Description:
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).
Title
- Brief Title: Study Evaluating Safety and Efficacy of UCART Targeting CS1 in Patients With Relapsed/Refractory Multiple Myeloma (MELANI-01)
- 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
Clinical Trial IDs
- ORG STUDY ID:
UCARTCS1A_01
- NCT ID:
NCT04142619
Conditions
- Relapsed/Refractory Multiple Myeloma
Interventions
Drug | Synonyms | Arms |
---|
UCARTCS1A | | Dose Escalation |
Purpose
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).
Trial Arms
Name | Type | Description | Interventions |
---|
Dose Escalation | Experimental | Several tested doses of UCARTCS1A until the Maximum Tolerated Dose (MTD) is identified. | |
Eligibility Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Maximum Eligible Age: | 64 Years |
Minimum Eligible Age: | 18 Years |
Eligible Gender: | All |
Healthy Volunteers: | No |
Primary Outcome Measures
Measure: | Safety of UCARTCS1A |
Time Frame: | 24 months. |
Safety Issue: | |
Description: | Incidence, nature and severity of adverse events and serious adverse events (SAEs) throughout the study. |
Secondary Outcome Measures
Measure: | Response Assessment |
Time Frame: | 24 months |
Safety Issue: | |
Description: | 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 |
Measure: | Duration of Response |
Time Frame: | 24 months |
Safety Issue: | |
Description: | 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] |
Measure: | Progression Free Survival |
Time Frame: | 24 months |
Safety Issue: | |
Description: | 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 |
Measure: | Overall Survival |
Time Frame: | 24 months |
Safety Issue: | |
Description: | From the first day of study treatment to the date of death from any cause, assessed up to Month 24 |
Details
Phase: | Phase 1 |
Primary Purpose: | Interventional |
Overall Status: | Recruiting |
Lead Sponsor: | Cellectis S.A. |
Trial Keywords
- Multiple Myeloma
- Chimeric Antigen Receptor T-Cell (CART-T) therapy
- Transcription Activator-Like Effector Nuclease (TALEN)
- Allogeneic
Last Updated
April 29, 2021