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

Last updated on October 20, 2019

FOR MORE INFORMATION
Study Location
Johns Hopkins Hospital
Baltimore, Maryland, 21287 United States
Contact
1-800-718-1021
Eligibility criteria
Condition
The disease, disorder, syndrome, illness, or injury that is being studied.
ATTR-PN
Sex
Females and Males
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
18-75 years
Inclusion criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

- Patient has successfully completed either Protocol Fx-006 or Fx1A-201.

- If female, patient is post-menopausal, surgically sterilized, or willing to use two
acceptable methods of birth control (i.e., hormonal contraceptive, intra-uterine
device, diaphragm with spermicide, condom with spermicide) throughout the study and
for 3 months from the end of the study. (A condom alone is not considered an
acceptable method of birth control.)

- Patient is, in the opinion of the investigator, willing and able to comply with the
investigational product regimen and all other study requirements.

Exclusion criteria
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details

- Patient has not successfully completed either Protocol Fx-006 or Fx1A-201.

- Chronic use of non-protocol approved non-steroidal anti-inflammatory drugs (NSAIDs),
defined as greater than 3 to 4 times/month. The following NSAIDs are allowed:
acetylsalicylic acid, etodolac, ibuprofen, indomethicin, ketoprofen, nabumetone,
naproxen, nimesulide, piroxicam, and sulindac.

- If female, patient is pregnant or breast feeding.

- Clinically significant medical condition that, in the opinion of the investigator,
would place the patient at an increased risk to participate in the study.

- The patient has received a liver or heart transplant prior to enrollment.

NCT00925002
Pfizer
Active, not recruiting
Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis

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Descriptive Information
Brief Title  ICMJE Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis
Official Title  ICMJE OPEN-LABEL SAFETY AND EFFICACY EVALUATION OF FX-1006A IN SUBJECTS WITH TRANSTHYRETIN (TTR) AMYLOIDOSIS
Brief Summary

This is a Phase 3, open-label study designed to obtain additional, long-term, open-label safety and efficacy data for Fx-1006A and to continue to provide patients with 20 mg oral Fx-1006A (soft gel capsule) who have completed either Protocol Fx-006 (a 1 year, open-label extension study to Protocol Fx-005 which is a randomized, double-blind, placebo-controlled, 18-month study to evaluate the safety and efficacy of Fx-1006A) or Protocol Fx1A-201 (a Phase 2, open-label study to evaluate TTR stabilization as well as the safety and tolerability of Fx-1006A) until market availability of Fx-1006A in individual patients' country of residence.

Patients who successfully complete Protocol Fx-006 or Fx1A-201 will report to the clinical unit on Day 0 (Baseline) to sign the informed consent form and determine their eligibility for Protocol Fx1A-303. In addition, on Day 0 (Baseline), patients will have their entrance criteria reviewed and medical history and demography for all patients will be obtained. The relevant end of study assessments from Protocols Fx1A-201 and Fx-006 will serve as Baseline assessments for Protocol Fx1A-303 if these examinations were performed within 30 days of Day 0 (Baseline). For any patient successfully completing Protocol Fx-006, the Karnofsky Performance Scale Index will be assessed and the cranial nerve and upper limb components of the NIS will be performed and combined with the NIS-LL data from the end of study visit from Protocol Fx-006.

If there is more than 30 days between the final study visit of Fx-006 or Fx1A-201 and Day 0 (Baseline) of Fx1A-303, all Day 0 study procedures will be performed (i.e., no data from the final study visits from the previous studies will be utilized).

Eligible patients will begin once-daily dosing with 20 mg Fx-1006A at home on Day 1 (i.e., first dose) and will return to the clinical unit for study visits every 6 months.

Adverse events (AEs) and concomitant medication use will be collected at each 6-month visit to the clinical unit and, if female, a urine pregnancy test will be performed. An abbreviated physical examination (including weight and vitals signs) will be conducted at every other 6 month visit. A telephone call will be made at 3-month intervals between clinic visits to assess safety and the use of concomitant medications.

For the evaluation of efficacy, the NIS, Norfolk QOL-DN, and Karnofsky Performance Scale Index will be performed on an annual basis (i.e., every other 6-month visit).

An end of study visit will occur upon patient withdrawal (for any reason), program discontinuation by the Sponsor, or upon market availability of Fx-1006A in individual patients' country of residence.

Detailed DescriptionNot Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE ATTR-PN
Intervention  ICMJE Drug: Tafamidis
20 mg oral Fx-1006A daily
Study Arms  ICMJE Active Comparator: Open-Label
Intervention: Drug: Tafamidis
Publications *


*   Includes publications given by the data provider as well as publications
identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: July 28, 2017)
93
Original Estimated Enrollment  ICMJE
 (submitted: June 17, 2009)
100
Estimated Study Completion Date  ICMJE December 31, 2021
Estimated Primary Completion DateDecember 31, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Patient has successfully completed either Protocol Fx-006 or Fx1A-201.
  • If female, patient is post-menopausal, surgically sterilized, or willing to use two acceptable methods of birth control (i.e., hormonal contraceptive, intra-uterine device, diaphragm with spermicide, condom with spermicide) throughout the study and for 3 months from the end of the study. (A condom alone is not considered an acceptable method of birth control.)
  • Patient is, in the opinion of the investigator, willing and able to comply with the investigational product regimen and all other study requirements.

Exclusion Criteria:

  • Patient has not successfully completed either Protocol Fx-006 or Fx1A-201.
  • Chronic use of non-protocol approved non-steroidal anti-inflammatory drugs (NSAIDs), defined as greater than 3 to 4 times/month. The following NSAIDs are allowed: acetylsalicylic acid, etodolac, ibuprofen, indomethicin, ketoprofen, nabumetone, naproxen, nimesulide, piroxicam, and sulindac.
  • If female, patient is pregnant or breast feeding.
  • Clinically significant medical condition that, in the opinion of the investigator, would place the patient at an increased risk to participate in the study.
  • The patient has received a liver or heart transplant prior to enrollment.
Sex/Gender  ICMJE
Sexes Eligible for Study:All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Brazil,   France,   Germany,   Italy,   Portugal,   Sweden,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00925002
Other Study ID Numbers  ICMJE FX1A-303
B3461023 ( Other Identifier: Alias Study Number )
2009-011535-12 ( EudraCT Number )
Has Data Monitoring CommitteeNo
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product:Yes
Studies a U.S. FDA-regulated Device Product:No
IPD Sharing Statement  ICMJE
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/da....
URL:https://www.pfizer.com/science/clinical_trials/trial_data_and_results/da...
Responsible PartyPfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director:Pfizer CT.gov Call CenterPfizer
PRS AccountPfizer
Verification DateMarch 2019

ICMJE     Data element required by the

International Committee of Medical Journal Editors
and the
World Health Organization ICTRP

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Contact a representative by phone, email, or visiting the study website. Please see the references below:

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