Back to resultsA Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma
Primary Purpose
Multiple Myeloma
Status
Active
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
CTX120
Sponsored by
About this trial
This is an interventional treatment trial for Multiple Myeloma focused on measuring CAR T, Allogeneic, Multiple myeloma, CRISPR-Cas9
Eligibility Criteria
Key Inclusion Criteria:
- 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.
Key Exclusion Criteria:
- 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.
Sites / Locations
- University of Chicago
- Oregon Health and Science University
- University of Pennsylvania
- Sarah Cannon Research Institute
- Royal Prince Alfred Hospital
- Peter MacCallum Cancer Centre
- University Health Network, Princess Margaret Cancer Centre
- Institut Catala d'Oncologia Hospital Germans Trias i Pujol
- Universidad de Navarra
- Hospital Universitario de Salamanca
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
CTX120
Arm Description
Administered by IV infusion following lymphodepleting chemotherapy.
Outcomes
Primary Outcome Measures
Part A (dose escalation): Incidence of adverse events
Adverse events defined as dose-limiting toxicities
Part B (cohort expansion): Objective response rate
Objective response rate per International Myeloma Working Group (IMWG) response criteria.
Secondary Outcome Measures
Progression Free Survival
Overall Survival
Full Information
NCT ID
NCT04244656
First Posted
January 24, 2020
Last Updated
August 28, 2023
Sponsor
CRISPR Therapeutics AG
1. Study Identification
Unique Protocol Identification Number
NCT04244656
Brief Title
A Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
January 22, 2020 (Actual)
Primary Completion Date
November 2026 (Anticipated)
Study Completion Date
January 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CRISPR Therapeutics AG
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Multiple Myeloma
Keywords
CAR T, Allogeneic, Multiple myeloma, CRISPR-Cas9
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
26 (Actual)
8. Arms, Groups, and Interventions
Arm Title
CTX120
Arm Type
Experimental
Arm Description
Administered by IV infusion following lymphodepleting chemotherapy.
Intervention Type
Biological
Intervention Name(s)
CTX120
Intervention Description
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.
Primary Outcome Measure Information:
Title
Part A (dose escalation): Incidence of adverse events
Description
Adverse events defined as dose-limiting toxicities
Time Frame
From CTX120 infusion up to 28 days post-infusion
Title
Part B (cohort expansion): Objective response rate
Description
Objective response rate per International Myeloma Working Group (IMWG) response criteria.
Time Frame
From CTX120 infusion up to 60 months post-infusion
Secondary Outcome Measure Information:
Title
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
Title
Overall Survival
Time Frame
From date of CTX120 infusion until date of death due to any cause, assessed up to 60 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
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.
Key Exclusion Criteria:
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Annie Weaver, PhD
Organizational Affiliation
CRISPR Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
University of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Sarah Cannon Research Institute
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37203
Country
United States
Facility Name
Royal Prince Alfred Hospital
City
Sydney
State/Province
New South Wales
ZIP/Postal Code
2050
Country
Australia
Facility Name
Peter MacCallum Cancer Centre
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3000
Country
Australia
Facility Name
University Health Network, Princess Margaret Cancer Centre
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X6
Country
Canada
Facility Name
Institut Catala d'Oncologia Hospital Germans Trias i Pujol
City
Badalona
State/Province
Barcelona
ZIP/Postal Code
08916
Country
Spain
Facility Name
Universidad de Navarra
City
Pamplona
State/Province
Navarra
ZIP/Postal Code
31008
Country
Spain
Facility Name
Hospital Universitario de Salamanca
City
Salamanca
ZIP/Postal Code
37007
Country
Spain
12. IPD Sharing Statement
Citations:
PubMed Identifier
33792221
Citation
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.
Results Reference
derived
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A Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma
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