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    Summary
    EudraCT Number:2017-003606-40
    Sponsor's Protocol Code Number:SHP633-301
    National Competent Authority:Finland - Fimea
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-05-18
    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 ConcernedFinland - Fimea
    A.2EudraCT number2017-003606-40
    A.3Full title of the trial
    A Randomized, Open-label, 24-Week Safety, Efficacy, and Pharmacokinetic Study of Teduglutide in Infants 4 to 12 Months of Age with Short Bowel Syndrome Who are Dependent on Parenteral Support

    Original PIP P/238/2010
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Teduglutide study in infants 4 - 12 months of age suffering from Short Bowel Syndrome
    A.4.1Sponsor's protocol code numberSHP633-301
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/245/2015
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorShire Human Genetic Therapies, 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 supportShire Human Genetic Therapies, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire
    B.5.2Functional name of contact pointShire Medical Monitor
    B.5.3 Address:
    B.5.3.1Street Address300 Shire Way
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.6E-mailClinicalTransparency@shire.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 Yes
    D.2.1.1.1Trade name Revestive
    D.2.1.1.2Name of the Marketing Authorisation holderShire Pharmaceuticals Ireland Ltd
    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/3/01/077
    D.3 Description of the IMP
    D.3.2Product code A16AX08
    D.3.4Pharmaceutical form Powder and solution 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 INNTeduglutide
    D.3.9.1CAS number 287714-30-1
    D.3.9.2Current sponsor codeSHP633 - ALX-0600
    D.3.9.3Other descriptive nameTEDUGLUTIDE
    D.3.9.4EV Substance CodeSUB31909
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.25
    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 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
    Short Bowel Syndrome (SBS)
    E.1.1.1Medical condition in easily understood language
    Short Bowel Syndrome (SBS)
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10049416
    E.1.2Term Short-bowel syndrome
    E.1.2System Organ Class 10017947 - Gastrointestinal disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    the objectives of this trial are to evaluate the safety, efficacy/pharmacodynamics and pharmacokinetics (PK) of teduglutide treatment in infants with short bowel syndrome (SBS) dependent on parenteral support.
    E.2.2Secondary objectives of the trial
    not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Informed consent by the parent or legal guardian.
    2. Male or female infant 4 to 12 months corrected gestational age at screening.
    3. Weight at least 5 kg and weight-for-length Z-score greater than -2 at screening and baseline.
    4. Short bowel syndrome with dependence on parenteral support to provide at least 50% of fluid or caloric needs.
    5. Stable Parenteral Nutrition (PN) requirements for at least 1 month prior to screening, defined as a ≤10% change in the weight-normalized parenteral total fluid and caloric intake, despite attempts to wean PN, notwithstanding transient instability for events such as sepsis or interruption of
    central venous access.
    6. This criteria was deleted.
    7. Parent or legal guardian understands and is willing and able to fully adhere to study requirements as defined in this protocol.
    E.4Principal exclusion criteria
    1. Previous treatment with teduglutide.
    2. Intestinal malabsorption due to a genetic condition, such as cystic fibrosis, microvillus inclusion disease, etc.
    3. Severe, known dysmotility syndrome, such as pseudo-obstruction or persistent, severe, active gastroschisis-related dysmotility, that is the primary contributing factor to feeding intolerance and inability to reduce parenteral support, prior to screening. Dysmotility is defined as severe if it is expected to limit the advancement of enteral feeding.
    4. Inability to advance oral or enteral feeding due to lack of access to the gut, such as oral aversion in the absence of a feeding tube.
    5. Intestinal obstruction or clinically significant intestinal stenosis.
    6. Major gastrointestinal surgical intervention, such as serial transverse enteroplasty or major intestinal resection or anastomosis, within 3 months prior to screening or planned during the study period.
    7. Unstable cardiac disease.
    8. Renal dysfunction, defined as estimated glomerular filtration rate <50 mL/min/1.73 m2.
    9. Biliary obstruction, stenosis, or malformation.
    10. Clinically significant pancreatic disease.
    11. Severe hepatic dysfunction or portal hypertension, defined by at least 2 of the following parameters:
    a. International normalized ratio (INR) >1.5 not corrected with parenteral vitamin K
    b. Platelet count <100×10^3/μL due to portal hypertension
    c. Presence of clinically significant gastric or esophageal varices
    d. Documented cirrhosis
    12. Persistent cholestasis defined as conjugated bilirubin >4 mg/dL (>68 μmol/L) over a 2 week period.
    13. More than 3 serious complications of intestinal failure (e.g., catheter-associated bloodstream infections, interruption of nutrition due to feeding intolerance, catheter-associated thrombosis, severe fluid or electrolyte disturbances) within 1 month prior to or during screening.
    14. A history of cancer or a known cancer predisposition syndrome, such as juvenile polyposis or Beckwith-Wiedemann syndrome, or first degree relative with early onset of gastrointestinal cancer (including hepatobiliary and pancreatic cancers).
    15. Concurrent treatment with glucagon-like peptide-1 (GLP-1); glucagon-like peptide-2 (GLP-2); insulin-like growth factor-1 (IGF-1); growth hormone, somatostatin, or analogs of these hormones; or glutamine.
    16. Participation in a clinical study using an experimental drug within 3 months or 5.5 half-lives of the experimental drug, whichever is longer.
    17. Known or suspected intolerance or hypersensitivity to the investigational product, closely-related compounds, or any of the stated ingredients.
    18. Any condition, disease, illness, or circumstance that, in the investigator’s opinion, puts the subject at any undue risk, prevents completion of the study, or interferes with analysis of the study results.
    E.5 End points
    E.5.1Primary end point(s)
    Reduction in weight-normalized PN fluid volume by at least 20% from baseline at Week 24/EOT (end of treatment)
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 24/EoT
    E.5.2Secondary end point(s)
    - Reduction in weight-normalized parenteral calories by at least 20% from baseline to Week 24/EOT
    - Achievement of enteral autonomy by week 24
    - Time to achieve enteral autonomy
    - Change in weight-normalized parenteral fluid volume from baseline to each visit
    - Change in weight-normalized parenteral calories from baseline to each visit
    - Change in weight-normalized enteral fluid volume from baseline to each visit
    - Change in weight-normalized enteral caloric intake from baseline to each visit
    - Increase in weight-normalized enteral fluid intake by at least 20% from baseline to Week 24/EOT
    - Increase in weight-normalized enteral caloric intake by at least 20% from baseline to Week 24/EOT

    Pharmacokinetics
    The pharmacokinetic endpoint is plasma teduglutide concentration at nominal time point.

    Safety
    Safety endpoints consist of the following:
    - Adverse events (AEs)
    - Physical examinations
    - Vital signs
    - Weight, length, head circumference, and weight-for-length Z-scores (corrected for gestational age)
    - Laboratory safety data (biochemistry and haematology)
    - Urine output
    - Stool (including mixed) output
    - Antibodies to teduglutide

    Health Economics and Outcomes Research
    Health economics and outcomes research (HEOR) endpoints include the following:
    - Cumulative number of hospitalization days during the study
    E.5.2.1Timepoint(s) of evaluation of this end point
    each visit and week 24/EoT
    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 No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic Yes
    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) 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 Yes
    E.8.1.7.1Other trial design description
    1 arm standard of care and 1 arm on teduglutide
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial2
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA10
    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 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 years3
    E.8.9.1In the Member State concerned months4
    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 months4
    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: 10
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 10
    F.1.1.5Children (2-11years) No
    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
    infants of corrected gestational age 4 to 12 months
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 10
    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)
    All subjects (standard or care arm and teduglutide arm) will be evaluated for an open-label extension study
    Subjects on teduglutide arm will go back to standard of care treatment after the end study participation if not eligible for the extension study
    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 Opinion2018-06-06
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-09-24
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