Study Evaluating Safety and Efficacy of UCART Targeting CS1 in Patients With Relapsed/Refractory Multiple Myeloma (MELANI-01)
- Conditions
- Relapsed/Refractory Multiple Myeloma
- Interventions
- Biological: UCARTCS1A
- Registration Number
- NCT04142619
- Lead Sponsor
- Cellectis S.A.
- Brief 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 11
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Dose Escalation UCARTCS1A Several tested doses of UCARTCS1A until the Maximum Tolerated Dose (MTD) is identified.
- Primary Outcome Measures
Name Time Method Safety of UCARTCS1A 24 months. Incidence, nature and severity of adverse events and serious adverse events (SAEs) throughout the study.
- Secondary Outcome Measures
Name Time Method Duration of Response 24 months 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\]
Progression Free Survival 24 months 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
Response Assessment 24 months 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
Overall Survival 24 months From the first day of study treatment to the date of death from any cause, assessed up to Month 24
Trial Locations
- Locations (8)
Winship Cancer Institute Emory University
🇺🇸Atlanta, Georgia, United States
Hackensack Meridian Health
🇺🇸Hackensack, New Jersey, United States
UCSF Medical Center- Helen Diller Family Comprehensive Cancer Center
🇺🇸San Francisco, California, United States
Sarah Cannon Research Institute - Tennessee Oncology
🇺🇸Nashville, Tennessee, United States
MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
Sarah Cannon Research Institute - Colorado Blood Cancer Institute
🇺🇸Denver, Colorado, United States
Mayo Clinical Cancer Center (MCCC)
🇺🇸Rochester, Minnesota, United States
Sarah Cannon Research Institute - Methodist Healthcare
🇺🇸San Antonio, Texas, United States