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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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EUCTR |
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Last refreshed on:
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24 July 2012 |
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Main ID: |
EUCTR2008-001262-87-PT |
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Date of registration:
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21/04/2008 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Scientific title:
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An Open-Label Extension of Study Fx-005 Evaluating Long-Term Safety and Clinical Outcomes of Fx-1006A in Patients with Transthyretin Amyloid Polyneuropathy - Not applicable |
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Date of first enrolment:
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04/07/2008 |
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Target sample size:
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107 |
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Recruitment status: |
Not Recruiting |
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URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-001262-87 |
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Study type:
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Interventional clinical trial of medicinal product |
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Study design:
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Controlled: no
Randomised: no
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: no
Other: no
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Phase:
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Countries of recruitment
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France
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Germany
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Portugal
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Sweden
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Contacts
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Key inclusion & exclusion criteria
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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:
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
Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
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Health Condition(s) or Problem(s) studied
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Transthyretin Amyloid Polyneuropathy (ATTR-PN)
MedDRA version: 9.1
Level: LLT
Classification code 10057949
Term: Familial amyloid polyneuropathy
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Intervention(s)
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Product Name: Fx-1006A
Product Code: Fx-1006A
Pharmaceutical Form: Capsule, soft
CAS Number: 951395-08-7
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-
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Primary Outcome(s)
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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
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Secondary Objective: - To determine the pharmacodynamic stabilization effect of Fx-1006A on human V30M transthyretin (TTR);
- To obtain additional pharmacokinetic samples for population pharmacokinetics analysis in this patient population.
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Main Objective: - To evaluate the long-term safety and tolerability of chronic administration of Fx-1006A in patients with Transthyretin Amyloid Polyneuropathy (ATTR-PN);
- To evaluate the long-term effects of Fx-1006A on disease progression in patients with ATTR-PN.
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Secondary ID(s)
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Fx-006
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Not applicable
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Source(s) of Monetary Support
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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