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    Summary
    EudraCT Number:2017-005006-35
    Sponsor's Protocol Code Number:RM-493-022
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2019-03-20
    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 ConcernedFrance - ANSM
    A.2EudraCT number2017-005006-35
    A.3Full title of the trial
    Long Term Extension Trial of setmelanotide (RM-493) for patients who have completed a trial of Setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway.
    Essai d'extension à long terme portant sur la setmélanotide (RM-493) chez des patients ayant terminé un essai utilisant la setmélanotide dans le traitement de l'obésité liée à des défauts génétiques en amont du récepteur aux mélanocortines de type 4 (MC4R) dans la voie leptine-mélanocortine
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Long Term Extension Trial of setmelanotide (RM-493) for patients who have completed a trial of Setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway
    A.4.1Sponsor's protocol code numberRM-493-022
    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 Pharmacueticals, 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 pointTara Parsons
    B.5.3 Address:
    B.5.3.1Street Address11th Floor, 500 Boylston Street
    B.5.3.2Town/ cityBoston
    B.5.3.3Post codeMA 02116
    B.5.3.4CountryUnited States
    B.5.4Telephone number+17817996621
    B.5.5Fax number+18572644299
    B.5.6E-mailtparsons@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 Yes
    D.2.5.1Orphan drug designation numberEU/3/16/1688 and EU/3/16/1703
    D.3 Description of the IMP
    D.3.1Product namesetmelanotide (preserved formulation)
    D.3.2Product code RM-493
    D.3.4Pharmaceutical form 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 INNsetmelanotide
    D.3.9.1CAS number 920014-72-8
    D.3.9.3Other descriptive nameRM-493
    D.3.9.4EV Substance CodeSUB182686
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10.0
    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
    Treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway
    E.1.1.1Medical condition in easily understood language
    treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary
    • To characterize safety and tolerability of setmelanotide in patients who have completed a previous trial on treatment of setmelanotide for obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway.
    E.2.2Secondary objectives of the trial
    Exploratory
    To assess the effect of setmelanotide, long term, on:
    • Maintenance or continued weight loss
    • Hunger
    • Percent change in body fat mass
    • Waist circumference
    • Percent change in total body mass, non-bone lean mass, and bone density
    • Fasting lipid (cholesterol and triglyceride) panel
    • Changes in quality of life and health status
    • Biomarkers predictive of a setmelanotide response and/or that change when the rate of weight loss may change later in the treatment regimen.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Quantitative Skin Color Measurement (Mexameter or similar device)
    For those Investigative Sites that performed skin color measurements in the index study, these measurements may continue in this extension study.
    The same spectrophotometric device should be utilized to quantitatively measure skin
    pigmentation due to melanin of each patient as described in the SOA. Measurements for a given
    patient are to be performed by the same staff member when possible. At each time point, skin
    pigmentation will be measured for each patient at the same 3 specified skin areas (central
    forehead, zygomatic process, and right upper buttock), to represent differing degrees of sun
    exposure. Each measurement will be obtained in triplicate, and averaged.
    In the event a patient experiences changes to skin coloration that are unresolved at the end of
    study, the patient may be asked to return for additional follow up assessments to document
    resolution.
    Specific instructions will be provided to those sites participating in this sub-study.
    E.3Principal inclusion criteria
    1. Patients aged 6 or older that have completed participation on active drug and demonstrated adequate safety in a previous setmelanotide study for obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway.
    2. Study participant and/or parent or guardian is able to communicate well with the investigator, to understand and comply with the requirements of the study, and to understand and sign the written informed consent/assent. The patient must assent/consent to participate in the trial.
    3. Female participants of child-bearing potential must agree to use contraception as outlined in the protocol. Female participants of non-childbearing potential, defined as surgically sterile (status post hysterectomy, bilateral oophorectomy, or bilateral tubal ligation), postmenopausal for at least 12 months (and confirmed with a screening FSH level in the postmenopausal lab range), or have delayed pubertal development and failure to have achieved menarche, do not require contraception during the study. Any female participant in this latter category of having failed to reach menarche upon study entry and who now suspects this status may have changed should promptly inform the investigator and undergo pregnancy testing.
    E.4Principal exclusion criteria
    1. Current, clinically significant disease, if severe enough to interfere with the study and/or would confound the results. Any such patients should be discussed with the sponsor prior to inclusion.
    2. Pregnant and/or breastfeeding women are excluded.
    3. Diagnosis of schizophrenia, bipolar disorder, personality disorder or other Diagnostic and Statistical Manual of Mental Disorders (DSM-III) disorders that the investigator believes will interfere significantly with study
    compliance.
    4. A Patient Health Questionnaire-9 (PHQ-9) score of ≥ 15.
    5. Any suicidal ideation of type 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS). Any lifetime
    history of a suicide attempt, or any suicidal behavior in the last month.
    6. Current, severe stable restrictive or obstructive lung disease as a consequence of extreme obesity, evidence of significant heart failure (NYHA Class 3 or greater), or oncologic disease, if these were severe enough to interfere with the study and/or would confound the results. Any such patients should be discussed with the sponsor prior to inclusion.
    7. History of significant liver disease or liver injury, or current liver assessment for a cause of abnormal liver tests [as indicated by abnormal liver function tests, alanine transaminase (ALT), aspartate transaminase (AST),
    alkaline phosphatase, or serum bilirubin (> 2.0 x upper limit of normal (ULN) for any of these tests)] for an
    etiology other than non-alcoholic fatty liver disease (NAFLD). Thus, any underlying etiology besides
    NAFLD, including diagnosed non-alcoholic steatohepatitis (NASH), other causes of hepatitis, or history of hepatic cirrhosis will be exclusionary, but the presence of NAFLD would not be exclusionary.
    8. History or presence of impaired renal function as indicated by clinically significant abnormal creatinine,
    blood urea nitrogen (BUN), or urinary constituents (e.g., albuminuria) or moderate to severe renal
    dysfunction as defined by the Cockroft Gault equation <30 mL/min if 17 years or older or by Schwartz if 16 years or less.
    9. History or close family history (parents or siblings) of skin cancer or melanoma, or patient history of ocularcutaneous albinism.
    10. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions, determined as part of a screening comprehensive skin evaluation performed by a qualified dermatologist. Any concerning lesions
    identified during the screening period will be biopsied and results known to be benign prior to enrollment. If the pretreatment biopsy results are of concern, the patient may need to be excluded from the study.
    11. Volunteer is, in the opinion of the Study Investigator, not suitable to participate in the study.
    12. Significant hypersensitivity to study drug.
    13. Inability to comply with injection regimen.
    14. Patients who have been placed in an institution through an official or court order, as well as those who are dependent on the sponsor, Investigator or study site.
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability of setmelanotide
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the study.
    E.5.2Secondary end point(s)
    The yearly mean percent change from baseline in body weight.

    Hunger will be assessed at each visit using a daily questionnaire as well as a set of two global questions.

    Yearly body composition including total body weight loss, fat loss, and non-bone lean mass.

    Waist circumference will be measured according to US National Heart Lung and Blood Institute criteria [2000 NHLBI] over time.

    Potential improvements in lipids (fasting cholesterol and triglycerides) will be assessed over time.

    Quality of life will be assessed yearly.

    Biomarkers predictive of a setmelanotide response and/or that change when the rate of weight loss may change later in the treatment regimen may be evaluated including biomarkers of metabolic and lipid metabolism changes.

    Additionally, as required by the Food and Drug Administration (FDA) for all CNS-active obesity medications, changes in depression/suicidality as assessed by the C- SSRS and PHQ-9 will be monitored.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Yearly unless stated otherwise in the protocol.
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    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) 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 No
    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.8.3 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 EEA15
    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
    Netherlands
    Spain
    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 years2
    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 years2
    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: 20
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 80
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    12-17 year olds.
    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 50
    F.4.2.2In the whole clinical trial 100
    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)
    It is anticipated that patients may continue setmelanotide treatment under this protocol until setmelanotide achieves marketing authorization and the patient can be switched over to commercially available setmelanotide without gaps in therapy. If marketing authorization is not achieved by the end of the extension trial (up to five years), a protocol amendment to extend the duration of this trial or entry into another clinical trial will be considered.
    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 Decision2019-11-20
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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