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    Summary
    EudraCT Number:2016-003730-25
    Sponsor's Protocol Code Number:SBT20-102
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-01-09
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2016-003730-25
    A.3Full title of the trial
    A Two Part Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of SBT-020 in Patients with Early Stage Huntington’s Disease.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the safety and efficacy of a new medicine SBT-020 in patients with Early Stage Huntington’s Disease.
    A.3.2Name or abbreviated title of the trial where available
    SBT-020 in HD
    A.4.1Sponsor's protocol code numberSBT20-102
    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 SponsorStealth Bio Therapeutics 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 supportStealth Biotherapeutics
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCentre for Human Drug Research
    B.5.2Functional name of contact pointPrincipal Investigator
    B.5.3 Address:
    B.5.3.1Street AddressZernikedreef 8
    B.5.3.2Town/ cityLeiden
    B.5.3.3Post code2333 CL
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31715246400
    B.5.5Fax number+31715246499
    B.5.6E-mailclintrials@chdr.nl
    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 nameSBT-020
    D.3.2Product code SBT-020
    D.3.4Pharmaceutical form Lyophilisate 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 INNSBT-020
    D.3.9.1CAS number 1795737-00-6
    D.3.9.2Current sponsor codeSBT-020-01
    D.3.9.3Other descriptive nameSS-20
    D.3.9.4EV Substance CodeSUB180075
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number5 to 25
    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 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
    Huntington's Disease
    E.1.1.1Medical condition in easily understood language
    Huntington's Disease
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10070668
    E.1.2Term Huntington's disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part 1:
    Primary Objective
    - To assess the safety of SBT-020 in early stage HD patients

    Part 2:
    Primary Objective
    - - To assess the safety and tolerability of longer term treatment with SBT-020 in early stage HD patients.
    E.2.2Secondary objectives of the trial
    Part 1
    - Effect of SBT-020 on mitochondrial function, measured by 31P-MRS in skeletal muscle.
    - the pk of SBT-020 in plasma.
    - The pk of SBT-020 (and SBT-020-related components) in urine
    - The effect of SBT-020 on mitochondrial function, by measuring MMP in isolated peripheral blood mononuclear cells (PBMCs).

    Part 2
    - Effect of SBT-020 on mitochondrial function, measured by 31P-MRS in skeletal muscle.
    - The effect of SBT-020 on mitochondrial function, measured by 31P-MRS in the brain.
    - To assess the plasma concentration of SBT-020 in plasma.
    - To investigate the effect of SBT-020 on mitochondrial function, by measuring the MMP in PBMCs.
    - The effect of SBT-020 on an exploratory set of urinary and plasma biomarkers related to mitochondrial function.
    - Effects of SBT-020 on cognition and other CNS functions, using the NeuroCart test battery.
    - Effects of SBT-020 on motor functioning, using the NeuroCart test battery and UHDRS



    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patient with a DNA confirmed diagnosis (CAG expansion of 36 or more repeats in the HTT gene) of HD
    2. At least 18 years of age
    3. Unified Huntington’s Disease Rating Scale (UHDRS) Total Motor Score (TMS) of 5 or more
    4. Unified Huntington’s Disease Rating Scale (UHDRS) Total Functional Capacity Score (TFC) of 7 or more
    5. PCr of at least 32.4 seconds, measured by dynamic 31P-MRS of the calf muscles.
    6. Absence of evidence of any significant active or chronic disease (apart from HD), following a detailed medical and surgical history, a complete physical examination including vital signs, 12-lead ECG, haematology, blood chemistry, and urinalysis, that might interfere with the study activities or patient’s safety by participating in the study, as judged by the investigator. Psychiatric comorbidities to HD (such as a major depressive disorder), are allowed under the scrutiny of the investigator.
    7. Agrees to refrain from making any new, major life-style changes (e.g. starting a new diet or changing exercise pattern)
    8. Must agree to use adequate methods of contraception. Female subjects of childbearing potential must use two adequate forms of contraception, one of which must be a barrier method for the duration of the study and for 30 days after the last dose. Male subjects with a partner of childbearing potential must use two adequate forms of contraception, one of which must be a barrier method, for the duration of the study and for 30 days after the last dose.
    9. Able to participate and willing to give written informed consent and to comply with the study restrictions.
    E.4Principal exclusion criteria
    1. Positive test for drugs of abuse, such as metamphetamines and cocaine, at screening or pre-dose, except those prescribed by a physician for treatment of intercurrent medical issues due to HD.
    2. History (within 3 months of screening) of alcohol consumption exceeding 2 standard drinks per day on average. Alcohol consumption will be prohibited during study confinement and at least 24 hours before screening and before each scheduled visit.
    3. History of active malignancy within the last 5 years, with the exception of localized or in situ carcinoma (e.g., skin basal or squamous cell carcinoma).
    4. Positive Hepatitis B surface antigen (HBsAg), Hepatitis C antibody (HCV Ab), or human immunodeficiency virus antibody (HIV Ab) at screening.
    5. Aspartate transaminase (AST), alanine transaminase (ALT), gamma glutamyl transferase (GGT) or total bilirubin levels >1.5 times the upper limit of normal at screening.
    6. eGFR < 60 mL/min (calculated by the Modification of Diet in Renal Disease equation) at Screening.
    7. Clinically significant abnormalities, as judged by the Investigator, in laboratory test results (including hepatic and renal panels, complete blood count, chemistry panel and urinalysis). In the case of uncertain or questionable results, tests performed during screening may be repeated before randomization to confirm eligibility or judged to be clinically irrelevant for HD patients.
    8. Participation in an investigational drug or device study within 3 months prior to screening.
    9. Loss or donation of blood over 500 mL within three months (males) or four months (females) prior to screening.
    10. Concomitant disease or condition that could interfere with, or for which the treatment of might interfere with, the conduct of the study, or that would, in the opinion of the Investigator, pose an unacceptable risk to the subject in this study.
    11. Presence of any contraindication to have MRI scans performed (e.g. claustrophobia, pacemaker, vascular clips etc.). MRI contraindications for both the 7 Tesla (part 1 and 2) and 3 T (part 2 only) MRIs will be assessed, using MRI contra-indications questionnaires and, if necessary, a plain radiograph
    12. Unwillingness to refrain from smoking more than half pack cigarettes per day (i.e. 10 units).
    13. Specific cardiac abnormalities on the resting ECG at screening: QTcF> 450 or < 300 msec, evidence of atrial fibrillation, atrial flutter, complete branch block, Wolf-Parkinson-White Syndrome, or cardiac pacemaker.
    14. Any confirmed significant allergic reactions (urticaria or anaphylaxis) against any drug, or multiple drug allergies (non-active hay fever is acceptable).
    15. Unwillingness or inability to comply with the study protocol for any other reason.

    E.5 End points
    E.5.1Primary end point(s)
    Tolerability / safety endpoints
    - AEs leading to premature discontinuation of study drug.
    - Treatment-emergent (S)AEs up to 5 pharmacokinetic half-lives after study drug discontinuation.
    - Change from baseline to End-of-Study in vital signs.
    - Treatment-emergent ECG abnormalities up to 5 pharmacokinetic half-lives after study drug discontinuation.
    - Treatment-emergent marked laboratory abnormalities up to 5 pharmacokinetic half-lives after study drug discontinuation.
    Pharmacokinetic endpoints
    Part 1:
    - Plasma SBT-020 PK profile.
    - Urinary SBT-020 (and SBT-020-related components) PK profile.
    Part 2:
    - Plasma SBT-020 concentrations.
    Pharmacodynamic endpoints
    Mitochondrial function
    - Mitochondrial function by 31P-MRS
    o Phosphocreatine recovery time (in seconds), measured by 31P-MRS in calf muscles
    o Difference between Pi/PCr ratio before, during and after visual stimulation, measured by 31P-MRS in the brain (Part 2 only)
    - Intensity of red-green fluorescence, measured by flow-cytometry in PBMCs
    Various exploratory plasma and urinary biomarkers for mitochondrial function
    Neuropsychological function (Part 2 only)
    - NeuroCart and paper and pencil assessments
    o total number of correct responses in 90 seconds on the Symbol Digit Modality Test (SDMT)
    o total number of correct responses in 45 seconds per trial on the Stroop colour word interference test
    o completion time in seconds and number of errors for each trial on the Trail Making Test (TMT)
    o total number correct on the Visual Verbal Learning Test (VVLT)
    o the total number of (commission and omission) errors and the mean reaction time of all correct response trials on the Sustained Attention to Response Task (SART) test
    o average performance (%) on the Adaptive Tracking task
    Motor function (Part 2 only)
    o total motor score (TMS) (range 0-124) on the UHDRS
    o total functional capacity score (TFC) score (range 0-13) on the UHDRS
    o mean tapping rate and standard deviation on the Finger tapping test
    o saccadic reaction time (seconds), saccadic peak velocity (degrees/second), saccadic inaccuracy (%) on the Saccadic eye movements test
    o percentage of time the eyes are in smooth pursuit of the target (%) on the Smooth pursuit test
    o antero-posterior sway (in mm) on the Bodysway test
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 1 - Day 42
    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 Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 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 trial4
    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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 years1
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 24
    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 state24
    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)
    None
    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 Decision2017-01-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-02-14
    P. End of Trial
    P.End of Trial StatusCompleted
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