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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04244656




Registration number
NCT04244656
Ethics application status
Date submitted
24/01/2020
Date registered
28/01/2020
Date last updated
6/11/2020

Titles & IDs
Public title
A Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma
Scientific 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
Secondary ID [1] 0 0
CRSP-ONC-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Multiple Myeloma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Other cancer types

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - CTX120

Experimental: CTX120 - Administered by IV infusion following lymphodepleting chemotherapy.


Other interventions: 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.

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part A (dose escalation): Incidence of adverse events - Adverse events defined as dose-limiting toxicities
Timepoint [1] 0 0
From CTX120 infusion up to 28 days post-infusion
Primary outcome [2] 0 0
Part B (cohort expansion): Objective response rate - Objective response rate per International Myeloma Working Group (IMWG) response criteria.
Timepoint [2] 0 0
From CTX120 infusion up to 60 months post-infusion
Secondary outcome [1] 0 0
Progression Free Survival
Timepoint [1] 0 0
From date of CTX120 infusion and date of disease progression or death due to any cause, assessed up to 60 months
Secondary outcome [2] 0 0
Overall Survival
Timepoint [2] 0 0
From date of CTX120 infusion until date of death due to any cause, assessed up to 60 months

Eligibility
Key inclusion criteria
Key

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
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
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 design
Purpose of the study
Treatment
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Research Site 5 - Sydney
Recruitment hospital [2] 0 0
Research Site 2 - Melbourne
Recruitment postcode(s) [1] 0 0
2050 - Sydney
Recruitment postcode(s) [2] 0 0
3000 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Illinois
Country [2] 0 0
United States of America
State/province [2] 0 0
Oregon
Country [3] 0 0
United States of America
State/province [3] 0 0
Tennessee
Country [4] 0 0
Canada
State/province [4] 0 0
Ontario
Country [5] 0 0
Spain
State/province [5] 0 0
Navarra

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
CRISPR Therapeutics AG
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT04244656
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ewelina Morawa, MD
Address 0 0
CRISPR Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Trials
Address 0 0
Country 0 0
Phone 0 0
+1 877-214-4634
Fax 0 0
Email 0 0
MedicalAffairs@crisprtx.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04244656