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    Summary
    EudraCT Number:2016-003826-18
    Sponsor's Protocol Code Number:111-206
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2018-05-30
    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 ConcernedUK - MHRA
    A.2EudraCT number2016-003826-18
    A.3Full title of the trial
    A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children with Achondroplasia, Age 0 to < 60 Months
    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 and Efficacy of BMN 111 in Infants and Young Children with Achondroplasia
    A.4.1Sponsor's protocol code number111-206
    A.5.4Other Identifiers
    Name:INDNumber:111299
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/379/2017
    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-mailclinicaltrials@bmrn.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/3/12/1094
    D.3 Description of the IMP
    D.3.1Product namemodified recombinant human C-type natriuretic peptide
    D.3.2Product code BMN 111
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation Yes
    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 INNvosoritide
    D.3.9.1CAS number 1480724-61-5
    D.3.9.2Current sponsor codeBMN 111
    D.3.9.3Other descriptive nameMODIFIED RHCNP
    D.3.9.4EV Substance CodeSUB120857
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.8
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/12/1094
    D.3 Description of the IMP
    D.3.1Product namemodified recombinant human C-type natriuretic peptide
    D.3.2Product code BMN 111
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation Yes
    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 INNvosoritide
    D.3.9.1CAS number 1480724-61-5
    D.3.9.2Current sponsor codeBMN 111
    D.3.9.3Other descriptive nameMODIFIED RHCNP
    D.3.9.4EV Substance CodeSUB120857
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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: 3
    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/3/12/1094
    D.3 Description of the IMP
    D.3.1Product namemodified recombinant human C-type natriuretic peptide
    D.3.2Product code BMN 111
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation Yes
    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 INNvosoritide
    D.3.9.1CAS number 1480724-61-5
    D.3.9.3Other descriptive nameMODIFIED RHCNP
    D.3.9.4EV Substance CodeSUB120857
    D.3.10 Strength
    D.3.10.1Concentration unit µg/kg microgram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboLyophilisate and solvent for solution 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
    E.1.1.1Medical condition in easily understood language
    Dwarfism
    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 primary objectives of the study are to:
    - Evaluate the safety and tolerability of BMN 111 in children age 0 to < 60 months with ACH
    - Evaluate the effect of BMN 111 on change from baseline in length/ height Z-score
    E.2.2Secondary objectives of the trial
    - Evaluate the effect of BMN 111 on change from baseline in AGV throughout the 52 weeks of the study
    - Evaluate the effect of BMN 111 on bone morphology/quality by x-ray and dual x-ray absorptiometry (DXA)
    - Evaluate the PK of BMN 111 in children age 0 to < 60 months with ACH
    - Evaluate hip function
    - Evaluate for hip, thigh, or knee pain, or change in gait from medical history
    - Evaluate the effect of BMN 111 on health related quality of life (HRQol), developmental status, and functional using age-specific QoL and functional independence questionnaires (Bayley-Scales of Infant and Toddler Development, Third eddition [Bayley III], Activity of Daily Living and Functional Independence Meaure (Wee-FIM), Infant Toddler Quality of Life Questionnaire (ITQOL), Child Behavior Checklist [CBCL]
    - Evaluate immunogenicity of BMN 111 and assess impact on safety, PK, and efficacy measures
    - Evaluate the effect of BMN 111 on bone metabolism and BMN 111 pharmacodynamic biomarkers
    Evaluate the effect of BMN 111 on growth parameters and body
    proportions, including change from baseline in upper:lower segment
    body ratio
    Evaluate the effect of BMN 111 on sleep apnea
    Evaluate the effect of BMN 111 on skull and brain morphology, including
    foramen magnum, ventricular and brain parenchymal dimensions
    Describe the incidence of surgical interventions, including cervical
    decompression, adenotonsillectomy,and tympanostomy
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Diagnosis of ACH, confirmed by genetic testing. If subjects had previous genetic testing, subjects must have a lab report from a certified laboratory with the study specific mutation documented.
    2. Age 0 to < 60 months, at study entry (Day 1)
    3. Cohort 1 and 2 subjects must have at least a 6-month period of pre-treatment growth assessment in Study 111-901 immediately before screening and have one documented measurement of height/body length a minimum of 6 months (+/- 10 days) prior to the screening visit for 111-206. Cohort 3 subjects must have a minimum of 3 months of observation prior to treatment. This observational period can be obtained either (1) via prior enrolment in Study 111-901 or (2) via enrolment in this Study 111-206 for a minimum of 3 months of non-treatment observation prior to commencement of treatment.
    4. Parent(s) or guardian(s) (and the subjects themselves, if required by local regulations or ethics committee) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure
    5. Willing and able to perform all study procedures as physically possible
    6. Parent(s) or caregiver(s) are willing to administer daily injections to the subjects and complete the required training.

    Inclusion Criteria for Cohort 3 Observation Period

    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 (if applicable) after the nature of the study has been explained and prior to performance of any research-related procedure.
    2.Birth to ≤ 3 months of age at study entry.
    3.Have ACH, documented by genetic testing
    4.Are willing and able to perform all study procedures as physically possible
    After completing observation period subjects must fulfil the general eligibility criteria prior to receiving treatment with study drug.
    E.4Principal exclusion criteria
    1. Have hypochondroplasia or short-stature condition other than achondroplasia (e.g., trisomy 21, pseudoachondroplasia, etc.)
    2. Subject weighs < 5.0 kg (Cohort 1 and 2) or < 4.0 kg (Cohort 3; treatment phase)
    3. Have any of the following:
    - Hypothyroidism or hyperthyroidism
    - Insulin-requiring diabetes mellitus
    - Autoimmune inflammatory disease (including celiac disease, systemic lupus erythematosus, juvenile dermatomyositis, scleroderma, etc.)
    - Inflammatory bowel disease
    - Autonomic neuropathy
    4. Have a history of any of the following:
    - Renal insufficiency defined as serum creatinine > 2 mg/dL
    - Chronic anemia or Hgb < 10.0 g/dL (based on screening clinical laboratory testing)
    - Baseline systolic blood pressure (BP) below age and gender specified normal range or recurrent symptomatic hypotension (defined as episodes of low BP generally accompanied by symptoms e.g., dizziness, fainting) or recurrent symptomatic orthostatic hypotension
    - Cardiac or vascular disease, including the following
    o Cardiac dysfunction (abnormal echocardiogram determined to be
    clinically significant by PI and medical monitor) at Screening Visit
    o Hypertrophic cardiomyopathy
    o Pulmonary hypertension
    o Congenital heart disease with ongoing cardiac dysfunction
    o Cerebrovascular disease
    o Aortic insufficiency or other clinically significant valvular dysfunction
    o Clinically significant atrial or ventricular arrhythmias
    5. Have a clinically significant finding or arrhythmia that indicates abnormal cardiac function or conduction or QTc-F > 450 msec on screening ECG
    6. Have evidence of cervicomedullary compression (CMC) likely to require surgical intervention within 60 days of Screening as determined by the Investigator and informed on the following assessments:
    - Physical exam (e.g., neurologic findings of clonus, opisthotonus, exaggerated reflexes, dilated facial veins)
    - Polysomnography (e.g., severe central sleep apnea)
    - MRI indicating presence of severe CMC or spinal cord damage
    7. Have an unstable medical condition likely to require surgical intervention in the next 6 months, or planned spine or long-bone surgery (i.e., surgery involving significant disruption of bone cortex) during the study period
    8. Have documented uncorrected Vitamin D deficiency: 25(OH)D ≤ 15 ng/mL (37.5 nmol/L)
    9. Require any other investigational product prior to completion of the study period
    10. Have received another investigational product or investigational medical device within 30 days prior to the Screening visit
    11. Have used any other investigational product or investigational medical device for the treatment of achondroplasia or short stature at any time
    12. Require current chronic therapy with antihypertensive medication or any medication that, in the investigator’s judgment, may compromise the safety or ability of the subject to participate in this clinical study
    13. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to screening, or long-term treatment (> 3 months) at any time
    14. Have had regular long-term treatment (> 1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma, or intranasal steroids, are acceptable) in the 12 months prior to screening.
    15. Have ever had CMC surgery (Cohorts 2 and 3 only), spine or long-bone surgery (i.e., surgery involving disruption of bone cortex) or have ever had bone-related surgery with chronic complications.
    NOTE: Subjects with prior cervicomedullary decompression may be allowed into Cohort 1 only after discussion and agreement with the Medical Monitor.
    16. Have ever had limb-lengthening surgery or plan to have limb-lengthening surgery during the study period.
    17. Have had a fracture of the long bones or spine within 6 months prior to screening.
    18. Have aspartate aminotransferase (AST) or alanine aminotransferase (ALT) or total bilirubin greater than upper limit of normal (ULN) at screening (except for subjects with a known history of Gilberts or newborns entering screening in Cohort 3).
    19. Have evidence of severe untreated sleep apnea; or have newly initiated sleep apnea treatment (eg, continuous positive airway pressure [CPAP] or sleep apnea-mitigating surgery) in the 2 months prior to screening.
    20. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
    21. Have known hypersensitivity to BMN 111 or its excipients
    22. Have a history of hip surgery or severe hip dysplasia.
    23. Have a history of clinically significant hip injury in the 30 days prior to screening.
    24. Have a history of slipped capital femoral epiphysis or avascular necrosis of the femoral head



    25. Have abnormal findings on baseline clinical hip exam or imaging assessments that are determined to be clinically significant as determined by the Investigator.
    26. Have a condition or circumstance that, in the view of the Investigator, places the subject at high risk for poor treatment compliance or for not completing the study. 27. Have any concurrent disease or condition that, in the view of the Investigator, would interfere with study participation or safety evaluations, for any reason

    Exclusion Criteria for Cohort 3 Observation Period

    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:
    •Hypothyroidism
    •Insulin-requiring diabetes mellitus
    •Autoimmune inflammatory disease (including celiac disease, lupus (SLE), juvenile dermatomyositis, scleroderma, and others)
    •Inflammatory bowel disease
    •Autonomic neuropathy
    3.Have an unstable clinical condition likely to lead to intervention during the course of the study, including progressive cervical medullary compression
    4.Have a history of any of the following:
    •Renal insufficiency
    •Anemia
    5.Have a history of cardiac or vascular disease, including the following:
    •Cardiac dysfunction
    •Hypertrophic cardiomyopathy
    •Congenital heart disease
    •Cerebrovascular disease, aortic insufficiency
    •Clinically significant atrial or ventricular arrhythmias
    6.Current treatment with antihypertensive medications, ACE inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium-channel blockers, cardiac glycosides, systemic anticholinergic agents, any medication that may impair or enhance compensatory tachycardia, drugs known to alter renal function that is expected to continue for the duration of the study
    7.Have had regular long-term treatment (> 1 month) with oral corticosteroids (low dose ongoing inhaled steroid for asthma is acceptable) in the previous 3 months
    8.Concomitant medication that prolongs the QT/QTc interval within 14 days or 5 half lives, whichever is longer, before the Screening visit
    9.Have used any other investigational product or investigational medical device for the treatment of ACH or short stature
    10.Planned or expected bone-related surgery (ie. surgery involving disruption of bone cortex), during the study period.
    11.Planned or expected to have limb-lengthening surgery during the study period.
    12.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.
    13.Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation
    After completing observation period subjects must fulfill the general eligibility criteria prior to recieving treatment with study drug.
    E.5 End points
    E.5.1Primary end point(s)
    - To assess the safety and tolerability of daily SC BMN 111 administered
    in children age 0 to < 60 months with ACH
    - To evaluate the effect of BMN 111 on length/height Z-score.
    E.5.1.1Timepoint(s) of evaluation of this end point
    -Duration of trial
    -Anthropometric Measurement: Screening/ Baseline, Day 1, Week 6, Week 13, Week 26, Week 39, Week 52
    -Imaging assessments (X-rays of the spine and entire lower extremities,
    DXA of total body less head and spine)
    -CBCL
    E.5.2Secondary end point(s)
    The secondary efficacy endpoints include:
    1) Change from baseline in AGV (annualized to cm/yr),
    2) Change from baseline in bone morphology/quality,
    3) PK sampling,
    4) hip function,
    5) hip, thigh, or knee pain, or change in gait from medical history,
    6) developmental/functional/QOL status
    7) Evaluate immunogenicity of BMN 111
    8) Biomarker samples to evaluate the effect of BMN 111 on bone metabolism and BMN 111 pharmacodynamic biomarkers. Growth
    parameters and body proportions, sleep apnea, skull and brain
    morphology, and clinical outcome assessments (developmental/functional/HRQoL status).
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) Anthropometric Measurement: Screening/ Baseline, Day 1, Week 6, Week 13, Week 26, Week 39, Week 52
    2) DXA and AP and lateral X-rays: Screening/ Baseline, Week 52
    3) Day 1, Week 13, Week 26, Week 39, Week 52
    4) Screening/ Baseline, Week 26, Week 52
    5) Screening/ Baseline, Day 1, Day 2, Day 3, Day 8, Week 3, Week 6, Week 13, Week 20, Week 26, Week 39, Week 52, Week 56
    6) Screening/ Baseline, Week 26, Week 52
    7) Day 1, Week 13, Week 26, Week 52
    8)
    - Blood and urine biomarkers: Screening/ Baseline, Day 1, Day 2, Day 3, Day 8, Week 3, Week 6, Week 13, Week 20, Week 26, Week 39, Week 52
    - Biomarker samples (optional): Week 6 and Week 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 Yes
    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 No
    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 trial2
    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 No
    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
    United Kingdom
    United States
    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
    LPLV
    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 months0
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    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: 70
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 3
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 37
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 30
    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 in this protocol will be under 5 years old, so not capable of giving consent
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 14
    F.4.2.2In the whole clinical trial 70
    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)
    Following completion of the study, subjects in both treatments groups may be eligible to receive BMN 111 in an open-label extension study, to assess safety and efficacy of BMN 111 over the longer term.
    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 Decision2018-07-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-09-12
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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