Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
ATTR-PN |
Drug: Tafamidis |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Open-label Safety And Efficacy Evaluation Of Fx-1006a In Patients With Transthyretin Amyloidosis (fx1a-303) |
- 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 ] [ Designated as safety issue: Yes ]
- 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 ] [ Designated as safety issue: Yes ]
| Estimated Enrollment: | 110 |
| Study Start Date: | August 2009 |
| Estimated Study Completion Date: | December 2021 |
| Estimated Primary Completion Date: | December 2021 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Active Comparator: Open-Label |
Drug: Tafamidis
20 mg oral Fx-1006A daily
|
Eligibility| Ages Eligible for Study: | 18 Years to 75 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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 LocationsPlease refer to this study by its ClinicalTrials.gov identifier: NCT00925002
| Contact: Pfizer CT.gov Call Center | 1-800-718-1021 |
| United States, Maryland | |
| Johns Hopkins Hospital | Active, not recruiting |
| Baltimore, Maryland, United States, 21287 | |
| Argentina | |
| FLENI (Fundacion Lucha contra las Enfermedades Neurologicas Infantiles) | Active, not recruiting |
| Buenos Aires, Argentina, C1428AQK | |
| FLENI Florencia Picone | Active, not recruiting |
| Ciudad de Buenos Aires, Argentina, C1428AQK | |
| Brazil | |
| Hospital Universitario Clementino Fraga Filho | Recruiting |
| Rio de Janeiro, RJ, Brazil, 21941-913 | |
| France | |
| CHU Henri Mondor | Active, not recruiting |
| Creteil cedex, France, 94010 | |
| Germany | |
| Universitatsklinikum Muenster | Active, not recruiting |
| Muenster, Germany, 48149 | |
| University Hospital Medizinische Klinik und Polikinik B | Active, not recruiting |
| Munster, Germany, 48129 | |
| Italy | |
| IRCCS - Policlinico San Matteo | Active, not recruiting |
| Pavia, Italy, 27100 | |
| Centro per lo Studio e la Cura delle Amiloidosi Sistemiche IRCCS Policlinico San Matteo | Active, not recruiting |
| Pavia, Italy, 27100 | |
| Portugal | |
| Hospital de Santa Maria | Active, not recruiting |
| Lisbon, Portugal, 1649-028 | |
| Serviço de Neurologia, piso 7, Hospital de Santa Maria | Active, not recruiting |
| Lisbon, Portugal, 1649-035 | |
| Unidade Clinica de Paramiloidose, EPE - Centro Hospitalar do Porto, Hospital Geral de Santo Antonio | Active, not recruiting |
| Porto, Portugal, 4099-001 | |
| Sweden | |
| Umea University Hospital | Active, not recruiting |
| Umea, SE, Sweden, 901 87 | |
| Neuroscience Center | Active, not recruiting |
| Umea, Sweden, 90185 | |
| Norrland's University Hospital | Active, not recruiting |
| Umea, Sweden, SE-901 85 | |
| Study Director: | Pfizer CT.gov Call Center | Pfizer |
More Information
Additional Information:
No publications provided
| Responsible Party: | Pfizer |
| ClinicalTrials.gov Identifier: | NCT00925002 History of Changes |
| Other Study ID Numbers: | FX1A-303, B3461023, 2009-011535-12 |
| Study First Received: | June 17, 2009 |
| Last Updated: | February 12, 2015 |
| Health Authority: | United States: Food and Drug Administration |
ClinicalTrials.gov processed this record on March 03, 2015