AL) Amyloidosis Undergoing High Dose Therapy and Autologous Hematopoietic Progenitor Cell Transplant
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
- Being tested
- Melphalan, Pegfilgrastim, Autologous Hematopoietic Progenitor Cell Transplant
- Keywords
- Melphalan, Autologous Hematopoietic Progenitor Cell Transplant, High Dose Therapy, 16-875
- Medical subject headings
- Multiple Myeloma, Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MelphalanDevice
PegfilgrastimDrug
Autologous Hematopoietic Progenitor Cell TransplantProcedure or surgery
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Device: Melphalan; Drug: Pegfilgrastim; Procedure: Autologous Hematopoietic Progenitor Cell Transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2016 (estimated)
- Study startSep 2016
- Primary completionSep 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple MyelomaStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Spectrum Pharmaceuticals, Inc
- Responsible party
- Sponsor
- Investigators
- Heather Landau, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02909036
- Sponsor's protocol ID
- 16-875
- Status last verified
- Oct 2026
- First submitted
- 19 Sep 2016
- Last updated
- 14 Oct 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacokinetic (PK)-Directed Dosing of Captisol Enabled Melphalan for Patients With Multiple Myeloma or Light Chain (AL) Amyloidosis Undergoing High Dose Therapy and Autologous Hematopoietic Progenitor Cell Transplant
- ClinicalTrials.gov ID
- NCT02909036
- Acronym / sponsor ID
- 16-875
- Conditions
- Multiple Myeloma, Amyloidosis
- Keywords
- Melphalan, Autologous Hematopoietic Progenitor Cell Transplant, High Dose Therapy, 16-875
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 46 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Collaborators
- Spectrum Pharmaceuticals, Inc
- Study start
- Sep 2016 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 21 Sep 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Melphalan | Experimental | Device: Melphalan, Drug: Pegfilgrastim, Procedure: Autologous Hematopoietic Progenitor Cell TransplantTest 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 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Age ≥18 and ≤ 75
- Patients must have either:
- 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.
- OR
- Light chain (AL) amyloidosis who may be newly diagnosed or previously treated
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Melphalan | Device | Melphalan |
| Pegfilgrastim | Drug | Melphalan |
| Autologous Hematopoietic Progenitor Cell Transplant | Procedure or surgery | Melphalan |
Primary outcome
Overall Response Rate (ORR)
Time frame: 1 day
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2026.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Heather Landau, MD principal investigator
Memorial Sloan Kettering Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Melphalan, Pegfilgrastim and Autologous Hematopoietic Progenitor Cell Transplant in people with Multiple Myeloma and Amyloidosis, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate (ORR) (time frame: 1 day). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026; AzTrials last refreshed its copy on 6 Nov 2026.