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

January 7, 2025 updated by: CRISPR Therapeutics AG

A Phase 1 Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Anti-BCMA Allogeneic CRISPR-Cas9-Engineered T Cells (CTX120) in Subjects With Relapsed or Refractory Multiple Myeloma

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

26

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2050
        • Royal Prince Alfred Hospital
    • Victoria
      • Melbourne, Victoria, Australia, 3000
        • Peter MacCallum Cancer Centre
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X6
        • University Health Network, Princess Margaret Cancer Centre
  • Spain
      • Salamanca, Spain, 37007
        • Hospital Universitario de Salamanca
    • Barcelona
      • Badalona, Barcelona, Spain, 08916
        • Institut Catala d'Oncologia Hospital Germans Trias i Pujol
    • Navarra
      • Pamplona, Navarra, Spain, 31008
        • Universidad de Navarra
  • United States
    • Illinois
      • Chicago, Illinois, United States, 60637
        • University of Chicago
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania
    • Tennessee
      • Nashville, Tennessee, United States, 37203
        • Sarah Cannon Research Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  1. Age ≥18 years.
  2. Relapsed or refractory multiple myeloma, as defined by IMWG response criteria and treatment with at least 2 prior lines of therapy.
  3. Eastern Cooperative Oncology Group performance status 0 or 1.
  4. Adequate renal, liver, cardiac and pulmonary organ function
  5. 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.

Key Exclusion Criteria:

  1. Prior allogeneic stem cell transplant (SCT).
  2. Less than 60 days from autologous SCT at time of screening and with unresolved serious complications.
  3. Prior treatment with any gene therapy or genetically modified cell therapy, including CAR T cells or natural killer cells, or BCMA-directed therapy.
  4. Evidence of direct central nervous system (CNS) involvement by multiple myeloma.
  5. 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.
  6. Unstable angina, clinically significant arrhythmia, or myocardial infarction within 6 months of enrollment.
  7. Active HIV, hepatitis B virus or hepatitis C virus infection.
  8. 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.
  9. Use of systemic anti-tumor therapy or investigational agent within 14 days prior to enrollment.
  10. Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy.
  11. Women who are pregnant or breastfeeding.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: CTX120
Administered by IV infusion following lymphodepleting chemotherapy.
Biological: CTX120
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Part A (dose escalation): Incidence of adverse events
Time Frame: From CTX120 infusion up to 28 days post-infusion
Adverse events defined as dose-limiting toxicities
From CTX120 infusion up to 28 days post-infusion
Part B (cohort expansion): Objective response rate
Time Frame: From CTX120 infusion up to 60 months post-infusion
Objective response rate per International Myeloma Working Group (IMWG) response criteria.
From CTX120 infusion up to 60 months post-infusion

Secondary Outcome Measures

Outcome Measure
Time Frame
Progression Free Survival
Time Frame: From date of CTX120 infusion and date of disease progression or death due to any cause, assessed up to 60 months
From date of CTX120 infusion and date of disease progression or death due to any cause, assessed up to 60 months
Overall Survival
Time Frame: From date of CTX120 infusion until date of death due to any cause, assessed up to 60 months
From date of CTX120 infusion until date of death due to any cause, assessed up to 60 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CRISPR Therapeutics AG

Investigators

  • Study Director: Annie Weaver, PhD, CRISPR Therapeutics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 22, 2020

Primary Completion (Actual)

January 4, 2024

Study Completion (Actual)

January 4, 2024

Study Registration Dates

First Submitted

January 24, 2020

First Submitted That Met QC Criteria

January 27, 2020

First Posted (Actual)

January 28, 2020

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

January 7, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CRSP-ONC-002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Multiple Myeloma

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Lithuania

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe