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

Multiple Myeloma Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04244656
• Other study ID #: CRSP-ONC-002
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: January 22, 2020
• Est. completion date: January 2027

Study information

• Verified date: August 2023
• Source: CRISPR Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 26
• Est. completion date: January 2027
• Est. primary completion date: November 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Multiple Myeloma
• Neoplasms, Plasma Cell

Intervention

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.

Locations

Country	Name	City	State
Australia	Peter MacCallum Cancer Centre	Melbourne	Victoria
Australia	Royal Prince Alfred Hospital	Sydney	New South Wales
Canada	University Health Network, Princess Margaret Cancer Centre	Toronto	Ontario
Spain	Institut Catala d'Oncologia Hospital Germans Trias i Pujol	Badalona	Barcelona
Spain	Universidad de Navarra	Pamplona	Navarra
Spain	Hospital Universitario de Salamanca	Salamanca
United States	University of Chicago	Chicago	Illinois
United States	Sarah Cannon Research Institute	Nashville	Tennessee
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
CRISPR Therapeutics AG

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Part A (dose escalation): Incidence of adverse events	Adverse events defined as dose-limiting toxicities	From CTX120 infusion up to 28 days post-infusion
Primary	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	Progression Free Survival		From date of CTX120 infusion and date of disease progression or death due to any cause, assessed up to 60 months
Secondary	Overall Survival		From date of CTX120 infusion until date of death due to any cause, assessed up to 60 months