In preparation

• Register: EUCTR
• Last refreshed on: 2 October 2012
• Main ID: EUCTR2009-011535-12-DE
• Date of registration: 18/05/2009
• Prospective Registration: Yes
• Primary sponsor: Pfizer Inc.
• Public title: In preparation
• Scientific title: Open-Label Safety and Efficacy Evaluation of Fx-1006A in Patients with Transthyretin Amyloidosis
• Date of first enrolment: 26/08/2009
• Target sample size: 110
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-011535-12
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Argentina, Brazil, France, Germany, Italy, Portugal, Sweden, United States

Contacts

• Name: Clinical Trials.gov Call Center
• Affiliation: Pfizer Inc.

• Name: Clinical Trials.gov Call Center
• Affiliation: Pfizer Inc.

Key inclusion & exclusion criteria

Inclusion criteria:
1. Patient has successfully completed either Protocol Fx-006 or Fx1A-201.
2. 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.).
3. Patient is, in the opinion of the investigator, willing and able to comply with the investigational product regimen and all other study requirements.
Sub-study:
4.Decrease in creatinine clearance to < 30 mL/minute, or the need for chronic dialysis.
5.Willing to provide written informed consent for this sub-study.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 110
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Patient has not successfully completed either Protocol Fx-006 or Fx1A-201.
2. 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.
3. If female, patient is pregnant or breast feeding.
4. Clinically significant medical condition that, in the opinion of the investigator, would place the patient at an increased risk to participate in the study.
5. An alanine transaminase (ALT) and aspartate transaminase, (AST) value >3X ULN that in the medical judgement of the investigator is due to reduced liver function or active disease.
6. The patient has received a liver or heart transplant.
Sub-study:
7. Unwilling to provide written informed consent for this sub-study.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Transthyretin Amyloidosis (ATTR)
MedDRA version: 14.1 Level: LLT Classification code 10057949 Term: Familial amyloid polyneuropathy System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Intervention(s)

Product Name: Tafamidis meglumine
Product Code: Fx-1006A
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: tafamidis
CAS Number: 951395-08-7
Current Sponsor code: Fx-1006A
Other descriptive name: [d-glucitol, 1-deoxy-1-(methylamino)-, 2-(3,5-dichlorophenyl)-6-benzoxazolecarboxylate (1:1)]
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: End of study

Primary end point(s): Safety endpoints:
- The incidence of treatment-emergent adverse events (TEAEs), physical examinations, clinical laboratory testing, use of concomitant medications and vital signs.

Efficacy endpoints:
- The Neuropathy Impairment Score (NIS)
- The Total Quality of Life (TQOL) score as measured using the Norfolk Quality of Life for diabetic neuropathy (QOL-DN) questionnaire
- The Karnofsky Performance Scale Index
-Assessment of Patient Ambulation

Main Objective: To obtain additional, long-term, open-label safety and efficacy data for Fx-1006A in patients with transthyretin (TTR) amyloidosis (ATTR).

Secondary Objective: To continue to provide the investigational product Fx-1006A until market availability or until 31 December 2012 or whichever is earliest, to patients with ATTR who have completed Protocol Fx-006 or Protocol Fx1A-201.

Secondary Outcome(s)

Secondary end point(s): None described at time of submission

Timepoint(s) of evaluation of this end point: Not applicable

Secondary ID(s)

Fx1A-303

NCT00925002

Source(s) of Monetary Support

Pfizer Inc.