Long-Term Follow-Up Study for Subjects Treated With P-BCMA-101

Study Overview
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Brief Summary
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Subjects are enrolled in this study following completion or early discontinuation from a Poseida sponsored or supported study of P-BCMA-101 T cells and will be followed for a total of 15 years post treatment from the last P-BCMA-101 treatment. Subjects will be monitored for safety and efficacy to assess the risk of delayed adverse events (AEs) and assess long-term efficacy, and PK and quantification of P-BCMA-101 T cells. Rimiducid may be administered as indicated.

Detailed Description
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Per Health Authorities guidelines for gene therapy medicinal products that utilize integrating vectors (FDA, 2006; Guidance for Industry, Gene Therapy Clinical Trials-Observing Subjects for Delayed Adverse Events), long term safety and efficacy follow up of treated subjects is required. Subjects are enrolled in this study following completion or early discontinuation from a Poseida sponsored or supported study of P-BCMA-101 T cells and will be followed for a total of 15 years post treatment from the last P-BCMA-101 treatment. Subjects will be monitored for safety and efficacy to assess the risk of delayed adverse events (AEs) and assess long-term efficacy, and PK and quantification of P-BCMA-101 T cells. Rimiducid may be administered as indicated. Study visits Subjects will only enter this protocol after completing or discontinuing from their primary P-BCMA-101 protocol. Once enrolled in this protocol a subject will return for regular follow-up depending on when they last received P-BCMA-101 on their primary protocol: Every 3 months until the end of the first year after P-BCMA-101 treatment Every 6 months until the end of the third year after P-BCMA-101 treatment Then yearly until the end of the 15th year after P-BCMA-101 treatment (ie. if a subject discontinues from their primary protocol 2 years after receiving P-BCMA-101, they will be entering this study at the beginning of the 3rd year, and will remain on this study for 13 years). Subjects will undergo serial assessment of safety, chemistry, hematology, and disease response as specified in the Schedule of Events. Subjects will further undergo a physical exam and medical history, and anti-myeloma medications, related AEs, new malignancies, new or exacerbated clinically significant neurologic, hematologic, rheumatologic or other autoimmune disorders will be recorded. After progressive disease (PD) has been confirmed for a subject after P-BCMA-101 administration, visits may be performed remotely (AEs collected by telephone and laboratory studies completed at a local facility).

Official Title
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Open Label, Multicenter, Long-Term Follow-Up Study for Subjects Treated With P-BCMA-101

Conditions
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Multiple Myeloma

Intervention / Treatment
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* Drug: Rimiducid may be administered as indicated


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects who have received P-BCMA-101 and completed or discontinued early from a Poseida sponsored treatment protocol. Subject has provided informed consent. Exclusion Criteria: None

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: P-BCMA-101 treated<br>Patients who received previous treatment with P-BCMA-101. Rimiducid may be administered as indicated. | Drug: Rimiducid may be administered as indicated<br>* Patients who received P-BCMA-101 in a previous trial will be evaluated in this trial for long term safety and efficacy. Rimiducid (safety switch activator) may be administered as indicated.<br>* Other names: Rimiducid (safety switch activator);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] | Incidence and severity of treatment-emergent adverse events to evaluate the long-term safety of P-BCMA-101 | Treatment with P-BCMA-101 through year 15 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Anti-myeloma effect of P-BCMA-101 (Response Rate) | Percentage of patients with complete response (CR), very good partial response (VGPR), or partial response (PR) according to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma | Treatment with P-BCMA-101 through year 15 | 
| Anti-myeloma effect of P-BCMA-101 (Time to Response) | Time from P-BCMA-101 administration to time of first documented response (PR or better) according to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma. | Treatment with P-BCMA-101 through year 15 | 
| Anti-myeloma effect of P-BCMA-101 (Duration of Response) | Time from complete response (CR), very good partial response (VGPR), or partial response (PR) to progressive disease according to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma. | Treatment with P-BCMA-101 through year 15 | 
| Anti-myeloma effect of P-BCMA-101 (Progression Free Survival) | Time from P-BCMA-101 treatment to progressive disease according to the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma, or death | Treatment with P-BCMA-101 through year 15 | 
| Anti-myeloma effect of P-BCMA-101 (Overall Survival) | Duration of survival from time of treatment with P-BCMA-101 | Treatment with P-BCMA-101 through year 15 | 
| Concentration of P-BCMA-101 cells | Concentration of P-BCMA-101 cells in blood and bone marrow over time | Treatment with P-BCMA-101 through year 15 | 
| Biomarkers for P-BCMA-101 (BCMA Cells) | The persistence of anti-tumor effect of P-BCMA-101 and its relationship to persistence of P-BCMA-101 cells, BCMA tumor surface expression and circulating BCMA. | Treatment with P-BCMA-101 through year 15 | 
| Biomarkers for P-BCMA-101 (Cell Count) | Absolute B and T lymphocyte count | Treatment with P-BCMA-101 through year 15 | 
| Biomarkers for P-BCMA-101 (Expansion) | Expansion and/or persistence of P-BCMA-101 T cells | Treatment with P-BCMA-101 through year 15 | 
| Incidence of adverse events related to rimiducid, if indicated | Incidence of adverse events related to rimiducid, if indicated | Rimiducid infusion through Year 15 after P-BCMA-101 infusion, if applicable | 
| Effect of rimiducid on grade of P-BCMA-101-related adverse events | Effect of rimiducid on grade of P-BCMA-101-related adverse events as assessed by CTCAE v4.03, if indicated. | Rimiducid infusion through Year 15 after P-BCMA-101 infusion, if applicable | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CAR-T cells