Clinical trial NCT04142619: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Relapsed/Refractory Multiple Myeloma
Keywords: Multiple Myeloma
Chimeric Antigen Receptor T-Cell (CART-T) therapy
Transcription Activator-Like Effector Nuclease (TALEN)
Allogeneic
Lead sponsor: Cellectis S.A.
Sponsor class: INDUSTRY
First posted: 2019-10-29
Enrollment: 11
Sex: ALL
Minimum age (years): 18
Maximum age (years): 64
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


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.

Primary outcomes:
measure: Safety of UCARTCS1A
timeFrame: 24 months.
Secondary outcomes:
measure: Response Assessment
timeFrame: 24 months
measure: Duration of Response
timeFrame: 24 months
measure: Progression Free Survival
timeFrame: 24 months
measure: Overall Survival
timeFrame: 24 months
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: UCSF Medical Center- Helen Diller Family Comprehensive Cancer Center
state: California
city: Denver
country: United States
facility: Sarah Cannon Research Institute - Colorado Blood Cancer Institute
state: Colorado
city: Atlanta
country: United States
facility: Winship Cancer Institute Emory University
state: Georgia
city: Rochester
country: United States
facility: Mayo Clinical Cancer Center (MCCC)
state: Minnesota
city: Hackensack
country: United States
facility: Hackensack Meridian Health
state: New Jersey
city: Nashville
country: United States
facility: Sarah Cannon Research Institute - Tennessee Oncology
state: Tennessee
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: San Antonio
country: United States
facility: Sarah Cannon Research Institute - Methodist Healthcare
state: Texas
Study officials:
References: