Long-term Follow-up Study of Lentiviral-based Gene-edited Immune Cell Therapy

Sponsor
Pell Bio-Med Technology Co., Ltd. (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05377307
Collaborator
(none)
49
3
180
16.3
0.1

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
  • Genetic: Pell's lentiviral-based gene-edited immune cell therapy

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

Study Type:
Observational
Anticipated Enrollment :
49 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Long-term Follow-up Study to Evaluate the Safety and Efficacy in Patients Who Have Ever Received Lentiviral-based Gene-edited Immune Cell Therapy
Anticipated Study Start Date :
Sep 1, 2022
Anticipated Primary Completion Date :
Sep 1, 2037
Anticipated Study Completion Date :
Sep 1, 2037

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

  1. 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

  1. 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

  2. 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

  3. 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

  4. 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

Ages Eligible for Study:
N/A and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
  1. Patients must have ever received Pell's lentiviral-based gene-edited immune cell as monotherapy or as combination therapy in clinical trials.

  2. The last lentiviral-based gene-edited immune cell infusion within 15 years.

  3. 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.
Responsible Party:
Pell Bio-Med Technology Co., Ltd.
ClinicalTrials.gov Identifier:
NCT05377307
Other Study ID Numbers:
  • PLLV-LTFU-401
First Posted:
May 17, 2022
Last Update Posted:
May 17, 2022
Last Verified:
May 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Pell Bio-Med Technology Co., Ltd.
Additional relevant MeSH terms:

Study Results

No Results Posted as of May 17, 2022