The long-term clinical study to evaluate the safety of the regenerative medicine and the gene therapy by the autologous transplantation of human LCAT gene transduced human pre-adipocyte

JRCT ID: jRCT2033240371

Registered date:03/10/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Familial LCAT deficiency
• Date of first enrollment: 03/10/2024
• Target sample size: 3
• Study type: Interventional
• Intervention(s): none (Evaluate long term safety after administration of the study product in a previous phase of the trial)

Outcome(s)

Primary Outcome

Occurrence of adverse events which cannot be ruled out in relation to the characteristics of this clinical trial product (human preadipocytes which the LCAT gene has been introduced by ex vivo retroviral vector)
(1) Appearance of RCR after administration of clinical trial products
(2) Tumorigenesis of the administration site after administration of the clinical trial product (complementarily evaluated by the following 2 items)
(A) Malignancy test (TK activity)
(B) MRI examination
(3) Appearance of anti-LCAT antibody after administration of clinical trial products
(4) Occurrence of unexpected serious adverse events other than (1) to (3)

The presence or absence of RCR will be evaluated for (1) and changes in MRI images will be evaluated for (2).
(3) The presence or absence of tumor development will be evaluated by making a comprehensive judgment based on the MRI images and the findings related to the safety evaluation of the 24-week clinical trial. For (3), laboratory values will be compared before and after administration of the investigational product to evaluate whether or not anti-LCAT antibodies are expressed.

Secondary Outcome

Secondary evaluation items related to safety:
(1) Type, frequency and severity of adverse events other than the main endpoints that occurred after obtaining consent
(2) Anti-FBS antibody
For (1), the presence or absence of abnormal findings at the lipectomy site will be included. For (2), laboratory values before and after administration of the study product will be compared, and the presence or absence of the expression of each antibody will be evaluated.

Secondary evaluation items related to efficacy:
Changes in blood biochemical test values are evaluated during the observation period.
(1) LCAT activity (artificial substrate method, self-substrate method, high-sensitivity activity measurement method)
(2) Blood LCAT concentration (ELISA)
(3) Cholesterylester/Total cholesterol ratio
(4) HDL-cholesterol
(5) Total-cholesterol
(6) LDL-cholesterol

Key inclusion & exclusion criteria

• Age minimum: >= 16age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1) Patients who participated in a 24-week clinical trial conducted prior to this study and were administered the investigational product
2) Patients who have obtained written consent from the patient or, in the case of a minor, written consent from the patient and the patient's surrogate for the initiation of the clinical trial

• Exclude criteria: 1) Patients deemed inappropriate by the investigator or subinvestigator

Related Information

• Primary Sponsor: Yokote Kotoro
• Secondary Sponsor: CellGenTech, Inc.