Ignite Creation Date:
2025-12-24 @ 3:23 PM
Ignite Modification Date:
2026-03-09 @ 1:59 AM
Study NCT ID:
NCT02507492
Status:
UNKNOWN
Last Update Posted:
2016-02-22
First Post:
2015-05-29
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
RM-493 Treatment Trial in Proopiomelanocortin (POMC) Deficient Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'C579663', 'term': 'setmelanotide'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 10}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2015-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2016-02', 'completionDateStruct': {'date': '2018-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2016-02-19', 'studyFirstSubmitDate': '2015-05-29', 'studyFirstSubmitQcDate': '2015-07-22', 'lastUpdatePostDateStruct': {'date': '2016-02-22', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2015-07-24', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2018-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Effect of RM-493 on Body weight', 'timeFrame': 'Day 1 through Day 84', 'description': 'Change of body weight after Treatment with RM-493.'}], 'secondaryOutcomes': [{'measure': 'Effect of RM-493 on metabolic serum parameters', 'timeFrame': 'Day 1 through Day 84', 'description': 'Measurement of the effect of RM-493 on metabolic serum parameters as measured by blood test.'}, {'measure': 'Effect of RM-493 on blood pressure', 'timeFrame': 'Day 1 through Day 84', 'description': 'Measurement of the effect of RM-493 on blood pressure by daily blood pressure measurements.'}, {'measure': 'Effect of RM-493 on hunger', 'timeFrame': 'Day 1 through Day 84', 'description': 'Measurement of the effect of RM-493 on hunger as measured by the Global Hunger Scale'}, {'measure': 'Effect of RM-493 on body composition/Energy expenditure', 'timeFrame': 'Day 1 through Day 84', 'description': 'Measurement of the effect of RM-493 on body composition/Energy expenditure'}, {'measure': 'Effect of RM-493 on weight loss after treatment continuation of 2 years', 'timeFrame': 'Day 1 through Day 730', 'description': 'Measurement of the effect of RM-493 on weight loss after treatment continuation of 2 years'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['POMC, LEPR, PCSK1'], 'conditions': ['Homozygous or Compound Heterozygous POMC, LEPR or PCSK1 Gene Mutation']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the effects of a once daily subcutaneous (SC) injection of RM-493, in subjects with POMC (propiomelanocortin) or other related rare genetic mutations, on body weight, metabolic function and blood pressure. Patients who respond during the initial 84 days of treatment can enter into long-term (2-year) extensions. The study drug (RM-493) will be administered in an unblinded fashion.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Written informed consent\n* Homozygous or compound heterozygous (different gene mutation on both alleles) POMC, LEPR or PCSK1 gene mutation\n* Obesity (BMI \\> 30 kg/m2; + 2 BMI SDS)\n* No other therapeutic option, which might cure the patient (e.g. bariatric surgery (see chapter 8))\n* Negative Pregnancy test\n* Highly effective contraception in women (defined as pearl index \\< 1), if necessary also for partners of test persons)\n* No participation in other clinical trials according to AMG (Arzneimittelgesetz) (2 months before and after) at the time of this trial\n* 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\n* sufficient kidney and liver function (Creatinine, ALT, AST)\n\n * normal values Alanine-Aminotransferase (ALT) (female): \\< 31 U/l\n * normal values Alanine-Aminotransferase (ALT) (male): \\< 41 U/l\n * normal values Aspartate-Aminotransferase (AST) (female \\> 17 years): \\< 35 U/l; (female \\< 17 years): 16- 46 U/l\n * normal values Aspartate-Aminotransferase (AST) (male \\> 17 years): \\< 50 U/l; (male \\< 17 years): 16-46 U/l\n * normal values bilirubins (male and female) up to 1,2 mg/dl\n * normal values Creatinine (female \\> 15 years): 0,51-0,95 mg/dl) ; (female \\< 15 years): 0,46-0,77 mg/dl\n * normal values Creatinine (male \\> 15 years): 0,67 - 1,17 mg/dl) ; (male \\< 15 years): 0,46-0,77 mg/dl\n\nExclusion Criteria:\n\n* Pregnancy or Breastfeeding\n* All contraindications against study medication (including auxiliary substances)\n* Interactions with study medication\n* Participation of the patient in a clinical study within the last 2 months\n* Intolerance against albumin\n* Concomitant diseases, impaired organ functions, except for known, concurrent GI disorders or other clinical findings expected in PCSK1 or LEPR gene disorders\n* Renal insufficiency (Creatinine \\> 0,95 mg/dl (female), \\> 1,17 mg/dl (male))\n* Impaired liver function (Bilirubins \\> 1.2 mg/dl)\n* Neurological / psychiatric diseases\n* HIV Infection\n* Active Hepatitis B or C\n* Melanoma or Melanoma occurrence in the family history\n* Non-compliance\n* Subjects who are legally detained in an official institution'}, 'identificationModule': {'nctId': 'NCT02507492', 'briefTitle': 'RM-493 Treatment Trial in Proopiomelanocortin (POMC) Deficient Patients', 'organization': {'class': 'OTHER', 'fullName': 'Charite University, Berlin, Germany'}, 'officialTitle': 'RM-493 Treatment Trial in Proopiomelanocortin (POMC) Deficient Patients', 'orgStudyIdInfo': {'id': 'RM-493-011'}, 'secondaryIdInfos': [{'id': '2014-002392-28', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'RM-493 Once Daily', 'description': 'Dose once daily in the morning', 'interventionNames': ['Drug: RM-493']}], 'interventions': [{'name': 'RM-493', 'type': 'DRUG', 'otherNames': ['setmelanotide'], 'armGroupLabels': ['RM-493 Once Daily']}]}, 'contactsLocationsModule': {'locations': [{'zip': '13353', 'city': 'Berlin', 'state': 'State of Berlin', 'status': 'RECRUITING', 'country': 'Germany', 'contacts': [{'name': 'Susanna Wiegand, PD Dr.', 'role': 'CONTACT', 'email': 'susanna.wiegand@charite.de', 'phone': '+49 30 450 566887'}, {'name': 'Peter Kühnen, Dr.', 'role': 'CONTACT', 'email': 'peter.kuehnen@charite.de', 'phone': '+49 30 450 450 666839'}, {'name': 'Heiko Krude, Prof. Dr.', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Peter Kühnen, Dr.', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'Charité - Universitätsmedizin Berlin', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}], 'centralContacts': [{'name': 'Susanna Wiegand, PD Dr.', 'role': 'CONTACT', 'email': 'susanna.wiegand@charite.de', 'phone': '+49 30 450 566887'}, {'name': 'Peter Kühnen, Dr.', 'role': 'CONTACT', 'email': 'peter.kuehnen@charite.de', 'phone': '+49 30 450 450 666839'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Charite University, Berlin, Germany', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Principal Investigator', 'investigatorFullName': 'PD Dr. Susanna Wiegand', 'investigatorAffiliation': 'Charite University, Berlin, Germany'}}}}