Genetically Engineered Cells (MUC1-Activated T-Cells) for the Treatment of MUC1 Positive Recurrent or Refractory Multiple Myeloma

NCT05411497 | Active Not Recruiting Phase 1 DMC FDA Drug

• 3 other identifiers: MC200806NCI-2022-0311121-001202
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 2

Brief Summary

This phase I trial tests the safety, side effects and best dose of MUC1-activated T cells in treating patients with multiple myeloma that has come back (relapsed) or does not respond to treatment (refractory) and is positive for expression of the MUC1 protein. T-cells are infection fighting blood cells that can kill cancer cells. MUC1-activated T-cells are made from the body's own T cells. The manufactured T-cells are made to target the MUC1 genetic marker and may help the body's immune system identify and kill cancer cells.

Conditions

Recurrent Plasma Cell Myeloma; Refractory Plasma Cell Myeloma

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events

  Up to 4 years

Secondary Outcomes (3)

• Clinical response

  Up to 4 years

• Progression-free survival

  From registration to disease progression or death due to any cause, assessed up to 4 years

• Overall survival

  From registration to death due to any cause, assessed up to 4 years

Other Outcomes (6)

• Pharmacodynamics, adoptive T cells immunophenotyping TCRVbeta

  Up to 4 years

• Pharmacodynamics, immune cell profiling (whole blood)

  Up to 4 years

• Pharmacodynamics, cytokines (plasma)

  Up to 4 years

Study Arms (1)

Treatment (cyclophosphamide, MUC1-activated T-cells)

EXPERIMENTAL

LD CHEMOTHERAPY: Patients receive cyclophosphamide IV over 60 minutes on days -5, -4, -3. ASCT: Patients receive MUC1-activated T-cells IV over 10 minutes to 1 hour on day 0.

Biological: Autologous MUC1-activated T-cells; Drug: Cyclophosphamide

Interventions

Autologous MUC1-activated T-cells BIOLOGICAL

Given IV

Also known as: Autologous MUC1-activated T-lymphocytes

Treatment (cyclophosphamide, MUC1-activated T-cells)

Cyclophosphamide DRUG

Given IV

Also known as: (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida, Ciclofosfamide, Cicloxal, Clafen, Claphene, CP monohydrate, CTX, CYCLO-cell, Cycloblastin, Cycloblastine, Cyclophospham, Cyclophosphamid monohydrate, Cyclophosphamide Monohydrate, Cyclophosphamidum, Cyclophosphan, Cyclophosphane, Cyclophosphanum, Cyclostin, Cyclostine, Cytophosphan, Cytophosphane, Cytoxan, Fosfaseron, Genoxal, Genuxal, Ledoxina, Mitoxan, Neosar, Revimmune, Syklofosfamid, WR- 138719

Treatment (cyclophosphamide, MUC1-activated T-cells)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \>= 18 years
• Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0-1
• MUC1 expression in multiple myeloma tumor cells verified by immunohistochemistry (IHC) or flow cytometry (FCM). Heterogeneous tumor expression of MUC1 is acceptable
• Relapsed or refractory multiple myeloma previously treated with or intolerant to at least three prior lines of therapy and be relapsed or intolerant to a proteasome inhibitor, an immune modulatory drug (IMiD), and a CD38 antibody. Patients must be at least 90 days since an autologous stem cell transplant, if performed
• Patients must have measurable disease per IMWG criteria on study entry, which must include at least one of the following:
• Serum M-spike \>= 0.5 g/dL Patients with IgA, IgM or IgD myeloma in whom serum protein electrophoresis is deemed unreliable for routine M-protein quantitation may be considered eligible if total serum IgA, IgM or IgD level is elevated above normal range and parallels disease course
• hour urine M-spike \>= 200 mg
• Involved serum free light chain (FLC) \>= 10 mg/L with an abnormal free light chain ratio
• Bone marrow plasma cells \> 30%
• Patients with extramedullary disease:
• lesion that has a single diameter of \>= 2 cm measured by computed tomography (CT) or magnetic resonance imaging (MRI) or the CT portion of the positron emission tomography (PET)/CT
• Skin lesions can be used if the area is \>= 2cm in at least one diameter and measured with a ruler
• Willingness and ability to provide written informed consent
• Willing to return to Mayo Clinic Hospital in Arizona (MCA) for follow-up during the active monitoring phase of the study
• Willingness to provide mandatory blood, bone marrow biopsy, and aspirate specimens for correlative research

You may not qualify if:

• Clinically unresolved central nervous system (CNS) metastases
• Prior treatment targeting MUC1
• Subjects with known plasma cell leukemia (PCL)
• Any of the following are excluded because this study involves an agent (CTX) that has known genotoxic, mutagenic and/or teratogenic effects:
• Pregnant persons
• Nursing persons
• Persons of childbearing potential who are unwilling to employ adequate birth control measures
• History of myocardial infarction \>= 6 months prior to registration, and/or congestive heart failure requiring ongoing treatment such as medications and/or an implanted defibrillator to control life-threatening arrhythmias.
• Failure to recover to grade 1 or baseline from acute, reversible effects of prior therapy regardless of interval since last treatment.
• EXCEPTION: Grade 1 peripheral (sensory) neuropathy that has been stable for at least 3 months since completion of prior treatment.
• Uncontrolled concurrent illness including, but not limited to:
• Inability to clear an ongoing or active infection
• Symptomatic congestive heart failure
• Unstable angina pectoris
• Uncontrolled psychiatric problems and/or difficult social situation

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (2)

Mayo Clinic Hospital in Arizona

Phoenix, Arizona, 85054, United States

Location

Mayo Clinic in Arizona

Scottsdale, Arizona, 85259, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Multiple Myeloma

Interventions

Cyclophosphamide

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Study Officials

• Leif Bergsagel, M.D.

  Mayo Clinic Hospital in Arizona

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 19, 2022
• First Posted: June 9, 2022
• Study Start: June 20, 2022
• Primary Completion (Estimated): August 18, 2027
• Study Completion (Estimated): August 18, 2028
• Last Updated: November 4, 2025

Record last verified: 2025-10

Locations

US (2)