Transthyretin (TTR) a 127-amino acid, tetrameric protein, primarily synthesized in the liver, is a secreted protein present in the blood and cerebrospinal fluid and is a carrier of thyroxine and retinol-binding protein-retinol (vitamin A) complex. Transthyretinassociated amyloidoses are diseases caused by dissociation of the transthyretin tetramer into monomers, which misfold, ultimately forming amyloid deposits in various organs.
Conditions
Interventions
Trade Name: Not applicable. No drug used.
Pharmaceutical Form:
Sponsors
Fold RX Pharmaceuticals, a Pfizer Company
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subject has symptomatic ATTR disease with documented Val30Met, Glu89Gln, Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, or Val122Ile mutation in the TTR gene. 2. Must be able to be a participant in the THAOS registry and are either currently XML File Identifier: HMmrmo7rsZGmYHxB4NUacap1qIE= Page 7/14 21/01/2014 enrolled or will be enrolled in THAOS prior to entry into this substudy. 3. Subject provides written informed consent to participate in this substudy. Subject must have already provided written informed consent to participate in the THAOS registry. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 30 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 15
Exclusion criteria
Exclusion criteria: 1. Subject has a diagnosis of primary or secondary amyloidosis. 2. Subject has a documented mutation in the TTR gene other than Val30Met, Glu89Gln, Phe64Leu, Thr60Ala, Ser50Arg, Ile107Val, Val20Ile, Ile68Leu, or Val122Ile. 3. Subject has received a liver transplant. 4. Subject does not have symptomatic ATTR disease. 5. Subject has received treatment with tafamidis prior to entry into this substudy. 6. Subjects using non-steroidal anti-inflammatory drugs (NSAIDs) that are not permitted in the protocol within 2 weeks prior to blood collection. ? The following NSAIDs are permitted: acetylsalicylic acid, etodolac, ibuprofen, indomethicin, ketoprofen, nabumetone, naproxen, nimesulide, piroxicam, and sulindac. 7. Subject received an investigational drug in another clinical investigational study within 30 days (or as determine by the local requirements, whichever is longer) or 5 half-lives prior to substudy enrollment and blood collection. 8. Participation in other studies (other than the THAOS survey) that include medication treatment within 6 months prior to substudy enrollment and blood collection. 9. Subjects who are investigational site staff members directly involved in the conduct of the trial and their family members, site staff members otherwise supervised by the Investigator, or subjects who are Pfizer employees directly involved in the conduct
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objective of this study is to collect blood samples which will be used to assist in development and validation of a biomarker assay for TTR amyloidoses.;Secondary Objective: Not Applicable;Primary end point(s): The objective of this study is to collect blood samples which will be used to assist in development and validation of a biomarker assay for TTR amyloidoses.;Timepoint(s) of evaluation of this end point: Samples will be retained until they are no longer needed for the development and validation of a biomarker assay of TTR. | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Not applicable;Secondary end point(s): Not applicable | — |
Countries
Argentina, Brazil, Italy, United States
Contacts
Public ContactClinician
Pfizer, Inc
Outcome results
None listed