Clinical trial NCT05685238: A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A (FRONTIER 4)
Official title: Open-label, Long-term Safety and Efficacy Study of Mim8 in Participants With Haemophilia A With or Without Inhibitors
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Haemophilia A
Haemophilia A With Inhibitors
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2023-01-17
Enrollment: 451
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

1. 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.
2. Male or female with diagnosis of congenital haemophilia A based on medical records
3. 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:

   1. 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,
   2. Participant has completed the end of treatment visit for study NN7769-4514, NN7769-4516 or NN7769-4728.
4. 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.

Exclusion Criteria:

1. 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.
2. Participant who has discontinued or been withdrawn from studies NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728.
3. Previous participation in this study. Participation is defined as signed informed consent.
4. Female who is pregnant, breast-feeding or intends to become pregnant.
5. 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).
6. 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.
7. Any planned major surgery, during part 1 of the study.
8. Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation.

Primary outcomes:
measure: Number of treatment emergent adverse events
timeFrame: From visit 1 (week 0) until end of study (up to 283 weeks)
Secondary outcomes:
measure: Number of injection site reactions
timeFrame: From visit 1 (week 0) until end of treatment (up to 262 weeks)
measure: Occurrence of anti Mim8 antibodies
timeFrame: From visit 1 (week 0) until end of treatment (up to 262 weeks)
measure: Number of treated bleeding episodes
timeFrame: From visit 1 (week 0) until end of treatment (up to 262 weeks)
measure: Number of treated spontaneous bleeding episodes
timeFrame: From visit 1 (week 0) until end of treatment (up to 262 weeks)
measure: Number of treated traumatic bleeding episodes
timeFrame: From visit 1 (week 0) until end of treatment (up to 262 weeks)
measure: Number of treated joint bleeding episodes
timeFrame: From visit 1 (week 0) until end of treatment (up to 262 weeks)
measure: Number of treated target joint bleeding episodes (arm 2)
timeFrame: From visit 1 (week 0) until end of treatment (up to 262 weeks)
measure: Mim8 plasma concentration
timeFrame: From visit 1 (week 0) until end of treatment (up to 262 weeks)
measure: Device handling using haemophilia device assessment tool (HDAT) (applicable for participants in arm 2 only)
timeFrame: From visit 8 (week 26) until end of treatment (up to 262 weeks)
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles - Endocrinology
state: California
city: Aurora
country: United States
facility: UC Denver Hemoph & Thrombo Ctr
state: Colorado
city: Miami
country: United States
facility: Univ of Miami/SCCC
state: Florida
city: Tampa
country: United States
facility: St Joseph's Children's Hospita
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare Atlanta
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa_Iowa City
state: Iowa
city: Detroit
country: United States
facility: Central Michigan University
state: Michigan
city: Cleveland
country: United States
facility: Univ Hosp Cleveland Med Ctr
state: Ohio
city: Dayton
country: United States
facility: Dayton Children Hemostati Ctr
state: Ohio
city: Hershey
country: United States
facility: Penn State MS Hershey Med Ctr
state: Pennsylvania
city: Philadelphia
country: United States
facility: St Christopher Hosp for Child
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt U Med Ctr_Nashville
state: Tennessee
city: Innsbruck
country: Austria
facility: Universitätsklinik für Innere Medizin V
city: Innsbruck
country: Austria
facility: Universitätsklinik für Innere Medizin V
city: Wien
country: Austria
facility: AKH - Klin. Abt. f. Haematologie u. Haemostaseologie
city: Bruxelles
country: Belgium
facility: Cliniques universitaires Saint-Luc - Service Hématologie
city: Leuven
country: Belgium
facility: UZ Leuven - Kindergeneeskunde
city: Sofia
country: Bulgaria
facility: UMHAT "Tsaritsa Yoanna-ISUL"
city: Hamilton
country: Canada
facility: McMaster University
state: Ontario
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Beijing
country: China
facility: Beijing Children's Hospital，Capital Medical University
state: Beijing
city: Guangzhou
country: China
facility: Haemotology, Nanfang Hospital, Southern Medical University
state: Guangdong
city: Wuhan
country: China
facility: Tongji Hospital, Tongji Medical College of HUST-Hematology
state: Hubei
city: Wuhan
country: China
facility: Tongji Hospital, Tongji Medical College of HUST
state: Hubei
city: Changsha
country: China
facility: Xiangya Hospital Central-South University
state: Hunan
city: Chengdu
country: China
facility: Chengdu Women's and Children's central hospital
state: Sichuan
city: Tianjin
country: China
facility: Institute of hematology and Blood Diseases Hospital, Tianjin-Hematology
state: Tianjin
city: Hangzhou
country: China
facility: Children's Hospital, Zhejiang University school of medicine
state: Zhejiang
city: Jinan
country: China
facility: Jinan Central Hospital
city: København Ø
country: Denmark
facility: Department of Haematology, 2081
city: Bron
country: France
facility: Hospices Civils de Lyon-Hopital Cardiologique Louis Pradel-1
city: Le Kremlin Bicetre Cedex
country: France
facility: Ap-Hp-Hopital de Bicetre-1
city: Lille
country: France
facility: Centre Hospitalier Universitaire de Lille-Institut Coeur Poumon
city: Nantes
country: France
facility: Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu
city: Berlin
country: Germany
facility: Vivantes Klinikum am Friedrichshain - Innere Medizin - Angiologie und Hämostaseologie
city: Bonn
country: Germany
facility: Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
city: Frankfurt am Main
country: Germany
facility: HZRM Haemophilie-Zentrum Rhein Main GmbH
city: Surat
country: India
facility: Nirmal Hospital Pvt. Ltd.
state: Gujarat
city: Mumbai
country: India
facility: Seth GS medical college and KEM Hospital
state: Maharashtra
city: Pune
country: India
facility: Sahyadri Clinical Research And Development Center
state: Maharashtra
city: Ludhiana
country: India
facility: Christian Medical College and Hospital
state: Punjab
city: Jaipur
country: India
facility: SMS Medical College & Hospital
state: Rajasthan
city: Ranipet
country: India
facility: CMCV
state: Tamil Nadu
city: Noida
country: India
facility: Post Graduate Institute of Child Health
state: Uttar Pradesh
city: Mumbai
country: India
facility: Seth GS medical college and KEM Hospital
city: Dublin
country: Ireland
facility: St James's CRF
state: Leinster
city: Tel-Hashomer
country: Israel
facility: Sheba MC The Israeli National Hemophilia Center
city: Firenze
country: Italy
facility: AOU Careggi Firenze
city: Milano
country: Italy
facility: Ist Clinico Humanitas Centro Trombosi e Malattie Emorragiche
city: Milano
country: Italy
facility: Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico
city: Napoli
country: Italy
facility: Azienda Ospedaliera di Rilievo Nazionale Santobono Pausilipon
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Ges
city: Rome
country: Italy
facility: A.O.U. Policlinico Umberto I
city: Aichi
country: Japan
facility: Nagoya University Hospital_Blood Transfusion
city: Gunma
country: Japan
facility: Ota Memorial Hospital_Pediatrics
city: Nara
country: Japan
facility: Nara Medical University Hospital_Pediatrics
city: Saitama
country: Japan
facility: Saitama Children's Med Centre_Hematology-Oncology
city: Tochigi
country: Japan
facility: Jichi Medical University Hospital_Hematology
city: Tochigi
country: Japan
facility: Jichi Medical University Hospital_Pediatrics
city: Tokyo
country: Japan
facility: National Center for Child Health and Development_Hematology
city: Tokyo
country: Japan
facility: Tokyo Medical Univ. Hospital_Laboratory Medicine
city: Tokyo
country: Japan
facility: Ogikubo Hospital_Pediatries & Blood
city: Daejeon
country: Korea, Republic of
facility: Daejeon Eulji Medical Center, Eulji University
city: Seoul
country: Korea, Republic of
facility: Severance Hospital, Yonsei University Health System
city: Seoul
country: Korea, Republic of
facility: Gangdong Kyung Hee University Hospital
city: Riga
country: Latvia
facility: Stradini Clinic of Oncology
city: Riga
country: Latvia
facility: Children University Clinical Hospital
city: Vilnius
country: Lithuania
facility: Vilnius University hospital Santaros klinikos
city: Vilnius
country: Lithuania
facility: Centre of Oncology and Hematology, Vilnius University
city: Kota Kinabalu
country: Malaysia
facility: Hospital Queen Elizabeth 1
state: Sabah
city: Ampang, Selangor
country: Malaysia
facility: Hospital Ampang
state: Selangor
city: Merida
country: Mexico
facility: Centro Multidisciplinario Para El Desarrollo Especializado De La Investigación Clínica En Yucatán S.C.P. (CEMDEICY S.C.P.)
city: Amsterdam
country: Netherlands
facility: Academisch Medisch Centrum
city: Utrecht
country: Netherlands
facility: UMC Utrecht, Van Creveldkliniek
city: Wroclaw
country: Poland
facility: Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego
state: Dolnoslaskie
city: Warszawa
country: Poland
facility: Instytut Hematologii i Transfuzjologii
state: Mazowieckie
city: Warszawa
country: Poland
facility: Intytut Hematologii i Transfuzjologii
state: Mazowieckie
city: Poznań
country: Poland
facility: Uniwersytecki Szpital Kliniczny W Poznaniu
state: Wielkopolskie
city: Krakow
country: Poland
facility: Szpital Uniwersytecki, Oddzial Kliniczny Hematologii
city: Lublin
country: Poland
facility: Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa
city: Coimbra
country: Portugal
facility: Unidade Local de Saúde de Coimbra, E.P.E.
city: Lisboa
country: Portugal
facility: Centro Hospitalar Lisboa Central - Hospital Dona Estefânia
city: Porto
country: Portugal
facility: Centro Hospitalar Universitario De Santo Antonio E.P.E
city: Porto
country: Portugal
facility: Unidade Local de Saude de Sao Joao E.P.E
city: Cluj-Napoca
country: Romania
facility: Institut Oncologic "Prof. Dr. Ion Chiricuta" Cluj Napoca
city: Riyadh
country: Saudi Arabia
facility: King Faisal Specialist Hospital & Research Centre, Riyadh
city: Belgrade
country: Serbia
facility: Clinical Centre of Serbia, Institute for Haematology
city: Novi Sad
country: Serbia
facility: Clinical Centre of Vojvodina
city: Martin
country: Slovakia
facility: Univerzitna Nemocnica Martin
city: Rimavska Sobota
country: Slovakia
facility: Vseobecna nemocnica Rimavska Sobota
city: Vranov nad Toplou
country: Slovakia
facility: Vranovska nemocnica, a.s.
city: Johannesburg
country: South Africa
facility: Wits Bara Clinical Trial Site
state: Gauteng
city: Parktown, Johannesburg
country: South Africa
facility: Charlotte Maxeke Johannesburg Academic Hospital
state: Gauteng
city: Barcelona
country: Spain
facility: Hospital Vall d'Hebron
city: Esplugues Llobregat
country: Spain
facility: Hospital Sant Joan de Déu
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Málaga
country: Spain
facility: Hospital Carlos Haya
city: Málaga
country: Spain
facility: Hospital Regional Universitario de Málaga
city: Bern
country: Switzerland
facility: Universitätsklinik für Hämatologie
city: Luzern 16
country: Switzerland
facility: Pädiatrische Onkologie-Hämatologie
city: St. Gallen
country: Switzerland
facility: Zentrum für Labormedizin
city: Zürich
country: Switzerland
facility: Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie
city: Taichung City
country: Taiwan
facility: Taichung Veterans General Hospital
city: Taichung
country: Taiwan
facility: Chung Shan Medical University Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Ankara
country: Turkey
facility: Gazi University
state: Beşevler/Ankara
city: Adana
country: Turkey
facility: Acibadem Adana Hastanesi
city: Ankara
country: Turkey
facility: Hacettepe Universitesi
city: Antalya
country: Turkey
facility: Akdeniz Universitesi
city: Bornova-IZMIR
country: Turkey
facility: Ege Universitesi Tip Fakultesi
city: Capa-ISTANBUL
country: Turkey
facility: Istanbul University Oncology Institute
city: Edirne
country: Turkey
facility: Trakya University
city: Belfast
country: United Kingdom
facility: Belfast City Hospital
city: Cardiff
country: United Kingdom
facility: Arthur Bloom Haemophilia Centre
city: London
country: United Kingdom
facility: Royal Free Haemophilia Comprehensive Care Center
city: London
country: United Kingdom
facility: Royal Free Haemophilia Comprehensive Care Centre
city: London
country: United Kingdom
facility: Evelina London Children's Hospital
city: London
country: United Kingdom
facility: St Thomas' Hospital
city: Sheffield
country: United Kingdom
facility: Royal Hallamshire Hospital
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Transparency dept. 2834
role: STUDY_DIRECTOR
References: