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    Summary
    EudraCT Number:2014-002392-28
    Sponsor's Protocol Code Number:RM-493-Supplementation-Therapy
    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:2014-08-04
    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 ConcernedGermany - BfArM
    A.2EudraCT number2014-002392-28
    A.3Full title of the trial
    Setmelanotide (RM-493) Treatment Trial in Patients with rare genetic disorders of obesity
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Setmelanotide (RM-493) Treatment Trial in Patients with rare genetic disorders of obesity
    A.3.2Name or abbreviated title of the trial where available
    RM-493-Supplementation-Therapy
    A.4.1Sponsor's protocol code numberRM-493-Supplementation-Therapy
    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 SponsorCharité Univeritaetsmedizin Berlin
    B.1.3.4CountryGermany
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportFunds of the Institute of Experimental Pediatric Endocrinology, Charité Universitaetsmedizin Berlin
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCharité - Universitätsmedizin Berlin , Campus Virchow-Klinikum
    B.5.2Functional name of contact pointInstitut Experimentelle Pädiatrie
    B.5.3 Address:
    B.5.3.1Street AddressAugustenburgerplatz 1
    B.5.3.2Town/ cityBerlin
    B.5.3.3Post code13353
    B.5.3.4CountryGermany
    B.5.4Telephone number4930450666839
    B.5.5Fax number4930450566916
    B.5.6E-mailpeter.kuehnen@charite.de
    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 nameRM-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 INNnone
    D.3.9.1CAS number 920014-72-8
    D.3.9.3Other descriptive nameRM-493
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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.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 nameRM-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.3.9.1CAS number 920014-72-8
    D.3.9.3Other descriptive nameSetmelanotide (RM-493)
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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
    Body weight is regulated within the hypothalamus. In rare cases mutations in genes, which are embedded in the signaling cascades of the hypothalamus lead to early onset severe obesity. POMC is one important gene and encodes the hormone Melanocortin (MSH), which regulates via the MC-4 receptor energy expenditure and satiety. Moreover within the pituitary POMC encodes the hormone ACTH. In total the failure of these hormone leads to obesity and secondary adrenal insufficiency in this POMC patients.
    E.1.1.1Medical condition in easily understood language
    Mutations in the gene POMC (LEPR, or PCSK1) leads to the lack of one important hormone, which is necessary to regulate body weight. For this reason the patients develop an early onset severe obesity.
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    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 objectives:
    • Body weight
    E.2.2Secondary objectives of the trial
    Secondary objectives:
    • Pubertal development
    • Metabolic serum parameters (insulin, liver function)
    • Blood pressure
    • Body-weight after a treatment duration of 2 years
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Written informed consent by the patients or the responsible relatives
    • Rare genetic disease populations in adults (≥18 years):
    o Homozygous or compound heterozygous (different gene mutation on both alleles) POMC, LEPR, MC4R or PCSK1 gene mutation
    o Heterozygous POMC, MC4R mutations
    o POMC hypermethylation (epigenetic) variant (>51.92 % POMC methylation intensity at the specific analysed POMC region)
    o Bardet-Biedl Syndrome
    o Alstrom’s Syndrome
    • Non-adult adolescent patients (≥ 12 years of age)
    o Homozygous or compound heterozygous POMC, LEPR or MC4R gene mutations
    o POMC hypermethylation (epigenetic) variant (> 35.79% POMC methylation intensity for individuals younger than 30 years at the specific analysed POMC region)
    o As substantial efficacy is shown in adult patients within each rare genetic disorder, then adolescent patients (greater than or equal to 12 years of age) can enter the study.
    • Obesity (BMI > 30 kg/m2; + 2 BMI SDS)
    • No other therapeutic option, which might cure the patient (e.g. bariatric surgery (see chapter 8))
    • Negative Pregnancy test
    • Highly effective contraception in women (defined as pearl index < 1), if necessary also for partners of test persons)
    • No participation in other clinical trials according to AMG (2 months before and after) at the time of this trial
    • Normal or minimally elevated blood pressure (measured in 24RR monitoring or similar methods) according the guidelines of the ESH (European Society of Hypertension) and Deutsche Hochdruckliga: systolic > 159 mmHg/diastolic 99 mmHg
    • sufficient kidney and liver function (Creatinine, ALT, AST)
    o normal values Alanin-Aminotransferase (ALT) (female): < 31 U/l
    o normal values Alanin-Aminotransferase (ALT) (male): < 41 U/l
    o normal values Aspartat-Aminotransferase (AST) (female > 17 years): < 35 U/l; (female < 17 years): 16-46 U/l
    o normal values Aspartat-Aminotransferase (AST) (male > 17 years): < 50 U/l; (male < 17 years): 16-46 U/l
    o normal values Bilirubine (male and female) up to 1,2 mg/dl
    o normal values Creatinine (female > 15 years): 0,51-0,95 mg/dl) ; (female < 15 years): 0,46-0,77 mg/dl
    o normal values Creatinine (male > 15 years): 0,67 – 1,17 mg/dl) ; (male < 15 years): 0,46-0,77 mg/dl
    E.4Principal exclusion criteria
    • Pregnancy or Breastfeeding
    • All contraindications against study medication (including auxiliary substances)
    • Interactions with study medication
    • Participation of the patient in a clincial study within the last 2 months
    • Intolerance against albumin
    • Concomitant diseases, impaired organ functions, except for known, concurrent GI disorders or other clinical findings expected in PCSK1 or LEPR or MC4R gene disorders
    • Renal insufficiency (Creatinine > 0.95 mg/dl (female), > 1.17 mg/dl (male))
    • Impaired liver function (Bilirubine > 1.2 mg/dl)
    • history of Neurological / psychiatric diseases
    • history of HIV Infection
    • history of Active Hepatitis B or C
    • Melanoma or Melanoma occurrence in the family history
    • Non-compliance
    • Subjects who are legally detained in an official institution
    • HIV Infection
    • Active Hepatitis B or C
    • Melanoma or Melanoma occurrence in the family history
    • Non-compliance
    • Subjects who are legally detained in an official institution
    E.5 End points
    E.5.1Primary end point(s)
    • Body weight course before and with RM-493 Treatment
    E.5.1.1Timepoint(s) of evaluation of this end point
    Body weight will be measured weekly throughout the study. The primary endpoint will be the body weight before and after the treatment period with RM-493.
    E.5.2Secondary end point(s)
    •• Pubertal development with RM-493 treatment
    • Changes of metabolic parameters (insulin, liver / kidney function)
    • Blood pressure
    • Body composition/Energy expenditure
    • Global Hunger Score (10 point scale)
    • Amendment: weight loss after treatment continuation of 2 years.
    • Changes of skin and nevi color
    • Psychological development of each patient during treatment
    period

    E.5.2.1Timepoint(s) of evaluation of this end point
    - the pubertal development will be examined just before and after the treatment period with RM-493 by a clinical examination and by performing a LHRH test.

    - before and after the treatment period there will be a blood collection in which metabolic parameters will be analyzed.

    - Blood pressure will be analyzed over 24h before and after the treatment period. Moreover, after each dosage escalation the blood pressure will be monitored carefully in the clinic for 12 hours following the new dose. Finally, patients will have blood pressure checked 3 times per day at home.

    the secondary endpoints were determined during the study visit every 6 months alternating with the 3-month visit;
    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 Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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.8.3 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 No
    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 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
    LVLS
    The trial will end after the last treatment period with RM-493 (max. 90 days) of the last of 2 patients.
    There will be a follow-up period until December 2020, in which the responsible doctor will be contacted once a year.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) Yes
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 2
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    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)
    After regular/premature termination the patients will be followed-up regularly in the outpatients clinics of the SPZ (Sozial-pädiatrisches Zentrum) at the Charité every 3 months or a specialized center for pediatric endocrinology.
    In the case of premature termination, the reason for withdrawal must be entered on the case report form (CRF) page and must be followed for safety and efficacy until 12 months after discontinuation.
    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 Decision2014-11-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-10-31
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-12-17
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