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    EudraCT Number:2016-002780-34
    Sponsor's Protocol Code Number:13VR3
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-09-13
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSweden - MPA
    A.2EudraCT number2016-002780-34
    A.3Full title of the trial
    An Open-Label, Long-Term Safety Study of A Long-acting Human Growth Hormone Somavaratan (VRS-317) in Children with Growth Hormone Deficiency
    The VISTA Study: Versartis Long-Term Safety Study of Somavaratan
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Long-Acting Growth Hormone (Somavaratan, VRS-317) in Children
    A.4.1Sponsor's protocol code number13VR3
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02068521
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation PlanP/065/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorVersartis, 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 supportVersartis, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationResearchPoint Global
    B.5.2Functional name of contact pointGlobal Project Manager
    B.5.3 Address:
    B.5.3.1Street Address5301 Southwest Parkway, Suite 100
    B.5.3.2Town/ cityAustin, TX
    B.5.3.3Post code78735
    B.5.3.4CountryUnited States
    B.5.4Telephone number+15124506175
    B.5.5Fax number+15123431684
    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 numberEMA/OD/074/13
    D.3 Description of the IMP
    D.3.1Product nameSomavaratan
    D.3.2Product code VRS-317
    D.3.4Pharmaceutical form 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 INNVRS-317
    D.3.9.1CAS number 1448335-08-7
    D.3.9.3Other descriptive nameVRS-317
    D.3.9.4EV Substance CodeSUB31926
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    E.1.1.1Medical condition in easily understood language
    Growth Hormone Deficiency
    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
    Evaluate the safety of somavaratan during long-term treatment
    E.2.2Secondary objectives of the trial
    Evaluate changes in pharmacodynamic responses (IGF-I and IGFBP-3), bone age, weight, height velocity, height standard deviation scores, metabolic parameters, pubertal development and anti-drug antibody responses during long-term somavaratan treatment
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusion Criteria for Subjects Completing a Previous Somavaratan Study:
    1.Completion of a somavaratan clinical study in pediatric subjects with GHD.
    2.Willing and able to comply with all study procedures.

    Inclusion Criteria for New Treatment Naïve and Switch Subjects:
    1.Chronological Age ≥ 3.0 years.
    2.Pre-pubertal status: Absent breast development in girls, testicular volume < 4.0 mL in boys.
    3.Diagnosis of GHD as documented by two or more GH stimulation test results ≤ 10.0 ng/mL. The use of prior GH stimulation test results is permitted providing the stimulatory agents, GH assay and test results are approved in writing by the Medical Monitor. In localities with different diagnostic criteria, any child meeting the GH stimulation test criteria described here will be considered eligible.
    4.In Sweden, a brain MRI must be obtained during Screening to rule out any contraindications to rhGH treatment (must be obtained within 6 months prior to enrollment).
    5.Normal thyroid function at Screening Visit in subjects not being treated for hypothyroidism. Subjects requiring thyroxine replacement must be considered adequately treated by the PI and Medical Monitor.
    6.Normal adrenal function (morning cortisol and/or local stimulation test) at Screening Visit or within 6 months of the Screening Visit, in subjects not being treated for adrenal insufficiency. Subjects with adrenal insufficiency must receive glucocorticoid treatment for a minimum of 4 weeks before study drug administration.
    7.Pathology relating to cause of GHD must be stable for at least 6 months prior to screening.
    8.Legally authorized representatives must be willing and able to give informed consent.
    E.4Principal exclusion criteria
    Exclusion Criteria for Subjects Completing a Previous Somavaratan Study:
    1.Withdrawal from a somavaratan clinical study in pediatric subjects with GHD.
    2.Use of certain medications with potential to alter responses to the test product including, but not limited to, IGF-I, GH-releasing hormone, and chronic anti-inflammatory doses of glucocorticoids.
    3.Presence of a significant medical condition if such a condition or its treatment can influence the response to study drug (e.g., diabetes, renal failure).
    Exclusion Criteria for New Treatment Naïve and Switch Subjects:
    1.Prior/concomitant treatment with any growth promoting agent (e.g., GH, IGF-I, GH releasing hormone (GHRH), gonadotrophins, sex steroids). Up to 10 day exposures to a growth promoting agent for diagnostic purposes are permitted if administered 30 or more days prior to screening.
    2.Current, significant disease (e.g., diabetes, cystic fibrosis, renal insufficiency). In all cases of concurrent disease, screening must be approved in writing by the Medical Monitor.
    3.Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome (e.g., Russell Silver, Prader Willi, Turner, etc.). Unconfirmed or suspected genetic variants will be considered individually.
    4.Birth weight and/or birth length less than 5th percentile for gestational age using gestational age growth charts.
    5.Prolonged daily (>14 days) use of anti-inflammatory doses of oral glucocorticoids.
    6.Prior history of malignancy.
    7.Treatment with an investigational drug in the 30 days prior to screening.
    8.Known allergy to constituents of the study drug formulation.
    9.Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening.
    10.Significant spinal abnormalities including scoliosis, kyphosis, Chiari malformation, and spina bifida variants.
    11.Significant abnormality in screening studies (as assessed by PI and Medical Monitor).
    12.Current social conditions which would prevent completion of study activities (e.g., planned family move to a distant location).
    13.History of pancreatitis or undiagnosed chronic abdominal pain.
    14.History of spinal or total body irradiation.
    15.Other pituitary hormone deficiencies that are not properly treated.
    16.Unwillingness to provide consent for participation in all trial activities.

    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is safety as assessed by physical examination, inspection of injection sites, vital signs, clinical laboratory determinations (including fasting glucose, insulin, and lipids), 12-lead ECGs (for new treatment naïve subjects and subjects not previously exposed to somavaratan), PK/PD assessments, and immunogenicity assessments.
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of study.
    E.5.2Secondary end point(s)
    Secondary growth endpoints are the annual assessment of height velocity, changes in height SDS, body weight, body mass index, bone age and pubertal status.
    Secondary PD endpoints include changes in IGF-I and IGFBP-3 expressed as concentrations and/or SD scores. These secondary endpoints may be further evaluated to determine potential differences in response due to age and gender, baseline conditions, as well as other measures.
    E.5.2.1Timepoint(s) of evaluation of this end point
    End of study.
    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. 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 Yes
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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
    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
    Last visit last subject
    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 days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    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: 400
    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. of subjects for this age range: 320
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 80
    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 No
    F.3.3.7Others Yes
    F. of other specific vulnerable populations
    Minors > 3.0 years.
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 400
    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)
    Subjects will be treated with VRS-317 until the product is commercially available or until the study is discontinued by the Sponsor, then they will receive medical care at the discretion of the investigator or their treating physician.
    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 Decision2016-11-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-09-28
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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