Transthyretin Amyloidosis (ATTR)
Conditions
Interventions
Treatment group:ART001 infusion therapy
Sponsors
Suzhou Dushu Lake Hospital
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Inclusion criteria
Inclusion criteria: 1. Male or female ; 2. At the time of signing the informed consent form, the age range was 18 to 80 years old (inclusive of the cutoff) 3. Diagnosis of TTR amyloidosis (ATTR) based on all of the following: a Documented TTR mutation (i.e., whole TTR gene sequencing information). b Documented tissue deposition of TTR amyloid by biopsy or by validated noninvasive imaging. 4. Subjects must meet the following laboratory criteria during Screening: a. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), total bilirubin (see exception for Gilbert`s Syndrome below), and international normalized ratio (INR) = upper limit of normal (ULN) range at Screening. b. Estimated glomerular filtration rate (GFR) > 45 mL/min/1.73m2 as measured by the Modification of Diet in Renal Disease equation at Screening. c. Platelet count = ULN; d. Normal activated partial thromboplastin time (aPTT), prothrombin time (PT), thrombin generation time (TGT), fibrinogen and d-dimer levels. e. N-terminal prohormone of brain natriuretic peptide (NT-proBNP) < 2,000 pg/mL. 5. Body weight of subject must between 45Kg to 90Kg at screening. 6. Female subjects should meet one of the following criteria: a. Postmenopausal women (defined as no menstruation for at least 12 consecutive months prior to screening with no other medical reasons). b. Underwent sterilization surgery at least 1 month before screening (including procedures such as hysterectomy, bilateral salpingectomy, and bilateral oophorectomy). 7. Male subjects with partners of child-bearing potential or who are pregnant must agree to using a condom prior to Screening and for 180 days after study drug administration.Male subjects must agree not to donate sperm for 180 days after study drug administration. 8. Must understand this study and sign the Informed Consent Form (ICF).
Exclusion criteria
Exclusion criteria: 1. Amyloidosis attributable to non-TTR protein, e.g., amyloid light-chain (AL) amyloidosis. 2. History of Hepatitis B or C infection or positive Hepatitis B surface antigen (HBsAg) or Hepatitis C Virus antibody (HCV Ab) test at Screening.History of positive human immunodeficiency virus (HIV) status. 3. Females of child-bearing potential or who are breastfeeding. 4. Use of any of the following TTR-directed therapy for ATTR within the specified timeframe: Patisiran (small interfering ribonucleic acid [siRNA] therapeutic formulated LNP),prior history of use. Inotersen (antisense oligonucleotide [ASO)]),prior history of use. Vutrisiran (investigational siRNA therapeutic GalNAc conjugate): prior history of use. Tafamidis (TTR stabilizer): last dose administered less than 14 days prior to study drug administration. Diflunisal (TTR stabilizer): last dose administered less than 3 days prior to study drug dosing. Doxycycline and/or tauroursodeoxycholic acid (TTR matrix solvent): last dose administered less than 14 days of study drug dosing. Any other investigational agent for the treatment of ATTR: last dose administered less than 30 days or 5 half-lives, whichever is longer, prior to study drug dosing; 5. Anticipated survival is less than 2 years, in the opinion of the Investigator; 6. Unwilling to comply with study procedures including follow-up as specified by the protocol or unwilling to cooperate fully with the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| TTR protein(prealbumin); | — |
Secondary
| Measure | Time frame |
|---|---|
| Blood routine;Blood biochemical;Coagulation parameter;Urinalysis;Thyroid Function Tests;CRP;Complement(C3,C4);Cytokine(IFN-?,TNF-a,IL-2,IL-4,IL-6,IL-10,MCP-1);Vital Signs;Physical Examination; | — |
Countries
China
Contacts
Public ContactYonggang Hao
Suzhou Dushu Lake Hospital
Outcome results
None listed