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    Summary
    EudraCT Number:2017-000701-21
    Sponsor's Protocol Code Number:111-901
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-04-27
    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 ConcernedGermany - BfArM
    A.2EudraCT number2017-000701-21
    A.3Full title of the trial
    A Multicenter, Multinational Clinical Assessment Study for Pediatric
    Patients with Achondroplasia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study looking at growth measurements of children with achondroplasia
    A.4.1Sponsor's protocol code number111-901
    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 SponsorBioMarin Pharmaceutical 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 supportBioMarin Pharmaceutical Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBioMarin Pharmaceutical Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address105 Digital Drive
    B.5.3.2Town/ cityNovato
    B.5.3.3Post code94949
    B.5.3.4CountryUnited States
    B.5.6E-mailMedInfo@bmrn.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP Role
    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.3.4Pharmaceutical form
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNvorsoritide
    D.3.9.4EV Substance CodeSUB120857
    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) 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Achondroplasia
    E.1.1.1Medical condition in easily understood language
    Achondroplasia
    E.1.1.2Therapeutic area Body processes [G] - Bones and nerves physological processes [G11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    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
    The objective of this study is to collect consistent baseline growth
    measurements on pediatric subjects being considered for subsequent
    enrollment in other future studies sponsored by BioMarin.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Individuals eligible to participate in this study must meet all of the following criteria:
    1. Parent(s) or guardian(s) willing and able to provide signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. Also, willing and able to provide written assent (as needed) after the nature of the study has been explained and prior to performance of any research-related procedure.
    2. Birth to ≤ 17 years of age at study entry.
    3. Have ACH, documented by clinical diagnosis
    4. Are ambulatory and able to stand without assistance (not applicable for children who are younger than 5 years of age and less than 104 cm in length)
    5. Are willing and able to perform all study procedures as physically possible
    E.4Principal exclusion criteria
    Individuals who meet any of the following exclusion criteria are not
    eligible to participate in the study:
    1. Have hypochondroplasia or short stature condition other than ACH
    (e.g., trisomy 21, pseudoachondroplasia)
    2. Have any of the following disorders:
    o Hypothyroidism
    o Insulin-requiring diabetes mellitus
    o Autoimmune inflammatory disease (including celiac disease, lupus
    (SLE), juvenile dermatomyositis, scleroderma, and others)
    o Inflammatory bowel disease
    o Autonomic neuropathy
    3. Have an unstable clinical condition likely to lead to intervention during
    the course ofthe study, including progressive cervical medullary
    compression
    4. Growth plates have fused
    5. Have a history of any of the following:
    o Renal insufficiency
    o Anemia
    6. Have a history of cardiac or vascular disease, including the following:
    o Cardiac dysfunction
    o Hypertrophic cardiomyopathy
    o Congenital heart disease
    o Cerebrovascular disease, aortic insufficiency
    o Clinically significant atrial or ventricular arrhythmias
    7. Current treatment with antihypertensive medications, ACE inhibitors,
    angiotensin IIreceptor blockers, diuretics, beta-blockers, calciumchannel
    blockers, cardiacglycosides, systemic anticholinergic agents, any
    medication that may impair orenhance compensatory tachycardia, drugs
    known to alter renal function that isexpected to continue for the
    duration of the study
    8. Have been treated with growth hormone, insulin-like growth factor 1
    (IGF-1), oranabolic steroids in the previous 6 months or long-term
    treatment (> 3 months) at anytime
    9. Have had regular long-term treatment (> 1 month) with oral
    corticosteroids (low-dose ongoing inhaled steroid for asthma is
    acceptable) in the previous 12 months
    10. Concomitant medication that prolongs the QT/QTc interval within 14
    days or5 half-lives, whichever is longer, before the Screening visit
    11. Have used any other investigational product or investigational
    medical device for the treatment of ACH or short stature
    12. Planned or expected bone-related surgery (ie. surgery involving
    disruption of bone cortex), during the study period. Subjects with
    previous bone-related surgery may enroll if surgery occurred at least 12
    months prior to the study and healing is complete without sequelae.
    13. Planned or expected to have limb-lengthening surgery during the
    study period.Subjects with previous limb- lengthening surgery may
    enroll if surgery occurred at least 18 months prior to the study and
    healing is complete without sequelae.
    14. Have any condition that, in the view of the Investigator, places the
    subject at high risk of poor compliance with the visit schedule or of not
    completing the study.
    15. Concurrent disease or condition that, in the view of the Investigator, would interferewith study participation.
    E.5 End points
    E.5.1Primary end point(s)
    Growth measurements at baseline and then subsequently at 3-month
    intervals
    E.5.1.1Timepoint(s) of evaluation of this end point
    Study completion (enrollment in another study), discontinuation of participation, or termination of the study
    E.5.2Secondary end point(s)
    Not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    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 No
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    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 Yes
    E.6.13.1Other scope of the trial description
    Interventional
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Clinical Assessment Study
    E.7.2Therapeutic exploratory (Phase II) No
    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 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 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.8.2.4Number 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    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
    Japan
    Turkey
    United States
    France
    Germany
    Spain
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years12
    E.8.9.2In all countries concerned by the trial months11
    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: 500
    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: 17
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 325
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Minors will be enrolled in this study.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 175
    F.4.2.2In the whole clinical trial 500
    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)
    Patient who have at least 6-month period of pretreatment growth assessment in Study 111-901 might be eligible to be enrolled in studies, 111-202 (phase 2, open-label, sequential cohort dose-escalation study) and 111-301 (phase 3 randomized, double-blind, placebo-controlled, multicenter study).
    Patients who have completed 24 months treatment in Study 111-202 have the option to participate in Study 111-205, a 5-year, open-label extension study.
    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 Decision2017-06-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-06-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-02-11
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