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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05560555
Recruitment Status : Completed
First Posted : September 29, 2022
Last Update Posted : December 27, 2022
Sponsor:
Information provided by (Responsible Party):
Pfizer

Brief 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.

Condition or disease Intervention/treatment
Hereditary Transthyretin Amyloidosis (ATTRv) Polyneuropathy Drug: Tafamidis

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Study Type : Observational
Actual Enrollment : 5 participants
Observational Model: Other
Time Perspective: Retrospective
Official Title: Tafamidis 61mg, Resultados en Amiloidosis ATTR Con afectación neurológica y Multisistémica - TRAMA
Actual Study Start Date : October 24, 2022
Actual Primary Completion Date : November 15, 2022
Actual Study Completion Date : November 15, 2022


Group/Cohort Intervention/treatment
Retrospective cohort ATTRv and ATTRwt patients enrolled in B3461028 and B3461045 studies in Spain Drug: Tafamidis
61 milligrams (mg) as received in studies B3461028 and B3461045




Primary Outcome Measures :
  1. Change from Baseline in Neurological Impairment score (NIS) [ Time Frame: Baseline, Month 12 ]

Secondary Outcome Measures :
  1. To describe extra-cardiac clinical features and clinical management of the Spanish ATTRv and ATTRwt patients with mixed phenotype treated with tafamidis 61 mg. [ Time Frame: Baseline through month 36 ]
  2. Change From Baseline in Neuropathy Impairment Score - Lower Limbs [NIS (LL) [ Time Frame: Baseline through Month 36 ]
  3. Change from Baseline in Norfolk Quality of Life- Diabetic Neuropathy (Norfolk QOL-DN) [ Time Frame: Baseline through month 36 ]
  4. Change From Baseline in composite autonomic symptom scale 31 (Compass-31) [ Time Frame: Baseline through month 36 ]
  5. Change from Baseline in Modified Body Mass Index (mBMI) [ Time Frame: Baseline through month 36 ]
  6. Change from Baseline in Sudomotor Response [ Time Frame: Baseline through month 36 ]


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Ages Eligible for Study:   18 Years to 99 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Study population: retrospective cohort 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 have kept going to their multisystemic follow-ups (neuro/ophtalmo/gastrointestinal) ≥12 months
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05560555


Locations
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Spain
Hospital Universitari de Bellvitge
Barcelona, Spain, 08907
Hospital Clinico San Carlos
Madrid, Spain, 28040
Hospital Son Llatzer
Palma de Mallorca, Spain, 07198
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
Additional Information:
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT05560555    
Other Study ID Numbers: B3461104
TRAMA ( Other Identifier: Alias Study Number )
First Posted: September 29, 2022    Key Record Dates
Last Update Posted: December 27, 2022
Last Verified: December 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Polyneuropathies
Amyloid Neuropathies, Familial
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Amyloid Neuropathies
Genetic Diseases, Inborn
Amyloidosis, Familial
Metabolism, Inborn Errors