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    Summary
    EudraCT Number:2016-000849-30
    Sponsor's Protocol Code Number:SHP633-304
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-06-16
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2016-000849-30
    A.3Full title of the trial
    A Prospective, Open-label, Long-term Safety and Efficacy Study of
    Teduglutide in Pediatric Patients with Short Bowel Syndrome Who
    Completed TED-C14-006

    Original PIP P/238/2010
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A long term extension study looking at the efficacy and safety of teduglutide in paediatric patients suffering from short bowel syndrome who took part in study TED-C14-006
    A.4.1Sponsor's protocol code numberSHP633-304
    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
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.4Pharmaceutical form Powder and solvent 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 codeALX-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 number5.0
    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.IMP: 2
    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 nameTeduglutide
    D.3.4Pharmaceutical form Powder and solvent 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 codeALX-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
    E.1.1.1Medical condition in easily understood language
    Short Bowel Syndrome
    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 20.0
    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 primary objective of the study is to evaluate the long-term safety and tolerability of teduglutide treatment in paediatric subjects with SBS who completed study TED-C14-006.
    E.2.2Secondary objectives of the trial
    The secondary objective of this study is to evaluate the long-term efficacy of teduglutide treatment in paediatric subjects with SBS who completed study TED-C14-006.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Study Inclusion Criteria:
    The subject will be considered eligible for the study if they meet all of the study inclusion criteria. Teduglutide treatment eligibility does not impact study eligibility.
    1. Subject provides written informed consent (subject, parent or legal guardian and, as appropriate, informed assent) to participate in the study before completing any study-related procedures.
    2. Subject completed the TED-C14-006 study (including subjects in the standard of care treatment arm).
    3. Subject understands and is willing and able to fully adhere to study requirements

    Teduglutide Eligibility Criteria:
    Subjects are eligible for teduglutide treatment if at least one (≥1) of the teduglutide treatment inclusion criteria, and none of the teduglutide treatment exclusion criteria, are met. In addition, the investigator and the subject (and/or parent or legal guardian, as appropriate) must agree to proceed with treatment.

    Teduglutide Treatment Inclusion Criteria:
    1. Subject is teduglutide-naïve, receiving parenteral support (PS), and unable to significantly reduce PS or advance enteral feeds (e.g., 10% or less change in PS or advance in feeds) for at least 3 months prior to and during the teduglutide pre-treatment visit, as assessed by the investigator. Transient instability for events such as interruption of central access or treatment for sepsis is allowed if the PS returns to within 10% of baseline prior to the event.
    2. Subject was previously treated with teduglutide and at least one of the following criteria is satisfied:
    a. Increasing PS requirements following teduglutide discontinuation.
    b. Decreased PS requirement during prior teduglutide treatment, followed by cessation of improvement after teduglutide discontinuation.
    c. Deteriorating nutritional status (e.g., weight loss or growth failure) despite maximal tolerated enteral nutrition following teduglutide discontinuation.
    d. Deteriorating fluid or electrolyte status despite maximal tolerated enteral fluid and electrolyte intake following teduglutide discontinuation.
    e. Severe diarrhoea related to teduglutide discontinuation.

    Follow-up Period Escape Criteria:
    At the discretion of the investigator, the follow-up period may be interrupted and the subject may proceed directly to the pre-treatment visit, if 1 of the following criteria is met:
    1. Increasing PS requirements following teduglutide discontinuation
    2. Deteriorating nutritional status (e.g., weight loss or growth failure) despite maximal tolerated enteral nutrition following teduglutide discontinuation.
    3. Deteriorating fluid or electrolyte status despite maximal tolerated enteral fluid and electrolyte intake following teduglutide discontinuation.
    4. Severe diarrhoea related to teduglutide discontinuation.
    E.4Principal exclusion criteria
    Study Exclusion Criteria:
    There are no exclusion criteria for this study.

    Teduglutide Treatment Exclusion Criteria:
    1. Body weight <10 kg at the pre-treatment visit.
    2. Unresected gastrointestinal (GI) polyp, known polyposis condition, pre-malignant change, or malignancy, in the GI tract.
    3. History of cancer in the previous 5 years except surgically curative skin cancers.
    4. Serial transverse enteroplasty or other major intestinal surgery within 3 months preceding the teduglutide pre-treatment visit. Insertion of a feeding tube, anastomotic ulcer repair, minor intestinal resections ≤10 cm, and endoscopic procedures are allowed.
    5. Intestinal or other major surgery planned or scheduled to occur during the 28-week cycle.
    6. Clinically significant intestinal stricture or obstruction.
    7. Clinically significant, active or recurrent pancreatic or biliary disease.
    8. Active, severe, or unstable, clinically significant hepatic impairment or injury, including the following laboratory values at the pre-treatment visit:
    a. Total bilirubin ≥2 × upper limit of normal (ULN)
    b. Aspartate aminotransferase (AST) ≥7 × ULN
    c. Alanine aminotransferase (ALT) ≥7 × ULN
    9. Renal dysfunction shown by results of an estimated glomerular filtration rate (eGFR) below 50 mL/min/1.73 m2 at the pre-treatment visit.
    10. Unstable cardiac disease, congenital heart disease or cyanotic disease, with the exception of subjects who had undergone ventricular or atrial septal defect repair, or patent ductus arteriosus (PDA) ligation.
    11. Participation in a clinical study using an experimental drug (other than glutamine, Omegaven or Smoflipid) within 3 months or 5.5 half-lives of the experimental drug, whichever is longer, prior to the pre-treatment visit and for the duration of the 28-week cycle.
    12. Treatment with analogs of glucagon-like peptide-1 (GLP-1), glucagon-like peptide-2 (GLP-2) (not including teduglutide), insulin-like growth factor-1 (IGF-1), or growth hormone, within 3 months preceding the teduglutide pretreatment visit.
    13. Treatment with octreotide or dipeptidyl peptidase 4 (DPP-4) inhibitors within 3 months prior to the pre-treatment visit.
    14. Known or suspected intolerance or hypersensitivity to the investigational product, closely-related compounds, or any of the stated ingredients.
    15. Known history of alcohol or other substance abuse within 1 year prior to the pre-treatment visit.
    16. Pregnant or lactating female subjects.
    17. Sexually active female subjects of child-bearing potential unwilling to use approved contraception during teduglutide treatment and for 30 days after the treatment period.
    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)
    Efficacy endpoints:
    - Reduction in PS volume of at least 20%
    - Absolute and relative change in PS volume
    - Complete weaning off PS
    - Change in days per week of PS
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of each teduglutide treatment period (week 24 or EOT), and each study visit.

    In addition, the Efficacy/PD endpoints will be analysed using the baseline of the Core study (TED-C14-006) and the baseline of each treatment cycle.
    E.5.2Secondary end point(s)
    Safety Endpoints
    - Adverse events, including those pertaining to GI symptoms
    - Vital signs, including temperature, heart rate, blood pressure
    - Body weight, height (or length), head circumference (up to 36 months of age) trends on growth charts, BMI; z-scores will be calculated for height (or length), weight, head circumference and BMI
    - Laboratory safety data (i.e., clinical chemistry, haematology, and urinalysis)
    - Urine output
    - Stool output
    - Antibodies to teduglutide
    - Gastrointestinal-specific testing (teduglutide treatment eligible subjects only) including colonoscopy or sigmoidoscopy, abdominal ultrasound, and FOBT, upper GI series with small bowel follow through

    Health Economics and Outcomes Research (HEOR) Endpoints:
    - Change in Pediatric Quality of Life Inventory (PedsQL) score
    - Change in PedsQL Family Impact Module score
    - Change in PedsQL Gastrointestinal Symptoms Module Sub-Scales scores:
     Food and Drink Limits
     Diarrhoea
    E.5.2.1Timepoint(s) of evaluation of this end point
    Each scheduled visit

    The HEOR endpoints will be analysed using the Baseline of each
    treatment cycle at approximately 12 week intervals (weeks 12 and 24 of each teduglutide treatment cycle, and every 12 weeks for subjects not on teduglutide).
    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 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 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
    arm for participants in non teduglutide treatment taking standard of care
    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 Yes
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    Finland
    Italy
    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 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: 34
    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: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 14
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 19
    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 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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 34
    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 or care
    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 Decision2017-08-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-07-31
    P. End of Trial
    P.End of Trial StatusCompleted
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