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    Summary
    EudraCT Number:2019-002754-22
    Sponsor's Protocol Code Number:TCC-201
    National Competent Authority:Ireland - HPRA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-09-03
    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 ConcernedIreland - HPRA
    A.2EudraCT number2019-002754-22
    A.3Full title of the trial
    ACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 52 weeks in prepubertal children with achondroplasia followed by an Open-Label Extension Period.
    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, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia followed by an Open-Label Extension Period
    A.3.2Name or abbreviated title of the trial where available
    ACcomplisH
    A.4.1Sponsor's protocol code numberTCC-201
    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 Growth Disorders 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 Growth Disorders 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 CNP 3.9 mg CNP-38/vial
    D.3.2Product code TransCon CNP
    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 INNC-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
    D.3.9.1CAS number 2413551-27-4
    D.3.9.2Current sponsor codeTransCon CNP
    D.3.9.3Other descriptive nameC-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
    D.3.9.4EV Substance CodeSUB193224
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3.9
    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) 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 No
    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 placeboPowder and solvent for solution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Achondroplasia (ACH) in prepubertal children
    E.1.1.1Medical condition in easily understood language
    Achondroplasia (skeletal growth disorder) in children
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 25.0
    E.1.2Level LLT
    E.1.2Classification code 10000452
    E.1.2Term Achondroplasia
    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
    In prepubertal children with achondroplasia (ACH) at 52 weeks
    • To determine the safety of once weekly subcutaneous (SC) doses of
    TransCon CNP
    • To evaluate the effect of once weekly SC doses of TransCon CNP on
    annualized height velocity (AHV)
    E.2.2Secondary objectives of the trial
    • To evaluate the effect of once weekly SC doses of TransCon CNP on body proportionality (upper to lower body segment ratio) in prepubertal children with ACH at 52 weeks.
    • To evaluate the pharmacokinetic (PK) properties of once weekly SC
    doses of TransCon CNP
    • To assess the potential immunogenic response to once weekly SC doses of TransCon CNP
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Clinical diagnosis of ACH with genetic confirmation
    2. Age between 2 to 10 years old (inclusive) at Screening Visit
    3. Prepubertal (Stage 1 breasts for girls or testicular volume < 4ml for boys) at Screening Visit
    4. Able to stand without assistance
    5. Caregiver willing and able to administer subcutaneous injections of study drug
    6. Written, signed informed consent of the parent(s) or legal guardian(s) of the participant and written assent of the participant as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC)
    E.4Principal exclusion criteria
    1.Clinically significant findings at Screening that:
    •are expected to require surgical intervention during participation in the trial or
    •are musculoskeletal in nature, such as Salter-Harris fractures and severe hip pain or
    •otherwise are considered by investigator or Medical Monitor to make a participant unfit to receive study drug or undergo trial related procedures
    2.Have received treatment (>3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time
    3.Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit
    4.Have received any study drug or device intended to affect stature or body proportionality at any time
    5.History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones
    6.History of any bone-related surgery that affects growth potential of long bones, such as orthopedic reconstructive urgery and osteotomy (Limb-lengthening with full recovery is allowed with a minimum of 12 months of bone healing. Foramen magnum decompression and laminectomy with full recovery are allowed with minimum of 6 months of bone healing. History of 8 plate epiphysiodesis is allowed, but the plates must have been removed prior to Screening with minimum 4 weeks of healing).
    7.Have a form of skeletal dysplasia other than ACH or known medical conditions that result in short stature or abnormal growth.
    8.History or presence of malignant disease, other than basal cell epithelioma/carcinoma or completely resected squamous skin cancer with no recurrence for 12 months per medical records
    9.History or presence of the following:
    •Chronic anemia (resolved iron deficiency anemia is allowed)
    •Significant cardiovascular disease per the judgement of the investigator, such as congenital heart disease (uncomplicated patent ductus arteriosus and atrial or ventricular septal defect with repair are allowed), aortic insufficiency, clinically significant arrhythmias, congestive heart failure with NYHA class II and above, or other conditions that impair regulation of blood pressure or heart rate
    •Condition that impacts hemodynamic stability (such as autonomic dysfunction, orthostatic intolerance)
    •History of chronic renal insufficiency
    •Chronic or recurrent illness that can affect hydration or volume status. This may include conditions associated with decreased nutritional intake
    or increased volume loss
    •Bone fracture within 6 months prior to Screening Visit (within 2 months for fracture of digits)
    •Any disease or condition that, in the opinion of the investigator, may make the participant unlikely to fully complete the trial, may confound interpretation of trial results, or presents undue risk from receiving study drug
    10.Child has significant electrocardiogram abnormalities, including evidence of a previous myocardial infarction, left ventricular hypertrophy, flat T waves (particularly in the inferior leads) or more
    than minor non-specific ST-T wave changes or:
    •QRS>90 milliseconds (msec)
    •QT interval corrected using Fridericia's formula (QTcF)>440 msec
    •PR interval>170 msec
    •Complete right or left bundle branch block
    11.Requires, or anticipated to require, chronic (>4 weeks) or repeated (more than twice per year) treatment with oral corticosteroids during participation in the trial (low and mid-dose inhaled corticosteroids are allowed. High-dose inhaled corticosteroids are not allowed.). See Appendix 4 for definition of high doses of inhaled corticosteroids.
    12.Use of medication known to prolong the QT/QTc interval within 7 days or 5 half-lives (whichever is longer) prior to the Screening Visit (see https://crediblemeds.org/ for the list of medications that are
    known to prolong the QT/QTc interval. Note: Only medications on the
    Known Risk list are excluded, not those on the Possible or Conditional Risk lists)
    13.Ongoing treatment with any medication that affects blood pressure or heart rate
    14.Known hypersensitivity to the components of the study drug [trehalose, tris(hydroxymethyl)aminomethane, succinate and PEG]
    15.Any other reason that in the opinion of the investigator would prevent the child from complying with the trial requirements or completing the trial
    E.5 End points
    E.5.1Primary end point(s)
    Safety Endpoints
    The following safety endpoints will be assessed for both blinded
    Randomized Period and Open-Label Extension Period:
    • Incidence of AEs
    • All blood chemistry, hematology, lipid panel, and urinalysis parameters
    • Vital sign measurements and physical examination assessments
    • 12-lead ECG
    • Radiographic findings from:
    - Bone age X-ray
    - DXA
    - AP standing lower extremity X-ray
    - AP and lateral spine X-ray
    • Incidence of anti-drug antibodies

    Efficacy Endpoints
    • AHV as measured at 52 weeks of weekly TransCon CNP treatment or placebo

    Pharmacokinetic Endpoints
    • Plasma concentration of Total CNP
    • Plasma concentration of Free CNP
    • Plasma concentration of mPEG and mPEG-linker
    E.5.1.1Timepoint(s) of evaluation of this end point
    Efficacy will be evaluated at 52 weeks but safety and PK will be evaluated at 52 weeks and during the open label extension
    E.5.2Secondary end point(s)
    • Change in upper to lower body segment ratio as measured at 52 weeks of weekly TransCon CNP treatment or placebo
    E.5.2.1Timepoint(s) of evaluation of this end point
    At 52 weeks of weekly TransCon CNP treatment or placebo
    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 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 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 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 Yes
    E.8.1.7.1Other trial design description
    Dose escalation trial
    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.8.2.4Number of treatment arms in the trial6
    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 EEA11
    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
    Australia
    Canada
    New Zealand
    United States
    Switzerland
    Austria
    Denmark
    Germany
    Ireland
    Portugal
    Spain
    United Kingdom
    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 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: 60
    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: 60
    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
    Subjects are children who are not able to give legal consent. Parents or
    legal guardian will sign the informed consent. Children 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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 60
    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)
    All subjects who successfully complete TCC-201 per protocol will be
    invited to screen for an open-label long-term safety trial. The objective
    of the open-label long-term safety trial is to assess the safety and
    efficacy of long-term treatment with weekly TransCon CNP. For any
    participant who chooses to not participate in the open-label long-term
    safety trial, correspondence with the participant's caregiver will be
    conducted at least 5 weeks after final dose to evaluate for AEs.
    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 Decision2019-11-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-15
    P. End of Trial
    P.End of Trial StatusOngoing
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