The 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

Phase 1

Recruiting

• Conditions: Familial LCAT deficiency; Corneal opacity, Renal dysfunction, Hemolytic anemia; D007863

• Registration Number: JPRN-jRCT2033200096
• Lead Sponsor: Yokote Koutaro

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-08-25
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 3

Inclusion Criteria

Patients who meet all of the following conditions.
1)Patients with a definitive diagnosis of familial LCAT deficiency based on the following diagnostic criteria
(1) LCAT gene mutation has been confirmed in genetic diagnosis (having amino acid residue substitution in LCAT protein)
(2) Low HDL-cholesterol (less than 25 mg/dL) is observed, and one or some of following complications typical for this disease is detected; corneal opacification, renal dysfunction (proteinuria), hemolytic anemia
(3) Blood LCAT activity (artificial substrate method) is below the detection limit (reference standard value 382-512U)
2)Patients older than 16.
3)Patient who provides written informed consent. If the patient is a minor, written consent must be obtained from the patient as well as a parent or guardian.

Exclusion Criteria

Patients who meet any of the following conditions are not included.
1)Patients who are not expected to express full-length LCAT protein due to frameshift or the appearance of stop codon by insertion/deletion mutation in LCAT gene
2)Patients whose mutant LCAT protein cannot be detected in blood by immunological method (ELISA or immunoprecipitation western blot)
3)Patients with advanced liver disease (fulminant hepatitis, cirrhosis, etc.) that affects lipid metabolism
4)Patients with advanced renal disease (transferred or planned to transfer to dialysis)
5)Patients who received LCAT protein replacement therapy by whole blood or plasma transfusion within 1 month before obtaining written consent.
6)Patients who are considered to be difficult to perform subcutaneous liposuction
7)Pregnant and lactating women. In the case of female patients who can become pregnant (all women except those who have undergone permanent contraception or those who have been postmenopausal*), those who do not agree to contraception during the clinical trial period (up to 24 weeks after administration). In addition, in the case of male patients, patients who do not agree to contraception during the clinical trial period (up to 24 weeks after administration).
8)Patients with other similar diseases that are not caused by LCAT deficiency and have low HDL-cholesterol (Apo A-1 abnormality/Tangier disease)
9)Patients with a history of hypersensitivity to the components of this clinical trial product or the components (fetal bovine serum and human albumin) used in the manufacturing process of this clinical trial product.
10)Other patients who are deemed unsuitable by the investigator or the sub-investigator

Study & Design

• Study Type: Interventional
• Study Design: Not specified