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Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis

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: NCT00925002
Recruitment Status : Active, not recruiting
First Posted : June 19, 2009
Last Update Posted : April 28, 2020
Sponsor:
Information provided by (Responsible Party):
Pfizer

Brief Summary:
This is a Phase 3, open-label study designed to obtain additional long-term safety and efficacy data for oral tafamidis (20 mg soft gelatin capsule) administered once daily (QD). In addition, this study continued to provide tafamidis to Val30Met subjects who had completed Protocol Fx-006 (a 1-year, open-label extension study to Protocol Fx-005 which was a randomized, double-blind, placebo-controlled, 18-month study to evaluate the safety and efficacy of tafamidis) or non-Val30Met subjects who had completed Protocol Fx1A-201 (a Phase 2, open-label study to evaluate TTR stabilization, safety, and tolerability of tafamidis) for up to 10 years or until subjects had access to tafamidis for ATTR-PN via prescription. Upon regulatory approval for the treatment of ATTR-PN in their respective country and access to prescription tafamidis, subjects may have been withdrawn from the study. Such subjects were considered study completers.

Condition or disease Intervention/treatment Phase
ATTR-PN Drug: Tafamidis Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 93 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: OPEN-LABEL SAFETY AND EFFICACY EVALUATION OF FX-1006A IN SUBJECTS WITH TRANSTHYRETIN (TTR) AMYLOIDOSIS
Actual Study Start Date : August 5, 2009
Estimated Primary Completion Date : May 30, 2020
Estimated Study Completion Date : May 30, 2020


Arm Intervention/treatment
Active Comparator: Open-Label Drug: Tafamidis
20 mg oral Fx-1006A daily




Primary Outcome Measures :
  1. Percentage of patients with a change from baseline in Neuropathy Impairment Score (NIS) [ Time Frame: Baseline up to 10 years ]
  2. Percentage of patients with a change from baseline in Total Quality of Life (TQOL) score [ Time Frame: Baseline up to 10 years ]
  3. Number or Percentage of patients with a change from baseline in Karnofsky Performance Scale Index [ Time Frame: Baseline up to 10 years ]
  4. Percentage of patients with a change in subject ambulation as measured by modified Polyneuropathy Disability (mPND) score [ Time Frame: Baseline up to 10 years ]

Secondary Outcome Measures :
  1. Incidence of treatment emergent adverse events from baseline through 10 years [ Time Frame: Baseline up to 10 years ]
  2. Number or percentage of patients with change from baseline in Clinical Laboratory parameters [ Time Frame: Baseline up to 10 years ]
  3. Number of patients with change in ECG parameters [ Time Frame: Baseline up to 10 years ]
  4. Number or percentage of patients with change from baseline in Vital sign measurements [ Time Frame: Baseline up to 10 years ]
  5. Descriptive summary of physical examination findings for patients through 10 years [ Time Frame: Baseline up to 10 years ]
  6. Descriptive summary of concomitant medication use for patients through 10 years [ Time Frame: Baseline up to 10 years ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion criteria:

  • Subject had successfully completed either Protocol Fx-006 or Fx-1A-201.
  • Male or female subjects with ATTR-PN who had not undergone liver or heart transplantation at time of enrollment.
  • If female, subject was post-menopausal, surgically sterilized, or willing to use an acceptable method of birth control

Key Exclusion criteria:

  • Chronic use of non-protocol approved non-steroidal anti-inflammatory drugs (NSAIDs)
  • Pregnant or breast feeding female subjects.
  • Clinically significant medical condition that, in the opinion of the investigator, would place the subject at an increased risk to participate in the study.
  • An alanine aminotransferase (ALT) and aspartate aminotransferase (AST) value >3 × upper limit of normal (ULN) that, in the medical judgment of the investigator, was due to reduced liver function or active liver disease.
  • Sexually active males with partners of childbearing potential not using highly effective contraception or not agreeing to continue highly effective contraception for at least 3 months after last dose of study drug.

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): NCT00925002


Locations
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United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Argentina
FLENI
Ciudad Autonoma de Buenos aires, Argentina, C1428AQK
Brazil
Hospital Universitário Clementino Fraga Filho -HUCFF Universidade Federal do Rio de Janeiro
Rio de Janeiro, RJ, Brazil, CEP 21941-913
France
CHU Henri Mondor
Creteil cedex, France, 94010
Germany
Universitatsklinikum Muenster
Muenster, Germany, 48149
Italy
Centro per lo Studio e la Cura delle Amiloidosi Sistemiche IRCCS - Policlinico San Matteo
Pavia, Italy, 27100
Dipartimento di Neurofisiopatologia
Pavia, Italy, 27100
Fondazione IRCCS Policlinico San Matteo
Pavia, Italy, 27100
IRCCS
Pavia, Italy, 27100
Portugal
Centro Hospitalar Lisboa Norte, EPE- Hospital de Santa Maria
Lisboa, Portugal, 1649-028
Unidade Clinica de Paramiloidose Centro Hospitalar do Porto, EPE - Hospital Geral de Santo António
Porto, Portugal, 4099-001
Sweden
Norrlands University Hospital
Umea, Sweden, 90185
Umea University Hospital
Umea, Sweden, 90187
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00925002    
Other Study ID Numbers: FX1A-303
B3461023 ( Other Identifier: Alias Study Number )
2009-011535-12 ( EudraCT Number )
First Posted: June 19, 2009    Key Record Dates
Last Update Posted: April 28, 2020
Last Verified: April 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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.
URL: 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: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases