Multiple MyelomaPhase 1NCT04244656
A Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma
A Phase 1 study testing CTX120 in people with Multiple Myeloma, run by CRISPR Therapeutics AG at 10 sites in 4 countries.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Multiple Myeloma
- What is being tested?
- CTX120
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 10 sitesin 4 countries
- Study dates
- 22 Jan 2020to 4 Jan 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
10 sites in 4 countries.
10 sites in 4 countries
United States4 sites
University of Chicago
Chicago, Illinois, 60637
Sarah Cannon Research Institute
Nashville, Tennessee, 37203
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Oregon Health and Science University
Portland, Oregon, 97239
Spain3 sites
Institut Catala d'Oncologia Hospital Germans Trias i Pujol
Badalona, Barcelona, 08916
Universidad de Navarra
Pamplona, Navarre, 31008
Hospital Universitario de Salamanca
Salamanca, 37007
Australia2 sites
Peter MacCallum Cancer Centre
Melbourne, Victoria, 3000
Royal Prince Alfred Hospital
Sydney, New South Wales, 2050
Canada1 site
University Health Network, Princess Margaret Cancer Centre
Toronto, Ontario, M5G 1X6
About this study
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
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.
- Conditions
- Multiple Myeloma
- Being tested
- CTX120
- Keywords
- CAR T, Allogeneic, Multiple myeloma, CRISPR-Cas9
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CTX120Biological
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.
| Study group | Role | What they receive |
|---|---|---|
| CTX120 | Experimental | Administered by IV infusion following lymphodepleting chemotherapy. Receives: Biological: CTX120 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A (dose escalation): Incidence of adverse events
Adverse events defined as dose-limiting toxicities
Measured: From CTX120 infusion up to 28 days post-infusion
Part B (cohort expansion): Objective response rate
Objective response rate per International Myeloma Working Group (IMWG) response criteria.
Measured: From CTX120 infusion up to 60 months post-infusion
Secondary outcomes (2)
Progression Free Survival
Measured: From date of CTX120 infusion and date of disease progression or death due to any cause, assessed up to 60 months
Overall Survival
Measured: From date of CTX120 infusion until date of death due to any cause, assessed up to 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2020
- Study start22 Jan 2020
- Primary completion4 Jan 2024
- Study completion4 Jan 2024
Why it stopped: Patients to be followed up in the CRSP-ONC-LTF study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Multiple MyelomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- CRISPR Therapeutics AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Annie Weaver, PhD, Study director, CRISPR Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04244656
- Sponsor's protocol ID
- CRSP-ONC-002
- Status last verified
- Jan 2025
- First submitted
- 24 Jan 2020
- Last updated
- 8 Jan 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 33792221
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04244656
- Acronym / sponsor ID
- CRSP-ONC-002
- Conditions
- Multiple Myeloma
- Keywords
- CAR T, Allogeneic, Multiple myeloma, CRISPR-Cas9
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Patients to be followed up in the CRSP-ONC-LTF study
- Enrolment
- 26 participants (actual)
- Lead sponsor
- CRISPR Therapeutics AGIndustry (pharma, biotech, devices)
- Study start
- 22 Jan 2020 (actual)
- Primary completion
- 4 Jan 2024 (actual)
- Study completion
- 4 Jan 2024 (actual)
- First posted
- 28 Jan 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CTX120 | Experimental | Biological: CTX120Administered by IV infusion following lymphodepleting chemotherapy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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 (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CTX120CTX120 B-cell maturation antigen (BCMA)-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components. | Biological | CTX120 |
Primary outcome
Part A (dose escalation): Incidence of adverse events
Time frame: From CTX120 infusion up to 28 days post-infusion
Adverse events defined as dose-limiting toxicities
Part B (cohort expansion): Objective response rate
Time frame: From CTX120 infusion up to 60 months post-infusion
Objective response rate per International Myeloma Working Group (IMWG) response criteria.
2 secondary outcomes
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
Overall Survival
Time frame: From date of CTX120 infusion until date of death due to any cause, assessed up to 60 months
Results and safety
The study was stopped early (reason given: Patients to be followed up in the CRSP-ONC-LTF study). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2025.
- Derived from this study: PubMed 33792221Bruno 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Annie Weaver, PhD study director
CRISPR Therapeutics
10 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CTX120 in people with Multiple Myeloma, run by CRISPR Therapeutics AG at 10 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Spain, Australia, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Patients to be followed up in the CRSP-ONC-LTF study). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part A (dose escalation): Incidence of adverse events (time frame: From CTX120 infusion up to 28 days post-infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04244656, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2025.