Ignite Creation Date:
2025-12-26 @ 10:57 AM
Ignite Modification Date:
2025-12-26 @ 10:57 AM
Study NCT ID:
NCT04204408
Status:
COMPLETED
Last Update Posted:
2025-12-19
First Post:
2019-12-17
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Research Study Investigating Mim8 in People With Haemophilia A
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR'], 'maskingDescription': 'Sponsor staff involved in the clinical trial is masked according to company standard procedures'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Part 1 placebo-controlled double-blind within cohorts (phase 1) Part 2 open-label (phase 2)'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 275}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-01-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'dispFirstSubmitDate': '2024-10-04', 'completionDateStruct': {'date': '2023-10-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-12-15', 'studyFirstSubmitDate': '2019-12-17', 'studyFirstSubmitQcDate': '2019-12-17', 'dispFirstPostDateStruct': {'date': '2024-10-08', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2025-12-19', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2019-12-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-10-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Part 1: Number of treatment emergent adverse events', 'timeFrame': 'From time of dosing (Day 1) to Week 16', 'description': 'Count'}, {'measure': 'Part 2: Number of treatment emergent adverse events', 'timeFrame': 'From time of first dosing (Day 1) to Week 12', 'description': 'Count'}, {'measure': 'Part 2, extension: Number of treatment emergent adverse events', 'timeFrame': 'From Week 12 up to Week 176 (16 weeks after last dose)', 'description': 'Count'}], 'secondaryOutcomes': [{'measure': 'Part 1: Number of injection site reactions', 'timeFrame': 'From time of dosing (Day 1) to Week 16', 'description': 'Count'}, {'measure': 'Part 1: Relative change in D-dimer', 'timeFrame': 'From baseline (Day 1) to Week 16', 'description': 'Percent'}, {'measure': 'Part 1: Relative change in prothrombin fragment 1 and 2', 'timeFrame': 'From baseline (Day 1) to Week 16', 'description': 'Percent'}, {'measure': 'Part 1: Relative change in fibrinogen', 'timeFrame': 'From baseline (Day 1) to Week 16', 'description': 'Percent'}, {'measure': 'Part 1: Relative change in platelets', 'timeFrame': 'From baseline (Day 1) to Week 16', 'description': 'Percent'}, {'measure': 'Part 1: Cmax, SD: the maximum concentration of Mim8 after a single dose', 'timeFrame': 'From baseline (Day 1) to Week 16', 'description': 'μg/mL'}, {'measure': 'Part 1: AUC0-inf, SD: the area under the Mim8 concentration-time curve from time 0 to infinity after a single dose', 'timeFrame': 'From baseline (Day 1) to Week 16', 'description': 'μg\\*day/mL'}, {'measure': 'Part 1: t1/2, SD: the terminal half-life of Mim8 after a single dose', 'timeFrame': 'From baseline (Day 1) to Week 16', 'description': 'Days'}, {'measure': 'Part 1: tmax, SD: the time to maximum concentration of Mim8 after a single dose', 'timeFrame': 'From baseline (Day 1) to Week 16', 'description': 'Days'}, {'measure': 'Part 1: Change in activated partial thromboplastin time', 'timeFrame': 'From baseline (Day 1) to Week 16', 'description': 'Seconds'}, {'measure': 'Part 2 (weekly and monthly dosing): Number of injection site reactions', 'timeFrame': 'From time of first dosing (Day 1) to Week 12', 'description': 'Count'}, {'measure': 'Part 2 (weekly and monthly dosing): Occurrence of anti-Mim8 antibodies', 'timeFrame': 'From baseline (Day 1) to Week 12', 'description': 'Count'}, {'measure': 'Part 2 (weekly and monthly dosing): Relative change in D-dimer', 'timeFrame': 'From baseline (Day 1) to Week 12', 'description': 'Percent'}, {'measure': 'Part 2 (weekly and monthly dosing): Relative change in prothrombin fragment 1 and 2', 'timeFrame': 'From baseline (Day 1) to Week 12', 'description': 'Percent'}, {'measure': 'Part 2 (weekly and monthly dosing): Relative change in fibrinogen', 'timeFrame': 'From baseline (Day 1) to Week 12', 'description': 'Percent'}, {'measure': 'Part 2 (weekly and monthly dosing): Relative change in platelets', 'timeFrame': 'From baseline (Day 1) to Week 12', 'description': 'Percent'}, {'measure': 'Part 2 PK session 2 (weekly dosing): Cmax, MD: the maximum concentration of Mim8 after multiple doses', 'timeFrame': 'From Day 57 to Day 64', 'description': 'μg/mL'}, {'measure': 'Part 2 PK session 2 (weekly dosing): AUCτ, MD: the area under the Mim8 concentration-time curve in the dosing interval after multiple doses', 'timeFrame': 'From Day 57 to Day 64', 'description': 'μg\\*day/mL'}, {'measure': 'Part 2 PK session 2 (monthly dosing): Cmax, MD: the maximum concentration of Mim8 after multiple doses', 'timeFrame': 'From Day 57 to Day 85', 'description': 'μg/mL'}, {'measure': 'Part 2 PK session 2 (monthly dosing): AUCτ, MD: the area under the Mim8 concentration-time curve in the dosing interval after multiple doses', 'timeFrame': 'From Day 57 to Day 85', 'description': 'μg\\*day/mL'}, {'measure': 'Part 2 (weekly dosing): Mean of maximum thrombin generation (peak height)', 'timeFrame': 'From Day 57 to Day 64', 'description': 'nM'}, {'measure': 'Part 2 (monthly dosing): Mean of maximum thrombin generation (peak height)', 'timeFrame': 'From Day 57 to Day 85', 'description': 'nM'}, {'measure': 'Part 2, extension: Number of injection site reactions', 'timeFrame': 'From Week 12 up to Week 176 (16 weeks after last dose)', 'description': 'Count'}, {'measure': 'Part 2, extension: Occurrence of anti-Mim8 antibodies', 'timeFrame': 'From Week 12 up to Week 176 (16 weeks after last dose)', 'description': 'Count'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Healthy Volunteers', 'Haemophilia A With or Without Inhibitors']}, 'referencesModule': {'references': [{'pmid': '41246456', 'type': 'DERIVED', 'citation': 'Chowdary P, Lentz SR, Gil L, Lopez-Jaime FJ, Windyga J, Ong Clausen WH, Laursen PN, Mahlangu J. FRONTIER1 multiple ascending dose extension: a safety, tolerability, pharmacokinetics, and pharmacodynamics study of Mim8 in people with hemophilia A. Res Pract Thromb Haemost. 2025 Oct 8;9(7):103207. doi: 10.1016/j.rpth.2025.103207. eCollection 2025 Oct.'}]}, 'descriptionModule': {'briefSummary': 'This study is investigating how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medication that will be used for prevention of bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected with a thin needle in the skin of the stomach, using a pen-injector.\n\nThe study will last for up to 44 months. It consists of a main phase (part 1 and part 2) and an extension phase. In part 1, participants will be injected only once with either Mim8 or a "dummy" medicine (placebo) - which one will be decided by chance. In part 2 and the extension phase participants will get an Mim8 injection weekly or monthly.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\nSingle ascending dose part 1:\n\n* Male, aged 18-45 years (both inclusive) at the time of signing informed consent\n* Considered to be generally healthy based on the medical history, physical examination, and the results of vital signs, electrocardiogram and clinical laboratory tests performed during the screening visit, as judged by the investigator\n\nMultiple ascending dose part 2:\n\n* Male, aged 12-64 years (both inclusive) at the time of signing informed consent (Germany and Japan have local requirements)\n* Diagnosis of congenital haemophilia A with FVIII activity below 1% based on medical records\n\nExploratory biomarker cohort:\n\n* Male, aged equal to or above 12 years at the time of signing informed consent (Germany and Japan have local requirements)\n* Diagnosis of congenital haemophilia A with FVIII activity below 1% based on medical recordsv\n\nExclusion Criteria:\n\nPart 1:\n\n* Factor VIII activity equal to or above 150% at screening\n* Increased risk of thrombosis, e.g. known history of personal or first degree relative(s) with unprovoked deep vein thrombosis\n* Any clinical signs or established diagnosis of venous or arterial thromboembolic disease\n\nPart 2:\n\n* Known congenital or acquired coagulation disorders other than haemophilia A\n* Increased risk of thrombosis as evaluated by the investigator. E.g. known history of personal or first degree relative(s) with unprovoked deep vein thrombosis with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing\n* Any clinical signs or established diagnosis of venous or arterial thromboembolic disease with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing\n* Advanced atherosclerotic disease (e.g. known history of ischemic heart disease, ischemic stroke) as evaluated by the investigator\n* Any autoimmune disease that may increase the risk of thrombosis\n* Receipt of emicizumab or drugs with similar modes of action within 5 half-lives before trial product administration\n* Ongoing or planned immune tolerance induction therapy\n\nExploratory biomarker cohort:\n\n* Known congenital or acquired coagulation disorders other than haemophilia A\n* Increased risk of thrombosis as evaluated by the investigator. E.g. known history of personal or first degree relative(s) with unprovoked deep vein thrombosis with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing\n* Any clinical signs or established diagnosis of venous or arterial thromboembolic disease with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing\n* Advanced atherosclerotic disease (e.g. known history of ischemic heart disease, ischemic stroke) as evaluated by the investigator\n* Any autoimmune disease that may increase the risk of thrombosis\n* Ongoing or planned immune tolerance induction therapy'}, 'identificationModule': {'nctId': 'NCT04204408', 'acronym': 'FRONTIER1', 'briefTitle': 'A Research Study Investigating Mim8 in People With Haemophilia A', 'organization': {'class': 'INDUSTRY', 'fullName': 'Novo Nordisk A/S'}, 'officialTitle': 'Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Subcutaneous Doses of NNC0365-3769 (Mim8) in Healthy Subjects and in Subjects With Haemophilia A With or Without Factor VIII Inhibitors', 'orgStudyIdInfo': {'id': 'NN7769-4513'}, 'secondaryIdInfos': [{'id': 'U1111-1227-4220', 'type': 'OTHER', 'domain': 'World Health Organization (WHO)'}, {'id': '2019-000465-20', 'type': 'REGISTRY', 'domain': 'European Medicines Agency (EudraCT)'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Single dose (part 1) Mim8', 'description': 'Blinded. Single doses in healthy volunteers. Dose escalation. In each of the 6 cohorts, 6 participants will receive Mim8.', 'interventionNames': ['Drug: NNC0365-3769 (Mim8)']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Single dose (part 1) placebo', 'description': 'Blinded. Single doses in healthy volunteers. In each of the 6 cohorts, 2 participants will receive placebo.', 'interventionNames': ['Drug: Placebo (Mim8)']}, {'type': 'EXPERIMENTAL', 'label': 'Multiple dose (part 2)', 'description': 'Open-label. There will be 4 cohorts receiving once-weekly doses (part 2 cohorts 1, 2, 3 and 5) and one cohort receiving once-monthly doses (part 2 cohort 4). Participants will continue into the part 2 extension on the same treatment regimen.', 'interventionNames': ['Drug: NNC0365-3769 (Mim8)']}], 'interventions': [{'name': 'NNC0365-3769 (Mim8)', 'type': 'DRUG', 'description': 'Mim8 administered subcutaneously (s.c., under the skin). The treatment period will consist of 12 once-weekly doses or 3 once-monthly doses', 'armGroupLabels': ['Multiple dose (part 2)', 'Single dose (part 1) Mim8']}, {'name': 'Placebo (Mim8)', 'type': 'DRUG', 'description': 'Mim8 placebo administered subcutaneously (s.c., under the skin)', 'armGroupLabels': ['Single dose (part 1) placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85016-7710', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': 'Arizona H&T Phoenix Child Hosp', 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital Los Angeles - Endocrinology", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': "Children's Healthcare Atlanta", 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Rush University Med. Cntr', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'facility': 'University of Iowa_Iowa City', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '48109', 'city': 'Ann Arbor', 'state': 'Michigan', 'country': 'United States', 'facility': 'University Of Michigan', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}, {'zip': '48201', 'city': 'Detroit', 'state': 'Michigan', 'country': 'United States', 'facility': "Children's Hospital of Michigan", 'geoPoint': {'lat': 42.33143, 'lon': -83.04575}}, {'zip': '48823', 'city': 'East Lansing', 'state': 'Michigan', 'country': 'United States', 'facility': 'Michigan State University', 'geoPoint': {'lat': 42.73698, 'lon': -84.48387}}, {'zip': '28204', 'city': 'Charlotte', 'state': 'North Carolina', 'country': 'United States', 'facility': 'St. Jude Clinic Novant Health', 'geoPoint': {'lat': 35.22709, 'lon': -80.84313}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Child's Hsp Med Ctr", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '45404', 'city': 'Dayton', 'state': 'Ohio', 'country': 'United States', 'facility': 'Dayton Children Hemostati Ctr', 'geoPoint': {'lat': 39.75895, 'lon': -84.19161}}, {'zip': '17033-2360', 'city': 'Hershey', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Penn State MS Hershey Med Ctr', 'geoPoint': {'lat': 40.28592, 'lon': -76.65025}}, {'zip': '37232', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Vanderbilt Hemostasis Thrombosis Clinic', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Versiti, CCBD', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}, {'zip': '6020', 'city': 'Innsbruck', 'country': 'Austria', 'facility': 'Universitätsklinik für Innere Medizin V', 'geoPoint': {'lat': 47.26266, 'lon': 11.39454}}, {'zip': 'A 6020', 'city': 'Innsbruck', 'country': 'Austria', 'facility': 'Universitätsklinik für Innere Medizin V', 'geoPoint': {'lat': 47.26266, 'lon': 11.39454}}, {'zip': '1527', 'city': 'Sofia', 'country': 'Bulgaria', 'facility': 'UMHAT Tsaritsa Yoanna - ISUL EAD, Pediatric clinical hematology and oncology', 'geoPoint': {'lat': 42.69751, 'lon': 23.32415}}, {'zip': '10117', 'city': 'Berlin', 'country': 'Germany', 'facility': 'Charité - Campus Charité Mitte - Charité Research Organisation GmbH', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'zip': '10249', 'city': 'Berlin', 'country': 'Germany', 'facility': 'Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'zip': '20124', 'city': 'Milan', 'state': 'MI', 'country': 'Italy', 'facility': 'Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '00161', 'city': 'Roma', 'country': 'Italy', 'facility': 'Policlinico Umberto I Sezione Ematologia', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'zip': '466-8560', 'city': 'Aichi', 'country': 'Japan', 'facility': 'Nagoya University Hospital_Blood Transfusion', 'geoPoint': {'lat': 32.51879, 'lon': 130.62158}}, {'zip': '634-8522', 'city': 'Nara', 'country': 'Japan', 'facility': 'Nara Medical University Hospital_Pediatrics', 'geoPoint': {'lat': 34.68505, 'lon': 135.80485}}, {'zip': '160-0023', 'city': 'Tokyo', 'country': 'Japan', 'facility': 'Tokyo Medical Univ. Hospital_Laboratory Medicine', 'geoPoint': {'lat': 35.6895, 'lon': 139.69171}}, {'zip': '60-569', 'city': 'Poznan', 'state': 'Greater Poland Voivodeship', 'country': 'Poland', 'facility': 'Uniwersytecki Szpital Kliniczny W Poznaniu', 'geoPoint': {'lat': 52.40692, 'lon': 16.92993}}, {'zip': '02-776', 'city': 'Warsaw', 'state': 'Masovian Voivodeship', 'country': 'Poland', 'facility': 'Instytut Hematologii i Transfuzjologii', 'geoPoint': {'lat': 52.22977, 'lon': 21.01178}}, {'zip': '30-688', 'city': 'Krakow', 'country': 'Poland', 'facility': 'Szpital Uniwersytecki, Oddzial Kliniczny Hematologii', 'geoPoint': {'lat': 50.06143, 'lon': 19.93658}}, {'zip': '2193', 'city': 'Parktown, Johannesburg', 'state': 'Gauteng', 'country': 'South Africa', 'facility': 'Charlotte Maxeke Johannesburg Academic Hospital'}, {'zip': '0699', 'city': 'Polokwane', 'state': 'Limpopo', 'country': 'South Africa', 'facility': 'Pietersburg Hospital', 'geoPoint': {'lat': -23.90449, 'lon': 29.46885}}, {'zip': '28046', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Hospital Universitario La Paz', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '29010', 'city': 'Málaga', 'country': 'Spain', 'facility': 'Hospital Regional Universitario de Málaga', 'geoPoint': {'lat': 36.72016, 'lon': -4.42034}}, {'zip': '46026', 'city': 'Valencia', 'country': 'Spain', 'facility': 'Hospital La Fe - Hemostasia y Trombosis', 'geoPoint': {'lat': 39.47391, 'lon': -0.37966}}, {'zip': '3010', 'city': 'Bern', 'country': 'Switzerland', 'facility': 'Universitätsklinik für Hämatologie', 'geoPoint': {'lat': 46.94809, 'lon': 7.44744}}, {'zip': '8091', 'city': 'Zurich', 'country': 'Switzerland', 'facility': 'Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie', 'geoPoint': {'lat': 47.36667, 'lon': 8.55}}, {'zip': '06230', 'city': 'Ankara', 'country': 'Turkey (Türkiye)', 'facility': 'Hacettepe Üniversitesi Hastanesi- Endokrinoloji', 'geoPoint': {'lat': 39.91987, 'lon': 32.85427}}, {'zip': '22030', 'city': 'Edirne', 'country': 'Turkey (Türkiye)', 'facility': 'Trakya Üniversitesi Tıp Fakültesi Hastanesi- Kardiyoloji', 'geoPoint': {'lat': 41.67719, 'lon': 26.55597}}, {'zip': '35100', 'city': 'Izmir', 'country': 'Turkey (Türkiye)', 'facility': 'Ege Üniversitesi Hastanesi- Hematoloji', 'geoPoint': {'lat': 38.41273, 'lon': 27.13838}}, {'zip': 'NW3 2QG', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Royal Free Haemophilia Comprehensive Care Centre', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'OX3 7LJ', 'city': 'Oxford', 'country': 'United Kingdom', 'facility': 'Oxford Haemophilia Comprehensive Care Center', 'geoPoint': {'lat': 51.75222, 'lon': -1.25596}}], 'overallOfficials': [{'name': 'Clinical Reporting Anchor and Disclosure (1452)', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Novo Nordisk A/S'}]}, 'ipdSharingStatementModule': {'url': 'http://novonordisk-trials.com', 'ipdSharing': 'YES', 'description': 'According to the Novo Nordisk disclosure commitment on novonordisk-trials.com'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Novo Nordisk A/S', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}