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    Summary
    EudraCT Number:2012-002787-27
    Sponsor's Protocol Code Number:ACP-001_CT-004
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-12-19
    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 ConcernedCzechia - SUKL
    A.2EudraCT number2012-002787-27
    A.3Full title of the trial
    A multicenter, Phase 2, randomized, open label, active-controlled, parallel-group study investigating the safety, tolerability, and efficacy of different dose levels of ACP-001 administered once weekly versus standard daily rhGH replacement therapy in pre-pubertal children with Growth Hormone Deficiency (GHD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of ACP-001, a long-acting human growth hormone product, for treatment of Growth Hormone Deficiency in pre-pubertal children
    A.4.1Sponsor's protocol code numberACP-001_CT-004
    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 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 A/S
    B.4.2CountryDenmark
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAccelsiors CRO and Consultancy Services Ltd
    B.5.2Functional name of contact pointClinical Trials Info
    B.5.3 Address:
    B.5.3.1Street Address103 Háros Str.
    B.5.3.2Town/ cityBudapest
    B.5.3.3Post code1222
    B.5.3.4CountryHungary
    B.5.4Telephone number+361299 00 91
    B.5.5Fax number+361299 00 96
    B.5.6E-mailclinicaltrials@accelsiors.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 nameACP-001
    D.3.2Product code ACP-001
    D.3.4Pharmaceutical form Powder 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 INNsomatropin
    D.3.9.2Current sponsor codeTransCon PEG hGH
    D.3.9.3Other descriptive nameTransiently PEGylated hGH prodrug
    D.3.9.4EV Substance CodeSUB20678
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10.0
    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 RoleComparator
    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 Yes
    D.2.1.1.1Trade name Genotropin
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameRecombinant human Growth Hormone (rhGH)
    D.3.2Product code n/a
    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 INNSOMATROPIN
    D.3.9.1CAS number 12629-01-5
    D.3.9.3Other descriptive namerecombinant DNA-derived human growth hormone
    D.3.9.4EV Substance CodeSUB10584MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5.3
    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 pre-pubertal 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 17.1
    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 compare the safety and PK/PD profile of three different ACP-001 doses to that of a commercially available standard daily rhGH formulation in pre-pubertal children with growth failure due to GHD.
    E.2.2Secondary objectives of the trial
    1.To evaluate the 6-month growth pattern (height velocity)
    2.To select a safe and efficacious dose for pivotal development
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Pre-pubertal children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage I aged:
    a. Boys: 3 years ≤ boy’s age ≤ 12 years
    b. Girls: 3 years ≤ girl’s age ≤ 11 years;
    2. Diagnosis of GHD confirmed by two different GH provocation tests, defined as a peak GH level of ≤ 10 ng/mL, determined by a central laboratory using a validated assay. One well documented historical test (with properly recorded sampling times and results as well as euthyroid status of the patient) performed within 3 months prior to Screening can be accepted to replace one GH stimulation test analyzed by the central laboratory;
    3. Bone age (BA) not greater than the chronological age;
    4. Impaired height and height velocity defined as:
    a. Height (HT) of at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS ≤ - 2.0) according to the CDC standards of 2000
    b. Height velocity (HV) of at least 1 SD below the mean HV for chronological age and sex (HV SDS ≤ - 1.0) according to the standards of Prader et al. of 1989, whereas the time between 2 height measurements is not less than 6 months;
    5. BMI within ±2 SD of the mean BMI for chronological age and sex according to the 2000 CDC standards;
    6. Baseline IGF-I level of at least 1 SD below the mean IGF-I level standardized for age and sex (IGF-I SDS ≤ -1.0) according to the central laboratory reference values;
    7. Normal fundoscopy at Screening (without signs/symptoms of intracranial hypertension);
    8. Children with multiple hormonal deficiencies must be on stable replacement therapy (stable dose and normal blood hormone levels) for other hypothalamo-pituitary axes for at least 3 months. Thyroid replacement therapy for thyroid hormone deficiency must be instituted at least 6 months (and be stable for at least 3 months) prior to Screening. Temporary adjustment of glucocorticoid replacement therapy, as appropriate, is acceptable;
    9. Normal 46 XX karyotype for girls;
    10. Written informed consent of the parent or legal guardian of the patient and written assent of the patient (if the patient is able to read, understand, and sign).
    E.4Principal exclusion criteria
    1. Children with a body weight below 11 kg;
    2. Children with hematocrit at Screening below 34%;
    3. Prior exposure to rhGH or IGF-I therapy;
    4. Children with past or present intracranial tumor growth as confirmed by a sellar MRI scan (with contrast) at Screening (if no MRI was performed within the last 6 months prior to Screening);
    5. Children born small for gestational age (SGA), i.e. birth weight and/or birth length < -2 SD for gestational age;
    6. Malnutrition, defined as:
    a. Serum albumin level below the lower limit of normal (LLN) according to the reference ranges of the central laboratory, and
    b. Serum iron below the lower limit of normal (LLN) according to the reference ranges of the central laboratory, and
    c. BMI < -2 SD for age and sex;
    7. Children with psychosocial dwarfism;
    8. Children with idiopathic short stature;
    9. Other causes of short stature such as coeliac disease (confirmed by anti-transglutaminase antibodies test), hypothyroidism, or rickets;
    10. Presence of anti-hGH binding antibodies at Screening;
    11. History or presence of malignant disease; any evidence of present tumor growth;
    12. Any clinically significant abnormality likely to affect growth or the ability to evaluate growth, e.g. chronic diseases like renal insufficiency, spinal cord irradiation, etc.;
    13. Patients with diabetes mellitus or impaired fasting sugar (based on WHO standards);
    14. Chromosomal abnormalities and medical syndromes (Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, SHOX mutations/deletions and skeletal dysplasias) with the exception of septo-optic dysplasia;
    15. Closed epiphyses;
    16. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids or methylphenidate for attention deficit hyperactivity disorder (ADHD) with the exception of hormone replacement therapies (thyroxine, hydrocortisone,
    desmopressin);
    17. Children requiring glucocorticoid therapy (e.g. asthma) who are taking a dose of greater than 400 μg/d of inhaled budesonide or equivalents for longer than 1 month during a calendar year;
    18. Major medical conditions and/or presence of contraindication to rhGH treatment;
    19. Known or suspected HIV-positive patient;
    20. Known hypersensitivity to the components of the study medication;
    21. The patient and/or the parent/legal guardian are likely to be noncompliant in respect to study conduct;
    22. Participation in any other trial of an investigational agent within 30 days prior to Screening.
    E.5 End points
    E.5.1Primary end point(s)
    Safety endpoints
    •Incidence of AEs;
    •Local tolerability (assessed by the patient and the investigator);
    •Incidence of anti-hGH antibody and incidence of treatment-emergent anti-PEG antibody formation;
    •IGF-I levels;
    •Parameters of glucose metabolism (fasting glucose and insulin level, HbA1c) and lipid parameters;
    •Hormone levels: thyroid status and cortisol levels;
    •All other hematology and biochemistry blood parameters;
    •Results of the physical examinations, vital sign measurements and ECG.

    Efficacy endpoints
    •The relationship between the dose of ACP-001 or daily rhGH and the annualized HV at 6 months;
    •Relationship between delta HT SDS and ACP-001 dose or daily rhGH dose after 3 and 6 months of treatment;
    •Annualized HV during treatment with ACP-001 at the end of 6 months, for each ACP-001 dose group and for the daily rhGH treatment group;
    •Comparison of annualized height velocity between ACP-001 and the corresponding dose of daily rhGH;
    •Change in HT SDS during treatment with ACP-001 at 3 and 6 months compared to baseline for each ACP-001 dose groups and for the daily rhGH treatment group;
    •Change in serum IGF-I levels during treatment with ACP-001, from baseline to end of 6 months for each ACP-001 dose group, compared between the ACP-001 dose groups and to the daily rhGH group;
    •IGF-I standard deviation scores (IGF-I SDS) and proportion of patients achieving normalization of serum IGF-I levels during treatment with ACP-001 (for each dose regimen) or daily rhGH, including comparisons between treatment groups;
    •Change in hGH levels during treatment with ACP-001, from baseline to end of 6 months compared between the ACP-001 dose groups and to the daily rhGH group.
    E.5.1.1Timepoint(s) of evaluation of this end point
    •The relationship between the dose of ACP-001 or daily rhGH and the annualized HV at 6 months;
    •Relationship between delta HT SDS and ACP-001 dose or daily rhGH dose after 3 and 6 months of treatment;
    •Annualized HV during treatment with ACP-001 at the end of 6 months;
    •Change in HT SDS during treatment with ACP-001 at 3 and 6 months compared to baseline;
    •Change in serum IGF-I levels during treatment with ACP-001, from baseline to end of 6 months for each ACP-001 dose group;
    •IGF-I standard deviation scores (IGF-I SDS) and proportion of patients achieving normalization of serum IGF-I levels during treatment with ACP-001 (for each dose regimen) or daily rhGH;
    •Change in hGH levels during treatment with ACP-001, from baseline to end of 6 months.
    E.5.2Secondary end point(s)
    PK and PD endpoints
    •PK profile of hGH from ACP-001 treated patients compared between ACP-001 dose groups and to the PK profile of hGH from the daily rhGH group during V1 and V3;
    •PK profile of TransCon PEG hGH during V1 and V3 (ACP-001 treated patients only);
    •PK profile of PEG during V1 and V3 (ACP-001 treated patients only);
    •PD profile of IGF-I and IGFBP-3 during V1 and V3 compared between the ACP-001 dose groups and to the daily rhGH group.
    E.5.2.1Timepoint(s) of evaluation of this end point
    All PK and PD endpoints will be evaluated during visits V1 and V3
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial4
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA21
    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
    Belarus
    Bulgaria
    Czech Republic
    Egypt
    France
    Germany
    Greece
    Hungary
    Poland
    Romania
    Russian Federation
    Slovenia
    Turkey
    Ukraine
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months8
    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 months2
    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 Yes
    F.1.1Number of subjects for this age range: 52
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 52
    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 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 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
    Patients are between 3-12 years old and therefore not able to give legal consent. Parents or legal guardian will sign the informed consent. Patients 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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 52
    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)
    Treatment after the trial period is at the discretion of the treating physician / investigator. Up to 6 months hGH from commercial and approved source will be provided in case treatment is otherwise not available.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-05-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-02-06
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-07-14
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