A Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma

NCT04244656 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: CRSP-ONC-002
• Study Type: interventional
• Target: 26
• Locations: 4 countries
• Sites: 10

Brief Summary

This is a single-arm, open-label, multicenter, Phase 1 study evaluating the safety and efficacy of CTX120 in subjects with relapsed or refractory multiple myeloma.

Conditions

Multiple Myeloma

Keywords

CAR T; Allogeneic; Multiple myeloma; CRISPR-Cas9

Outcome Measures

Primary Outcomes (2)

• Part A (dose escalation): Incidence of adverse events

  Adverse events defined as dose-limiting toxicities

  From CTX120 infusion up to 28 days post-infusion

• Part B (cohort expansion): Objective response rate

  Objective response rate per International Myeloma Working Group (IMWG) response criteria.

  From CTX120 infusion up to 60 months post-infusion

Secondary Outcomes (2)

• Progression Free Survival

  From date of CTX120 infusion and date of disease progression or death due to any cause, assessed up to 60 months

• Overall Survival

  From date of CTX120 infusion until date of death due to any cause, assessed up to 60 months

Study Arms (1)

CTX120

EXPERIMENTAL

Administered by IV infusion following lymphodepleting chemotherapy.

Biological: CTX120

Interventions

CTX120 BIOLOGICAL

CTX120 B-cell maturation antigen (BCMA)-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components.

CTX120

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.
• Relapsed or refractory multiple myeloma, as defined by IMWG response criteria and treatment with at least 2 prior lines of therapy.
• Eastern Cooperative Oncology Group performance status 0 or 1.
• Adequate renal, liver, cardiac and pulmonary organ function
• Female subjects of childbearing potential and male subjects must agree to use acceptable method(s) of contraception from enrollment through at least 12 months after CTX120 infusion.

You may not qualify if:

• Prior allogeneic stem cell transplant (SCT).
• Less than 60 days from autologous SCT at time of screening and with unresolved serious complications.
• Prior treatment with any gene therapy or genetically modified cell therapy, including CAR T cells or natural killer cells, or BCMA-directed therapy.
• Evidence of direct central nervous system (CNS) involvement by multiple myeloma.
• History or presence of clinically relevant CNS pathology such as a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, any autoimmune disease with CNS involvement.
• Unstable angina, clinically significant arrhythmia, or myocardial infarction within 6 months of enrollment.
• Active HIV, hepatitis B virus or hepatitis C virus infection.
• Previous or concurrent malignancy, except basal cell or squamous cell skin carcinoma, adequately resected and in situ carcinoma of cervix, or a previous malignancy that was completely resected and has been in remission for ≥5 years.
• Use of systemic anti-tumor therapy or investigational agent within 14 days prior to enrollment.
• Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy.
• Women who are pregnant or breastfeeding.

Sponsors & Collaborators

• CRISPR Therapeutics AG: lead

Study Sites (10)

University of Chicago

Chicago, Illinois, 60637, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Sarah Cannon Research Institute

Nashville, Tennessee, 37203, United States

Location

Royal Prince Alfred Hospital

Sydney, New South Wales, 2050, Australia

Location

Peter MacCallum Cancer Centre

Melbourne, Victoria, 3000, Australia

Location

University Health Network, Princess Margaret Cancer Centre

Toronto, Ontario, M5G 1X6, Canada

Location

Institut Catala d'Oncologia Hospital Germans Trias i Pujol

Badalona, Barcelona, 08916, Spain

Location

Universidad de Navarra

Pamplona, Navarre, 31008, Spain

Location

Hospital Universitario de Salamanca

Salamanca, 37007, Spain

Location

Related Publications (1)

• Bruno B, Wasch R, Engelhardt M, Gay F, Giaccone L, D'Agostino M, Rodriguez-Lobato LG, Danhof S, Gagelmann N, Kroger N, Popat R, Van de Donk NWCJ, Terpos E, Dimopoulos MA, Sonneveld P, Einsele H, Boccadoro M. European Myeloma Network perspective on CAR T-Cell therapies for multiple myeloma. Haematologica. 2021 Aug 1;106(8):2054-2065. doi: 10.3324/haematol.2020.276402.

  PMID: 33792221 DERIVED

MeSH Terms

Conditions

Multiple Myeloma

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

Study Officials

• Annie Weaver, PhD

  CRISPR Therapeutics

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: January 24, 2020
• First Posted: January 28, 2020
• Study Start: January 22, 2020
• Primary Completion: January 4, 2024
• Study Completion: January 4, 2024
• Last Updated: January 8, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (4); ES (3); CA (1); AU (2)