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    Summary
    EudraCT Number:2018-004842-40
    Sponsor's Protocol Code Number:K020-218
    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:2019-06-04
    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 ConcernedGermany - BfArM
    A.2EudraCT number2018-004842-40
    A.3Full title of the trial
    A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with
    a Urea Cycle Disorder with Inadequate Control on Standard of Care
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects with Urea Cycle Disorder with an inadequate control of the disease with current standard drugs.
    A.4.1Sponsor's protocol code numberK020-218
    A.5.4Other Identifiers
    Name:IND NumberNumber:138734
    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 SponsorKaleido Biosciences
    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 supportKaleido Biosciences
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKaleido Biosciences
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street Address65 Hayden Avenue
    B.5.3.2Town/ cityLexington, MA
    B.5.3.3Post code02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1617999 2152
    B.5.6E-mailquality@kaleido.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameKB195
    D.3.2Product code KB195
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNKB195
    D.3.9.1CAS number none
    D.3.9.2Current sponsor codeKB195
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number9
    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
    Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care
    E.1.1.1Medical condition in easily understood language
    Subjects with a Urea Cycle Disorder with inadequate control on standard treatments.
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10080020
    E.1.2Term Urea cycle disorder
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic 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 efficacy of KB195 in ammonia lowering, when added to standard of care (SOC), in subjects with a urea cycle disorder (UCD)
    E.2.2Secondary objectives of the trial
    • Evaluate the safety and tolerability of KB195 in subjects with a UCD
    • Determine the proportion of fasting plasma ammonia concentrations above the upper limit of normal (ULN) before and at the end of treatment
    • Evaluate the effect of concomitant KB195 administration on indicators of nitrogen-binding therapy (NBT) delivery and activity, as applicable, in the subset of subjects whose ard of care (SOC) includes NBT
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    In the subset of subjects whose SOC includes nitrogen-binding therapy (NBT), to determine the effect, if any, of concomitant KB195 administration on indicators of NBT delivery and activity, as applicable.
    E.3Principal inclusion criteria
    1. Is able and willing, or has a legally authorized representative (LAR) who is able and willing, to give written informed consent. When the LAR provides informed consent, the subject must also provide written assent if able.
    2. Is willing to comply with specified procedures as stated in the protocol
    3. Has any confirmed UCD other than N-acetylglutamate synthase (NAGS) deficiency, with the diagnosis confirmed through genetic testing, enzymatic analysis, or analysis of ammonia and amino acid concentrations, as appropriate for the UCD subtype; documentation may be from the historical record
    4. Is male or female, 18 to 65 years of age (inclusive)
    5. Has a body mass index (BMI) ≥ 20.0 and < 40.0 kg/m2
    6. Has evidence of poorly controlled disease on the current SOC, as evidenced by:
    a. One plasma ammonia concentration > ULN within 6–12 months before Day -1 and 1 plasma ammonia concentration > ULN during the Screening Period, OR
    b. At least 2 plasma ammonia concentrations > ULN within 6 months of Screening (1 of which can be during Screening)
    7. If NBT is part of SOC, is on a stable dose and regimen of NBT for at least 4 weeks before Screening and the dose is expected to remain stable during the study
    8. Is willing to maintain a stable diet throughout the course of the study, and is willing to continue usual exercise routine
    9. Is willing to continue taking any current supplements and vitamins (with the exception of prebiotic or probiotic supplements) that the subject is currently taking, for the duration of the study
    10. If taking probiotics or prebiotics, is on a stable dose and regimen for at least 4 weeks before Screening and the dose and regimen are expected to remain stable during the study
    11. Has negative urine screen for drugs of abuse at Screening
    12. If subject is female: subject is surgically sterile or post-menopausal (12 months with no menses without an alternate medical cause); or if subject is a female of childbearing potential (FCBP), must have a negative serum pregnancy test at Screening, must not be lactating, and must agree to abstain from sexual activity or agree to use a highly effective method of contraception for the duration of the study and for 30 days after the last dose of KB195
    13. If subject is male and has a female partner of childbearing potential, must agree to abstain from sexual activity or agree to use a highly effective contraceptive method for the duration of the study and for 90 days after the last dose of KB195
    E.4Principal exclusion criteria
    1. Is at high risk for metabolic decompensation, as indicated by:
    - More than 4 hospitalizations within 1 year before the Screening Visit for management of acute metabolic decompensation due to UCD, OR
    - An ammonia concentration of ≥ 100 μmol/L with clinical signs or symptoms during Screening (if a subject meets all other study requirements, at the PI’s discretion the subject may be re-screened 1 time after their ammonia is controlled and clinical symptoms have resolved), OR
    - Other evidence of metabolic instability as judged by the PI.
    2. Has had a substantive change (as judged by the PI) in diet or any other aspect of UCD management within 4 weeks before the Screening Visit
    3. Has used a systemic anti-infective (for example, an antibiotic, antifungal, or antiviral product) within 4 weeks before the Screening Visit, or use is anticipated during the study (if a subject fails this criterion and meets all other study requirements, at the PI’s discretion and after consultation with the Sponsor, the subject may be re-screened 1 time)
    4. Has been diagnosed with Citrullinemia Type II
    5. Is receiving any systemically administered immunosuppressant medication on a chronic basis at a dose > 10 mg/day of prednisone or equivalent
    6. Has changed the use or dose of any drug or other compound to modulate GI motility (including stool softeners, laxatives, bismuth, or fiber supplements), within 4 weeks before the Screening Visit, or the use or dose is expected change during the course of the study. Note that proton pump inhibitors and antacids are permitted, provided they are taken at baseline and continued throughout the study. (If a subject fails this criterion and meets all other study requirements, at the PI’s discretion the subject may be re-screened 1 time)
    7. Has a history of or active GI or liver disease that may affect assessment of the efficacy, safety, or tolerability of KB195, including but not limited to the following: chronic diarrhea, malabsorption syndrome, inflammatory bowel disease, irritable bowel syndrome, liver cirrhosis or failure, autoimmune disease, or GI malignancy
    8. Has a clinically significant or uncontrolled concomitant illness that would put the subject at risk or jeopardize the objectives of the study
    9. Has a prior solid organ transplantation including liver transplantation, or is anticipated to receive a liver transplant during study participation
    10. Has used an investigational drug, product, or device within 30 days before the Screening Visit; or is enrolled in another investigational drug, product, or device study within 30 days before the Screening Visit
    11. Has a contraindication, sensitivity, or known allergy to any ingredient of the study drug
    12. Is considered, in the opinion of the PI, to likely be a poor attendee or unlikely for any reason to be able to comply with the study procedures (for example, planned procedures or traveling that would occur during the duration of the study)
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of subjects who achieve a ≥15% reduction from baseline in fasting plasma ammonia at the end of treatment
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day -1, Day 55 +/- 1d
    E.5.2Secondary end point(s)
    • Proportion of subjects normalizing their fasting plasma ammonia concentrations from above the ULN at baseline to below ULN at end of treatment
    • Change from baseline to end of treatment in the 24-hour area under the concentrationtime
    curve (AUC24) of plasma ammonia
    • Number of subjects experiencing adverse events (AEs)
    • Number of subjects experiencing serious AEs (SAEs)
    • Change from baseline to end of treatment in vital signs and safety laboratory values
    • Change from baseline to end of treatment in Gastrointestinal Tolerability Questionnaire (GITQ) scores
    • Change from baseline to end of treatment in Bristol Stool Scale (BSS) scoring
    • Change from baseline to end of treatment in plasma concentrations of sodium benzoate (NaB), and plasma and urine concentration of phenylbutyric acid (PBA), phenylacetic acid (PAA), and/or phenylacetylglutamine (PAGN) for applicable subjects
    E.5.2.1Timepoint(s) of evaluation of this end point
    Fasting ammonia: D -1, D55
    AUC24: D-1 to D1, D55-56
    Blood chemistry and hematology, urinalysis : screening (once between D-28 and D-2), D-1, D28 ±2, D56 (end of treatment), D84 ±2 and at early withdrawal (if applicable).
    Gastrointestinal Tolerability Questionnaire (GITQ), Bristol Stool Scale (BSS) and Adverse Event (AE) review at the following visits throughout the study: D-1, D28 ±2, D55 ±1, D56 (end of treatment), D84 ±2, Early withdrawal (if 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 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 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 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.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    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
    Belgium
    Canada
    France
    Germany
    Mexico
    Netherlands
    Saudi Arabia
    Spain
    Switzerland
    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
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    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 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: 29
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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
    Included in the study if able and willing, or has a legally authorized representative (LAR) who is able and willing, to give written informed consent. When the LAR provides informed consent, the subject must also provide written assent if able.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 29
    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)
    Standard of care for the patient per 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 Decision2019-09-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-11-25
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-10-04
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