Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   40665   clinical trials with a EudraCT protocol, of which   6637   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2018-004058-11
    Sponsor's Protocol Code Number:RM-493-023
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-04-11
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2018-004058-11
    A.3Full title of the trial
    A Phase 3 trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and AlstrC6m syndrome (AS) Patients with Moderate to Severe Obesity
    Ensayo de fase III de setmelanotida (RM-493), un agonista del receptor de melanocortina-4 (MC4R), en pacientes con síndrome de Bardet-Biedl (SBB) y síndrome de Alström (SA) con obesidad de moderada a grave
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Setmelanotide (RM-493) trial in Bardet-Biedl Syndrome (BBS) and AlstrC6m syndrome (AS) Patients with Moderate to Severe Obesity
    Ensayo de setmelanotida (RM-493), en pacientes con síndrome de Bardet-Biedl (SBB) y síndrome de Alström (SA) con obesidad de moderada a grave
    A.4.1Sponsor's protocol code numberRM-493-023
    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 pointMatt Webster
    B.5.3 Address:
    B.5.3.1Street Address500 Boylston Street, 11th Floor
    B.5.3.2Town/ cityBoston
    B.5.3.3Post codeMA 02116
    B.5.3.4CountryUnited States
    B.5.4Telephone number001508 789 1527
    B.5.6E-mailmwebster@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 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.3.9.2Current sponsor codeRM-493
    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
    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
    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
    Obesity and hyperphagia in patients with Bardet-Biedl Syndrome or AlstrC6m syndrome
    Obesidad e hiperfagia en pacientes con sindrome de Bardet-Biedl o Sindrome de Alstrom
    E.1.1.1Medical condition in easily understood language
    Obesity and hyperphagia in patients with Bardet-Biedl Syndrome or AlstrC6m syndrome
    Obesidad e hiperfagia en pacientes con sindrome de Bardet-Biedl o Sindrome de Alstrom
    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
    To assess the effect of setmelanotide on the proportion of patients (b %12 years of age at baseline) treated with setmelanotide for approximately (~) 52 weeks who achieve a clinically meaningful reduction from baseline (ie, b %10%) in body weight.
    Evaluar el efecto de la setmelanotida en la proporción de pacientes (de ≥ 12 años de edad en el inicio) tratados con setmelanotida durante aproximadamente (~) 52 semanas que lograron una reducción clínicamente significativa respecto al inicio (es decir, ≥ 10 %) de su peso corporal.
    E.2.2Secondary objectives of the trial
    Key Secondary
    • To assess the effect of setmelanotide on the proportion of all patients who achieve a b %10% reduction from baseline in body weight after ~52 weeks of treatment with setmelanotide.
    • To assess the effect of setmelanotide on mean percent change in body weight (in patients b %12 years of age at baseline) compared with baseline after ~52 weeks of treatment with setmelanotide..
    • To assess the effect of setmelanotide on the proportion of patients who achieve a b %25% improvement in the weekly average of the daily hunger score (in patients b %12 years of age at baseline) after ~52 weeks of treatment with setmelanotide.
    • To assess the effect of setmelanotide on the mean percent change from baseline in the weekly average of the daily hunger score (in patients b %12 years of age at baseline) after ~52 weeks of treatment with setmelanotide.
    • Evaluar el efecto de la setmelanotida en la proporción de todos los pacientes que lograron una reducción del ≥ 10 % respecto al inicio del peso corporal después de ~ 52 semanas de tratamiento con setmelanotida.
    • Evaluar el efecto de la setmelanotida sobre la media del porcentaje de cambio del peso corporal (en pacientes de ≥ 12 años de edad en el inicio) entre el inicio y después de ~ 52 semanas de tratamiento con setmelanotida.
    • Evaluar el efecto de la setmelanotida en la proporción de pacientes que alcanzaron una mejora de ≥25 % en la media semanal de la escala de hambre diaria (en pacientes de ≥ 12 años de edad en el inicio) después de ~ 52 semanas de tratamiento con setmelanotida.
    • Evaluar el efecto de la setmelanotida sobre la media del porcentaje de cambio desde el inicio de la media semanal de la escala de hambre diaria (en pacientes de ≥12 años de edad en el inicio) después de ~ 52 semanas de tratamiento con setmelanotida
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. BBS clinical diagnosis as per Beales, 1999 (with either 4 primary features or 3 primary and 2 secondary features, as described below):
    - Primary Diagnostic Criteria
    • Rod cone dystrophy
    • Learning disabilities
    • Polydactyly
    • Hypogonadism in males
    • Obesity
    • Renal anomalies
    - Secondary Diagnostic Criteria:
    • Speech disorder/delay
    • Mild spasticity (especially lower limbs)
    • Strabismus/cataracts/astigmatism
    • Diabetes mellitus
    • Brachydactyly/syndactyly
    • Dental crowding/hypodontia/small roots/high arched palate
    • Developmental delay
    • Left ventricular hypertrophy/congenital heart disease
    • Polyuria/polydipsia (nephrogenic diabetes insipidus)
    • Hepatic fibrosis
    • Ataxia/poor coordination

    Or AS diagnosis as per Marshall, 2007 (using major and minor age adjusted criteria, as described below).
    For patients 6 to b $14 years of age - Minimum Required: 2 major criteria or 1 major and 3 minor criteria, as follow:
    - Major Diagnostic Criteria
    • Mutation of ALMS1 and/or family history of AS
    • Vision (nystagmus, photophobia, diminished acuity, if old enough for testing: cone dystrophy by ERG)
    - Minor Diagnostic Criteria
    • Obesity and/or insulin resistance and/or T2DM
    • History of DCM/CHF
    • Hearing loss
    • Hepatic dysfunction
    • Renal failure
    • Advanced bone age

    For patients b %15 years of age - Minimum Required: 2 major and 2 minor criteria or 1 major and 4 minor criteria, as listed below:
    - Major Diagnostic Criteria:
    • Mutation of ALMS1 and/or family history of AS
    • Vision (history of nystagmus in infancy/childhood, legal blindness, cone and rod dystrophy by ERG)
    - Minor Diagnostic Criteria:
    • Obesity and/or insulin resistance and/or T2DM
    • History of DCM/CHF
    • Hearing loss
    • Hepatic dysfunction
    • Renal failure
    • Short stature
    • Males: hypogonadism
    • Females: irregular menses

    Note: at least 90% of patients with BBS and 100% of patients with AS must have genetically confirmed diagnosis at the time of enrollment
    • A genetically confirmed diagnosis of BBS is defined as a homozygous or compound heterozygous loss-of-function mutation in BBS genes; patients without a genetically confirmed BBS diagnosis must be reviewed with the Sponsor medical monitor prior to enrollment.
    • A genetically confirmed diagnosis of AS is defined as a homozygous or compound heterozygous loss-of-function mutation in the ALMS1 gene.
    Once 10% of patients without a confirmed BBS diagnosis have been enrolled in the study, sites will be notified that only genetically confirmed BBS patients may be enrolled.

    2. b %6 years of age.
    3. Obese, defined as BMI b %30 kg/m2 for patients b %16 years of age or weight >97th percentile for age and sex on growth chart assessment for patients 6 to 15 years of age.
    4. 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 is able to understand and sign the written informed consent/assent.
    5. Female participants of child-bearing potential must be confirmed non-pregnant and 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), post-menopausal for at least 12 months (and confirmed with a screening follicle stimulating hormone (FSH) level in the post-menopausal lab range), orfailure to have progressed to Tanner Stage V and/or failure to have achieved menarche, do not require contraception during the study.
    6. Male participants with female partners of childbearing potential must agree to use a double barrier method contraception if they become sexually active during the study or within 90 days following their participation in the study. Male patients must also not donate sperm during and for 90 days following their participation in the study.
    1. Diagnóstico clínico de SBB según Beales, 1999 (con 4 características primarias o 3 primarias y 2 secundarias de la tabla que aparece a continuación)
    • Criterios de diagnóstico primarios (principales)
    • Distrofia de conos rojos
    • Polidactilia
    • Obesidad
    • Discapacidades en el aprendizaje
    • Hipogonadismo en varones
    • Anomalias renales

    Criterios de diagnóstico secundarios
    • Trastornos de retraso en el habla
    • Estrabismo/cataratas/astigmatismo
    • Braquidactilia/sindactilia
    • Espasticidad leve
    • Diabetes Mellitus
    • Apiñamiento dental/Hipodontia/raíces pequeñas/paladar ojival
    • Retraso en el desarrollo
    • Poliuria/Polidipsia (diabetes insípida nefrogénica)
    • Ataxia/pobre coordinación
    • Hipertrofia ventricular izquierda/enfermedad cardiaca congénita
    • Fibrosis hepática

    O diagnóstico de SA según Marshall, 2007 (usando criterios ajustados principales y menores de la tabla que aparece a continuación).
    Criterios diagnósticos
    Para pacientes de 6 a 14 años de edad. Minimo requerido: 2 criterios principales o 1 criterio principal y 3 secundarios.
    Criterios principales
    • Mutación de ALMS1 y/o antecedentes familiares de SA
    • Visión (nistagmo, fotofobia, agudeza visual disminuida, si es lo bastante mayor para hacerle pruebas: distrofia de conos detectada mediante ERG)
    Criterios menores
    • Obesidad y/o resistencia a la insulina y/o DM2
    • Antecedentes de MCD/ICC
    • Pérdida de oído
    • Disfunción hepática
    • Insuficiencia renal
    • Edad ósea avanzada
    Para pacientes de ≥ 15 años de edad,Mínimo requerido: 2 criterios principales y 2 criterios menores o 1 criterio principal y 4 criterios menores
    Criterios principales
    • Mutación de ALMS1 y/o antecedentes familiares de SA
    • Visión (antecedentes de nistagmo en la infancia/niñez, ceguera legal, distrofia de conos y bastones detectada mediante ERG)
    Criterios menores
    • Obesidad y/o resistencia a la insulina y/o DM2
    • Antecedentes de MCD/ICC
    • Pérdida de oído
    • Disfunción hepática
    • Insuficiencia renal
    • Baja estatura
    • Varones: hipogonadismo
    • Mujeres: periodos irregulares e/o hiperandrogenismo
    Nota: al menos el 90 % de los pacientes con SBB y el 100 % de los pacientes con SA deben tener un diagnóstico genéticamente confirmado en el momento de la inclusión
    • Se define el diagnóstico genéticamente confirmado de SBB como una mutación homocigótica o heterocigótica compuesta de pérdida de función en los genes BBS (del SBB); los pacientes sin un diagnóstico genéticamente confirmado de SBB serán revisados con el supervisor médico del promotor antes de la inclusión.
    • Se define el diagnóstico genéticamente confirmado de SA como una mutación homocigótica o heterocigótica compuesta de pérdida de función en el gen ALMS1
    Una vez el 10 % de los pacientes sin un diagnóstico genéticamente confirmado de SBB hayan sido incluidos en el estudio, se notificará a los centros que solo podrán incluirse a pacientes con diagnóstico genéticamente confirmado de SBB.
    2. > =6 años de edad.
    3. Obesos, definidos como con IMC ≥30 kg/m2 para pacientes ≥16 años de edad o peso >97.º percentil por edad y sexo respecto a la gráfica de evaluación del crecimiento para pacientes de entre 6 y 15 años de edad.
    4. El participante en el estudio y/o su progenitor o tutor puede comunicarse bien con el investigador, puede comprender y cumplir con los requisitos del estudio y pueden comprender y firmar el consentimiento/asentimiento informado por escrito.
    5. Las participantes (femeninas) con capacidad de concebir deben tener prueba negativa de embarazo confirmada y aceptar utilizar anticonceptivos como se definen en el protocolo. Las participantes (femeninas) sin capacidad de concebir, definidas como quirúrgicamente estériles (estado posterior a histerectomía, ooforectomía bilateral o ligadura bilateral de trompas), posmenopáusicas durante al menos 12 meses (y confirmado mediante niveles de FSH en el rango considerado como posmenopáusico por el laboratorio en la selección), o que no hayan progresado al estadio V de Tanner y/o que aún no hayan alcanzado la menarquia, no necesitarán anticonceptivos durante el estudio.
    6. Los participantes masculinos con parejas femeninas con capacidad de concebir, deben consentir en utilizar un método de barrera doble si son sexualmente activos durante el estudio o dentro de los 90 días siguientes a su participación en el estudio. Los participantes masculinos tampoco deben donar esperma durante y hasta 90 días después de su participación en el estudio.
    E.4Principal exclusion criteria
    1. Recent intensive (within 2 months) diet and/or exercise regimen with or without the use of weight loss agents (including herbal medications) that has resulted in >2% weight loss. These patients may be reconsidered approximately 1 month after cessation of such intensive regimens.
    2. Current or prior (within prior 2 months) use of any medication, including those approved to treat obesity, that could impact the efficacy results of this study (eg, orlistat, lorcaserin, phentermine-topiramate, naltrexone-bupropion, liraglutide). Patients on a stable dose and regimen (for at least 2 months) of medication to treat attention deficit hyperactivity disorder (ADHD) may be enrolled in the study as long as they agree to remain on the same dose and regimen during the study.
    3. Prior gastric bypass surgery resulting in >10% weight loss durably maintained from the baseline pre-operative weight with no evidence of weight regain. Specifically, patients may be considered if surgery was not successful, resulted in <10% weight loss compared to pre-operative baseline weight, or there is clear evidence of weight regain after an initial response to bariatric surgery. All patients with a history of bariatric surgery must be discussed with, and receive approval from, the Sponsor prior to enrollment.
    4. Diagnosis of schizophrenia, bipolar disorder, personality disorder or other Diagnostic and Statistical Manual of Mental Disorders fifth edition (DSM-V) disorders that the Investigator believes will interfere significantly with study compliance. Neurocognitive disorders affecting ability to consent will not be disqualifying as long as an appropriate guardian able to give consent has been appointed.
    5. In patients with no significant neurocognitive deficits:
    • A PHQ-9 score of b %15 and/or
    • Any suicidal ideation of type 4 or 5 on the C-SSRS, any lifetime history of a suicide attempt, or any suicidal behavior in the last month.
    6. Current, clinically significant pulmonary, cardiac, or oncologic disease considered severe enough to interfere with the study and/or confound the results. Any patient with a potentially clinically significant disease should be reviewed with the Sponsor to determine eligibility.
    7. History of significant liver disease or liver injury, or a current liver assessment due to abnormal liver tests (as indicated by abnormal liver function tests, alanine transaminase [ALT], aspartate transaminase [AST], alkaline phosphatase, or serum bilirubin >1.5 the 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 is exclusionary, but the presence of NAFLD is not be exclusionary. 8. Moderate to severe renal dysfunction defined as <30 mL/min (Appendix
    11.6).
    9. History or close family history (parents or siblings) of skin cancer or melanoma (excluding non-invasive basal or squamous cell lesion), or patient history of ocular-cutaneous albinism.
    10. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion), determined as part of comprehensive skin evaluation performed by a qualified dermatologist during screening. Any concerning lesions identified during the screening period will be biopsied and results must be known to be benign prior to enrollment. If the pre-treatment biopsy results are of concern, the patient should be excluded from the study.
    11. Patient is, in the opinion of the Study Investigator, not suitable to participate in the study.
    12. Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing.
    13. Significant hypersensitivity to study drug.
    14. Inability to comply with QD injection regimen.
    1. Dieta intensiva reciente (dentro de los últimos dos meses) y/o régimen de ejercicio con o sin el uso de agentes para perder peso (incluyendo medicación a base de hierbas) que haya resultado en una pérdida de peso de >2 %. Se podrá volver a considerar a estos pacientes aproximadamente un mes después de que dejen dicho régimen intensivo.
    2. Uso actual o previo (dentro de los últimos dos meses) de cualquier medicamento, incluyendo los aprobados para tratar la obesidad, que pudiera tener influencia sobre los resultados de eficacia de este estudio (p. ej., orlistat, lorcaserina, fentermina-topiramato, naltrexona-bupropion, liraglutida). Se podrá incluir a pacientes con dosis y régimen estable (durante al menos 2 meses) de medicación para tratar el trastorno por déficit de atención e hiperactividad (TDAH) en el estudio siempre y cuando estén de acuerdo en mantener la misma dosis y régimen durante el estudio.
    3. Cirugía previa de baipás gástrico con un resultado de una pérdida de peso >10 % mantenida duraderamente desde el peso en el inicio antes de la operación sin evidencia de nueva ganancia de peso. Específicamente, se podrá considerar a los pacientes si la cirugía no tuvo éxito, dio como resultado una pérdida de peso de <10 % en comparación con el peso en el inicio antes de la operación, o hay una clara evidencia de nueva ganancia de peso después de una respuesta inicial a la cirugía bariátrica. Todos los pacientes con historial de cirugía bariátrica deben ser analizados y recibir la aprobación del promotor antes de ser incluidos.
    4. Diagnóstico de esquizofrenia, trastorno bipolar, trastorno de personalidad u otro trastorno de los recogidos en el Manual diagnóstico y estadístico de trastornos mentales (Diagnostic and Statistical Manual of Mental Disorders, DSM-V) que el investigador crea interferirá significativamente con el cumplimiento del estudio. Los trastornos neurocognitivos que afecten la capacidad de consentir no serán descalificativos siempre y cuando se haya designado un tutor adecuado capaz de otorgar consentimiento.
    5. En pacientes sin déficits neurocognitivos significativos:
    • Una puntuación en el Cuestionario de salud del paciente 9 (Patient Health Questionnaire-9, PHQ-9) ≥15 y/o
    • Cualquier ideación de suicidio de tipo 4 o 5 en la C-SSRS, cualquier antecedente de intento de suicidio a lo largo de su vida, o cualquier comportamiento suicida en el último mes.
    6. Enfermedad actual clínicamente significativa pulmonar, cardiaca u oncológica considerada lo bastante grave como para interferir con el estudio y/o confundir los resultados. Cualquier paciente con una enfermedad potencial y clínicamente significativa debe ser revisado con el promotor para determinar su idoneidad.
    7. Historial de enfermedad o lesión hepática significativa, o evaluación actual del hígado debida a pruebas hepáticas anómalas (tal como indican las pruebas funcionales hepáticas anómalas de alanina transaminasa [ALT], aspartato transaminasa [AST], fosfatasa alcalina, o bilirrubina sérica [>1,5 el límite superior de la normalidad o LSN] para cualquiera de estos análisis) para una etiología diferente a la enfermedad de hígado graso no alcohólico (EHGNA). Así pues, cualquier etiología subyacente aparte de la EHGNA, incluyendo esteatohepatitis no alcohólica (EHNA) diagnosticada, otras causas de hepatitis, o antecedentes de cirrosis hepática serán motivo de exclusión, pero la presencia de EHGNA no sería excluyente.
    8. Disfunción renal moderada o grave según la definición de la ecuación de Cockroft Gault <30 ml/min.
    9. Historial o antecedentes de familiares cercanos (padres o hermanos) de cáncer de piel o melanoma (excluyendo las lesiones no invasivas basales o de células escamosas), o antecedentes del paciente de albinismo óculo-cutáneo.
    10. Hallazgos dermatológicos significativos relacionados con lesiones cutáneas de melanoma o premelanoma (excluyendo las lesiones no invasivas basales o de células escamosas), determinados como parte de una evaluación integral de cribado para patología cutánea realizada por un dermatólogo cualificado. Se realizará una biopsia a cualquier lesión de interés identificada durante la fase de selección y los resultados benignos deberán ser confirmados antes de la inclusión. Si los resultados de la biopsia antes del tratamiento presentan problemas, el paciente deberá ser excluido del estudio.
    11. El paciente no será, según la opinión del investigador del estudio, adecuado para participar en el estudio.
    12. Participación en cualquier estudio clínico con un fármaco/dispositivo experimental dentro de los 3 meses anteriores al primer día de administración.
    13. Hipersensibilidad significativa al fármaco del estudio.
    14. Incapacidad de cumplir con el régimen de inyección diario.
    E.5 End points
    E.5.1Primary end point(s)
    • The proportion of patients (12 years of age at baseline) who achieve a 10% reduction from baseline in body weight(ie, are responders) after ~52 weeks of treatment with setmelanotide.
    La proporción de pacientes (de ≥ 12 años de edad en el inicio) que consigue una reducción del ≥10% desde el inicio de su peso corporal (es decir, "responden") después de ~ 52 semanas de tratamiento con setmelanotida.
    E.5.1.1Timepoint(s) of evaluation of this end point
    after ~52 weeks of treatment with setmelanotide
    después de ~ 52 semanas de tratamiento con setmelanotida
    E.5.2Secondary end point(s)
    • The proportion of all patients, regardless of age at baseline, who achieve a 10% reduction from baseline in body weight (ie, are responders) after ~52 weeks of treatment with setmelanotide.
    • Mean percent change from baseline in body weight (in patients b %12 years of age at baseline) after ~52 weeks of treatment with setmelanotide.
    • The proportion of patients who achieve a b %25% improvement in the weekly average of the daily hunger score (in patients b %12 years of age at baseline) after ~52 weeks of treatment with setmelanotide.
    • Mean percent change from baseline in the weekly average of the daily hunger score (in patients b %12 years of age at baseline) after ~52 weeks of treatment with setmelanotide.
    • La proporción de todos los pacientes, independientemente de la edad que tengan en el inicio, que consigue una reducción del ≥ 10 % desde el inicio de su peso corporal (es decir, "responden") después de ~ 52 semanas de tratamiento con setmelanotida.
    • Media del porcentaje de cambio desde el inicio del peso corporal (en pacientes de ≥ 12 años de edad en el inicio) después de ~ 52 semanas de tratamiento con setmelanotida.
    • La proporción de pacientes que consigue una mejoría del ≥ 25 % en la media semanal de la escala de hambre diaria (en pacientes de ≥ 12 años de edad en el inicio) después de ~ 52 semanas de tratamiento con setmelanotida.
    • Media del porcentaje de cambio desde el inicio de la media semanal de la escala de hambre diaria (en pacientes de ≥ 12 años de edad en el inicio) después de ~ 52 semanas de tratamiento con setmelanotida
    E.5.2.1Timepoint(s) of evaluation of this end point
    after ~52 weeks of treatment with setmelanotide
    después de ~ 52 semanas de tratamiento con setmelanotida
    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 Yes
    E.6.7Pharmacodynamic Yes
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    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
    Doble ciego y abierto
    Double blind and open label
    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 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 No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Canada
    France
    Netherlands
    Spain
    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
    LVLS
    Ultimo paciente ultima visita
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months9
    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: 30
    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: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 15
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2019-04-11. Yes
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 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)
    Patients may be entered into a separate extension study if they are benefitting from the IMP.
    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 Decision2019-04-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-02-28
    P. End of Trial
    P.End of Trial StatusOngoing
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2021 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA