Severe familial hypercholesterolemia (mainly homozygotes) undergoing LDL-apheresis
Conditions
Interventions
Allogeneic adipose tissue-derived multi-lineage progenitor cells are isolated from donors and cultured. Total 1.6x10^5/kg-1x10^7/kg cells in the heparin-added lactated Ringer aree transplanted into li
Sponsors
Osaka University Graduate School of Medicine
Osaka University Hospital Medical Center for Translational Research
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: The patient who meets the following criteria is enrolled. 1) The patient diagnosed with familial hypercholesterolemia (based on the diagnostic criteria of the Japanese arteriosclerosis society) 2) The patient who undergoes LDL-apheresis 3) The patient who is older than or equal to 20 years old and younger than 80 years when obtained informed consents are obtained 4) The patient who can understand the contents of the study, have the ability for agreement and write a written agreement The patient whose relative (blood relationships in the sixth degree), spouse or relative (by marriage in the third degree) can meet the eligibility criteria of the cell donor and agree to become a cell donor with a written agreement.
Exclusion criteria
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 Nile 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end-point about the safety 1) Safety evaluation about the immunosuppressive drug use Correlation of dose immunosuppressive drug and renal function, blood sugar level, and affection of the infectious disease are evaluated 2) Evaluation of transplanted ADMPC in the systemic circulation The STR-PCR is examined in the systemic circulation before and after transplantation. Secondary end-point about the efficacy 1) Decrease in LDL-C, changes in serum lipid (T-cho, HDL-C, TG, Lp(a)) (1) The rate of change in LDL-C (actual value), LDL-C(Friedewald formula) among 8-11weeks, 13-16 weeks and 17-20 weeks after transplantation (2) The change over time in LDL-C (actual value), LDL-C(Friedewald formula), T-cho, HDL-C, TG and Lp(a) (3) The association with reascension of LDL-C (actual value) and LDL-C (Friedewald formula) after LDL-apheresis and the transplanted cell counts is examined. (4) The association with the change of LDL-C (actual value) and LDL-C (Friedewald formula) in 8-11 weeks, 13-16 weeks and 17-20 weeks and the transplanted cell counts is examined. 2) Secession possibility of LDL-apheresis 3) The association of the background of donor (age, sex and weight) and the quantity of collected adipose tissue is evaluated. | — |
Countries
Japan
Contacts
Public ContactMasahiro Koseki
Osaka University Graduate School of Medicine Cardiology
Outcome results
None listed