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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00409175
• Date of registration: 06/12/2006
• Prospective Registration: Yes
• Primary sponsor: Pfizer
• Public title: Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
• Scientific title: Safety and Efficacy of Orally Administered Fx-1006A in Patients With Familial Amyloid Polyneuropathy (FAP): A Randomized, Double-blind, Placebo-controlled Study
• Date of first enrolment: January 2007
• Target sample size: 128
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00409175
• Study type: Interventional
• Phase: Phase 2/Phase 3

Countries of recruitment

Argentina, Brazil, France, Germany, Portugal, Spain, Sweden, United Kingdom, United States

Contacts

• Name: Jeff Packman
• Affiliation: FoldRx Pharmaceuticals, Inc.

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Familial Amyloid Polyneuropathy

Intervention(s)

Drug: Fx-1006A

Drug: Placebo

Primary Outcome(s)

Change From Baseline in Norfolk Quality of Life- Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 18 [Time Frame: Baseline, Month 18]

Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 18 [Time Frame: Month 18]

Secondary Outcome(s)

Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Domain Scores at Month 6, 12 and 18 [Time Frame: Baseline, Month 6, 12, 18]

Change From Baseline in Neuropathy Impairment Score- Lower Limb (NIS-LL) Score at Month 6, 12 and 18 [Time Frame: Baseline, Month 6, 12, 18]

Change From Baseline in Modified Body Mass Index (mBMI) at Month 6, 12 and 18 [Time Frame: Baseline, Month 6, 12, 18]

Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 6 and 12 [Time Frame: Month 6, 12]

Change From Baseline in Summated 3 Score for Small Nerve Fiber Function at Month 6, 12 and 18 [Time Frame: Baseline, Month 6, 12, 18]

Change From Baseline in Summated 7 Score for Large Nerve Fiber Function at Month 6, 12 and 18 [Time Frame: Baseline, Month 6, 12, 18]

Percentage of Participants With Stabilized Transthyretin (TTR) Tetramer [Time Frame: Week 8, Month 6, 12, 18]

Change From Baseline in Norfolk Quality of Life - Diabetic Neuropathy (QOL-DN) Total Quality of Life (TQOL) Score at Month 6 and 12 [Time Frame: Baseline, Month 6, 12]

Secondary ID(s)

B3461020

FX-005

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 17/12/2012
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00409175