Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045.

Polyneuropathy Clinical Trial

— TRAMA  

Official title:

Tafamidis 61mg, Resultados en Amiloidosis ATTR Con afectación neurológica y Multisistémica - TRAMA

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05560555
• Other study ID #: B3461104
• Secondary ID: TRAMA
• Status: Completed
• Start date: October 24, 2022
• Est. completion date: November 15, 2022

Study information

• Verified date: December 2022
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

A study of patients with hereditary transthyretin amyloidosis (ATTRv) and wild-type transthyretin amyloidosis (ATTRwt) that have been enrolled in B3461028 and B3461045 studies in Spain - exposed to tafamidis 61mg for ≥12 months with polyneuropathy (PN) have kept going to their multisystemic follow-ups (neuro/ophtalmo/gastrointestinal) ≥12 months.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 5
• Est. completion date: November 15, 2022
• Est. primary completion date: November 15, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 99 Years

Eligibility

Inclusion Criteria:

• Treatment with tafamidis 61 mg = 12 months
• Neurological follow up = 12 months
• Diagnosis of transthyretin amyloidosis with polyneuropayhy (ATTR-PN) based on one of

the following:

• Amplitude reduction in, at least, 2 nerves under normal value, excluding median nerve OR 50% amplitude reduction in, at least, 2 nerves on the basal value of the patient, excluding median nerve OR 2 abnormal tests detecting thin fibers alterations (through Sudoscan, RR Interval análisis, etc..)

Exclusion Criteria:

• Treatment with tafamidis 61 mg < 12 months
• Neurological follow up < 12 months
• Other diagnosis for polyneuropathy

Related Conditions & MeSH terms

• Amyloid Neuropathies, Familial
• Amyloidosis
• Polyneuropathies
• Polyneuropathy

Intervention

Drug:
• Tafamidis
61 milligrams (mg) as received in studies B3461028 and B3461045

Locations

Country	Name	City	State
Spain	Hospital Universitari de Bellvitge	Barcelona
Spain	Hospital Clinico San Carlos	Madrid
Spain	Hospital Son Llatzer	Palma de Mallorca

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from Baseline in Neurological Impairment score (NIS)		Baseline, Month 12
Secondary	To describe extra-cardiac clinical features and clinical management of the Spanish ATTRv and ATTRwt patients with mixed phenotype treated with tafamidis 61 mg.		Baseline through month 36
Secondary	Change From Baseline in Neuropathy Impairment Score - Lower Limbs [NIS (LL)		Baseline through Month 36
Secondary	Change from Baseline in Norfolk Quality of Life- Diabetic Neuropathy (Norfolk QOL-DN)		Baseline through month 36
Secondary	Change From Baseline in composite autonomic symptom scale 31 (Compass-31)		Baseline through month 36
Secondary	Change from Baseline in Modified Body Mass Index (mBMI)		Baseline through month 36
Secondary	Change from Baseline in Sudomotor Response		Baseline through month 36

External Links

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