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Subcutaneous Daratumumab, Once Weekly Carfilzomib, and Dexamethasone (DKd) in Patients With High-Risk Smoldering Multiple Myeloma

Phase 2
Active, not recruiting
Conditions
Multiple Myeloma
Interventions
Registration Number
NCT04933539
Lead Sponsor
National Cancer Institute (NCI)
Brief Summary

Background:

Multiple myeloma (MM) is a tumor in which malignant plasma cells accumulate in the bone marrow. It can cause organ damage and is not curable. Researchers want to see if a combination drug treatment can help.

Objective:

To try to prevent or slow down developing MM and its associated organ damage by treating it while still in the smoldering phase with a mix of drugs known as DKd.

Eligibility:

People ages 18 and older with smoldering MM that is at high risk of converting to symptomatic MM.

Design:

Participants will be screened with:

Medical history

Physical exam

Blood and urine tests

Bone survey (x-rays of their bones)

Spinal magnetic resonance imaging

Bone marrow biopsy (a needle is used to remove bone marrow from their hipbone)

Electrocardiogram (to check heart function)

Lung function tests

Treatment will be given in 28-day cycles. Participants will get daratumumab by injection under the skin. They will get carfilzomib intravenously (IV) through a tube inserted in a vein. They will get dexamethasone as oral tablets or as an IV. They will get all 3 drugs for 8 or 12 cycles. Then they will get daratumumab alone for up to 24 cycles. They may have stem cells collected.

Participants will have frequent study visits. At these visits, they will repeat some screening tests. They will complete questionnaires. They will have imaging scans. For these scans, they may receive an oral or IV contrast.

Participants will have a follow-up visit 30 days after treatment ends. Then they will have visits every 3-12 months. They will be followed on this study for life.

Detailed Description

Background:

Smoldering multiple myeloma (SMM) is a precursor condition to MM defined by the clinical parameters of M-protein \>=3.0 g/dL or bone marrow plasma cells \>=10%, and absence of end organ disease.

Patients with high-risk SMM have a risk of progression to MM of 72-75% in 5 years with median time to progression of \<2 years.

The current standard of care for SMM is close follow-up without treatment, until symptomatic MM develops. However, International Myeloma Working Group (IMWG) recommends Preventive clinical trials need to be considered for patients with high risk smoldering myeloma.

Carfilzomib is a proteasome inhibitor and daratumumab is an anti-CD38 monoclonal antibody, both with potent anti-MM effects.

Objectives:

To assess the remission rate of daratumumab, carfilzomib, and dexamethasone (DKd) in patients with high-risk (HR) smoldering multiple myeloma (SMM) by determining the minimal residual disease (MRD) negative complete response (CR) rate by up to 12 cycles of induction therapy using flow cytometry.

Eligibility:

SMM according to the IMWG definition; i.e.:

Serum M-protein \>=3 g/dl and/or bone marrow plasma cells \>=10 % and \<60%

Absence of anemia: Hemoglobin \>10 g/dl

Absence of renal failure: serum creatinine \<2.0 mg/dL.

Absence of hypercalcemia: Ca \<10.5 mg/dl or 2.62 mmol/L

Absence of lytic bone lesions

\<=1 focal lesion on MRI

Involved/un-involved light chain ratio \<100

High-risk SMM per Mayo Clinic, Spanish PETHEMA, or the Rajkumar, Landgren, Mateos criteria

Age \>=18 years

Eastern Cooperative Oncology Group (ECOG) performance status 0-2

Adequate laboratory parameters

Design:

Single arm trial of combination therapy (daratumumab, carfilzomib, and dexamethasone) followed by daratumumab maintenance monotherapy (DKd-D) for high-risk SMM.

Participants will receive 8 cycles of DKd induction combination therapy. After 8 cycles, participants who have not attained an MRD negative remission will receive 4 additional cycles of DKd. Each cycle consists of 28-days.

After 4 cycles of induction therapy, transplant eligible participants may choose to undergo stem cell collection for storage.

After induction with DKd, participants will receive daratumumab maintenance therapy for 24 cycles.

Participants will have routine blood work with SPEP and free light chains at the start of each cycle during the induction phase. Laboratory evaluations may be spread out to every 3-6 months during the maintenance and follow-up phases.

Pre-treatment, post-treatment and follow-up bone marrow biopsies will be obtained for confirmation of diagnosis, response and correlative studies.

Participants will also undergo evaluation for MRD at regular interval time points, using multi-parametric flow cytometry, FDG PET-CT, and Diffusion Weighted Whole Body (DW-MRI).

The statistical analysis of the primary endpoint, MRD negativity, will be performed at the end of induction therapy.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
14
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Arm 1DexamethasoneDaratumumab SC (Cycles 1-2: Days 1, 8, 15, 22; Cycles 3-6: Days 1, 15; Cycles =7: Days 1 of the 28-day cycle); Carfilzomib IV (Days 1, 8, 15 of the 28-day cycle); Dexamethasone PO/IV (Days 1, 8, 15, 22 of the 28-day cycle)
Arm 1CarfilzomibDaratumumab SC (Cycles 1-2: Days 1, 8, 15, 22; Cycles 3-6: Days 1, 15; Cycles =7: Days 1 of the 28-day cycle); Carfilzomib IV (Days 1, 8, 15 of the 28-day cycle); Dexamethasone PO/IV (Days 1, 8, 15, 22 of the 28-day cycle)
Arm 1DaratumumabDaratumumab SC (Cycles 1-2: Days 1, 8, 15, 22; Cycles 3-6: Days 1, 15; Cycles =7: Days 1 of the 28-day cycle); Carfilzomib IV (Days 1, 8, 15 of the 28-day cycle); Dexamethasone PO/IV (Days 1, 8, 15, 22 of the 28-day cycle)
Primary Outcome Measures
NameTimeMethod
Response Rate12 cycles

To assess the remission rate of DKd in participants with high-risk (HR) SMM by determining the minimal residual disease (MRD) negative complete response (CR) by flow cytometry (10-5 sensitivity) rate after 12 cycles of therapy

Secondary Outcome Measures
NameTimeMethod
Biochemical and Symptomatic progression free survival (PFS)every 6-8 weeks

Using Kaplan-Meier method, considering those who biochemically progress or die and those who clinically progress or die, and censoring those who do not

Toxicity evaluation of DKdthrough 30 days post treatment

Descriptive statistics to determine safety of receiving KRd with daratumumab maintenance

Overall Response Rateevery 6-8 weeks

The fraction of participants who experience a PR, VGPR, CR or sCR after DKd determined by dividing the number of responders by the total evaluable patients.

Duration of Responseevery 6-8 weeks

Time from PR or better until the time of PD. Kaplan-Meier will be utilized

Durability of MRD negative complete response (CR)through 3 years post-treatment

Sustained negative CR rate 1, 2, 3 years.

Overall survival (OS)ongoing

Determined using the Kaplan-Meier method

Trial Locations

Locations (1)

National Institutes of Health Clinical Center

🇺🇸

Bethesda, Maryland, United States

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