Long-term Follow-up Study of Lentiviral-based Gene-edited Immune Cell Therapy
Study Details
Study Description
Brief Summary
According to health authorities guidances (FDA 2006, EMA(European Medicines Agency) 2009) for gene therapy clinical trials, observing subjects for delayed adverse events for 15 years is recommended. This purpose of this long-term follow-up study is to evaluate the safety and efficacy in patients who have ever received lentiviral-based gene-edited immune cells which are manufactured by Pell Bio-Med Technology Co. Ltd.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Detailed Description
After completion or early withdraw from the other treatment protocol, patients should be enrolled into this long-term follow-up study. If patients do not enter this study right after leaving the treatment protocol, they may have the option to enter this long-term follow-up study at any time within 15 years after the last lentiviral-based gene-edited immune cell infusion.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Group A After completion or early withdraw from the treatment protocol, patients will be enrolled into this long-term follow-up study. If patients do not enter this study right after leaving the treatment protocol, they may have the option to enter this study at any time within 15 years after the last lentiviral-based gene-edited immune cell infusion. |
Genetic: Pell's lentiviral-based gene-edited immune cell therapy
No study drug or other planned treatment will be administered. Subjects who previously received Pell's lentiviral-based gene-edited immune cell therapy will be evaluated the safety and efficacy.
|
| Group B Some patients may require joining other Pell's gene-edited immune cell therapy study during participating in this long-term follow-up study. For such case, the patient could be enrolled into the new treatment protocol. Meanwhile, the patient can be remaining in this long-term follow-up protocol as an inactive participant. |
Genetic: Pell's lentiviral-based gene-edited immune cell therapy
No study drug or other planned treatment will be administered. Subjects who previously received Pell's lentiviral-based gene-edited immune cell therapy will be evaluated the safety and efficacy.
|
Outcome Measures
Primary Outcome Measures
- To assess delayed adverse events which are suspected related to previous gene-edited immune cell therapy [15 years]
• Proportion of patients with any events of the following items which are suspected related to previous gene-edited immune cell therapy. New malignancies New incidence or exacerbation of a pre-existing neurologic disorder New incidence or exacerbation of a prior rheumatologic or other autoimmune disorder New incidence of a hematologic disorder, including hypogammaglobulinemia New incidence of infection (potentially product-related) Other than the above adverse events, which are suspected related to gene-edited immune cell therapy judged by the investigator
Secondary Outcome Measures
- Monitor for Replication Competent of Lentivirus (RCL) [15 years]
Proportion of patients with detectable RCL in peripheral blood by VSV-G(Vesicular stomatitis virus G) qPCR
- Monitor the persistence of gene-edited immune cells in peripheral blood(By qPCR) [15 years]
Proportion of patients with detectable transgene level in peripheral blood by qPCR
- Monitor the persistence of gene-edited immune cells in peripheral blood(By Flowcytometry) [5 years]
Persistence of gene-edited immune cells in peripheral blood using flow cytometry
- To assess the long-term efficacy of gene-edited immune cells [15 years]
Proportion of patients with relapse or progress among patients who didn't progress or relapse at study entry/reentry Incidence of death
Eligibility Criteria
Criteria
Inclusion Criteria:
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Patients must have ever received Pell's lentiviral-based gene-edited immune cell as monotherapy or as combination therapy in clinical trials.
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The last lentiviral-based gene-edited immune cell infusion within 15 years.
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Patient/patient's parent/legal guardian is capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Exclusion Criteria:
There are no specific exclusion criteria for this study.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Kaohsiung Medical University Chung-Ho Memorial Hospital | Kaohsiung | Taiwan | 807377 | |
| 2 | National Taiwan University Hospital | Taipei | Taiwan | 10025 | |
| 3 | Taipei Veterans General Hospital | Taipei | Taiwan | 112201 |
Sponsors and Collaborators
- Pell Bio-Med Technology Co., Ltd.
Investigators
- Study Director: Chien-Ting Lin, MD, Pell Bio-Med Technology Co., Ltd.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- PLLV-LTFU-401