Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis
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| ClinicalTrials.gov Identifier: NCT00925002 |
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Recruitment Status :
Active, not recruiting
First Posted : June 19, 2009
Last Update Posted : March 27, 2018
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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 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 |
| Arm | Intervention/treatment |
|---|---|
| Active Comparator: Open-Label |
Drug: Tafamidis
20 mg oral Fx-1006A daily |
- 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 ]
- 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 ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00925002
| 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 | |
| Study Director: | Pfizer CT.gov Call Center | Pfizer |
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| 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 Key Record Dates |
| 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:
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Amyloidosis Proteostasis Deficiencies Metabolic Diseases |