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    Summary
    EudraCT Number:2015-002548-15
    Sponsor's Protocol Code Number:VTS301
    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:2015-09-02
    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 ConcernedUK - MHRA
    A.2EudraCT number2015-002548-15
    A.3Full title of the trial
    A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects with Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects with Niemann-Pick Type C1 Disease
    A.4.1Sponsor's protocol code numberVTS301
    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 SponsorVtesse, Inc. a Mallinckrodt Pharmaceutical Company
    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 supportVtesse, Inc. a Mallinckrodt Pharmaceutical Company
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVtesse, Inc. a Mallinckrodt Pharmaceutical Company
    B.5.2Functional name of contact pointSusan VanMeter, MD
    B.5.3 Address:
    B.5.3.1Street Address1425 US Route 206
    B.5.3.2Town/ cityBedminster
    B.5.3.3Post codeNJ 07921
    B.5.3.4CountryUnited States
    B.5.4Telephone number001240.278.6860
    B.5.6E-mailSusan.VanMeter@mnk.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/3/13/1124
    D.3 Description of the IMP
    D.3.1Product nameVTS-270
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNHP-β-CD
    D.3.9.1CAS number 128446-35-5
    D.3.9.2Current sponsor codeHP-β-CD
    D.3.9.3Other descriptive name2-HYDROXYPROPYL-BETA-CYCLODEXTRIN
    D.3.9.4EV Substance CodeSUB21143
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease
    E.1.1.1Medical condition in easily understood language
    Niemann-Pick Type C1 (NPC1) Disease
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A:
    The primary study objective in Part A was to select the dose of VTS-270 to be used in Part B and Part C. Dose selection criteria included safety and tolerability including a thorough audiologic evaluation. Preliminary efficacy data was provided to the Dose Selection Committee (DSC) to assist, if necessary, in dose selection.

    Part B:
    The primary study objective in Part B is to evaluate, in a double-blind sham-controlled design, the progression of the neurologic manifestations of NPC1 disease following 52 weeks of treatment for subjects treated with VTS-270 compared to sham control, using the following assessments:
    •The NPC-SS composite which consists of the sum of 4 components of the Neimann Pick Type C Severity Scale (NPC-SS): ambulation, fine motor, cognition, and swallowing.
    •The blinded Clinician-Global Impression of Change (CGIC)

    Part C:
    The primary study objective in Part C is to evaluate the long-term safety, tolerability, and efficacy of VTS 270.
    E.2.2Secondary objectives of the trial
    Part A:
    There are no secondary objectives for Part A.

    Part B:
    The secondary objectives for Part B are divided into 2 categories: key secondary objective and other secondary objectives.
    The key secondary objective for Part B is to evaluate the progression of the neurologic manifestations of NPC1 disease using the total NPC-SS score, excluding the hearing domain and auditory brainstem response (ABR) modifier results, following 52 weeks of treatment for subjects treated with VTS-270 compared to sham controls and the Caregiver CGIC.

    Part C:
    The secondary study objectives in Part C are to:
    1. Assess the safety and tolerability of the B. Braun Celsite Spinal Access Port utilized to administer VTS-270 (device safety and tolerability substudy).
    2. Assess the plasma and CSF PK of VTS-270 and trough HP-β-CD concentration in subjects receiving the 900 mg dose of VTS-270 via the B. Braun Celsite Spinal Access Port (device PK substudy).
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    (1) Part C European Site-Specific Device Safety and Tolerability Substudy and (2) European Site-Specific Device PK Substudy.
    The both sub-studies are included into the main Protocol v11.0 dated 24-Aug-2018.

    Objectives for substudy (1):
    Assess the safety and tolerability of the B. Braun Celsite Spinal Access Port utilized to administer VTS-270 in approximately 6 subjects who are currently enrolled in VTS301 Part C.

    Objectives for substudy (2):
    Assess the plasma and CSF PK of VTS-270 and trough HP-β-CD concentration in subjects receiving the 900 mg dose of VTS-270 via the B. Braun Celsite Spinal Access Port.

    E.3Principal inclusion criteria
    1. Male or female subjects, 4 to 21 years of age at time of screening with onset of neurological symptoms prior to 15 years of age.
    2. Diagnosis of NPC1 determined by one of the following:
    a. Two NPC1 mutations;
    b. Positive filipin staining or oxysterol testing and at least one NPC1 mutation;
    c. Vertical supranuclear gaze palsy (VSNGP) in combination with either:
    i. One NPC1 mutation, or
    ii. Positive filipin staining or oxysterol levels consistent with NPC1 disease and no NPC2 mutations.
    3. Subject or parent/guardian must provide written informed consent to participate in the study. In addition to parental consent, assent to participate must also be sought from minor children.
    4. Ability to undergo a LP and IT drug administration under monitored anesthesia care (conscious sedation) or if medically necessary, general anesthesia.
    5. An NPC SS score of 0 through 4, inclusive, on the cognition component, and 1 through 4 in two or more of the following components: ambulation, fine motor skills, or swallowing.
    6. Total NPC SS score of 10 or greater.
    7. Subjects with adequately controlled seizures may qualify for enrollment. Subjects with a history of seizures should have a stable
    pattern of seizure activity and be on a stable dose and regimen of antiepileptic medication during the 3 months prior to screening without
    change in dose or regimen up to and including Study Day 0.
    8. If taking miglustat, must have been on a stable dose for the past 6 to 8 weeks and be willing to remain on a stable dose for the duration of participation in Parts A and B of the study. Alternatively, subjects may elect to discontinue miglustat use and be eligible for trial entry after
    undergoing a minimum 6 week washout period prior to Study Day 0.
    9. Agree to discontinue all nonprescription supplements such as coenzyme Q10, curcumin, cinnamon, fish oil supplements, high dose vitamin D (>500 mIU/day), N-acetylcystine, acetylleucine, or gingko biloba at least 1 month prior to first dose (Study Day 0). Note: Daily administration of an age-appropriate multivitamin is allowed.
    10. Agree to discontinue any other investigational treatments for NPC including, for example, vorinostat or arimoclomol, acetylleucine, or
    curcumin at least 3 months prior to first dose (Study Day 0).
    11. Females of childbearing potential (not surgically sterile) must use a medically acceptable method of contraception and must agree to continue use of this method for the duration of the study and for 30 days after participation in the study. Acceptable methods of contraception include barrier method with spermicide, intrauterine device, steroidal contraceptive in conjunction with a barrier method, or abstinence.
    12. Subject or caregiver must possess the ability, per the Investigator, to understand and comply with protocol requirements including clinical outcome measurements and instructions for the entire duration of the study.
    13. Caregiver, parent, guardian or responsible adult must be able and willing to accompany the subject to study visits.

    Part C
    1. Subject has completed Part B, meets the criteria for dose reduction for a second time, or meets the criteria for the rescue option
    OR
    2. Subject is a current participant in the NIH phase 1/2a open-label study and
    a. Subject agrees to convert from the dose of VTS-270 currently receiving as a subject in the NIH phase 1/2a protocol to the dose chosen for Parts B and C of this study 900 mg;
    b. Subject agrees to convert from the monthly dosing regimen used in the NIH phase 1/2a protocol to an every 2 week dosing regimen.
    c. In instances where NIH phase 1/2a subjects eligible to enroll into Part C are unable to convert from their current NIH phase 1/2a dose or monthly regimen, the investigator must receive prior written authorization from the sponsor for the subject to enter Part C of the study on an amended
    dose and/or regimen.
    OR
    3. Subject has received prior written authorization from Vtesse to enroll directly into Part C.
    4. Females of childbearing potential (not surgically sterile) must use a medically acceptable method of contraception and must agree to continue use of this method for the duration of the study and for 30 days after participation in the study. Acceptable methods of contraception include barrier method with spermicide, intrauterine device, steroidal contraceptive in conjunction with a barrier method, abstinence, or same-sex partner.
    5. Subject or parent/guardian must provide written informed consent to participate in the study. In addition to parental consent, assent to
    participate must also be sought from minor children.

    E.4Principal exclusion criteria
    1. Exclusion criteria as assessed by NPC SS:
    a. Unable to walk, wheelchair dependent (ambulation score=5)
    b. Needs a nasogastric tube or gastric tube for all feedings (swallowing=5). Note: Nasogastric or gastric tube use
    for supplemental feeding or medication administration is permitted and will not exclude a subject from this trial.
    c. Severe dysmetria (fine motor score=5)
    d. Minimal cognitive function (cognition score=5)
    2. Body weight < 15 kg.
    3. Prior treatment with IV HP-β-CD for NPC1 disease, unless the subject has undergone a minimum 3-month washout period prior to Study Day 0. Note: Any prior IT administration of HP-β-CD for the treatment of NPC1 disease will exclude a subject from enrollment.
    Treatment of other medical conditions with drug preparations containing HP-β-CD as an excipient (inactive ingredient) is acceptable and will not exclude a subject from immediate entry into this trial (no washout period required).
    4. Status epilepticus occurring within 3 months of screening and/or seizure frequency that cannot be quantified.
    5. Subjects on typical or atypical antipsychotics for treatment of psychosis. Note: Use of antipsychotic medication for treatment of other
    disorders (e.g., attention deficit hyperactivity disorder) will not exclude a subject from this trial.
    6. History of hypersensitivity reactions to any product containing HP-β-CD.
    7. Prior treatment with any other investigational product within 3 months prior to first dose (Study Day 0).
    8. Female subjects who are pregnant or nursing.
    9. Subjects with suspected infection of the central nervous system (CNS) or any systemic infection.
    10. Spinal deformity that could impact the ability to perform a LP.
    11. Skin infection in the lumbar region within 2 months of study entry.
    12. Neutropenia, defined as an absolute neutrophil count (ANC) of less than 1.5 x 109/L.
    13. Thrombocytopenia (platelet count less than 75 x 109/L).
    14. Activated partial thromboplastin time (aPTT) or prothrombin time (PT) prolonged by greater than 1.5 times the upper limit of normal (ULN) or known history of a bleeding disorder.
    15. Evidence of obstructive hydrocephalus or normal pressure hydrocephalus.
    16. Recent use of anticoagulants (in past 2 weeks prior to first dose [Study Day 0].
    17. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) greater than 4 times the ULN.
    18. Anemia: Hemoglobin more than 2 standard deviations below normal for age and gender.
    19. Estimated glomerular filtration rate (eGFR) < 60 mL/minute/1.73m2 calculated using the modified Schwartz formula (2009) for subjects aged 4 through 17 years old or using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula for subjects aged 18 years or older.
    20. Active pulmonary disease, oxygen requirement, or clinically significant history of decreased blood oxygen saturation, pulmonary therapy, or requiring active suction.
    21. Subjects who, in the opinion of the investigator, are unable to comply with the protocol or have medical conditions that would potentially increase the risk of participation.
    22. Life expectancy less than one year.
    E.5 End points
    E.5.1Primary end point(s)
    Part A:
    After all subjects in Part A had received 4 IT infusions at the randomized dose levels or sham LP, including 2 weeks of observation following the fourth dosing, all safety and efficacy data were provided to the DSC. The DSC included 2 neurologists with expertise in NPC1 disease who were also allowed to participate in the conduct of the study. The DSC also included a physician who was not allowed to be otherwise involved in the conduct of the study.
    The single dose selected for Parts B and C of the study was based on identification of the highest dose with acceptable safety and tolerability, including results of audiologic testing, as determined by the DSC. All data available through the 8-week time point were evaluated by the DSC in an unblinded fashion. These data included information on all AEs and SAEs through 8 weeks. Results of efficacy assessments were also provided to the committee. On the basis of these data, the DSC recommended a dose of 900 mg to be administered every 2 weeks for the remainder of the trial for subjects randomized to receive the active treatment. This dose was selected for its appropriate balance of safety, tolerability, and potential for efficacy.
    If at any time following the second dose of study drug during Part A of the study, a subject was reported to have clinically significant hearing loss (CTCAE grade 3 or higher), this was reviewed in an unblinded manner by the DSC, which could have made recommendations in light of other available ototoxicity data from the study. Such individuals were to have repeat pure tone audiometry (PTA) and distortion product otoacoustic emission (DPOAE) testing as part of the hearing loss AE evaluation. Such recommendations could have included, for example, continuing on the current dose scheme, decreasing that subject's dose by 300 to 600 mg, decreasing all subjects in a specific dose cohort by 300 to 600 mg, discontinuing that subject from the study, or discontinuing that dose cohort.

    Primary Efficacy Endpoint for Part B:
    The co-primary efficacy analysis will be based on:
    • Change from baseline in NPC-SS composite score at Week 52 in the group treated with VTS-270 vs. the sham control group.
    • Blinded Clinician CGIC at Week 52 in the group treated with VTS-270 vs the sham control group.

    Primary Endpoints in Part C:
    • The change from baseline to each assessment in a composite outcome that is the sum of the ambulation, cognition, fine motor, and swallowing components of the NPC-SS
    • The change from baseline to each assessment in total NPC-SS with the hearing domain and ABR modifiers removed
    • Proportion of responders (defined as no change or improvement on NPC-SS total score with hearing domain and ABR modifiers removed) at each assessment
    • Proportion of Blinded Clinician-CGIC (defined as a score of no change, minimally improved, moderately improved, or markedly improved) at each assessment compared to baseline
    • Summary of AEs, concomitant medications, physical examinations, audiologic examination, and clinical laboratories
    • Change from baseline in the EQ-5D-3L questionnaire at each assessment
    • Change from baseline to each assessment in each of the 9 clinical domains of the NPC-SS
    • Change from baseline to each assessment in the total NPC-SS with hearing domain and ABR modifier included
    • Time to one point increase (worsening) in NPC-SS composite score
    • The composite NPC-SS mean annualized rate of change (slope) from baseline to each assessment
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part A: 26 weeks
    Part B: 52 weeks
    Part C: until the investigator considers VTS-270 to no longer be
    beneficial, VTS-270 receives marketing authorization, or the
    development program is discontinued.
    E.5.2Secondary end point(s)
    Part B:
    The secondary outcomes for Part B are divided into 2 categories: Key secondary outcome and other secondary outcomes.
    The key secondary endpoints are:
    • Change from baseline at Week 52 in the total NPC–SS score with the hearing domain and ABR modifiers removed in the group treated with VTS-270 vs. the sham control group.
    • Proportion of Caregiver CGIC responders (defined as a score of no change, minimally improved, moderately improved or markedly improved) at Week 52 in the group treated with VTS-270 vs. the sham control group

    Other (non-key) secondary endpoints:
    • Summary of AEs, concomitant medications, physical examinations, audiologic examination, and clinical laboratories
    • Change from baseline in the EQ-5D-3L questionnaire at Week 52
    • Proportion of subjects in each group, treated for at least 6 months who qualified for the rescue option.
    • Change from baseline to Week 52 in each of the 9 clinical domains of the NPC-SS
    • Change from baseline to Week 52 in the total NPC-SS with hearing domain and ABR modifier included
    • Time to one point increase (worsening) in NPC-SS composite score
    • The composite NPC-SS mean annualized rate of change (slope) from baseline to Week 52
    • Change from baseline in the TUG test at Week 52
    • Change from baseline in the 9-hole peg test at Week 52

    Part C:
    Secondary endpoints for Part C will be evaluated for the device safety and tolerability substudy for the first nine port infusions:
    • Summary of wound check, examination of port site and catheter track at access port infusion visits
    • Intracranial pressure via LP
    • Summary of plasma and CSF pharmacokinetic parameters following a single dose of VTS-270 as well as trough HP-β-CD.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part A: 26 weeks
    Part B: 52 weeks
    Part C: until the investigator considers VTS-270 to no longer be
    beneficial, VTS-270 receives marketing authorization, or the
    development program is discontinued.



    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 No
    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 Yes
    E.6.13.1Other scope of the trial description
    Part C (Substudies): Safety and tolerability of the B. Braun Celsite Access Port System and Pharmacokinetics-Substudy
    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) Yes
    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 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) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Parts A and B of the trial are sham-controlled. Part C is open labelled.
    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 EEA9
    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
    France
    Germany
    Spain
    Turkey
    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 years5
    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 years5
    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: 35
    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: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 16
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 51
    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)
    Part C will be an open-label extension phase of the study. Part A and B and NIH phase 1/2a subjects who transition into
    Part C will receive treatment with VTS-270 until
    • The Investigator considers VTS-270 to no longer be beneficial to the
    subject
    • VTS-270 receives marketing authorization
    • The development program is discontinued
    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 Decision2015-10-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-11-09
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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