Relapsed/Refractory Multiple MyelomaPhase 1NCT04142619
Study Evaluating Safety and Efficacy of UCART Targeting CS1 in Patients With Relapsed/Refractory Multiple Myeloma (MELANI-01)
A Phase 1 study testing UCARTCS1A in people with Relapsed/Refractory Multiple Myeloma, run by Cellectis S.A. at 8 sites in United States.
Trial summary
This is a Phase I, FIH, open-label, dose escalation study evaluating Safety and Efficacy of UCART targeting CS1 in patients with Relapsed or Refractory Multiple Myeloma (MM). The purpose of this study is to evaluate the safety and clinical activity of UCARTCS1A and to determine the Maximum Tolerated Dose (MTD). Read more about this study
- Who is it for?
- Ages 18 to 64 years
- Conditions the study is for
- Relapsed/Refractory Multiple Myeloma
- What is being tested?
- UCARTCS1A
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 8 sitesUnited States
- Study dates
- 21 Nov 2019to 18 Jun 2023
- 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 to 64 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Patients with confirmed diagnosis of active multiple myeloma (as defined by International Myeloma Working Group [IMWG] criteria) who have relapsed/refractory disease after and have received at least 3 prior lines of prior therapy.
- Eastern Cooperative Oncology Group Performance Status of 0 or 1;
- No previous treatment with investigational gene targeting CS1 or chimeric antigen receptor therapy targeting CS1
- Adequate organ function, including bone marrow, renal, hepatic, pulmonary, and cardiac function based on the last assessment performed within the screening period.
- Other criteria may apply.
Who cannot join
- Previous treatment with investigational gene therapy targeting CS1 or chimeric antigen receptor therapy targeting CS1;
- Any cellular therapy (other than autologous or allogenic HSCT) within 60 days prior to enrollment;
- Prior treatment with rituximab or other anti-CD20 therapy within 3 months
- Any known active or uncontrolled infection
- Autologous hematopoietic stem cell transplantation (HSCT) within 12 weeks prior to enrollment; any cellular therapy (other than autologous) within 60 days prior to enrollment; prior allogeneic HSCT.
- Seropositive for Hepatitis C virus or positive for Hepatitis B surface antigen or core antibody.
- Presence of active and clinically relevant central nervous system disorder, such as epilepsy, generalized seizure disorder, paresis, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, cerebellar disease, or organic brain syndrome.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Winship Cancer Institute Emory University
Atlanta, Georgia, 30322
Sarah Cannon Research Institute - Colorado Blood Cancer Institute
Denver, Colorado, 80218
Hackensack Meridian Health
Hackensack, New Jersey, 07601
MD Anderson Cancer Center
Houston, Texas, 77030
Sarah Cannon Research Institute - Tennessee Oncology
Nashville, Tennessee, 37203-1625
Mayo Clinical Cancer Center (MCCC)
Rochester, Minnesota, 55905
Sarah Cannon Research Institute - Methodist Healthcare
San Antonio, Texas, 78229-6306
UCSF Medical Center- Helen Diller Family Comprehensive Cancer Center
San Francisco, California, 94115
About this study
Official title: Phase I, Open-label Dose-escalation Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of UCARTCS1A (Allogenic Engineered T-cells Expressing Anti-CS1 Chimeric Antigen Receptor) Administered in Patients With Relapsed/Refractory Multiple Myeloma
This is a Phase I, FIH, open-label, dose escalation study evaluating Safety and Efficacy of UCART targeting CS1 in patients with Relapsed or Refractory Multiple Myeloma (MM). The purpose of this study is to evaluate the safety and clinical activity of UCARTCS1A and to determine the Maximum Tolerated Dose (MTD).
- Conditions
- Relapsed/Refractory Multiple Myeloma
- Being tested
- UCARTCS1A
- Keywords
- Multiple Myeloma, Chimeric Antigen Receptor T-Cell (CART-T) therapy, Transcription Activator-Like Effector Nuclease (TALEN), Allogeneic
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UCARTCS1ABiological
Allogenic engineered T-cells expressing anti- CS1 Chimeric Antigen Receptor
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation | Experimental | Several tested doses of UCARTCS1A until the Maximum Tolerated Dose (MTD) is identified. Receives: Biological: UCARTCS1A |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of UCARTCS1A
Incidence, nature and severity of adverse events and serious adverse events (SAEs) throughout the study.
Measured: 24 months.
Secondary outcomes (4)
Response Assessment
At Day 35, Day 56 (M2), Day 84 (M3), Follow-up [Q3M up to Month 24; i.e., Month 3, Month 6, Month 9, Month 12, Month 15, Month 18, Month 21 and Month 24
Measured: 24 months
Duration of Response
Time Frame: From the date of the initial response to the date of disease progression or death from any cause, whichever occurs first, assessed up to Month 24]
Measured: 24 months
Progression Free Survival
From the first day of study treatment to the date of disease progression or death from any cause, whichever occurs first, assessed up to Month 24
Measured: 24 months
Overall Survival
From the first day of study treatment to the date of death from any cause, assessed up to Month 24
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2019
- Study start21 Nov 2019
- Primary completion18 Jun 2023
- Study completion18 Jun 2023
Why it stopped: The trial was discontinued due to sponsor's decision and not a consequence of any safety concern.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed/Refractory 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
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cellectis S.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04142619
- Sponsor's protocol ID
- UCARTCS1A_01
- Status last verified
- Sep 2023
- First submitted
- 25 Oct 2019
- Last updated
- 22 Sep 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I, Open-label Dose-escalation Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of UCARTCS1A (Allogenic Engineered T-cells Expressing Anti-CS1 Chimeric Antigen Receptor) Administered in Patients With Relapsed/Refractory Multiple Myeloma
- ClinicalTrials.gov ID
- NCT04142619
- Acronym / sponsor ID
- UCARTCS1A_01
- Conditions
- Relapsed/Refractory Multiple Myeloma
- Keywords
- Multiple Myeloma, Chimeric Antigen Receptor T-Cell (CART-T) therapy, Transcription Activator-Like Effector Nuclease (TALEN), Allogeneic
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The trial was discontinued due to sponsor's decision and not a consequence of any safety concern.
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Cellectis S.A.Industry (pharma, biotech, devices)
- Study start
- 21 Nov 2019 (actual)
- Primary completion
- 18 Jun 2023 (actual)
- Study completion
- 18 Jun 2023 (actual)
- First posted
- 29 Oct 2019 (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 Phase I, FIH, open-label, dose escalation study evaluating Safety and Efficacy of UCART targeting CS1 in patients with Relapsed or Refractory Multiple Myeloma (MM). The purpose of this study is to evaluate the safety and clinical activity of UCARTCS1A and to determine the Maximum Tolerated Dose (MTD).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation | Experimental | Biological: UCARTCS1ASeveral tested doses of UCARTCS1A until the Maximum Tolerated Dose (MTD) is identified. |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Patients with confirmed diagnosis of active multiple myeloma (as defined by International Myeloma Working Group [IMWG] criteria) who have relapsed/refractory disease after and have received at least 3 prior lines of prior therapy.
- Eastern Cooperative Oncology Group Performance Status of 0 or 1;
- No previous treatment with investigational gene targeting CS1 or chimeric antigen receptor therapy targeting CS1
- Adequate organ function, including bone marrow, renal, hepatic, pulmonary, and cardiac function based on the last assessment performed within the screening period.
- Other criteria may apply.
Key exclusion criteria (7 listed)
- Previous treatment with investigational gene therapy targeting CS1 or chimeric antigen receptor therapy targeting CS1;
- Any cellular therapy (other than autologous or allogenic HSCT) within 60 days prior to enrollment;
- Prior treatment with rituximab or other anti-CD20 therapy within 3 months
- Any known active or uncontrolled infection
- Autologous hematopoietic stem cell transplantation (HSCT) within 12 weeks prior to enrollment; any cellular therapy (other than autologous) within 60 days prior to enrollment; prior allogeneic HSCT.
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 |
|---|---|---|
| UCARTCS1AAllogenic engineered T-cells expressing anti- CS1 Chimeric Antigen Receptor | Biological | Dose Escalation |
Primary outcome
Safety of UCARTCS1A
Time frame: 24 months.
Incidence, nature and severity of adverse events and serious adverse events (SAEs) throughout the study.
4 secondary outcomes
Response Assessment
Time frame: 24 months
Duration of Response
Time frame: 24 months
Progression Free Survival
Time frame: 24 months
Overall Survival
Time frame: 24 months
Results and safety
The study was stopped early (reason given: The trial was discontinued due to sponsor's decision and not a consequence of any safety concern.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2023.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing UCARTCS1A in people with Relapsed/Refractory Multiple Myeloma, run by Cellectis S.A. at 8 sites in United States.
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 7 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 22 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. 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: The trial was discontinued due to sponsor's decision and not a consequence of any safety concern.). 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?
Safety of UCARTCS1A (time frame: 24 months.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 22 Sep 2023.