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Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)

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: NCT02146378
Recruitment Status : Active, not recruiting
First Posted : May 23, 2014
Last Update Posted : July 7, 2020
Sponsor:
Information provided by (Responsible Party):
Pfizer

Brief Summary:
The purpose of this study is to understand safety (e.g., occurrence of adverse drug reactions [ADRs]) and efficacy data on the long-term use of Vyndaqel Capsules (hereinafter referred to as Vyndaqel) in all patients who received this drug under actual use conditions after its marketing.

Condition or disease Intervention/treatment
Transthyretin Familial Amyloid Poluneuropathy Drug: Vyndaqel

Detailed Description:
Both prospective and retrospective is acceptable All the patients whom an investigator prescribes Vyndaqel should be registered

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Study Type : Observational
Actual Enrollment : 495 participants
Observational Model: Case-Only
Time Perspective: Other
Official Title: VYNDAQEL DRUG USE INVESTIGATION (REGULATORY POST MARKETING COMMITMENT PLAN)
Actual Study Start Date : January 10, 2014
Estimated Primary Completion Date : April 11, 2022
Estimated Study Completion Date : April 11, 2022

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
Vyndaqel Drug: Vyndaqel
20mg/day




Primary Outcome Measures :
  1. The incidence of adverse drug reaction in this study [ Time Frame: 3 years ]

Secondary Outcome Measures :
  1. Change from baseline on the following scale; NIS, QOL-DN, mBMI [ Time Frame: 3 years ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
The subjects of this surveillance are all patients who received Vyndaqel.
Criteria

Inclusion Criteria:

The subjects of this surveillance are all patients who received Vyndaqel.

Exclusion Criteria:

Patients not receive Vyndaqel.


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): NCT02146378


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: NCT02146378    
Other Study ID Numbers: B3461042
First Posted: May 23, 2014    Key Record Dates
Last Update Posted: July 7, 2020
Last Verified: July 2020
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: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases