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    Summary
    EudraCT Number:2012-003561-17
    Sponsor's Protocol Code Number:ECP-002
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-04-18
    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 ConcernedUK - MHRA
    A.2EudraCT number2012-003561-17
    A.3Full title of the trial
    A Phase 2 open-label, dose-escalation study to evaluate the safety, pharmacokinetics, immunogenicity and pharmacodynamics/efficacy of EDI200, an EDA-A1 replacement protein, administered to male infants with X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase 2 study to evaluate the safety of EDI200 administered to male infants with X-Linked Hypohydrotic Ectodermal Dysplasia (XLHED).
    A.4.1Sponsor's protocol code numberECP-002
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01775462
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorEdimer Pharmaceuticals, Inc.
    B.1.3.4CountryUnited States
    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 supportEdimer Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEdimer Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address55 Cambridge Parkway, Suite 102W
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02142
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16177584305
    B.5.5Fax number+18663344240
    B.5.6E-mailramsey@edimerpharma.com
    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 numberEU/3/05/334
    D.3 Description of the IMP
    D.3.2Product code EDI200
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNHuman immunoglobulin G1 constant region - human ectodysplasin-A1 receptor-binding domain fusion protein
    D.3.9.2Current sponsor codeEDI200
    D.3.9.3Other descriptive nameFc:EDA-A1 fusion protein
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    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 No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    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 Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    X-linked hypohidrotic ectodermal dysplasia (XLHED) is caused by inherited defects in the ectodysplasin (EDA) gene that disrupt synthesis and/or function of the primary translational product EDA-A1. The absence of normal EDA-A1 expression results in sweat and secretory gland hypoplasia predisposing XLHED-affected infants to serious and potentially life-threatening hyperthermia and pneumonia.
    E.1.1.1Medical condition in easily understood language
    EDI200 is developed as a treatment of a rare genetic disorder, X-linked Hypohidrotic Ectodermal Dysplasia (XLHED) so called Christ-Siemens-Touraine Syndrome.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10072592
    E.1.2Term Hypohidrotic ectodermal dysplasia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the safety, pharmacokinetics and immunogenicity of EDI200 administered to XLHED-affected neonates
    E.2.2Secondary objectives of the trial
    • To assess the pharmacodynamics/efficacy of EDI200 administered to XLHED-affected neonates
    • To compare clinical data and medical history obtained from untreated male siblings to that of the XLHED-affected neonates receiving study drug
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects for study drug administration must meet all of the following criteria to be enrolled:

    1. Male with genetic confirmation of an XLHED diagnosis.
    2. Subject must be at least 48 hours of age and no older than 14 days.
    3. Subject will have reached term (defined as 37 weeks gestation or older) prior to receiving first dose study drug.
    4. Written informed consent of both parents (if reasonably available) must be obtained for treatment of their XLHED-affected male infant.
    5. Neither mother nor the XLHED-affected male infant known to have received an investigational study drug in the 9 months prior to study subject enrollment in this study.
    6. No major medical issues that the PI considers a contraindication to participation.

    Male siblings of subjects receiving study drug must meet all of the following criteria to be enrolled in the natural history sub-study (no age limit involved):

    1. Provide written informed consent/assent.
    2. A full or half-sibling of a study subject where the study subject has received at least one dose of study drug in the Phase 2 XLHED Neonate Study and has not yet completed the study.
    3. No major medical issues that the PI considers a contraindication to participation.
    E.4Principal exclusion criteria
    Subjects for study drug administration who meet any of the following criteria cannot be enrolled in this study:

    1. Medically significant postnatal complications or congenital anomalies outside of those considered to be associated with the diagnosis of XLHED.

    Male siblings of subjects receiving study drug who meet any of the following criteria cannot be enrolled in the natural history sub-study:

    1. Known hypersensitivity to pilocarpine or pilocarpine-like muscarinic agonists.
    2. Known hypersensitivity to lidocaine or lidocaine-like agents.
    3. Presence of pacemaker.
    4. Subjects who are not able or are not willing to comply with the procedures of this protocol.
    5. Subject has a condition, which in the opinion of the PI would not allow for safe conduct of the study.
    E.5 End points
    E.5.1Primary end point(s)
    Treated neonates
    1. Safety laboratory measurements
    2. Physical examination with weight and vital signs
    3. Immunogenicity
    4. Pharmacokinetics
    5. Adverse events and concommitant medication
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Safety laboratory measurements:
    Baseline, treatment days 1, 16, 21 and 2 and 6 months after treatment

    2. Physical examination with weight and vital signs
    Full physical examination: Baseline, treatment days 21, and 2, 4 and 6 months after treatment
    Brief physical examination: TD 0, 1, 4, 7, 11, 14 and 15

    3. Immunogenicity:
    Baseline, treatment days 16 and 2 and 6 months after treatment

    4. Pharmacokinetics:
    Day 0: Baseline, 15 min, 3, 8, 24, 48 hours
    Day 14: prior to treatment, 15 min, 3, 18, 48, 168 hours, 2 and 6 months after treatment

    5. Adverse events and concommitant medication:
    at each visit
    E.5.2Secondary end point(s)
    Treated neonates:
    1. Growth and development
    2. Infections and hospitalizations
    3. Dental radiographs
    4. Antenatal ultrasound results for tooth bud development
    5. Facial photographs
    6. Sweat duct number
    7. Sweat rate
    8. Thermoregulation
    9. Dry eye evaluation
    10. Skin biopsy for expression profile of molecular markers

    Untreated siblings:
    1. Medical history
    2. Physical examination with vial signs and weight
    3. Growth development
    4. Infections/hospitalizations
    5. Dentition
    6. Facial development
    7. Sweat duct number
    8. Sweat rate
    9. Pulmonary function
    10. eNO level
    11. Dry eye assessment
    E.5.2.1Timepoint(s) of evaluation of this end point
    Treated neonates
    1. Growth and development
    Baseline and 2, 4 and 6 months after treatment

    2. Infections and hospitalizations:
    Captured under adverse events at each study visit

    3. Dental radiographs:
    Baseline

    4. Antenatal ultrasound:
    Only if available as part of obsteric care

    5. Facial photographs:
    Baseline and 6 months after treatment

    6. Sweat duct number:
    Baseline and 2 and 6 months after treatment

    7. Sweat rate:
    Baseline and 2 and 6 months after treatment

    8. Thermoregulation:
    Baseline and treatment day 21

    9. Dry eye evaluation:
    Baseline and 2 and 6 months after treatment

    10. Skin biopsy:
    Baseline and treatment days 1, 15

    Untreated siblings
    Medical history: study enrollment

    Endpoints (1-11):
    Prior to study completion of affected sibling
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    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 EEA3
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United States
    E.8.7Trial has a data monitoring committee Yes
    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
    End of study: Last visit of the last subject undergoing the trial (6 month follow up)
    Studienende: Letzte Untersuchung des letzten Studienteilnehmers (Follow-up Untersuchung nach 6 Monaten)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 10
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 10
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Already captured under F.1.1
    Study subjects will be newborns, thus are incapable of giving consent personally due to their age.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 10
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    From 6 months onward (end of data collection in the Phase 2 Study), the EDI200-exposed infants will be enrolled in a long-term extension study with yearly safety and age-appropriate PD/efficacy evaluations.
    Postnatal studies in both mice and dogs demonstrated support for the clinical development of EDI200 as a therapeutic to be administered to XLHED-affected patients in the neonatal period or earlier. Administration of EDI200 after the neonatal period is not supported by animal studies.
    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 Decision2013-06-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-01-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-03-17
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