Study type

Study topic

Human medicinal product
Disease /health condition

Study type

Non-interventional study

Scope of the study

Disease epidemiology

Data collection methods

Secondary use of data
Non-interventional study

Non-interventional study design

Other

Non-interventional study design, other

Descriptive analysis of a sub-set of THAOS - a multi-center longitudinal, observational, non-interventional survey
Study drug and medical condition

Study drug International non-proprietary name (INN) or common name

TAFAMIDIS MEGLUMINE

Medical condition to be studied

Hereditary neuropathic amyloidosis
Population studied

Short description of the study population

The study population for the described analyses will be the sub-set of patients enrolled in the THAOS registry who have a non-V30M mutation and have at least
12 months of pre-treatment data prior to receiving tafamidis. The data from all non-V30M patients enrolled in THAOS that are prescribed tafamidis will be evaluated at the time of starting tafamidis. Those that have approximately 12 months or more of pre-treatment data and a modified polyneuropathy disability (mPND) score <3 at the start of the treatment period will be included in the cohort.

Patients must meet all of the following inclusion criteria to be eligible for inclusion in the analysis cohort:
1. Patient has symptomatic peripheral and/or autonomic neuropathy with documented non-V30M mutation.
2. Must be a participant in the THAOS registry.
3. Patient must have already provided written informed consent to participate in the THAOS registry
4. Patient must be prescribed tafamidis and have >12 months of pre-treatment data in the THAOS registry. Given the non-interventional nature of this study, patient visit schedules are according to standard of care and timing of visits will vary. Although the intent is that the pre-treatment data period will be >12 months, a minimum of 9 months will be permitted.
5. Patient must have a visit recorded in the THAOS registry within 3 weeks prior to, or 3 week after starting tafamidis (the start of treatment visit).
6. Patient must have an mPND score of <3 (does not require assistance with ambulation) at the start of tafamidis treatment.


Patients meeting any of the following criteria will not be included in the analysis cohort:
1. Patient has a documented V30M mutation.
2. Patient has received a liver transplant.
3. Patient does not have symptomatic TTR-FAP.
4. Patient has chronically (>3-4 times a month) used non-steroidal anti-inflammatory drugs (NSAIDs), other than acetylsalicylic acid, etodolac, ibuprofen, indomethicin, ketoprofen, nabumetone, naproxen, nimesulide, piroxicam, and sulindac.
5. Patient received an investigational drug, other than tafamidis, in another clinical investigational study.

Age groups

Adults (18 to < 46 years)
Adults (46 to < 65 years)
Adults (65 to < 75 years)
Adults (75 to < 85 years)
Adults (85 years and over)

Estimated number of subjects

50
Study design details

Main study objective

compare disease progression over at least 12 months of pre‑treatment (patients will receive standard of care according to their treating physician) with disease progression over 12 months of tafamidis administration in symptomatic TTR-FAP patients with non‑V30M mutations

Outcomes

disease progression, effect of tafamidis on disease progression, evaluate safety of tafamidis in TTR-FAP

Data analysis plan

Conclusions regarding the effects of tafamidis will be made based on a preponderance of evidence, including consistency and directionality, across all the endpoints. Descriptive statistics will be provided for each period (the standard of care period and the tafamidis treatment period) for each neuropathy endpoint.