Safety and Efficacy of Orally Administered Fx-1006A in Patients with Familial Amyloid Polyneuropathy (FAP): a Phase II/III, Randomised, Double-Blind, Placebo-Controlled Study

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2006-002792-41-SE

Enrollment

120

Registered

2006-09-01

Start date

2006-10-30

Completion date

Unknown

Last updated

2012-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Familial Amyloid Polyneuropathy (FAP). MedDRA version: 9.1 Level: LLT Classification code 10057949 Term: Familial amyloid polyneuropathy

Interventions

Product Code: Fx-1006A Pharmaceutical Form: Capsule, soft Current Sponsor code: Fx-1006A Other descriptive name: N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium

2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- Pharmaceutical form of the placebo: Capsule, soft Route of adm

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Documented amyloid by biopsy - Documented V30M TTR mutation - Peripheral and/or autonomic neuropathy with a Karnofsky Performance Status >50. - Aged not less than 18 through 75 years - Female patients must be post-menopausal, surgically sterilised, or willing to use two acceptable methods of birth control (ie. a 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.) If male with a female partner of child bearing potential, willing to use two acceptable methods of birth control for the duration of the study. For both females and males, 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. 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: - 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 - Patients has primary amyloidosis - Patient is pregnant or breast feeding - Prior liver transplantation - No recordable sensory threshold for vibration perception in both feet, as measured by CASE IV - Patients with positive results for HBsAg, anti-HCV, and/or HIV - Renal insuffiency (creatinine clearance 2 times upper limit of normal that in the medical judgement of the Investigator are due to reduced liver function or active liver disease. - NHYA Class >3 - Other causes of sensorimotor neuropathy (B12 deficiency, Diabetes Mellitus, HIV treated with retroviral medications, thyroid disorders, alcohol abuse and chronic inflammatory diseases) - Comorbidity anticipated to limit survival to less than 18 months - Patient has received an investigational drug/device and/or participated in another clinical investigation within 60 days before baseline (Day 0).

Design outcomes

Primary

Measure	Time frame
Main Objective: - To evaluate the effect of Fx-1006A on disease progression in patients with Familial Amyloid Polyneuropathy (FAP). - To evaluate the safety and tolerability of chronic administration of Fx-1006A in patients with FAP.;Secondary Objective: - To determine the pharmacodynamic stablisation effect of Fx-1006A on human V30M TTR. - To determine the population pharmacokinetics in patients with FAP.;Primary end point(s): Co-primary efficacy end points: - Response to treatment at Month 18, as indicated by either improvement (decrease from baseline) or stabilisation (change from baseline of 0 to Grade 3 AE - Incidence of patients experiencing treatment-emergent >Grade 3 clinical laboratory findings. - 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 discontinuing from the study because of clinical or laboratory adverse events.	—

Measure

Time frame

Main Objective: - To evaluate the effect of Fx-1006A on disease progression in patients with Familial Amyloid Polyneuropathy (FAP). - To evaluate the safety and tolerability of chronic administration of Fx-1006A in patients with FAP.;Secondary Objective: - To determine the pharmacodynamic stablisation effect of Fx-1006A on human V30M TTR. - To determine the population pharmacokinetics in patients with FAP.;Primary end point(s): Co-primary efficacy end points: - Response to treatment at Month 18, as indicated by either improvement (decrease from baseline) or stabilisation (change from baseline of 0 to Grade 3 AE - Incidence of patients experiencing treatment-emergent >Grade 3 clinical laboratory findings. - 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 discontinuing from the study because of clinical or laboratory adverse events.

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Countries

Germany, Portugal, Spain, Sweden, United Kingdom

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026