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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00791492
• Date of registration: 13/11/2008
• Prospective Registration: No
• Primary sponsor: Pfizer
• Public title: An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy
• Scientific title: An Open-Label Extension Of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy
• Date of first enrolment: July 2008
• Target sample size: 86
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00791492
• Study type: Interventional
• Phase: Phase 2/Phase 3

Countries of recruitment

Argentina, Brazil, France, Germany, Portugal, Sweden

Contacts

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

Key inclusion & exclusion criteria

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.



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

Health Condition(s) or Problem(s) studied

ATTR-PN

Familial Amyloid Polyneuropathy

Intervention(s)

Drug: Fx-1006A

Primary Outcome(s)

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

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

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

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

Secondary Outcome(s)

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

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

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

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

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

Intraepidermal Nerve Fiber (IENF) Density [Time Frame: Baseline]

Change From Baseline in N-Terminal Prohormone Brain Natriuretic Peptide (NT-proBNP) Concentration at Week 6, Month 3, 6 and 12 [Time Frame: Baseline, Week 6, Month 3, 6, 12]

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

Change From Baseline in Troponin I Concentration at Week 6, Month 3, 6 and 12 [Time Frame: Baseline, Week 6, Month 3, 6, 12]

Secondary ID(s)

B3461021

FX-006

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/NCT00791492