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    Summary
    EudraCT Number:2021-004313-39
    Sponsor's Protocol Code Number:TCH-306EXT
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2023-01-30
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2021-004313-39
    A.3Full title of the trial
    A Multicenter, Open-Label, Extension Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults with Growth Hormone Deficiency
    Studio Multicentrico, in Aperto, di Estensione, per Valutare l’Efficacia a Lungo Termine e la Sicurezza di Lonapegsomatropina in Adulti con Deficit dell’Ormone della Crescita
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An extension study to check the safety of once weekly lonapegsomatropin when given for a long time in adults with growth hormone deficiency.
    Studio di estensione per verificare la sicurezza di lonapegsomatropina una volta alla settimana quando somministrata per lungo tempo negli adulti con deficit dell'ormone della crescita.
    A.3.2Name or abbreviated title of the trial where available
    N/A
    N/A
    A.4.1Sponsor's protocol code numberTCH-306EXT
    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 number70222244
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/19/2213
    D.3 Description of the IMP
    D.3.1Product namelonapegsomatropin drug product
    D.3.2Product code [ACP-011]
    D.3.4Pharmaceutical form Powder and solvent for solution for injection/infusion
    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.3.9.1CAS number 1934255-39-6
    D.3.9.2Current sponsor codeACP-011
    D.3.9.4EV Substance CodeSUB197278
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    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 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 Yes
    D.2.5.1Orphan drug designation numberEU/3/19/2213
    D.3 Description of the IMP
    D.3.1Product namelonapegsomatropin
    D.3.2Product code [ACP-011]
    D.3.4Pharmaceutical form Powder and solvent for solution for injection/infusion
    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.3.9.1CAS number 1934255-39-6
    D.3.9.2Current sponsor codeACP-011
    D.3.9.4EV Substance CodeSUB197278
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    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 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
    Adult Growth Hormone Deficiency (AGHD)
    deficit dell’ormone della crescita (GHD/AGHD)
    E.1.1.1Medical condition in easily understood language
    Lack of growth hormone in the body
    Mancanza di ormone della crescita nel corpo
    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 evaluate the safety of once-weekly lonapegsomatropin in adults with GHD previously treated in trial TCH-306.
    valutare la sicurezza della lonapegsomatropina una volta alla settimana negli adulti con deficit dell’ormone della crescita (GHD/AGHD) precedentemente trattati nello studio TCH-306.
    E.2.2Secondary objectives of the trial
    1. To evaluate the efficacy of once-weekly lonapegsomatropin in adults with GHD.
    2. To evaluate the pharmacodynamics (PD) of once-weekly lonapegsomatropin in adults with GHD.
    3. To evaluate the pharmacokinetics (PK) of once-weekly lonapegsomatropin in adults with GHD.
    1. Valutare l'efficacia di lonapegsomatropina una volta alla settimana negli adulti con GHD.
    2. Per valutare la farmacodinamica (PD) di lonapegsomatropina una volta alla settimana negli adulti con GHD.
    3. Valutare la farmacocinetica (PK) di lonapegsomatropina una volta alla settimana negli adulti con GHD.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signing of the trial specific informed consent.
    2. Completion of the treatment period and Visit 7 assessments of trial TCH-306, including collection and upload of Visit 7 DXA scan.
    3. Fundoscopy at Visit 7 in trial TCH-306 without signs/symptoms of intracranial hypertension or diabetic retinopathy stage 2 / moderate or above.
    1. Firma del consenso informato specifico dello studio.
    2. Completamento del periodo di trattamento e valutazione della visita 7 dello studio TCH-306, inclusa la raccolta e il caricamento della scansione DXA della visita 7.
    3. Fundoscopy alla visita 7 nello studio TCH - 306 senza segni/sintomi di ipertensione intracranica o retinopatia diabetica fase 2 / moderata o superiore.
    E.4Principal exclusion criteria
    1. Diabetes mellitus if any of the following are met:
    a. Poorly controlled diabetes, defined as HbA1C higher than 7.5% according to central laboratory at Visit 6 in trial TCH-306
    b. Use of diabetes mellitus drugs other than metformin and/or dipeptidyl peptidase-4 (DPP-4) inhibitors
    2. Active malignant disease or history of malignancy. Exceptions are:
    a. Resection of in situ carcinoma of the cervix uteri
    b. Complete eradication of squamous cell or basal cell carcinoma of the skin
    3. Known history of hypersensitivity and/or idiosyncrasy to the investigational product (somatropin or excipients)
    4. Female who is pregnant, plans to become pregnant, or is breastfeeding
    5. Female participant of childbearing potential (i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile) not willing throughout the trial to use
    contraceptives as required by local law or practice. Details included in Appendix 4/section 10.4 of this protocol
    6. Male participant not willing throughout the trial to use contraceptives as required by local law or practice. Details included in Appendix 4/ section 10.4 of this protocol
    7. Any disease or condition that, in the judgement of the investigator, may make the participant unlikely to comply with the requirements of the protocol or any condition that presents undue risk from the investigational product or trial procedures
    8. eGFR <60 mL/min/1.73m² determined based on Modification of Diet in Renal Disease (MDRD) equation using serum creatinine from the central laboratory at screening
    9. Hepatic transaminases (ie, AST or ALT) >3 times the upper limit of normal according to the central laboratory at screening
    1. Diabete mellito se si verifica una delle seguenti condizioni:
    a. Diabete scarsamente controllato, definito come HbA1c superiore al 7,5% secondo central laboratorio alla visita 6 nella prova TCH-306
    b. Uso di farmaci per il diabete mellito diversi dalla metformina e / o dalla dipeptidil peptidasi-4 (DPP-4) inibitori
    2. Malattia maligna attiva o storia di malignità. Le eccezioni sono:
    a. Resezione del carcinoma in situ della cervice uterina
    b. Completa eradicazione del carcinoma a cellule squamose o basali della pelle
    3. Anamnesi nota di ipersensibilità e / o idiosincrasia al prodotto sperimentale (somatropina o eccipienti)
    4. Donna che è incinta, sta pianificando una gravidanza o sta allattando
    5. Partecipante femminile in età fertile (cioè fertile, dopo il menarca e fino a diventare post-menopausa a meno che non sia permanentemente sterile) non disposto durante la prova ad usare contraccettivi come richiesto dalla legge o dalla pratica locale. Dettagli inclusi nell'appendice 4 / sezione 10.4 del presente protocollo
    6. Partecipante maschio non disposto durante la prova per usare i contraccettivi come richiesto dal locale legge o pratica. Dettagli inclusi nell'appendice 4 / sezione 10.4 del presente protocollo
    7. Qualsiasi malattia o condizione che, a giudizio dello sperimentatore, possa rendere il partecipante è improbabile che rispetti i requisiti del protocollo o qualsiasi condizione che presenti indebite rischio derivante dal prodotto sperimentale o dalle procedure di sperimentazione
    8. eGFR < 60 mL/min/1, 73m2 determinato in base alla modifica della dieta nell'equazione della malattia renale (MDRD) utilizzando la creatinina sierica del laboratorio centrale allo screening
    9. Transaminasi epatiche (cioè, AST o ALT) > 3 volte il limite superiore del normale secondo il laboratorio centrale allo screening
    E.5 End points
    E.5.1Primary end point(s)
    • Adverse events (AEs)
    • Laboratory values
    • Vital signs
    • Immunogenicity
    • 12-lead electrocardiogram (ECGs)
    • Fundoscopy
    * Eventi avversi (AE)
    * Valori di laboratorio
    * Segni vitali
    * Immunogenicità
    * elettrocardiogramma a 12 derivazioni (Ecg)
    * Fundoscopia
    E.5.1.1Timepoint(s) of evaluation of this end point
    throughout the treatment period of 52 weeks
    durante il periodo di trattamento di 52 settimane
    E.5.2Secondary end point(s)
    Efficacy endpoints (as assessed by dual-X-rayabsorptiometry
    (DXA):
    • Trunk percent fat
    • Trunk fat mass
    • Total body lean mass

    PD endpoints:
    • IGF-1 values and IGF-1 SDS

    PK endpoints:
    • hGH, lonapegsomatropin, and mPEG values
    Endpoint di efficacia (valutati mediante assorbimetria a doppio raggio X
    (DXA):
    * Tronco per cento di grasso
    * Massa grassa del tronco
    * Massa magra totale del corpo

    Endpoint PD:
    * IGF-1 valori e IGF-1 SDS

    Endpoint PK:
    * hGH, lonapegsomatropin, e mPEG valori
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy endpoints At week 52
    PD endpoints throughout the treatment period of 52 weeks
    PK endpoints from week 17 to week 52
    Endpoint di efficacia alla settimana 52
    Endpoint PD durante il periodo di trattamento di 52 settimane
    Endpoint PK dalla settimana 17 alla settimana 52
    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 Yes
    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 No
    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 Yes
    E.8.1.7.1Other trial design description
    In aperto
    open-label extension trial
    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.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA48
    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
    Australia
    Canada
    Israel
    Japan
    New Zealand
    United States
    France
    Poland
    Netherlands
    Romania
    Spain
    Germany
    Greece
    Italy
    Armenia
    Georgia
    Slovakia
    Turkey
    Ukraine
    United Kingdom
    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
    ultima visita dell'ultimo soggeto
    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 months0
    E.8.9.1In the Member State concerned days30
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months1
    E.8.9.2In all countries concerned by the trial days9
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 195
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 45
    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 state40
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 240
    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)
    Access to the trial drug after trial end will not be offered to any of the participants. Effective drugs for the indication are commercially available.
    L'accesso al farmaco di prova dopo la fine dello studio non sarà offerto a nessuno dei partecipanti. Farmaci efficaci per l'indicazione sono disponibili in commercio.
    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 Decision2023-08-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-07-17
    P. End of Trial
    P.End of Trial StatusOngoing
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