Clinical trial NCT04801329: Korean Post-marketing Surveillance Vyndamax® Capsules for the Treatment of Transthyretin Amyloid Cardiomyopathy
Official title: KOREAN POST-MARKETING SURVEILLANCE VYNDAMAX (Registered) CAPSULES FOR THE TREATMENT OF TRANSTHYRETIN AMYLOID CARDIOMYOPATHY (ATTR-CM)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: ATTR-CM (Transthyretin Amyloid Cardiomyopathy)
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2021-03-17
Enrollment: 100
Sex: ALL
Minimum age (years): 19
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study will be performed in patients who have received at least 1 dose of Vyndamax Capsules for the following indications as per local product label.(Treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization)

Brief summary: This non-interventioal study will be performed by design of post-marketing surveillance (PMS) as an additional pharmacovigilance activity of the Risk Management Plan (RMP) for Vyndamax® Capsules, which is required by the Ministry of Food and Drug Safety (MFDS) according to the local regulation. This post-marketing surveillance will investigate the safety and effectiveness of Vyndamax® Capsules as the treatment of transthyretin amyloid cardiomyopathy during 10 years under the setting of routine practice in Korea.


Eligibility criteria: \*Inclusion Criteria:

Patients must meet all of the following inclusion criteria to be eligible for inclusion in the study:

* 1. Adult patients with the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM)
* 2. Patients to whom Vyndamax® Capsules is prescribed for the treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM)
* 3. Evidence of a personally signed and dated informed consent document indicating that the patient (or a legally acceptable representative) has been informed of all pertinent aspects of the study.

  * Exclusion criteria

Patients meeting any of the following criteria will not be included in the study according to the local product label:

* 1. Patient with hypersensitivity or case history to tafamidis or to any of the excipients in the product
* 2. This product contains sorbitol (E420). Patients with rare hereditary problems of fructose intolerance should not take this medicine.
* 3. Patient who has a contraindication to Vyndamax® Capsules according to the approved local product label

Primary outcomes:
measure: The incidence of Adverse Events (AEs)
timeFrame: Baseline through 6 months of treatment
Secondary outcomes:
measure: Change from baseline in the New York Heart Association (NYHA) class at Month 6
timeFrame: Baseline, month 6
measure: Change from baseline in the 6 Minute Walk Distance (6MWD) at Month 6
timeFrame: Baseline, month 6
measure: Global assessment by investigator at Month 6
timeFrame: Baseline, month 6
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Pfizer
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: