The purpose of this substudy is to describe the collection of bloodsamples to be used for development and validation of a biomarker assay.

• Conditions: Transthyretin (TTR) a 127-amino acid, tetrameric protein, primarilysynthesized 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. Transthyretinassociatedamyloidoses are diseases caused by dissociation of the transthyretintetramer into monomers, which misfold, ultimately forming amyloid deposits in various organs.; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2013-004090-28-IT
• Lead Sponsor: Fold RX Pharmaceuticals, a Pfizer Company

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-03-31
• Last Update Posted: 11 April 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 45

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
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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

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified