Longterm Follow-up of Subjects Treated With bb2121

NCT02786511 | Completed

• 1 other identifier: LTF-305
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 9

Brief Summary

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.

Conditions

Multiple Myeloma

Outcome Measures

Primary Outcomes (6)

• 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

Study Arms (1)

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

Interventions

Safety and efficacy assessments DRUG

Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term implications of autologous transplant

Subjects with multiple myeloma

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Celgene: lead

Study Sites (9)

Stanford Cancer Center

Palo Alto, California, 94305, United States

Location

National Cancer Institute

Bethesda, Maryland, 20892, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02144, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

Mayo Clinic Cancer Center

Rochester, Minnesota, 55905, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Mount Sinai Medical Center

New York, New York, 10029, United States

Location

Sarah Cannon Research Institute

Nashville, Tennessee, 37203, United States

Location

MeSH Terms

Conditions

Multiple Myeloma

Interventions

Safety

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Accident Prevention; Accidents; Public Health; Environment and Public Health

Study Officials

• Kristen Hege

  Celgene Corporation

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 26, 2016
• First Posted: June 1, 2016
• Study Start: April 28, 2016
• Primary Completion: October 11, 2019
• Study Completion: October 11, 2019
• Last Updated: January 22, 2020

Record last verified: 2020-01

Locations

US (9)