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    Summary
    EudraCT Number:2006-002792-41
    Sponsor's Protocol Code Number:Fx-005
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2007-06-20
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2006-002792-41
    A.3Full title of the trial
    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
    A.3.2Name or abbreviated title of the trial where available
    Not applicable
    A.4.1Sponsor's protocol code numberFx-005
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberNot applicable
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorFoldRx Pharmaceuticals Limited
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEMEA/OD/032/06
    D.3 Description of the IMP
    D.3.2Product code Fx-1006A
    D.3.4Pharmaceutical form Capsule, soft
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeFx-1006A
    D.3.9.3Other descriptive nameN-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium;2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Information not present in EudraCT
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Information not present in EudraCT
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, soft
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Familial Amyloid Polyneuropathy (FAP)
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10057949
    E.1.2Term Familial amyloid polyneuropathy
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - 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.
    E.2.2Secondary objectives of the trial
    - To determine the pharmacodynamic stabilisation effect of Fx-1006A on human V30M transthyretin (TTR).
    - To determine the population pharmacokinetics in patients with FAP.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusion Criteria for All Patients:
    -Patient is aged ≥18 through 75 years.
    -If female, patient is post-menopausal, surgically sterilized, or willing to use two acceptable methods of birth control (i.e., 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 childbearing potential, willing to use two acceptable methods 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.

    Inclusion Criteria for Patients with Positive Biopsy
    -Patient has amyloid documented by biopsy (in accordance with institutional site standard of care)
    -Patient has documented V30M TTR mutation (procedure detailed in a Laboratory Manual).
    -Patient has peripheral and/or autonomic neuropathy with a Karnofsky Performance Status ≥50.

    Inclusion Criteria for Patients with Negative or No Biopsy
    -Patient has documented V30M TTR mutation (procedure detailed in a Laboratory Manual).
    -Patient has a clear family history with first degree relative with a diagnosis of FAP with progressive, sensorimotor polyneuropathy and V30M TTR mutation.
    -Patient has peripheral and/or autonomic neuropathy with a Karnofsky Performance Status ≥ 50
    E.4Principal exclusion criteria
    Exclusion Criteria for All Patients
    -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.
    -Patient has primary amyloidosis.
    -If female, patient is pregnant or breast feeding.
    -Patient has received prior liver transplantation.
    -Patient has no recordable sensory threshold for vibration perception in both feet, as measured by CASE IV.
    -Patients with positive results for hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (HCV), and/or human immunodeficiency virus (HIV).
    -Patient has renal insufficiency (creatinine clearance < 30 ml/min).
    -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.
    -Patient has a New York Heart Association (NYHA) Functional Classification ≥III
    -Patient has a co-morbidity anticipated to limit survival to less than 18 months.
    -Patient received an investigational drug/device and/or participated in another clinical investigational study within 60 days before Baseline.

    Exclusion Criterion for Patients with Positive Biopsy
    -Patient has other causes of sensorimotor neuropathy (B12 deficiency, Diabetes Mellitus, HIV treated with retroviral medications, thyroid disorders, alcohol abuse, and chronic inflammatory diseases).

    Exclusion Criterion for Patients with Negative or No Biopsy
    -Patient has other cause(s) of sensorimotor neuropathy, such as:
    • Vitamin B12 deficiency confirmed at Screening
    • Diabetes Mellitus confirmed by elevated HbA1c at Screening
    • Chemotherapeutic or other associated chronic drug therapy
    • Heavy metal or environmental toxin exposure
    • HIV confirmed by positive HIV antibody at Screening
    • Thyroid disorders, confirmed by abnormal thyroid function at Screening
    • Alcohol abuse confirmed by medical history
    E.5 End points
    E.5.1Primary end point(s)
    Co-primary efficacy endpoints:

    - Response to treatment at Month 18, as indicated by either improvement (decrease from baseline) or stabilisation (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 18 months in the Total Quality of Life (TQOL) score, as measured by the Norfolk QOL-DN.

    Secondary efficacy endpoints:
    - Response to treatment, as indicated by either improvement or stabilisation in the NIS-LL score, at Months 6 and 12.
    - Change from Baseline to Months 6, 12, and 18 in NIS-LL.
    - Change from Baseline to 6 and 12 months in the TQOL score, as measured by the Norfolk QOL-DN.
    - Change from Baseline to 6, 12 and 18 months in the five domains of the Norfolk QOL-DN.
    - Change from Baseline through Month 18 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 through Month 18 in heat pain and cooling thresholds as measured by Quantitative Sensory Testing (QST) utilizing CASE IV.
    - Change from Baseline through Month 18 in modified Body Mass Index (mBMI).
    - TTR stabilization through Month 18, as measured by a validated immunoturbidimetric assay.

    Safety endpoints:
    - Incidence of patients experiencing treatment-emergent serious adverse events.
    - Incidence of patients experiencing treatment-emergent  Grade 3 adverse events.
    - 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.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans Information not present in EudraCT
    E.7.1.2Bioequivalence study Information not present in EudraCT
    E.7.1.3Other Information not present in EudraCT
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The end of the study is defined as the last scheduled protocol activity of the last subject in the study. Follow-up visits for adverse events ongoing at the last scheduled study activity or for repeat laboratory assessments are considered to be part of the subject's ongoing medical care and not study assessments.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months9
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 115
    F.4.2.2In the whole clinical trial 120
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2007-10-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2007-09-06
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2009-06-30
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