CompletedNCT01383759Updated Apr 7, 2020Results posted

Bortezomib/Dexamethasone (BD), Followed By Autologous Stem Cell Transplantation and Maintenance Bortezomib/Dexamethasone For the Initial Treatment of Monoclonal Immunoglobulin Deposition Disease (MIDD) Associated With Multiple Myeloma and AL Amyloidosis

An interventional study of Bortezomib/Dexamethasone (BD), Followed By Autologous STC & Maintenance Bortezomib/Dexamethasone in Light Chain Deposition Disease (LCDD or MIDD), Light Chain and Heavy Chain Deposition Disease (LHCDD or MIDD) and Monoclonal Immunoglobulin Deposition Disease (MIDD), sponsored by Memorial Sloan Kettering Cancer Center. Completed at 5 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-04-07.

Sponsored by Memorial Sloan Kettering Cancer Center · Not applicable, Interventional, and Treatment

Phase: Not applicable
Study type: Interventional
Enrollment: 20
Allocation: Not applicable
Ages: 18 Years and older
Sex: All

Study summary

The goal of this clinical trial is to determine the toxicity and also the efficacy of a treatment that includes the following treatment: Two medications, bortezomib and dexamethasone (or BD), followed by autologous stem cell transplantation, and a prolonged course of treatment with bortezomib and dexamethasone after transplantation. This type of treatment has been very effective in multiple myeloma. However, there is little experience with this treatment in patients who have Monoclonal Immunoglobulin Deposition Disease (MIDD) or amyloidosis. The investigators and others have treated patients who have MIDD and amyloidosis with bortezomib and autologous stem cell transplantation and have had success with this treatment. But the combination of autologous transplant with BD given before and after the transplant is a new way of treating these diseases, which the investigators believe will be very effective.

Conditions studied

Light Chain Deposition Disease (LCDD or MIDD)
Light Chain and Heavy Chain Deposition Disease (LHCDD or MIDD)
Monoclonal Immunoglobulin Deposition Disease (MIDD)
Amyloidosis

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Keywords

BORTEZOMIB (VELCADE)
CYCLOPHOSPHAMIDE (CYTOXAN)
DEXAMETHASONE
MELPHALAN
11-061

In context

Multiple Myeloma

3,632 studies on the registry are indexed under Multiple Myeloma; 745 are open to participants now.

This study's enrollment of 20 is below the median of 41 across 2,907 interventional studies indexed under Multiple Myeloma.

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Lead sponsor

Memorial Sloan Kettering Cancer Center is the lead sponsor of 1,930 studies on the registry; 328 are open to participants now.

Of its 129 completed or terminated interventional studies of FDA-regulated products, 66 (51%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Age > or = to 18
New diagnosis of MIDD or AL amyloidosis based on pathologic findings confirmed at Memorial Sloan Kettering Cancer Center.
Patients must show the ability to understand the investigational nature of the treatment and to give voluntary informed consent before performance of any study-related procedure not part of normal medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to future medical care.
Female subject is either postmenopausal for at least 1 year before the screening visit, is surgically sterilized or if they are of childbearing potential, agree to practice 2 effective methods of contraception from the time of signing the informed consent form through 30 days after the last dose of bortezomib, or agree to completely abstain from heterosexual intercourse.
Male subjects, even if surgically sterilized (i.e., status post-vasectomy) must agree to 1 of the following: practice effective barrier contraception during the entire study treatment period and through a minimum of 30 days after the last dose of study drug, or completely abstain from heterosexual intercourse.
Adequate organ function defined as follows: Absolute granulocytes > 1,000/mm3 and platelets > 70,000/mm3, unless low granulocyte and platelets counts are due to multiple myeloma; total bilirubin \< 1.5 ULN; AST, ALT, and alkaline phosphatase \< 3 times upper limit of laboratory normal; LVEF > 50% by MUGA or ECHO (the method used at baseline must be used for later monitoring); DLCO > 50 % confirmed at MSKCC; elevated creatinine is not a contraindication to enrollment
Performance status (ECOG) \< or = to 2

Exclusion criteria

Exclusion Criteria:

Patient has received other investigational drugs with 14 days before enrollment
Prior initial treatment chemotherapy for MIDD, AL amyloidosis or multiple myeloma with the exception of one cycle of high dose dexamethasone
Prior bortezomib treatment
Myocardial infarction within 6 months prior to enrollment or New York Heart Association Class III or IV heart failure (see Appendix 20.2), uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities. Prior to study entry, any ECG abnormality at screening has to be documented by the investigator as not medically relevant.
Pregnant or lactating women are ineligible. A pregnancy test will be performed on each fertile premenopausal female 2 weeks prior to entry into the study. Treatment may not begin until the results of the pregnancy test are ascertained. All patients (men and women) must agree to use medically approved contraceptive measures for at least 4 weeks before starting therapy, during therapy, and for at least 3 months after therapy has stopped.
Pre existing neuropathy, sensory or neuropathic pain findings, grade 2 or higher on the NCI CTC neurotoxicity scale.
Concurrent active malignancy other than non melanoma skin cancers or carcinoma in situ of the cervix. Patients with previous malignancies, but which have not required anti tumor treatment within the preceding 24 months will be allowed to enter the trial. Patients with a history of a T1a or b prostate cancer (detected incidentally at TURP and comprising less than 5% of resected tissue) may participate if the PSA has remained within normal limits since TURP.
Patients with known HIV positivity or AIDS related illness. This is based upon the possibility of increasing HIV viral load with therapy
Any other medical condition or reason that, in the principal investigator's opinion, makes the patient unsuitable to participate in a clinical trial
Patients with a history of hypersensitivity reactions attributed to bortezomib, boron, or mannitol.
Radiation therapy within 3 weeks before randomization. Enrollment of subjects who require concurrent radiotherapy (which must be localized in its field size) should be deferred until the radiotherapy is completed and 3 weeks have elapsed since the last date of therapy.

Study design

Phase: Not applicable
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 20 participants (actual)

Study arms

Experimental
Bortezomib/Dexamethasone (BD) , STC & Maintenance BD
This is a pilot study to gain information and estimate the toxicity/tolerability of 1-3 cycles of BD, followed by HDM/ASCT, and maintenance therapy with BD in patients with MIDD associated with multiple myeloma and AL amyloidosis.
Drug: Bortezomib/Dexamethasone (BD), Followed By Autologous STC & Maintenance Bortezomib/Dexamethasone

Interventions

DrugBortezomib/Dexamethasone (BD), Followed By Autologous STC & Maintenance Bortezomib/Dexamethasone
The treatment has three phases: 1) Initial treatment phase: This phase consists of 1-3 21-day-cycles of a combination regimen that includes bortezomib 1.3 mg/m2, IV or Subcutaneous Injection (SQ), on days 1, 4, 8, and 11; and dexamethasone 40 mg PO or IV, on days 1, 4, 8, and 11. Stem cell mobilization and HDM/ASCT. Post-ASCT consolidation/maintenance treatment phase: This phase consists of six cycles of bortezomib 1.3 mg/m2, IV or (SQ) with dexamethasone 20 mg PO or IV administered on days 1, 8, 15, and 22 every 12 weeks +/- 2 weeks. Long Term Follow Up will cease when all patients on study have fulfilled the requirements for at least five follow up appointments.

What researchers measure

Primary outcomes

Percentage of Participants Experiencing Progression Free Survival at 12 Months
of a 3-phase comprehensive treatment approach including induction with BD followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.
Time frame: 12 months
Participants Evaluated for Toxicity
Toxicities will be assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), version 4.0.
Time frame: 2 years

Secondary outcomes

To Estimate the Hematologic Response Rate
\[Complete Response (CR) (Normalization of the free light chain (FLC)levels and ratio; Negative serum and urine ; \<5% plasma cells in bone marrow), Very Good Partial Response (VGPR) (Reduction in the dFLC (difference between involved \[iFLC\] and uninvolved FLC) to\<4mg/dl) and Partial Response (PR)\] (\>/= 50% reduction in the dFLC), achieved at 12 month, and at 24 months post-initiation of treatment following the 3-phase comprehensive treatment approach.
Time frame: 2 years
Organ Response - Cardiac Involvement at 12 Months
12 months post-initiation of treatment following the 3 phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.
Time frame: 12 months
Progression Free Survival at 24 Months
following the 3-phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.
Time frame: 24 months
Organ Response - Cardiac Involvement at 24 Months
24 months post-initiation of treatment following the 3 phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.
Time frame: 24 months
Organ Response - Renal Involvement at 12 Months
12 months post-initiation of treatment following the 3 phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.
Time frame: 12 months
Organ Response - Renal Involvement at 24 Months
24 months post-initiation of treatment following the 3 phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.
Time frame: 24 months

Results

Posted Mar 30, 2020

Participant flow

Participant flow — Overall Study
Milestone	Amyloidosis	Monoclonal Ig DepositionDisease (MIDD)
Started	19	1
Completed	19	1
Not completed	0	0

Outcome measures

PrimaryPercentage of Participants Experiencing Progression Free Survival at 12 Months

of a 3-phase comprehensive treatment approach including induction with BD followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.

Time frame:: 12 months
Reported as:: Number · percentage of patients

Percentage of Participants Experiencing Progression Free Survival at 12 Months
percentage of patients	Amyloidosis
Percentage of Participants Experiencing Progression Free Survival at 12 Months	84 (69 to 99)

PrimaryParticipants Evaluated for Toxicity

Toxicities will be assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), version 4.0.

Time frame:: 2 years
Reported as:: Count of participants · Participants

Participants Evaluated for Toxicity
Participants	Amyloidosis	Monoclonal Ig DepositionDisease (MIDD)
Participants Evaluated for Toxicity	19	1

SecondaryTo Estimate the Hematologic Response Rate

\[Complete Response (CR) (Normalization of the free light chain (FLC)levels and ratio; Negative serum and urine ; \<5% plasma cells in bone marrow), Very Good Partial Response (VGPR) (Reduction in the dFLC (difference between involved \[iFLC\] and uninvolved FLC) to\<4mg/dl) and Partial Response (PR)\] (\>/= 50% reduction in the dFLC), achieved at 12 month, and at 24 months post-initiation of treatment following the 3-phase comprehensive treatment approach.

Time frame:: 2 years
Reported as:: Count of participants · Participants

To Estimate the Hematologic Response Rate
Participants	Amyloidosis	Monoclonal Ig DepositionDisease (MIDD)
Complete Response (CR)	7	0
Partial Response (PR)	4	0
Progression of Disease (POD)	1	0
Stable Disease (SD)	1	0
Very Good Partial Response (VGPR)	6	1

SecondaryOrgan Response - Cardiac Involvement at 12 Months

12 months post-initiation of treatment following the 3 phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.

Time frame:: 12 months
Reported as:: Count of participants · Participants

Organ Response - Cardiac Involvement at 12 Months
Participants	Amyloidosis
Participants without cardiac involvement	7
Cardiac involvement, improved	8
Cardiac involvement, progressed	2
Cardiac involvement, died during induction	2

SecondaryProgression Free Survival at 24 Months

following the 3-phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.

Time frame:: 24 months
Reported as:: Number · percentage of patients

Progression Free Survival at 24 Months
percentage of patients	Amyloidosis
Progression Free Survival at 24 Months	68 (50 to 93)

SecondaryOrgan Response - Cardiac Involvement at 24 Months

24 months post-initiation of treatment following the 3 phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.

Time frame:: 24 months
Reported as:: Count of participants · Participants

Organ Response - Cardiac Involvement at 24 Months
Participants	Amyloidosis
Participants without cardiac involvement	7
Cardiac involvement, improved	8
Cardiac involvement, progressed	2
Cardiac involvement, died during induction	2

SecondaryOrgan Response - Renal Involvement at 12 Months

Time frame:: 12 months
Reported as:: Count of participants · Participants

Organ Response - Renal Involvement at 12 Months
Participants	Amyloidosis
No renal involvment	7
Renal involvment, improved	9
Renal involvment, profressed	2
Renal involvment, died	1

SecondaryOrgan Response - Renal Involvement at 24 Months

Time frame:: 24 months
Reported as:: Count of participants · Participants

Organ Response - Renal Involvement at 24 Months
Participants	Amyloidosis
No renal involvement	7
Renal Involvement, improved	9
Normal Renal Function after Kidney Transplant	2

Adverse events

Collected over 1 year. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Amyloidosis	7/19 (36.8%)	17/19 (89.5%)	19/19 (100%)
Monoclonal Ig DepositionDisease (MIDD)	0/1 (0%)	1/1 (100%)	1/1 (100%)

Most frequent serious events

Showing 10 of 24

Most frequent serious events
Event	Amyloidosis	Monoclonal Ig DepositionDisease (MIDD)
Hip fractureInjury, poisoning and procedural complications	0/19	1/1
Upper gastrointestinal hemorrhageGastrointestinal disorders	1/19	1/1
HypotensionVascular disorders	4/19	0/1
Acute kidney injuryRenal and urinary disorders	3/19	0/1
SyncopeNervous system disorders	2/19	0/1
DehydrationMetabolism and nutrition disorders	2/19	0/1
Cardiac arrestCardiac disorders	2/19	0/1
Lung infectionInfections and infestations	2/19	0/1
Upper respiratory infectionInfections and infestations	2/19	0/1
FallInjury, poisoning and procedural complications	1/19	0/1

Most frequent other events

Showing 10 of 102

Most frequent other events
Event	Amyloidosis	Monoclonal Ig DepositionDisease (MIDD)
Alanine aminotransferase increasedInvestigations	13/19	1/1
Alkaline phosphatase increasedInvestigations	17/19	1/1
Aspartate aminotransferase increasedInvestigations	16/19	1/1
Back painMusculoskeletal and connective tissue disorders	1/19	1/1
CoughRespiratory, thoracic and mediastinal disorders	2/19	1/1
Creatinine increasedInvestigations	12/19	1/1
DiarrheaGastrointestinal disorders	8/19	1/1
HyperglycemiaMetabolism and nutrition disorders	19/19	1/1
HyperkalemiaMetabolism and nutrition disorders	13/19	1/1
HypernatremiaMetabolism and nutrition disorders	8/19	1/1

Baseline characteristics

Age, Continuous
Age, Continuous(years)	Amyloidosis	Monoclonal Ig DepositionDisease (MIDD)	Total
Median	60 (38 to 73)	63 (63 to 63)	60 (38 to 73)

Sex: Female, Male
Sex: Female, Male(Participants)	Amyloidosis	Monoclonal Ig DepositionDisease (MIDD)	Total
Female	6	0	6
Male	13	1	14

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	Amyloidosis	Monoclonal Ig DepositionDisease (MIDD)	Total
Hispanic or Latino	0	0	0
Not Hispanic or Latino	19	1	20
Unknown or Not Reported	0	0	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	Amyloidosis	Monoclonal Ig DepositionDisease (MIDD)	Total
American Indian or Alaska Native	0	0	0
Asian	1	0	1
Native Hawaiian or Other Pacific Islander	0	0	0
Black or African American	1	0	1
White	16	1	17
More than one race	0	0	0
Unknown or Not Reported	1	0	1

Region of Enrollment
Region of Enrollment(Participants)	Amyloidosis	Monoclonal Ig DepositionDisease (MIDD)	Total
United States	19	1	20

Study locations

5 sites

Memorial Sloan Kettering Basking Ridge
Basking Ridge, New Jersey 07920, United States
Memorial Sloan Kettering Commack
Commack, New York 11725, United States
Memorial Sloan Kettering Westchester
Harrison, New York 10604, United States
Memorial Sloan Kettering Cancer Center
New York, New York 10065, United States
Memorial Sloan Kettering Rockville Centre
Rockville Centre, New York, United States

References and documents

Study documents

Protocol and statistical analysis plan · Jan 30, 2019

Documents are hosted by the registry — open the source record to download them.

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Apr 7, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT01383759
Lead sponsor: Memorial Sloan Kettering Cancer Center
Collaborators: Millennium Pharmaceuticals, Inc.
Responsible party: Sponsor
First posted: Jun 28, 2011
Start date: Jun 24, 2011
Primary completion: Apr 30, 2019
Completion: Apr 30, 2019
Results posted: Mar 30, 2020
Last update: Apr 7, 2020

Study contacts

Hani Hassoun, MD

principal investigator · Memorial Sloan Kettering Cancer Center

View the source record on ClinicalTrials.gov ↗

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