You are here

An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy

Last updated on December 16, 2017

FOR MORE INFORMATION
Study Location
FLENI Departamento de Hepatología y Transplante de Organos
Buenos Aires, , Argentina
Contact
1-800-718-1021
Eligibility criteria
Condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Familial Amyloid Polyneuropathy, 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

Male and non-pregnant female patients meeting all of the following criteria are eligible
for enrollment in this study:

- Patient has completed the Month 18 visit of Study Fx-005.

- If female, patient is post-menopausal, surgically sterilized, or willing to use an
acceptable method of birth control (i.e., a hormonal contraceptive, intra-uterine
device, diaphragm with spermicide, condom with spermicide) throughout the study. (A
condom alone is not considered an acceptable method of birth control.) If male with a
female partner of childbearing potential, willing to use an acceptable method of birth
control for the duration of the study. For both females and males, acceptable birth
control must be used for at least 3 months after the last dose of study medication.

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

- Patient agrees not to participate in another investigational drug or device study
while participating in this open-label extension study.

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

Patients meeting any of the exclusion criteria will not be enrolled in the study:

- Chronic use of non-steroidal anti-inflammatory drugs (NSAIDs), defined as greater than
3-4 times/month (ibuprofen and nimesulide will be permitted).

- If female, patient is pregnant or breast feeding.

- Patient has liver function test abnormalities: alanine transaminases (ALT) and/or
aspartate transaminases (AST) >2 times upper limit of normal (ULN) that in the medical
judgment of the investigator are due to reduced liver function or active liver
disease.

NCT00791492
Pfizer
Completed
An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy

NEED INFO?

Questions about a trial? Call or email to reach a Pfizer Clinical Trial Contact Center

Pfizer Clinical Trials Contact Center

1-800-718-1021

[email protected]

call now

Try a new search

Search for Clinical Trials by condition, keyword or trial number. Share your location or enter your zip code, city, or country to find studies near you.

Similar Trials

Familial Persistent Pulmonary Hypertension
NCT01720524
All Genders
0+
Years
Multiple Sites
Transthyretin (TTR) Amyloid Cardiomyopathy
NCT02791230
All Genders
18+
Years
Multiple Sites
Transthyretin Mutations, Transthyretin Amyloidosis
NCT00628745
All Genders
18+
Years
Multiple Sites
Hypertension, Angina Pectoris, Hypercholesterolemia, Familial Hypercholesterolemia
NCT01107743
All Genders
An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy
An Open-Label Extension Of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy

This study is designed to determine the long-term safety and tolerability of Fx-1006A as well as the effects of Fx-1006A on clinical outcomes in patients with ATTR-PN.

All patients who enroll in this extension study will receive once-daily oral 20 mg Fx-1006A for 12 months; therefore, patients randomized to placebo in Study Fx-005 will cross over to active drug (Fx-1006A 20 mg) during this study. However, patients and their families as well as clinical Investigators and their clinical site staff will remain blinded to the original Fx-005 treatment assignment. It is intended that there will be no interruption in study medication administration between the two studies. The majority of safety and clinical outcomes assessments will be identical to those evaluated in Study Fx-005. Additional assessments for this open-label extension study include 24-hour Holter monitoring and skin biopsy for IENF; patients will be required to provide written informed consent to participate in this open-label extension study prior to having these additional procedures performed.

The values obtained from procedures and evaluations conducted during the Month 18 visit of Study Fx-005 will be used as the Baseline values for this open-label extension study. The Baseline assessments of IENF and Holter monitoring may be conducted at either day of the Month 18 visit days of Study Fx-005, but prior to the first Fx-1006A dose in this open-label extension study. Clinic Visits will be conducted at Week 6 (± 2 days), and Month 3 (± 1 week), Month 6 (± 2 weeks), and Month 12 (± 2 weeks). Monthly telephone contacts (± 1 week of the scheduled date) will be made during months in which no investigative site visits are scheduled (Months 2, 4, 5, 7, 8, 9, 10, and 11) for assessment of adverse events and concomitant medications.

Neurological evaluation by NIS-LL will be performed at Months 6 and 12. The NIS-LL will be assessed by utilizing the average of two successive NIS-LL clinical assessment scores obtained at least 24 hours apart within a one week period for each study visit. A dedicated neurologist will be required to perform NIS-LL scoring across all time-points for each individual patient enrolled in the study.

Quality of life utilizing the Norfolk QOL-DN will be assessed at Months 6 and 12 (based on the total score as well as the five individual domains of the questionnaire).

QST (utilizing CASE IV), NCS, HRDB, mBMI, and echocardiography will be conducted at Months 6 and 12. Holter monitoring will be conducted at Baseline and Months 6 and 12. Biopsies for IENF will be obtained at Baseline only. Assessments of troponin I and NT-pro-BNP levels will be made at each study visit.

Blood samples for pharmacokinetic assessments (Fx-1006A concentrations as well as calculated steady-state parameters) and pharmacodynamic assessments (TTR stabilization) will be collected at Week 6 and Months 6 and 12.

Safety and tolerability will be assessed throughout the study. Vital signs, 12-lead ECG, blood and urine samples for clinical laboratory tests (serum chemistry, hematology, coagulation panel, urinalysis, and urine pregnancy testing), adverse events, and concomitant medications will be assessed at each study visit. Eye examinations (including fundal photography) will be conducted at Months 6 and 12. Abbreviated physical examinations will be conducted at Week 6, and Months 3 and 6, and a complete physical examination will be conducted at Month 12.

All patients will be contacted by telephone 30 days (± 1 week) after the last dose of study medication for assessment of adverse events and concomitant medications.

Patients who complete the Month 12 visit of this open-label study may be allowed to continue receiving Fx-1006A under a compassionate use program.

Patients who discontinue from the study at any time after enrollment (i.e., early termination) will have final safety assessments performed at the time of discontinuation. Any patient discontinuing after the Month 6 visit will have all safety and clinical outcomes assessments scheduled for the Month 12 visit performed.

Not Provided
Interventional
Phase 2
Phase 3
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Familial Amyloid Polyneuropathy
  • ATTR-PN
Drug: Fx-1006A
Fx-1006A 20mg soft gelatin capsule administered orally once daily (at the same time each day) for 12 months.
Experimental: Fx-1006A
Intervention: Drug: Fx-1006A
Waddington Cruz M, Amass L, Keohane D, Schwartz J, Li H, Gundapaneni B. Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy. Amyloid. 2016 Sep;23(3):178-183. Epub 2016 Aug 5.


*   Includes publications given by the data provider as well as publications
identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
86
October 2010
August 2008   (Final data collection date for primary outcome measure)

Inclusion Criteria:

Male and non-pregnant female patients meeting all of the following criteria are eligible for enrollment in this study:

  • Patient has completed the Month 18 visit of Study Fx-005.
  • If female, patient is post-menopausal, surgically sterilized, or willing to use an acceptable method of birth control (i.e., a hormonal contraceptive, intra-uterine device, diaphragm with spermicide, condom with spermicide) throughout the study. (A condom alone is not considered an acceptable method of birth control.) If male with a female partner of childbearing potential, willing to use an acceptable method of birth control for the duration of the study. For both females and males, acceptable birth control must be used for at least 3 months after the last dose of study medication.
  • Patient is, in the opinion of the investigator, willing and able to comply with the study medication regimen and all other study requirements.
  • Patient agrees not to participate in another investigational drug or device study while participating in this open-label extension study.

Exclusion Criteria:

Patients meeting any of the exclusion criteria will not be enrolled in the study:

  • Chronic use of non-steroidal anti-inflammatory drugs (NSAIDs), defined as greater than 3-4 times/month (ibuprofen and nimesulide will be permitted).
  • If female, patient is pregnant or breast feeding.
  • Patient has liver function test abnormalities: alanine transaminases (ALT) and/or aspartate transaminases (AST) >2 times upper limit of normal (ULN) that in the medical judgment of the investigator are due to reduced liver function or active liver disease.
Sexes Eligible for Study: All
18 Years to 75 Years   (Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
Argentina,   Brazil,   France,   Germany,   Portugal,   Sweden
 
 
NCT00791492
FX-006
B3461021
No
Not Provided
Not Provided
Pfizer
Pfizer
Not Provided
Study Director: Jeff Packman FoldRx Pharmaceuticals, Inc.
Pfizer
November 2012

ICMJE     Data element required by the

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

FOR MORE INFORMATION

Contact a representative by phone, email, or visiting thte study website. To get updates and notications about this trail, sign up using the form below.

BY PHONE

Pfizer Clinical Trials Contact Center

1-800-718-1021

BY EMAIL

Contact

[email protected]



Call Now