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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00925002
First received: June 17, 2009
Last updated: April 11, 2014
Last verified: April 2014
  Purpose

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.


Condition Intervention Phase
ATTR-PN
Drug: Tafamidis
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Open-Label Safety and Efficacy Evaluation Of Fx-1006a In Subjects With Transthyretin Amyloidosis

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • To obtain additional, long-term, open-label safety and efficacy data for Fx-1006A in patients with transthyretin (TTR) amyloidosis (ATTR) [ Time Frame: 10 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • To continue to provide the investigational product Fx-1006A until its market availability to patients with ATTR who have completed Protocol Fx-006 or Protocol Fx1A-201 [ Time Frame: 10 years ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 110
Study Start Date: August 2009
Estimated Study Completion Date: December 2019
Estimated Primary Completion Date: December 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Open-Label Drug: Tafamidis
20 mg oral Fx-1006A daily

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00925002

Locations
United States, Maryland
Pfizer Investigational Site
Baltimore, Maryland, United States, 21287
Argentina
Pfizer Investigational Site
Buenos Aires, Argentina, C1428AQK
Pfizer Investigational Site
Ciudad de Buenos Aires, Argentina, C1428AQK
Brazil
Pfizer Investigational Site
Rio de Janeiro, RJ, Brazil, 21941-913
Pfizer Investigational Site
Rio de Janeiro, Brazil, CEP 21941-913
France
Pfizer Investigational Site
Creteil Cedex, France, 94010
Germany
Pfizer Investigational Site
Muenster, Germany, 48149
Pfizer Investigational Site
Munster, Germany, 48129
Italy
Pfizer Investigational Site
Pavia, Italy, 27100
Portugal
Pfizer Investigational Site
Lisbon, Portugal, 1649-035
Pfizer Investigational Site
Lisbon, Portugal, 1649-028
Pfizer Investigational Site
Porto, Portugal, 4099-001
Sweden
Pfizer Investigational Site
Umea, SE, Sweden, 901 87
Pfizer Investigational Site
Umea, Sweden, 90185
Pfizer Investigational Site
Umea, Sweden, SE-901 85
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00925002     History of Changes
Other Study ID Numbers: FX1A-303, B3461023
Study First Received: June 17, 2009
Last Updated: April 11, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on April 17, 2014