A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A (FRONTIER 4)
- Conditions
- Haemophilia A With InhibitorsHaemophilia A
- Interventions
- Registration Number
- NCT05685238
- Lead Sponsor
- Novo Nordisk A/S
- Brief Summary
This study is looking at how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used to avoid bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). When and how often the participants will receive Mim8 in this study depends on the treatment participant receives in the current Mim8 study participant is taking part in. The study will last for up to 5.5 years. The duration of the study depends on when the participant enrolled in this study. The study will end if Mim8 is approved and marketed in participant's country during the study, or the study will end in 2028, whichever comes first. Mim8 will be injected under the skin with a thin needle either once a week, once every two weeks or once a month. Participants will get up to 262 injections; the number of injections depends on how often participants will get injections. While taking part in this study, there are some restrictions about what medicine participants can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 451
-
Informed consent obtained before any study related activities. Study related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
-
Male or female with diagnosis of congenital haemophilia A based on medical records
-
Ongoing participation in study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728 at the time of transfer. Participant should qualify either of the following criteria:
- Participant from study NN7769-4513, who has participated in the extension part of the study for at least 12 weeks prior to enrolment in study NN7769-4532, or,
- Participant has completed the end of treatment visit for study NN7769-4514, NN7769-4516 or NN7769-4728.
-
Participant and/or participant's parent(s)/participant's Legally acceptable representative (LAR) willingness and ability to comply with scheduled visits and study procedures, including the completion of diary.
- Any disorder, except for conditions associated with haemophilia A, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol.
- Participant who has discontinued or been withdrawn from studies NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728.
- Previous participation in this study. Participation is defined as signed informed consent.
- Female who is pregnant, breast-feeding or intends to become pregnant.
- Female of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures or as required by local regulation or practice).
- Participation (i.e., signed informed consent) in any interventional, clinical study (except from study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728) of an approved or non-approved investigational medicinal product.
- Any planned major surgery, during part 1 of the study.
- Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Arm 1 Mim8 Participants entering from the multiple ascending dose (MAD) part of study NN7769-4513. In part 1, participants will receive Mim8 prophylaxis (PPX) once every two weeks (Q2W) with subcutaneous (s.c.) administration using enhanced cartridge for 26 weeks. In part 2, participants will receive Mim8 PPX once-weekly (QW), Q2W or once-monthly (QM) with s.c. administration using enhanced cartridge or DV3407 pen-injector once it is approved. Arm 2 Mim8 Participants entering from study NN7769-4514 or NN7769-4516. In part 1, participants will receive Mim8 PPX QW or QM with s.c. administration using DV3407 pen-injector for 26 weeks. In part 2, participants will receive Mim8 PPX QW, Q2W or QM with s.c. administration using DV3407 pen-injector.
- Primary Outcome Measures
Name Time Method Number of treatment emergent adverse events From visit 1 (week 0) until end of study (up to 283 weeks) Measured as count of events.
- Secondary Outcome Measures
Name Time Method Number of treated traumatic bleeding episodes From visit 1 (week 0) until end of treatment (up to 262 weeks) Measured as count of bleeds.
Mim8 plasma concentration From visit 1 (week 0) until end of treatment (up to 262 weeks) Measured as micrograms per milliliter (µg/mL).
Number of treated bleeding episodes From visit 1 (week 0) until end of treatment (up to 262 weeks) Measured as count of bleeds.
Number of treated spontaneous bleeding episodes From visit 1 (week 0) until end of treatment (up to 262 weeks) Measured as count of bleeds.
Number of treated joint bleeding episodes From visit 1 (week 0) until end of treatment (up to 262 weeks) Measured as count of bleeds.
Number of treated target joint bleeding episodes (arm 2) From visit 1 (week 0) until end of treatment (up to 262 weeks) Measured as count of bleeds.
Device handling using haemophilia device assessment tool (HDAT) (applicable for participants in arm 2 only) From visit 8 (week 26) until end of treatment (up to 262 weeks) Measured as percentage of participants.
Number of injection site reactions From visit 1 (week 0) until end of treatment (up to 262 weeks) Measured as count of reactions.
Occurrence of anti Mim8 antibodies From visit 1 (week 0) until end of treatment (up to 262 weeks) Measured as count of participants.
Trial Locations
- Locations (118)
Evelina London Children's Hospital
🇬🇧London, United Kingdom
Children's Hospital Los Angeles - Endocrinology
🇺🇸Los Angeles, California, United States
UC Denver Hemoph & Thrombo Ctr
🇺🇸Aurora, Colorado, United States
Univ of Miami/SCCC
🇺🇸Miami, Florida, United States
St Joseph's Children's Hospita
🇺🇸Tampa, Florida, United States
Children's Healthcare Atlanta
🇺🇸Atlanta, Georgia, United States
Rush University Medical Center
🇺🇸Chicago, Illinois, United States
University of Iowa_Iowa City
🇺🇸Iowa City, Iowa, United States
Central Michigan University
🇺🇸Detroit, Michigan, United States
Univ Hosp Cleveland Med Ctr
🇺🇸Cleveland, Ohio, United States
Dayton Children Hemostati Ctr
🇺🇸Dayton, Ohio, United States
Penn State MS Hershey Med Ctr
🇺🇸Hershey, Pennsylvania, United States
St Christopher Hosp for Child
🇺🇸Philadelphia, Pennsylvania, United States
Vanderbilt U Med Ctr_Nashville
🇺🇸Nashville, Tennessee, United States
Universitätsklinik für Innere Medizin V
🇦🇹Innsbruck, Austria
AKH - Klin. Abt. f. Haematologie u. Haemostaseologie
🇦🇹Wien, Austria
Cliniques universitaires Saint-Luc - Service Hématologie
🇧🇪Bruxelles, Belgium
UZ Leuven - Kindergeneeskunde
🇧🇪Leuven, Belgium
UMHAT "Tsaritsa Yoanna-ISUL"
🇧🇬Sofia, Bulgaria
McMaster University
🇨🇦Hamilton, Ontario, Canada
The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
Beijing Children's Hospital，Capital Medical University
🇨🇳Beijing, Beijing, China
Haemotology, Nanfang Hospital, Southern Medical University
🇨🇳Guangzhou, Guangdong, China
Tongji Hospital, Tongji Medical College of HUST-Hematology
🇨🇳Wuhan, Hubei, China
Tongji Hospital, Tongji Medical College of HUST
🇨🇳Wuhan, Hubei, China
Xiangya Hospital Central-South University
🇨🇳Changsha, Hunan, China
Chengdu Women's and Children's central hospital
🇨🇳Chengdu, Sichuan, China
Institute of hematology and Blood Diseases Hospital, Tianjin-Hematology
🇨🇳Tianjin, Tianjin, China
Children's Hospital, Zhejiang University school of medicine
🇨🇳Hangzhou, Zhejiang, China
Jinan Central Hospital
🇨🇳Jinan, China
Department of Haematology, 2081
🇩🇰København Ø, Denmark
Hospices Civils de Lyon-Hopital Cardiologique Louis Pradel-1
🇫🇷Bron, France
Ap-Hp-Hopital de Bicetre-1
🇫🇷Le Kremlin Bicetre Cedex, France
Centre Hospitalier Universitaire de Lille-Institut Coeur Poumon
🇫🇷Lille, France
Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu
🇫🇷Nantes, France
Vivantes Klinikum am Friedrichshain - Innere Medizin - Angiologie und Hämostaseologie
🇩🇪Berlin, Germany
Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
🇩🇪Bonn, Germany
HZRM Haemophilie-Zentrum Rhein Main GmbH
🇩🇪Frankfurt am Main, Germany
Nirmal Hospital Pvt. Ltd.
🇮🇳Surat, Gujarat, India
Seth GS medical college and KEM Hospital
🇮🇳Mumbai, India
Sahyadri Clinical Research And Development Center
🇮🇳Pune, Maharashtra, India
Christian Medical College and Hospital
🇮🇳Ludhiana, Punjab, India
SMS Medical College & Hospital
🇮🇳Jaipur, Rajasthan, India
CMCV
🇮🇳Ranipet, Tamil Nadu, India
Post Graduate Institute of Child Health
🇮🇳Noida, Uttar Pradesh, India
St James's CRF
🇮🇪Dublin, Leinster, Ireland
Sheba MC The Israeli National Hemophilia Center
🇮🇱Tel-Hashomer, Israel
AOU Careggi Firenze
🇮🇹Firenze, Italy
Ist Clinico Humanitas Centro Trombosi e Malattie Emorragiche
🇮🇹Milano, Italy
Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Azienda Ospedaliera di Rilievo Nazionale Santobono Pausilipon
🇮🇹Napoli, Italy
Ospedale Pediatrico Bambino Ges
🇮🇹Roma, Italy
A.O.U. Policlinico Umberto I
🇮🇹Rome, Italy
Nagoya University Hospital_Blood Transfusion
🇯🇵Aichi, Japan
Ota Memorial Hospital_Pediatrics
🇯🇵Gunma, Japan
Nara Medical University Hospital_Pediatrics
🇯🇵Nara, Japan
Saitama Children's Med Centre_Hematology-Oncology
🇯🇵Saitama, Japan
Jichi Medical University Hospital_Hematology
🇯🇵Tochigi, Japan
Jichi Medical University Hospital_Pediatrics
🇯🇵Tochigi, Japan
National Center for Child Health and Development_Hematology
🇯🇵Tokyo, Japan
Tokyo Medical Univ. Hospital_Laboratory Medicine
🇯🇵Tokyo, Japan
Ogikubo Hospital_Pediatries & Blood
🇯🇵Tokyo, Japan
Daejeon Eulji Medical Center, Eulji University
🇰🇷Daejeon, Korea, Republic of
Severance Hospital, Yonsei University Health System
🇰🇷Seoul, Korea, Republic of
Gangdong Kyung Hee University Hospital
🇰🇷Seoul, Korea, Republic of
Stradini Clinic of Oncology
🇱🇻Riga, Latvia
Children University Clinical Hospital
🇱🇻Riga, Latvia
Vilnius University hospital Santaros klinikos
🇱🇹Vilnius, Lithuania
Centre of Oncology and Hematology, Vilnius University
🇱🇹Vilnius, Lithuania
Hospital Queen Elizabeth 1
🇲🇾Kota Kinabalu, Sabah, Malaysia
Hospital Ampang
🇲🇾Ampang, Selangor, Selangor, Malaysia
Centro Multidisciplinario Para El Desarrollo Especializado De La Investigación Clínica En Yucatán S.C.P. (CEMDEICY S.C.P.)
🇲🇽Merida, Mexico
Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego
🇵🇱Wroclaw, Dolnoslaskie, Poland
Instytut Hematologii i Transfuzjologii
🇵🇱Warszawa, Mazowieckie, Poland
Academisch Medisch Centrum
🇳🇱Amsterdam, Netherlands
Intytut Hematologii i Transfuzjologii
🇵🇱Warszawa, Mazowieckie, Poland
Uniwersytecki Szpital Kliniczny W Poznaniu
🇵🇱Poznań, Wielkopolskie, Poland
Szpital Uniwersytecki, Oddzial Kliniczny Hematologii
🇵🇱Krakow, Poland
UMC Utrecht, Van Creveldkliniek
🇳🇱Utrecht, Netherlands
Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa
🇵🇱Lublin, Poland
Unidade Local de Saúde de Coimbra, E.P.E.
🇵🇹Coimbra, Portugal
Centro Hospitalar Lisboa Central - Hospital Dona Estefânia
🇵🇹Lisboa, Portugal
Centro Hospitalar Universitario De Santo Antonio E.P.E
🇵🇹Porto, Portugal
Unidade Local de Saude de Sao Joao E.P.E
🇵🇹Porto, Portugal
Institut Oncologic "Prof. Dr. Ion Chiricuta" Cluj Napoca
🇷🇴Cluj-Napoca, Romania
Clinical Centre of Vojvodina
🇷🇸Novi Sad, Serbia
King Faisal Specialist Hospital & Research Centre, Riyadh
🇸🇦Riyadh, Saudi Arabia
Clinical Centre of Serbia, Institute for Haematology
🇷🇸Belgrade, Serbia
Hospital Sant Joan de Déu
🇪🇸Esplugues Llobregat, Spain
Univerzitna Nemocnica Martin
🇸🇰Martin, Slovakia
Vseobecna nemocnica Rimavska Sobota
🇸🇰Rimavska Sobota, Slovakia
Vranovska nemocnica, a.s.
🇸🇰Vranov nad Toplou, Slovakia
Wits Bara Clinical Trial Site
🇿🇦Johannesburg, Gauteng, South Africa
Charlotte Maxeke Johannesburg Academic Hospital
🇿🇦Parktown, Johannesburg, Gauteng, South Africa
Hospital Vall d'Hebron
🇪🇸Barcelona, Spain
Hospital Universitario La Paz
🇪🇸Madrid, Spain
Hospital Carlos Haya
🇪🇸Málaga, Spain
Hospital Regional Universitario de Málaga
🇪🇸Málaga, Spain
Universitätsklinik für Hämatologie
🇨🇭Bern, Switzerland
Pädiatrische Onkologie-Hämatologie
🇨🇭Luzern 16, Switzerland
Zentrum für Labormedizin
🇨🇭St. Gallen, Switzerland
Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie
🇨🇭Zürich, Switzerland
Taichung Veterans General Hospital
🇨🇳Taichung City, Taiwan
Chung Shan Medical University Hospital
🇨🇳Taichung, Taiwan
National Taiwan University Hospital
🇨🇳Taipei, Taiwan
Gazi University
🇹🇷Ankara, Beşevler/Ankara, Turkey
Acibadem Adana Hastanesi
🇹🇷Adana, Turkey
Hacettepe Universitesi
🇹🇷Ankara, Turkey
Akdeniz Universitesi
🇹🇷Antalya, Turkey
Ege Universitesi Tip Fakultesi
🇹🇷Bornova-IZMIR, Turkey
Istanbul University Oncology Institute
🇹🇷Capa-ISTANBUL, Turkey
Trakya University
🇹🇷Edirne, Turkey
Belfast City Hospital
🇬🇧Belfast, United Kingdom
Arthur Bloom Haemophilia Centre
🇬🇧Cardiff, United Kingdom
Royal Free Haemophilia Comprehensive Care Center
🇬🇧London, United Kingdom
Royal Free Haemophilia Comprehensive Care Centre
🇬🇧London, United Kingdom
St Thomas' Hospital
🇬🇧London, United Kingdom
Royal Hallamshire Hospital
🇬🇧Sheffield, United Kingdom