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

Sponsor
CRISPR Therapeutics AG (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04244656
Collaborator
(none)
80
Enrollment
10
Locations
1
Arm
83.3
Anticipated Duration (Months)
8
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

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.

Condition or DiseaseIntervention/TreatmentPhase
  • Biological: CTX120
Phase 1

Detailed Description

The study may enroll approximately 80 subjects in total.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
80 participants
Allocation:
N/A
Intervention Model:
Sequential Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
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
Actual Study Start Date :
Jan 22, 2020
Anticipated Primary Completion Date :
Nov 1, 2026
Anticipated Study Completion Date :
Jan 1, 2027

Arms and Interventions

ArmIntervention/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.

Outcome Measures

Primary Outcome Measures

  1. Part A (dose escalation): Incidence of adverse events [From CTX120 infusion up to 28 days post-infusion]

    Adverse events defined as dose-limiting toxicities

  2. Part B (cohort expansion): Objective response rate [From CTX120 infusion up to 60 months post-infusion]

    Objective response rate per International Myeloma Working Group (IMWG) response criteria.

Secondary Outcome Measures

  1. Progression Free Survival [From date of CTX120 infusion and date of disease progression or death due to any cause, assessed up to 60 months]

  2. Overall Survival [From date of CTX120 infusion until date of death due to any cause, assessed up to 60 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

SiteCityStateCountryPostal Code
1University of ChicagoChicagoIllinoisUnited States60637
2Oregon Health and Science UniversityPortlandOregonUnited States97239
3University of PennsylvaniaPhiladelphiaPennsylvaniaUnited States19104
4Sarah Cannon Research InstituteNashvilleTennesseeUnited States37203
5Royal Prince Alfred HospitalSydneyNew South WalesAustralia2050
6Peter MacCallum Cancer CentreMelbourneVictoriaAustralia3000
7University Health Network, Princess Margaret Cancer CentreTorontoOntarioCanadaM5G 1X6
8Institut Catala d'Oncologia Hospital Germans Trias i PujolBadalonaBarcelonaSpain08916
9Universidad de NavarraPamplonaNavarraSpain31008
10Hospital Universitario de SalamancaSalamancaSpain37007

Sponsors and Collaborators

  • CRISPR Therapeutics AG

Investigators

  • Study Director: Ewelina Morawa, MD, CRISPR Therapeutics

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
CRISPR Therapeutics AG
ClinicalTrials.gov Identifier:
NCT04244656
Other Study ID Numbers:
  • CRSP-ONC-002
First Posted:
Jan 28, 2020
Last Update Posted:
Oct 8, 2021
Last Verified:
Oct 1, 2021
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by CRISPR Therapeutics AG
Additional relevant MeSH terms:

Study Results

No Results Posted as of Oct 8, 2021