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    Summary
    EudraCT Number:2022-004107-32
    Sponsor's Protocol Code Number:RM-493-030
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2023-01-23
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2022-004107-32
    A.3Full title of the trial
    A Phase 2, Open-Label 20-Week Study to Evaluate the Safety and Efficacy of Setmelanotide in Subjects with Hypothalamic Obesity
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study designed to evaluate the body weight response to setmelanotide in patients with hypothalamic obesity.
    A.4.1Sponsor's protocol code numberRM-493-030
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04725240
    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 pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street Address222 Berkeley Street, Suite 1200
    B.5.3.2Town/ cityBoston
    B.5.3.3Post code02116
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1857-264-4280
    B.5.5Fax number+1857-264-4299
    B.5.6E-mailclinicaltrials@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 Yes
    D.2.1.1.1Trade name IMCIVREE
    D.2.1.1.2Name of the Marketing Authorisation holderRhythm Pharmaceuticals Inc
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 namesetmelanotide
    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.3.9.2Current sponsor codeRM-493
    D.3.9.3Other descriptive nameIMCIVREE
    D.3.9.4EV Substance CodeSUB192416
    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
    Hypothalamic Obesity
    E.1.1.1Medical condition in easily understood language
    “Hypothalamic obesity” refers to excess weight gain that may follow from an injury to the hypothalamus, a region of the brain.
    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
    The primary objective was to evaluate the change in body weight in response to setmelanotide administered subcutaneously (SC) daily in patients with HO.
    E.2.2Secondary objectives of the trial
    The secondary objectives were to evaluate changes in parameters of body weight, BMI, waist circumference, and hunger in response to setmelanotide in patients with HO and to evaluate the safety and tolerability of setmelanotide in patients with HO.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet all of the following criteria to be eligible for study participation:
    -Patient has documented evidence of HO, including:
    -Recent evidence of hypothalamic injury on magnetic resonance imaging (MRI); AND
    -Diagnosis of craniopharyngioma or other non-malignant brain tumor affecting the hypothalamic region; AND
    -Has undergone surgery, or chemotherapy, or radiation ≥6 months and ≤15 years before Screening.
    -Patient has either unilateral hypothalamic lesions or bilateral hypothalamic lesions, as assessed by MRI.
    -Aged 6 to 40 years.
    -Obesity, documented by a BMI ≥35 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and gender for patients 6 to <18 years of age.
    -Documented increase in BMI (change from pre-surgery baseline in BMI z-score ≥0.2 for patients <18 years of age or BMI >5% for patients >18 years of age) either during the first 6 months following surgery or within 1 year before surgery AND still present at Screening.
    -More than 6 months after the end of post-tumor treatment, including chemotherapy, surgery, or radiation.
    -Highly effective contraception throughout the study and for 90 days following the study.
    -Ability to communicate well with the Investigator, understand and comply with the requirements of the study, and understand and sign the written informed consent, or, for patients aged <18 years, a parent/legal guardian that can sign.
    -If receiving hormone replacement therapy, the dose of such therapy has remained stable for at least 2 months prior to Screening.
    E.4Principal exclusion criteria
    Patients meeting any of the following criteria are not eligible for study participation:
    -Weight gain >5% in the previous 3 months.
    -Weight loss ≥2% in the previous 3 months.
    -Note: Dietary and/or exercise regimens, with or without the use of medications, supplements, or herbal treatments associated with weight loss are allowed if:
    -the regimen and/or dose has been stable for at least 3 months prior to randomization
    -the patient has not experienced weight loss ≥2% during the previous 3 months, AND
    -the patient intends to keep the regimen and/or dose stable throughout the course of the study.
    -Bariatric surgery or procedure within the last 6 months.
    -Diagnosis of severe psychiatric disorders
    -HbA1c >10.0% at Screening.
    -Current, clinically significant pulmonary, cardiac, or oncologic disease considered severe enough to interfere with the study and/or confound the results.
    -Glomerular filtration rate (GFR) <30mL/min/1.73m^2 during Screening.
    -Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions.
    -History or close family history (parents or siblings) of skin cancer or melanoma
    -Participation in any clinical study with an investigational drug/device within 3 months, prior to the first setmelanotide dose.
    -Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
    -Inability to comply with once daily (QD) injection regimen.
    -Pregnant and/or breastfeeding, or desiring to become pregnant during this trial.
    -Cognitive impairment that, in the Investigator's opinion, precludes participation to the study and completions of study procedures or questionnaires.
    -Patient is, in Investigator's opinion, otherwise not suitable to participate in the study.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint was defined as the proportion of patients who achieve at least 5% BMI reduction from baseline at ~16 weeks of treatment with setmelanotide.
    E.5.1.1Timepoint(s) of evaluation of this end point
    ~16 weeks of treatment with setmelanotide.
    E.5.2Secondary end point(s)
    -The proportion of patients, aged ≥ 6 to < 18 years, with ≥ 0.2 reduction of BMI Z-score along with the proportion of patients aged ≥ 18 years with 5% reduction of body weight from baseline after 16 weeks of setmelanotide treatment.
    -The proportion of patients aged ≥ 18 years with ≥ 5% reduction of body weight from baseline after 16 weeks of setmelanotide treatment.
    -The change from baseline in waist circumference (cm) in patients aged ≥ 18 years after 16 weeks of setmelanotide treatment
    -The change in hunger in response to 16 weeks of setmelanotide treatment as measured by change from baseline in daily and global hunger scores


    E.5.2.1Timepoint(s) of evaluation of this end point
    ~16 weeks of treatment with setmelanotide.
    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 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.8.2.4Number of treatment arms in the trial1
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years1
    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: 12
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 3
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 15
    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)
    None
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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