Study Evaluating Safety and Efficacy of UCART Targeting CS1 in Patients With Relapsed/Refractory Multiple Myeloma (MELANI-01)

Sponsor

Cellectis S.A. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04142619

Collaborator

(none)

Enrollment

Locations

Arm

35.4

Anticipated Duration (Months)

3.6

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Relapsed/Refractory Multiple Myeloma	Biological: UCARTCS1A	Phase 1

Study Design

Study Type:

Interventional

Anticipated Enrollment :

18 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Nov 21, 2019

Anticipated Primary Completion Date :

May 18, 2022

Anticipated Study Completion Date :

Nov 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Dose Escalation Several tested doses of UCARTCS1A until the Maximum Tolerated Dose (MTD) is identified.	Biological: UCARTCS1A Allogenic engineered T-cells expressing anti- CS1 Chimeric Antigen Receptor

Arm

Intervention/Treatment

Experimental: Dose Escalation

Several tested doses of UCARTCS1A until the Maximum Tolerated Dose (MTD) is identified.

Biological: UCARTCS1A

Allogenic engineered T-cells expressing anti- CS1 Chimeric Antigen Receptor

Outcome Measures

Primary Outcome Measures

Safety of UCARTCS1A [24 months.]
Incidence, nature and severity of adverse events and serious adverse events (SAEs) throughout the study.

Secondary Outcome Measures

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
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
Overall Survival [24 months]
From the first day of study treatment to the date of death from any cause, assessed up to Month 24

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 64 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UCSF Medical Center- Helen Diller Family Comprehensive Cancer Center	San Francisco	California	United States	94115
2	Mayo Clinical Cancer Center (MCCC)	Rochester	Minnesota	United States	55905
3	Hackensack Meridian Health	Hackensack	New Jersey	United States	07601
4	Weill Cornell Medical College	New York	New York	United States	10065
5	MD Anderson Cancer Center	Houston	Texas	United States	77030