Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis
| Status: | Active, not recruiting |
|---|---|
| Conditions: | Hematology |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 3/7/2019 |
| Start Date: | August 5, 2009 |
| End Date: | December 31, 2021 |
OPEN-LABEL SAFETY AND EFFICACY EVALUATION OF FX-1006A IN SUBJECTS WITH TRANSTHYRETIN (TTR) AMYLOIDOSIS
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.
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.
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.
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