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The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
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    The EU Clinical Trials Register currently displays   41228   clinical trials with a EudraCT protocol, of which   6756   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .
     
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    Summary
    EudraCT Number:2017-003410-20
    Sponsor's Protocol Code Number:TransCon_hGH_CT-301EXT
    National Competent Authority:Greece - EOF
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-04-19
    Trial 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 ConcernedGreece - EOF
    A.2EudraCT number2017-003410-20
    A.3Full title of the trial
    enliGHten: A Multicenter, Phase 3, Long-term, Open-label Trial Investigating Safety and Efficacy of TransCon hGH Administered Once-Weekly in Children with Growth Hormone Deficiency (GHD) Who Have Completed a Prior TransCon hGH Clinical Trial
    enliGHten: Πολυκεντρική, φάσης 3, μακροχρόνια, ανοικτή μελέτη για την διερεύνηση της ασφαλείας και της αποτελεσματικότητας της TransCon hGH χορηγούμενης μια
    φορά την εβδομάδα σε παιδιά με ανεπάρκεια αυξητικής ορμόνης (GHD) που έχουν ολοκληρώσει προηγούμενη κλινική μελέτη με TransCon hGH
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A long-term extension trial of TransCon hGH, a sustained-release recombinant human growth hormone product, for treatment of Growth Hormone Deficiency in prepubertal children
    A.3.2Name or abbreviated title of the trial where available
    enliGHten
    enliGHten
    A.4.1Sponsor's protocol code numberTransCon_hGH_CT-301EXT
    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 SponsorAscendis Pharma Endocrinology Division A/S
    B.1.3.4CountryDenmark
    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 supportAscendis Pharma Endocrinology Division A/S
    B.4.2CountryDenmark
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAscendis Pharma A/S
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressTuborg Boulevard 12
    B.5.3.2Town/ cityHellerup
    B.5.3.3Post codeDK-2900
    B.5.3.4CountryDenmark
    B.5.4Telephone number004570222244
    B.5.6E-mailclinhelpdesk@ascendispharma.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameTransCon hGH (ACP-011)
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNlonapegsomatropin
    D.3.9.3Other descriptive nameTRANSCON PEG40 HGH
    D.3.9.4EV Substance CodeSUB187256
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number12.1
    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.IMP: 2
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameTransCon hGH (ACP-011)
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNlonapegsomatropin
    D.3.9.3Other descriptive nameTRANSCON PEG40 HGH
    D.3.9.4EV Substance CodeSUB187256
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number24.2
    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
    Growth hormone deficiency (GHD) in prepubertal children
    E.1.1.1Medical condition in easily understood language
    Lack of growth hormone in the body
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10056438
    E.1.2Term Growth hormone deficiency
    E.1.2System Organ Class 10014698 - Endocrine disorders
    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 long-term safety of weekly TransCon hGH in children with GHD previously treated in a phase 3 TransCon hGH trial
    E.2.2Secondary objectives of the trial
    - To assess annualized height velocity (HV) with long-term dosing of weekly TransCon hGH treatment
    - To assess the proportion of subjects with IGF-1 standard deviation score (SDS) in the normal range of 0.0 to +2.0 with long-term dosing of weekly TransCon hGH treatment
    - To evaluate the change in height standard deviation scores (ΔHSDS) with long-term dosing of weekly TransCon hGH treatment
    - To determine the incidence of antibodies against TransCon hGH (anti-hGH and anti-PEG) with long-term dosing of weekly TransCon hGH treatment
    - To assess the preference for weekly TransCon hGH or daily Genotropin
    - To assess the treatment satisfaction of weekly TransCon hGH over time
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Children who have completed a prior phase 3 TransCon hGH trial
    2) Children who have not permanently discontinued investigational product in the prior trial
    3) Written, signed, informed consent of the parent or legal guardian of the subject and written assent of the subject as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC)
    E.4Principal exclusion criteria
    1) Poorly-controlled diabetes mellitus (HbA1c ≥ 8.0%) or diabetic complications
    2) Evidence of closed epiphyses, defined as bone age > 14.0 years for females or > 16.0 years for males
    3) Major medical conditions unless approved by Medical Expert
    4) Known hypersensitivity to the components of the trial medication
    5) Likely to be non-compliant with respect to trial conduct (in regard to the subject and/or the parent/legal guardian/caregiver)
    6) Pregnancy
    7) Any other reason that in the opinion of the investigator would prevent the subject from completing participation or following the trial schedule
    E.5 End points
    E.5.1Primary end point(s)
    Safety Endpoints
    The safety endpoints as measured throughout the long-term dosing of weekly TransCon hGH treatment include the following:
     Incidence of AEs
     Incidence of antibodies against hGH, including neutralizing antibodies
     Incidence of antibodies against PEG
     Incidence of antibodies against TransCon hGH
     Incidence of IGF-1 SDS > 2.0, > 3.0
     Parameters of HbA1c and lipids
     Hormone levels, including thyroid status and morning cortisol
     All other hematology and chemistry parameters
     Vital sign measurements
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the trial
    E.5.2Secondary end point(s)
    Efficacy Endpoints
    The efficacy endpoints of long-term weekly TransCon hGH treatment include the following:
     Annualized HV
     ΔHSDS
     Proportion of subjects with IGF-1 SDS of 0 to +2.0. Additionally, cut points of -2.0 to +2.0 and -1.0 to +2.0 will be assessed.
     IGF-1 SDS
     IGFBP-3 SDS
    Pharmacodynamic Endpoint
     Serum IGF-1 SDS at 5 days ±1 day post-dose
    Other Endpoints
     Preference for weekly TransCon hGH or daily Genotropin treatment
     Satisfaction with weekly TransCon hGH
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the trial
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic Yes
    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 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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 EEA10
    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
    Armenia
    Australia
    Belarus
    Bulgaria
    Canada
    Georgia
    Greece
    Italy
    New Zealand
    Poland
    Romania
    Russian Federation
    Turkey
    Ukraine
    United States
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    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: 300
    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) No
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 199
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 100
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 Yes
    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
    Subjects are toddlers, children and adolescents, and therefore not able to give legal consent. Parents or legal guardian will sign the informed consent. Children and adolescents will sign as well in case they are able to read, understand and sign.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 300
    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)
    Once participation in this extension trial is completed, there will be no follow-up visit except as required to follow serious adverse event (SAEs) that are determined to be related to the study drug.
    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 Decision2018-06-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-04-27
    P. End of Trial
    P.End of Trial StatusOngoing
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