Transthyretin Amyloid Polyneuropathy (ATTR-PN) MedDRA version: 9.1 Level: LLT Classification code 10057949 Term: Familial amyloid polyneuropathy
Conditions
Interventions
2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Sponsors
FoldRx Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Inclusion Criteria for male and non-pregnant female patients: - Patient has completed the Month 18 visit of Study Fx-005. - If female, patient is post-menopausal, surgically sterilized, or willing to use an acceptable method of birth control (i.e., a hormonal contraceptive, intra-uterine device, diaphragm with spermicide, condom with spermicide) throughout the study. (A condom alone is not considered an acceptable method of birth control.) If male with a female partner of childbearing potential, willing to use an acceptable method of birth control for the duration of the study. For both females and males, acceptable birth control must be used for at least 3 months after the last dose of study medication. - Patient is, in the opinion of the investigator, willing and able to comply with the study medication regimen and all other study requirements. - Patient agrees not to participate in another investigational drug or device study while participating in this open-label extension 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 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients meeting any of the exclusion criteria will not be enrolled in the study: - Chronic use of non-protocol approved non-steroidal anti-inflammatory drugs (NSAIDs), defined as greater than 3-4 times/month. The following NSAID are allowed: acetylsalicylic acid, etodolac, ibuprofen, indomethicin, ketoprofen, nabumetone, naproxen, nimesulide, piroxicam, and sulindac. - If female, patient is pregnant or breast feeding. - Patient has liver function test abnormalities: alanine transaminases (ALT) and/or aspartate transaminases (AST) >2 times upper limit of normal (ULN) that in the medical judgment of the investigator are due to reduced liver function or active liver disease
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): Safety endpoints: - Incidence of patients experiencing treatment-emergent serious adverse events. - Incidence of patients experiencing treatment-emergent = Grade 3 adverse events. - Incidence of patients with treatment-emergent echocardiography (ECHO) findings considered by the Investigator to be clinically significant. - Incidence of patients with treatment-emergent electrocardiogram (ECG) findings considered by the Investigator to be clinically significant. - Incidence of patients with treatment-emergent Holter Monitor findings considered by the Investigator to be clinically significant. - Incidence of patients discontinuing from the study because of clinical or laboratory adverse events. Efficacy endpoints: - Response to treatment at Months 6 and 12, as indicated by either improvement (decrease from Baseline) or stabilization (change from baseline of 0 to < 2) in the Neurologic Impairment Score – Lower Limb (NIS-LL) score. The NIS-LL score for each study visit will be based on the average of two scores taken at least 24 hours apart within a one-week period. - Change from Baseline to Months 6 and 12 in the Total Quality of Life (TQOL) score, as measured by the Norfolk QOL-DN. - Change from Baseline to Months 6 and 12 in NIS-LL - Change from Baseline to Months 6 and 12 in the five domains of the Norfolk QOL-DN - Change from Baseline to Months 6 and 12 in “+7 composite score” as measured by nerve conduction studies (NCS), vibration detection threshold (VDT) and heart rate response to deep breathing (HRDB) - Change from Baseline to Months 6 and 12 in heat, pain and cooling thresholds as measured by Quantitative Sensory Testing (QST) utilizing CASE IV - Change from Baseline to Months 6 and 12 in modified Body Mass Index (mBMI) - Change from Baseline to Week 6 and Months 3, 6, and 12 in troponin I and NT-pro-BNP levels - IENF density at Baseline - TTR stabilization at Months 6 and 12, as measured by a validated immunoturbidimetric assay ;Main O | — |
Countries
France, Germany, Portugal, Sweden
Outcome results
None listed