Personalized Vaccine in Treating Patients With Smoldering Multiple Myeloma

NCT ID: NCT03631043

Last Updated: 2025-12-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

EARLY_PHASE1

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-12-21

Study Completion Date

2025-12-10

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This early phase I trial studies the side effects of personalized vaccine in treating patients with smoldering multiple myeloma. Vaccines made from a person's blood and bone marrow may help the body build an effective immune response to kill cancer cells.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

PRIMARY OBJECTIVES:

I. To demonstrate that developing a custom vaccine for smoldering multiple myeloma (SMM) is feasible.

II. To show that a custom peptide-based vaccine in smoldering multiple myeloma is safe.

SECONDARY OBJECTIVES:

I. To determine the intensity and longevity of antigen specific T-cell mediated immune responses to the neoantigen vaccine.

II. Time to progression to multiple myeloma (TTM) at the end of the follow up period (18 months).

III. Duration of response. IV. Clinical benefit rate (minor response \[MR\] or better) after 6 cycles of vaccine treatment per modified International Myeloma Working Group (IMWG) criteria for multiple myeloma (MM).

V. Overall survival.

EXPLORATORY OBJECTIVES:

I. Rate of minimal residual disease (MRD) negativity at complete remission (CR), if achieved. MRD assessment will be based on bone marrow aspirates.

II. Molecular profiling (including whole exome sequencing, gene expression profiling and ribonucleic acid (RNA) sequencing of tumor/bone marrow samples) and cellular (including flow cytometry) profiling at baseline using bone marrow aspirate samples and peripheral blood.

III. Immunophenotypic characterization of dendritic, T-, B-, natural killer (NK)- and NKT-cells, and inhibitory/activation markers on tumor cells at baseline (bone marrow and peripheral blood), day 1 of each cycle (peripheral blood only) and at completion of 6 cycles of therapy (bone marrow and peripheral blood) in bone marrow aspirate samples and/or peripheral blood.

IV. Perform mass spectrometry-based proteomics analysis on 15 myeloma cell lines to identify shared human leukocyte antigen (HLA) class I-restricted antigens that can be targeted with immunotherapy.

OUTLINE: Patients are assigned to 1 of 2 stages.

STAGE I: Patients undergo collection of blood and bone marrow for making the vaccine. Patients then receive personalized vaccine subcutaneously (SC) on days 1 and 15 of cycles 1-2 and on day 1 of cycles 3-6. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

STAGE II: Patients undergo collection of blood and bone marrow for making the vaccine. Patients then receive personalized vaccine SC on days 1 and 15 of cycles 1-2 and on day 1 of cycles 3-6. Patients also receive lenalidomide orally (PO) on days 1-21. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 3 months for 12 months.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Smoldering Plasma Cell Myeloma

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Stage I (personalized vaccine)

Patients undergo collection of blood and bone marrow for making the vaccine. Patients then receive personalized vaccine SC on days 1 and 15 of cycles 1-2 and on day 1 of cycles 3-6. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

Group Type EXPERIMENTAL

Biopsy Specimen Radiography

Intervention Type PROCEDURE

Undergo collection of blood and bone marrow

Vaccine Therapy

Intervention Type BIOLOGICAL

Given personalized vaccine SC

Stage II (personalized vaccine, lenalidomide)

Patients undergo collection of blood and bone marrow for making the vaccine. Patients then receive personalized vaccine SC on days 1 and 15 of cycles 1-2 and on day 1 of cycles 3-6. Patients also receive lenalidomide PO on days 1-21. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

Group Type EXPERIMENTAL

Biopsy Specimen Radiography

Intervention Type PROCEDURE

Undergo collection of blood and bone marrow

Lenalidomide

Intervention Type DRUG

Given PO

Vaccine Therapy

Intervention Type BIOLOGICAL

Given personalized vaccine SC

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Biopsy Specimen Radiography

Undergo collection of blood and bone marrow

Intervention Type PROCEDURE

Lenalidomide

Given PO

Intervention Type DRUG

Vaccine Therapy

Given personalized vaccine SC

Intervention Type BIOLOGICAL

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Biospecimen Radiography Specimen Radiography CC-5013 CC5013 CDC 501 Revlimid

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Adult patients with intermediate or high-risk smoldering multiple myeloma (SMM) are eligible
* Patients must have histologically confirmed SMM based on the following criteria. Both criteria must be met: (a) Serum monoclonal protein (IgG or IgA) \>= 3 g/dL or urinary monoclonal protein \>=500 mg per 24 hours and/or clonal bone marrow plasma cells more or equal to 10% (b) Absence of myeloma defining events or amyloidosis
* Additionally, patients must meet criteria for intermediate or high risk of progression to multiple myeloma by Programa para el Estudio de la Terapeutica en Hemopatía Maligna (PETHEMA) criteria (patients must have at least 1 risk factors present):

* \>= 95% abnormal plasma cells/total plasma cells in bone marrow compartment. (This is measured as a percentage of the total abnormal versus normal plasma cells in the bone marrow compartment using standard flow cytometry of the bone marrow aspirate. Having \>= 95% abnormal plasma cells/total plasma cells constitutes a risk factor for progression to multiple myeloma by PETHEMA criteria)
* Immunoparesis (The patient having low uninvolved immunoglobulins in peripheral blood, for example if a patient has IgA smoldering multiple myeloma, then either having a low IgM and/or low IgG will qualify as a risk factor for progression to multiple myeloma) \*1 of 2 risk factors: intermediate risk for progression at a rate of \~50% at 5 years \*2 of 2 risk factors: high risk for progression at a rate of 72% at 5 years
* Creatinine clearance \>= 40 ml/min using the modification of diet in renal disease (MDRD) equation
* Eastern Cooperative Oncology Group (ECOG) performance status 0-2
* Absolute neutrophil count (ANC) \>= 1.0 x 10\^9/L
* Hemoglobin \>= 10 g/dL
* Platelet count \>= 50 x 10\^9/L
* Platelet and blood transfusions are allowed on protocol. Growth factors, including granulocyte colony stimulating factors and erythropoietin are allowed
* Bilirubin \< 1.5 x the upper limit of normal (ULN)
* Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 3.0 x ULN
* Subjects must be able to give informed consent

Exclusion Criteria

* Evidence of myeloma defining events due to underlying plasma cell proliferative disorder meeting at least one of the following

* Hypercalcemia: serum calcium \> 0.25 mmol/L (\> 1 mg/dL) higher than the upper limit of normal or \> 2.75 mmol/L (\> 11 mg/dL)
* Renal Insufficiency: creatinine clearance \< 40 ml/min or serum creatinine \> 2 mg/dL
* Anemia: hemoglobin value \< 10 g/dL
* Bone lesions: one or more osteolytic lesions on skeletal radiography, computerized tomography (CT) or 2-deoxy-2\[F-18\] fluoro-D-glucose positron emission tomography CT (PET-CT)
* Prior or concurrent systemic treatment for SMM

* Bisphosphonates are permitted
* Treatment with corticosteroids is not permitted (allowed for physiologic doses)
* Radiotherapy is not permitted
* Prior treatment for smoldering multiple myeloma with chemotherapy agents approved for the treatment of multiple myeloma is not permitted
* Plasma cell leukemia
* Pregnant or lactating females
* Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment
* Has a known history of active TB (Bacillus tuberculosis)
* Has a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer
* Has an active infection requiring systemic therapy
* Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator
* Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial
* Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 60 days after the last dose of trial treatment
* Has a known history of human immunodeficiency virus (HIV) (HIV 1/2 antibodies)
* Has known active hepatitis B (e.g., hepatitis B surface antigen \[HBsAg\] reactive) or hepatitis C (e.g., hepatitis C virus \[HCV\] ribonucleic acid \[RNA\] \[qualitative\] is detected)
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

M.D. Anderson Cancer Center

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Krina Patel

Role: PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

M D Anderson Cancer Center

Houston, Texas, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Informed Consent Form

View Document

Related Links

Access external resources that provide additional context or updates about the study.

http://www.mdanderson.org

University of Texas MD Anderson Cancer Center Website

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2018-0345

Identifier Type: -

Identifier Source: org_study_id

NCI-2018-01614

Identifier Type: REGISTRY

Identifier Source: secondary_id

2018-0345

Identifier Type: OTHER

Identifier Source: secondary_id