Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis

Study Description

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Brief Summary:

This is a Phase 3, open-label study designed to obtain additional, long-term, open-label safety and efficacy data for Fx-1006A and to continue to provide patients with 20 mg oral Fx-1006A (soft gel capsule) who have completed either Protocol Fx-006 (a 1 year, open-label extension study to Protocol Fx-005 which is a randomized, double-blind, placebo-controlled, 18-month study to evaluate the safety and efficacy of Fx-1006A) or Protocol Fx1A-201 (a Phase 2, open-label study to evaluate TTR stabilization as well as the safety and tolerability of Fx-1006A) until market availability of Fx-1006A in individual patients' country of residence.

Patients who successfully complete Protocol Fx-006 or Fx1A-201 will report to the clinical unit on Day 0 (Baseline) to sign the informed consent form and determine their eligibility for Protocol Fx1A-303. In addition, on Day 0 (Baseline), patients will have their entrance criteria reviewed and medical history and demography for all patients will be obtained. The relevant end of study assessments from Protocols Fx1A-201 and Fx-006 will serve as Baseline assessments for Protocol Fx1A-303 if these examinations were performed within 30 days of Day 0 (Baseline). For any patient successfully completing Protocol Fx-006, the Karnofsky Performance Scale Index will be assessed and the cranial nerve and upper limb components of the NIS will be performed and combined with the NIS-LL data from the end of study visit from Protocol Fx-006.

If there is more than 30 days between the final study visit of Fx-006 or Fx1A-201 and Day 0 (Baseline) of Fx1A-303, all Day 0 study procedures will be performed (i.e., no data from the final study visits from the previous studies will be utilized).

Eligible patients will begin once-daily dosing with 20 mg Fx-1006A at home on Day 1 (i.e., first dose) and will return to the clinical unit for study visits every 6 months.

Adverse events (AEs) and concomitant medication use will be collected at each 6-month visit to the clinical unit and, if female, a urine pregnancy test will be performed. An abbreviated physical examination (including weight and vitals signs) will be conducted at every other 6 month visit. A telephone call will be made at 3-month intervals between clinic visits to assess safety and the use of concomitant medications.

For the evaluation of efficacy, the NIS, Norfolk QOL-DN, and Karnofsky Performance Scale Index will be performed on an annual basis (i.e., every other 6-month visit).

An end of study visit will occur upon patient withdrawal (for any reason), program discontinuation by the Sponsor, or upon market availability of Fx-1006A in individual patients' country of residence.

Condition or disease	Intervention/treatment	Phase
ATTR-PN	Drug: Tafamidis	Phase 3

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	93 participants
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	Open-label Safety And Efficacy Evaluation Of Fx-1006a In Subjects With Transthyretin (Ttr) Amyloidosis
Actual Study Start Date :	August 5, 2009
Estimated Primary Completion Date :	December 31, 2021
Estimated Study Completion Date :	December 31, 2021

Arms and Interventions

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Arm	Intervention/treatment
Active Comparator: Open-Label	Drug: Tafamidis; 20 mg oral Fx-1006A daily

Outcome Measures

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Primary Outcome Measures :

1. To obtain additional, long-term, open-label safety and efficacy data for Fx-1006A in patients with transthyretin (TTR) amyloidosis (ATTR) [ Time Frame: 10 years ]

Secondary Outcome Measures :

1. To continue to provide the investigational product Fx-1006A until its market availability to patients with ATTR who have completed Protocol Fx-006 or Protocol Fx1A-201 [ Time Frame: 10 years ]

Eligibility Criteria

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Ages Eligible for Study:	18 Years to 75 Years (Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

• Patient has successfully completed either Protocol Fx-006 or Fx1A-201.
• 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.)
• Patient is, in the opinion of the investigator, willing and able to comply with the investigational product regimen and all other study requirements.

Exclusion Criteria:

• Patient has not successfully completed either Protocol Fx-006 or Fx1A-201.
• 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.
• If female, patient is pregnant or breast feeding.
• Clinically significant medical condition that, in the opinion of the investigator, would place the patient at an increased risk to participate in the study.
• The patient has received a liver or heart transplant prior to enrollment.

Contacts and Locations

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Locations

United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Argentina
FLENI
Ciudad Autonoma de Buenos aires, Argentina, C1428AQK
Brazil
Hospital Universitário Clementino Fraga Filho -HUCFF Universidade Federal do Rio de Janeiro
Rio de Janeiro, RJ, Brazil, CEP 21941-913
France
CHU Henri Mondor
Creteil cedex, France, 94010
Germany
Universitatsklinikum Muenster
Muenster, Germany, 48149
Italy
Centro per lo Studio e la Cura delle Amiloidosi Sistemiche IRCCS - Policlinico San Matteo
Pavia, Italy, 27100
Dipartimento di Neurofisiopatologia
Pavia, Italy, 27100
Fondazione IRCCS Policlinico San Matteo
Pavia, Italy, 27100
IRCCS
Pavia, Italy, 27100
Portugal
Centro Hospitalar Lisboa Norte, EPE- Hospital de Santa Maria
Lisboa, Portugal, 1649-028
Unidade Clinica de Paramiloidose, EPE - Centro Hospitalar do Porto, Hospital Geral de Santo Antonio
Porto, Portugal, 4099-001
Sweden
Norrlands University Hospital
Umea, Sweden, 90185
Umea Universtiy Hospital
Umea, Sweden, 90187

Sponsors and Collaborators

Pfizer

Investigators

Study Director:	Pfizer CT.gov Call Center	Pfizer

More Information

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Additional Information:

To obtain contact information for a study center near you, click here. 

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Amass L, Li H, Gundapaneni BK, Schwartz JH, Keohane DJ. Influence of baseline neurologic severity on disease progression and the associated disease-modifying effects of tafamidis in patients with transthyretin amyloid polyneuropathy. Orphanet J Rare Dis. 2018 Dec 17;13(1):225. doi: 10.1186/s13023-018-0947-7.

Barroso FA, Judge DP, Ebede B, Li H, Stewart M, Amass L, Sultan MB. Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years. Amyloid. 2017 Sep;24(3):194-204. doi: 10.1080/13506129.2017.1357545. Epub 2017 Jul 31.

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.

Responsible Party:	Pfizer
ClinicalTrials.gov Identifier:	NCT00925002 History of Changes
Other Study ID Numbers:	FX1A-303; B3461023 ( Other Identifier: Alias Study Number ); 2009-011535-12 ( EudraCT Number )
First Posted:	June 19, 2009
Last Update Posted:	March 27, 2018
Last Verified:	March 2018

Studies a U.S. FDA-regulated Drug Product:		Yes
Studies a U.S. FDA-regulated Device Product:		No

Additional relevant MeSH terms:

Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases