Longterm Follow-up of Subjects Treated With bb2121

This is a multi-center, non-randomized, open label, longterm safety and efficacy follow-up study for subjects who have been treated with bb2121 in the Phase 1 clinical parent study, that evaluated the safety and efficacy of bb2121 in subjects with relapsed or refractory B cell maturation antigen (BCMA)-expressing multiple myeloma.

bb2121 is defined as autologous T lymphocytes (T cells) transduced ex vivo with anti-BCMA02 CAR lentiviral vector encoding the chimeric antigen receptor (CAR) targeted to human BCMA suspended in cryopreservative solution. bb2121 is administered in subjects 1 time (or retreated if retreatment criteria are met) in parent clinical study. No investigational treatment will be administered in this study.

After completing the parent study, eligible subjects will be followed for up to 15 years after their last bb2121 infusion in the parent study.

Study Overview

• Status: Completed
• Conditions: Multiple Myeloma
• Intervention / Treatment: Drug: Safety and efficacy assessments

Detailed Description

The LTF-305 study has completed enrollment and is scheduled to be closed. All patients participating in this study have discontinued from follow-up or have been transferred into the GC-LTFU-001 study for further observation (similar to time frames established in the LTF-305).

• Study Type: Observational
• Enrollment (Anticipated): 50

Contacts and Locations

Study Locations

• United States
  • California
    • Palo Alto, California, United States, 94305
      • Stanford Cancer Center
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • National Cancer Institute
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Beth Israel Deaconess Medical Center
    • Boston, Massachusetts, United States, 02115
      • Dana Farber Cancer Institute
    • Boston, Massachusetts, United States, 02144
      • Massachusetts General Hospital
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic Cancer Center
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Hackensack University Medical Center
  • New York
    • New York, New York, United States, 10029
      • Mount Sinai Medical Center
  • Tennessee
    • Nashville, Tennessee, United States, 37203
      • Sarah Cannon Research Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Subjects with multiple myeloma who were administered bb2121 in Study CRB-401

Description

Inclusion Criteria:

• Provision of written informed consent for this study by subjects
• Were administered bb2121 in the parent clinical study
• Able to comply with the study requirements

Exclusion Criteria:

• Subject has disease progression AND subject has undetectable VCN (<0.0003 vector copies per diploid genome) in peripheral blood cells for 2 consecutive measurements at least 1 month apart, at least 12 months after drug product infusion

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Subjects with multiple myeloma; Subjects treated with ex vivo gene therapy in a bluebird bio sponsored trial who agree to participate in this study.	Drug: Safety and efficacy assessments; Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term implications of autologous transplant

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival		15 years post-drug product infusion
Monitoring for all Adverse Events, including Serious Adverse Events, related to the drug product		15 years post-drug product infusion
Monitoring for all Serious Adverse Events including any new malignancy or new diagnosis of a neurologic, rheumatologic, or hematologic disorder that is clinically significant		5 years post-drug product infusion
Monitoring for Multiple Myeloma-specific response according to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma	Subjects without disease progression will be evaluated for at least 5 years post-drug product infusion if VCN is undetectable, and up to 15 years post-drug product infusion if VCN remains detectable.	5-15 years post-drug product infusion
Progression Free Survival	Subjects without disease progression will be evaluated for at least 5 years post-drug product infusion if VCN is undetectable, and up to 15 years post-drug product infusion if VCN remains detectable.	5-15 years post-drug product infusion
Monitoring for Vector Copy Number (VCN)	Subjects without disease progression will be evaluated for at least 5 years post-drug product infusion if VCN is undetectable, and up to 15 years post-drug product infusion if VCN remains detectable.	5-15 years post-drug product infusion

Collaborators and Investigators

• Sponsor: Celgene
• Investigators: Study Director: Kristen Hege, Celgene Corporation

Study record dates

Study Major Dates

• Study Start (ACTUAL): April 28, 2016
• Primary Completion (ACTUAL): October 11, 2019
• Study Completion (ACTUAL): October 11, 2019

Study Registration Dates

• First Submitted: May 26, 2016
• First Submitted That Met QC Criteria: May 31, 2016
• First Posted (ESTIMATE): June 1, 2016

Study Record Updates

• Last Update Posted (ACTUAL): January 22, 2020
• Last Update Submitted That Met QC Criteria: January 20, 2020
• Last Verified: January 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Immunoproliferative Disorders; Hematologic Diseases; Hemorrhagic Disorders; Hemostatic Disorders; Paraproteinemias; Blood Protein Disorders; Neoplasms, Plasma Cell; Multiple Myeloma
• Other Study ID Numbers: LTF-305