Clinical Trials /

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

NCT04142619

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).

Related Conditions:
  • Multiple Myeloma
Recruiting Status:

Recruiting

Phase:

Phase 1

Trial Eligibility

Document

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

DrugSynonymsArms
UCARTCS1ADose 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

NameTypeDescriptionInterventions
Dose EscalationExperimentalSeveral tested doses of UCARTCS1A until the Maximum Tolerated Dose (MTD) is identified.
  • UCARTCS1A

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