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    Summary
    EudraCT Number:2014-005623-27
    Sponsor's Protocol Code Number:RM-131-009
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-02-17
    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 ConcernedSweden - MPA
    A.2EudraCT number2014-005623-27
    A.3Full title of the trial
    A Phase 2b, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of RM-131 Administered to Patients with Vomiting Symptoms and Moderate to Severe Diabetic Gastroparesis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A controlled clinical trial to study RM-131 (relamorelin) in patients with vomiting symptoms and diabetic gastroparesis
    A.4.1Sponsor's protocol code numberRM-131-009
    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 SponsorRhythm Pharmaceuticals, 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 supportRhythm Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRhythm Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointInformation
    B.5.3 Address:
    B.5.3.1Street Address855 Boylston Street
    B.5.3.2Town/ cityBoston
    B.5.3.3Post codeMA 02116
    B.5.3.4CountryUnited States
    B.5.4Telephone number+18572644280
    B.5.5Fax number+18572644299
    B.5.6E-mailffiedorek@rhythmtx.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 nameRelamorelin
    D.3.2Product code RM-131
    D.3.4Pharmaceutical form Solution for injection in pre-filled syringe
    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 INNNone
    D.3.9.1CAS number 945416-05-7
    D.3.9.2Current sponsor codeRM-131
    D.3.9.3Other descriptive nameRELAMORELIN
    D.3.9.4EV Substance CodeSUB171935
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number100
    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 in pre-filled syringe
    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
    Vomiting symptoms in patient with moderate to severe diabetic gastroparesis
    E.1.1.1Medical condition in easily understood language
    Vomiting symptoms in patient with moderate to severe diabetic gastroparesis
    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 17.1
    E.1.2Level PT
    E.1.2Classification code 10051153
    E.1.2Term Diabetic gastroparesis
    E.1.2System Organ Class 10017947 - Gastrointestinal disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • Evaluate the effect of RM-131 on vomiting episodes in patients with diabetic gastroparesis (DG).
    E.2.2Secondary objectives of the trial
    • Evaluate the effects of RM-131 on 4 key symptoms of gastroparesis (nausea, early satiety, bloating, and abdominal pain) assessed with a composite score derived from the Diabetic Gastroparesis Symptom Severity Diary (DGSSD).
    • Evaluate the effect of RM-131 on vomiting severity (as assessed in the DGSSD).
    • Evaluate the effect of RM-131 on gastric emptying (GE).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. T1DM or T2DM with stable glycemic control and HbA1c ≤11% at screening.
    2. DG, defined as at least a 3-month history of symptoms suggestive of gastroparesis on an ongoing basis (e.g., vomiting, nausea, early satiety, bloating, or epigastric or abdominal pain).
    3. GCSI-DD score ≥2.6 at least once during the Screening Period (Visits 1-2).
    4. At least 2 vomiting episodes during the ~2 weeks prior to the first screening visit (Visit 1), as ascertained by patient history.
    5. Delayed GE confirmed at screening by abnormal GEBT (AB Diagnostics), defined as GE half-time (t1/2) ≥79 minutes (the 80th percentile of normative data). At least 50% of patients enrolled will have a t1/2 ≥97 minutes (i.e., the 95th percentile).
    6. Stable concomitant medications, defined as no changes in regimen for at least 2 weeks prior to Visit 2 (daily adjustments of insulin doses are permitted).
    7. No use of metoclopramide, erythromycin, domperidone, or other GI motility agents, or anti-emetics for at least 2 weeks prior to Visit 2, and willingness to remain off these medications (except as used as part of protocol specific rescue medication) during the course of the clinical trial.
    8. Body mass index >18 kg/m2.
    9. If female, has a negative serum or urine pregnancy test and is not lactating. For females able to bear children, a hormonal (i.e., oral, implantable, or injectable) and single-barrier method, or a double-barrier method of birth control must be used throughout the study. Female patients unable to bear children must have this documented in the eCRF (i.e., tubal ligation, hysterectomy, or post-menopausal [defined as a minimum of 1 year since
    the last menstrual period]). Post-menopausal status will be confirmed by measurement of follicle stimulating hormone (FSH).
    10. Able to provide written informed consent prior to any study procedures and willing and able to comply with study procedures.

    Additional inclusion criteria for randomization after the 2-week single-blind placebo run-in period:
    11. Compliance with the completion of the DGSSD and study drug injections, defined as approximately 80% diary completions and approximately 80% administration of injections, during the 2-week single-blind placebo run-in period. For those patients whose compliance is measured to be <80%, the final decision to randomize a patient will be made by the Investigator and the Sponsor (or designee).
    12. At least one vomiting episode at any time during the 2-week single-blind placebo run-in period, as recorded in the DGSSD.
    E.4Principal exclusion criteria
    1. Currently receiving parenteral feeding or presence of a nasogastric or other enteral tube for feeding or decompression.
    2. History of gastric surgery such as fundoplication, gastrectomy, gastric pacemaker placement, vagotomy, or bariatric procedure. (A history of diagnostic endoscopy is not exclusionary.)
    3. History of pyloric injection of botulinum toxin within 6 months of screening.
    4. Patients with clinical suspicion of upper GI obstruction (e.g., peptic stricture) must have been evaluated per standard of care and obstruction ruled out before screening.
    5. Currently taking opiates, or expecting to use opiates during the course of the clinical trial.
    6. Currently taking GLP-1 agonists, SGLT2 inhibitors or pramlintide.
    7. Allergic or intolerant of egg, wheat, milk, or algae, as these are components of the GEBT study meal. (Gluten-free crackers can be provided.)
    8. History of anorexia nervosa, binge-eating, or bulimia within 5 years of screening.
    9. ALT or AST > 2 × upper limit of normal (ULN) at Visit 1.
    10. History of intestinal malabsorption or pancreatic exocrine disease.
    11. Requires hemodialysis or has end-stage renal disease.
    12. History of human immunodeficiency virus (HIV) infection.
    13. Clinically significant neurologic or psychiatric disorders that are likely to impact compliance with protocol requirements.
    14. Poor venous access or inability to tolerate venipuncture.
    15. Participation in a clinical study within the 30 days prior to dosing in the present study.
    16. Any other reason that, in the Investigator’s opinion, would confound proper interpretation of the study or expose a patient to unacceptable risk, including renal, hepatic or cardiopulmonary disease, or significant acute ECG abnormalities.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is:
    • Longitudinal analysis of the number of weekly vomiting episodes, averaged across the 12-week treatment period.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Weekly
    E.5.2Secondary end point(s)
    The key secondary efficacy endpoint is:
    • Longitudinal analysis of changes from baseline over time in weekly averages of the 4 individual daily symptom scores (e.g., the 4 subjective symptoms) using a global statistic.
    Secondary efficacy endpoints are:
    • Longitudinal analysis of changes from baseline over time in weekly average of the 4- symptom composite of subjective symptoms of DG (nausea, early satiety, bloating, and epigastric or abdominal pain).
    • Longitudinal analysis of weekly average vomiting episodes.
    • Longitudinal analysis of changes from baseline over time in weekly averages of the other individual daily symptom scores (e.g., the 4 subjective symptoms).
    • Longitudinal analysis of changes from baseline over time in weekly averages of vomiting severity.
    • Longitudinal analysis of changes from baseline over time in weekly averages of the 5 individual daily symptom scores (e.g., the 4 subjective symptoms and vomiting endpoints) using a global statistic.
    • Gastric emptying as measured by the GEBT half-time.

    Other tertiary and exploratory endpoints will include PK, PK/PD, weight, glucose, and analysis of different global statistical approaches. Effects of RM-131 on symptoms of gastroparesis will be evaluated using the DGSSD. Exploratory assessments of symptoms may also utilize Symptom and Global Assessment data.

    Safety endpoints include:
    • Physical examination, including assessment of the injection site, weight and vital signs
    • Clinical laboratory safety tests:
    • Complete blood count (CBC) with automated differential
    • Chemistry panel (including sodium, potassium, chloride, carbon dioxide (CO2), albumin, glucose, calcium, total protein, aspartate aminotransferase (AST), ALT, GGT, creatine kinase (CK), alkaline phosphatase, total bilirubin, direct bilirubin, blood urea nitrogen (BUN), creatinine, uric acid, lactate dehydrogenase (LDH), cholesterol and triglycerides. (Procedures for follow-up of abnormal liver function tests are provided in Appendix 11.6.)
    • Urinalysis (microscopic review if indicated)
    • HbA1c
    • AE review
    • Concomitant medication review
    • ECG evaluations
    • Anti-RM-131 antibody measurements
    • IGF-1 and prolactin levels
    E.5.2.1Timepoint(s) of evaluation of this end point
    Weekly
    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 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 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 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 EEA23
    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
    Germany
    Israel
    Poland
    Sweden
    United Kingdom
    United States
    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
    LPLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months16
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial months16
    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: 265
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 100
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 63
    F.4.2.2In the whole clinical trial 395
    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.
    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 Decision2015-04-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-03-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-06-09
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