Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis

NCT00409175

Last updated date
Study Location
MGH Neuropathy Laboratory
Boston, Massachusetts, 02114, United States
Contact
1-800-718-1021

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Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Familial Amyloid Polyneuropathy
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

1. Amyloid documented by biopsy.

2. Documented V30M TTR mutation.

3. Peripheral and/or autonomic neuropathy with a Karnofsky Performance Status ≥50.

4. Patient is 18-75 years old.

5. If female, patient is post-menopausal, surgically sterilized, or willing to use 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, birth control must be used for at least 3 months after the last dose of study medication.

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

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


1. Chronic use of non-steroidal anti-inflammatory drugs (NSAIDs).


2. Primary amyloidosis.


3. If female, patient is pregnant or breast feeding.


4. Prior liver transplantation.


5. No recordable sensory threshold for vibration perception in both feet, as measured by
CASE IV.


6. Positive results for hepatitis B surface antigen (HBsAg), anti-hepatitis C virus
(HCV), and/or human immunodeficiency virus (HIV).


7. Renal insufficiency or liver function test abnormalities.


8. New York Heart Association (NYHA) Functional Classification ≥III.


9. Other causes of sensorimotor neuropathy (B12 deficiency, Diabetes Mellitus, HIV
treated with retroviral medications, thyroid disorders, alcohol abuse, and chronic
inflammatory diseases).


10. Co-morbidity anticipated to limit survival to less than 18 months.


11. Patient received an investigational drug/device and/or participated in another
clinical investigational study within 60 days before Baseline.

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Familial Amyloid PolyneuropathySafety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
NCT00409175
  1. Boston, Massachusetts
  2. Ciudad de Buenos Aires, Buenos Aires Province
  3. Rio de Janeiro, Southeast
  4. Le Kremlin Bicêtre, Ile de France
  5. Munster, Nordrhein-Westfalen
  6. Lisboa, Lisbon
  7. Porto, Norte
  8. Barcelona, Catalunya
  9. Umea, Vasterbotten County
  10. London,
ALL GENDERS
18 Years+
years
MULTIPLE SITES
Advanced Information
Descriptive Information
Brief Title  ICMJE Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
Official Title  ICMJE Safety and Efficacy of Orally Administered Fx-1006A in Patients With Familial Amyloid Polyneuropathy (FAP): A Randomized, Double-blind, Placebo-controlled Study
Brief Summary

This study will examine whether Fx-1006A is effective in halting the progression of Familial Amyloid Polyneuropathy (FAP).

Deposition of TTR amyloid is associated with a variety of human diseases. Deposition of amyloid fibrils of variant TTR (primarily V30M) in peripheral nerve tissue produces the condition called FAP.

The prevention of the formation of amyloid by stabilization of the TTR native state should constitute an effective therapy for amyloid diseases. Therapeutic intervention with a TTR stabilizer drug, such as Fx-1006A, is hypothesized to stop progression of the disease in FAP patients. FAP is a uniformly fatal disease and Fx-1006A is intended to halt the relentless neurological deterioration FAP patients experience.

This Phase 2/3 study will enroll early to mid-stage FAP patients in order to interrupt and stabilize the disease at a point in time where progression of motor and autonomic dysfunction can be maximally effected. Male and female patients with FAP with documented V30M TTR mutation will receive Fx-1006A or placebo once daily for a period of eighteen (18) months.

Detailed Description

Deposition of TTR amyloid is associated with a variety of human diseases. Deposition of amyloid fibrils of variant TTR (primarily V30M) in peripheral nerve tissue produces the condition called FAP.

The prevention of the formation of amyloid by stabilization of the TTR native state should constitute an effective therapy for amyloid diseases. Therapeutic intervention with a TTR stabilizer drug, such as Fx-1006A, is hypothesized to stop progression of the disease in FAP patients. FAP is a uniformly fatal disease and Fx-1006A is intended to halt the relentless neurological deterioration FAP patients experience.

This Phase 2/3 study will enroll early to mid-stage FAP patients in order to interrupt and stabilize the disease at a point in time where progression of motor and autonomic dysfunction can be maximally effected. Male and female patients with FAP with documented V30M TTR mutation will receive Fx-1006A or placebo once daily for a period of eighteen (18) months.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Familial Amyloid Polyneuropathy
Intervention  ICMJE
  • Drug: Fx-1006A
    Fx-1006A 20mg or matched placebo once daily (at the same time each day) for a period of 18 Months
  • Drug: Placebo
    Fx-1006A 20mg or matched placebo once daily (at the same time each day) for a period of 18 Months
Study Arms  ICMJE
  • Experimental: 1.
    Fx-1006A
    Intervention: Drug: Fx-1006A
  • Placebo Comparator: 2.
    Placebo
    Intervention: Drug: Placebo
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 Completed
Actual Enrollment  ICMJE
 (submitted: May 20, 2011)
128
Original Enrollment  ICMJE
 (submitted: December 6, 2006)
120
Actual Study Completion Date  ICMJE May 2009
Actual Primary Completion Date May 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Amyloid documented by biopsy.
  2. Documented V30M TTR mutation.
  3. Peripheral and/or autonomic neuropathy with a Karnofsky Performance Status ?50.
  4. Patient is 18-75 years old.
  5. If female, patient is post-menopausal, surgically sterilized, or willing to use 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, birth control must be used for at least 3 months after the last dose of study medication.
  6. Patient is, in the opinion of the investigator, willing and able to comply with the study medication regimen and all other study requirements.

Exclusion Criteria:

  1. Chronic use of non-steroidal anti-inflammatory drugs (NSAIDs).
  2. Primary amyloidosis.
  3. If female, patient is pregnant or breast feeding.
  4. Prior liver transplantation.
  5. No recordable sensory threshold for vibration perception in both feet, as measured by CASE IV.
  6. Positive results for hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (HCV), and/or human immunodeficiency virus (HIV).
  7. Renal insufficiency or liver function test abnormalities.
  8. New York Heart Association (NYHA) Functional Classification ?III.
  9. Other causes of sensorimotor neuropathy (B12 deficiency, Diabetes Mellitus, HIV treated with retroviral medications, thyroid disorders, alcohol abuse, and chronic inflammatory diseases).
  10. Co-morbidity anticipated to limit survival to less than 18 months.
  11. Patient received an investigational drug/device and/or participated in another clinical investigational study within 60 days before Baseline.
Sex/Gender  ICMJE
Sexes Eligible for Study:All
Ages  ICMJE 18 Years to 75 Years   (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,   Portugal,   Spain,   Sweden,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00409175
Other Study ID Numbers  ICMJE FX-005
B3461020
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director:Jeff PackmanFoldRx Pharmaceuticals, Inc.
PRS Account Pfizer
Verification Date November 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP