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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01383759
• Date of registration: 27/06/2011
• Prospective Registration: No
• Primary sponsor: Memorial Sloan Kettering Cancer Center
• Public title: 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
• Scientific title: Pilot Study of 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
• Date of first enrolment: June 24, 2011
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01383759
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: Hani Hassoun, MD
• Affiliation: Memorial Sloan Kettering Cancer Center

Key inclusion & exclusion criteria

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consent may be withdrawn by the subject at any time without prejudice to future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:

- 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Light Chain Deposition Disease (LCDD or MIDD)

Monoclonal Immunoglobulin Deposition Disease (MIDD)

Light Chain and Heavy Chain Deposition Disease (LHCDD or MIDD)

Amyloidosis

Intervention(s)

Drug: Bortezomib/Dexamethasone (BD), Followed By Autologous STC & Maintenance Bortezomib/Dexamethasone

Primary Outcome(s)

Percentage of Participants Experiencing Progression Free Survival at 12 Months [Time Frame: 12 months]

Participants Evaluated for Toxicity [Time Frame: 2 years]

Secondary Outcome(s)

Progression Free Survival at 24 Months [Time Frame: 24 months]

Organ Response - Renal Involvement at 12 Months [Time Frame: 12 months]

Organ Response - Renal Involvement at 24 Months [Time Frame: 24 months]

To Estimate the Hematologic Response Rate [Time Frame: 2 years]

Organ Response - Cardiac Involvement at 12 Months [Time Frame: 12 months]

Organ Response - Cardiac Involvement at 24 Months [Time Frame: 24 months]

Secondary ID(s)

11-061

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Millennium Pharmaceuticals, Inc.

Results

• Results available: Yes
• Date Posted: 30/03/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01383759