Clinical trial NCT04244656: 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
Overall status: TERMINATED
Phase: PHASE1
Conditions: Multiple Myeloma
Keywords: CAR T
Allogeneic
Multiple myeloma
CRISPR-Cas9
Lead sponsor: CRISPR Therapeutics
Sponsor class: INDUSTRY
First posted: 2020-01-28
Enrollment: 26
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Part A (dose escalation): Incidence of adverse events
timeFrame: From CTX120 infusion up to 28 days post-infusion
measure: Part B (cohort expansion): Objective response rate
timeFrame: From CTX120 infusion up to 60 months post-infusion
Secondary outcomes:
measure: Progression Free Survival
timeFrame: From date of CTX120 infusion and date of disease progression or death due to any cause, assessed up to 60 months
measure: Overall Survival
timeFrame: From date of CTX120 infusion until date of death due to any cause, assessed up to 60 months
Other outcomes:
Locations:
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Nashville
country: United States
facility: Sarah Cannon Research Institute
state: Tennessee
city: Sydney
country: Australia
facility: Royal Prince Alfred Hospital
state: New South Wales
city: Melbourne
country: Australia
facility: Peter MacCallum Cancer Centre
state: Victoria
city: Toronto
country: Canada
facility: University Health Network, Princess Margaret Cancer Centre
state: Ontario
city: Badalona
country: Spain
facility: Institut Catala d'Oncologia Hospital Germans Trias i Pujol
state: Barcelona
city: Pamplona
country: Spain
facility: Universidad de Navarra
state: Navarra
city: Salamanca
country: Spain
facility: Hospital Universitario de Salamanca
Study officials:
affiliation: CRISPR Therapeutics
name: Annie Weaver, PhD
role: STUDY_DIRECTOR
References:
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.