A phase I clinical trial evaluating the safety of allogeneic Adipose Tissue-derived multilineage progenitor cells-transplantation therapy in severe familial hypercholesterolemia patients

Phase 1

• Conditions: Severe familial hypercholesterolemia (mainly homozygotes) undergoing LDL-apheresis

• Registration Number: JPRN-UMIN000020591
• Lead Sponsor: Osaka University Graduate School of Medicine

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-01-16
• Study Completion: 2018-07-26
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Complete: follow-up complete
• Sex: All
• Target Recruitment: 4

Inclusion Criteria

Not provided

Exclusion Criteria

The patients corresponding to any of the following criteria are excluded from the study. 1) The patients who have or are suspected to have a malignant tumor or a precancerous lesion; or have the past history of those within five years 2) The patients who have a coronary disease or cerebrovascular disorder, which require any treatment. 3) The patients who have renal dysfunction, liver dysfunction and blood disorders. 4) The patient who have diabetes mellitus or have more than 6.5% HbA1c. 5) The patient who have an active infectious disease. 6) The patient who have any of infectious disease such as HBV (carrier and the existing infected patient), HCV, HTLV-I (ATLA), HIV, parvoviral B19, Treponema pallidum, gonococcus, tuberculosis, cytomegalovirus, EB virus, one of waist e viruses. 7) The patient who requires to have any treatment (surgical care) affecting the liver function test during the study 8) The patient Person with the communicable spongy encephalopathic and doubt and dementia 9) The pregnant patient, the possible pregnant patient or the patient who wishes to have pregnancy during a study 10) The patient who gives breast-feeding 11) The patient who has the past of an alcoholism or the drug dependency within six months before registration 12) The patient who are diagnosed by the doctor operating this study not to be able to recognize the significance of the clinical study from a mental disease. 13) Others, the patient who are diagnosed not to be eligible to be enrolled into this study by the doctor.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Safety evaluation 1) Systemic adverse events (1) Allergic reaction, rejection reaction and thrombosis by cell-transplantation Fever, eosinophilia, and abnormality of PT-INR, D-Dimer, AST, ALT (2) Complications in the blood vessel by cell-transplantation Bleeding, presence and severity of embolism 2) Adverse events of the puncture site Hematoma, presence and disease severity of the infectious disease 3) Other adverse events Presence, name, type, disease severity, frequency and period of adverse events are evaluated. 4) Other safety evaluations Vital signs (blood pressure, heart rate, body temperature), electrocardiograms, blood test and urinalysis are evaluated.