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NCT05685238PHASE3Recruiting

A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A

Novo Nordisk A/S

Start Date

2/13/2023

Completion Date

6/30/2028

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). 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 June 2028, whichever comes first. Participants will get up to 262 injections; the number of injections depends on how often participants will get injections and how long time participants take part in the study. 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

Age Range: No minimum to No maximum

Arm 1 \& 2: 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. Arm 3: 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 severe haemophilia A (endogenous FVIII activity less than (\<) 1 percentage \[%\]) with or without FVIII inhibitors based on medical records. 3. Aged \<1 year at the time of signing informed consent. 4. Body weight greater than or equal to (≥) 3.2 kilograms at the time of signing informed consent. 5. previously untreated patients (PUPs) or minimally treated patients (MTPs) (i.e., up to 5 days of exposure to haemophilia-related treatment such as plasma-derived FVIII, recombinant FVIII, fresh frozen plasma, cryoprecipitate, or whole blood products). 6. Full-term pregnancy (gestational age ≥37 weeks). 7. Participant's parent(s)/LAR(s) willingness and ability to comply with scheduled visits and Arm 3 (infant) procedures, including the completion of diary and patient reported outcome (PRO) questionnaire. 8. Participants \<3 months of age must show no signs of active intracranial haemorrhage at screening. This is confirmed by cranial ultrasound performed according to local practice and regardless of delivery method. 9. Receipt of vitamin K prophylaxis (as per local standard practice). 10. Availability of historical results in medical records for: 1. activated partial thromboplastin time (aPTT) 2. FVIII levels. 11. Availability of historical results in medical records or pre-dose sample taken for: 1. fibrinogen 2. haematology parameters 3. biochemistry parameters (aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT), bilirubin and creatinine). Exclusion criteria 1. Known or suspected hypersensitivity to trial product or related products. 2. Previous participation in study 4532. Participation is defined as signed informed consent. 3. Participation (i.e., signed informed consent) in any interventional clinical study with receipt of the last dose within 6 months (or 5 half-lives of the investigational medicinal product, whichever is shorter) before planned enrolment. 4. Exposure to non-factor haemostatic products for bleeding prophylaxis within 6 months (or 5 half-lives of the medicinal product, whichever is shorter) before planned enrolment. 5. Known congenital or acquired coagulation disorders other than haemophilia A. 6. Other conditions (e.g., autoimmune disease) or laboratory abnormality that may increase the risk of bleeding or thrombosis, as evaluated by the investigator. Any disorder, except for conditions associated with haemophilia A, that in the investigator's opinion might jeopardise the participant's safety or compliance with the protocol. 7. Lack of adequate parental/legally acceptable representative (LAR) support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary. 8. Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease. 9. Any planned major surgery, during part 1 of Arm 3 (infant). For definition of major surgery. 10. Immune tolerance induction planned to take place after treatment initiation. 11. Hepatic dysfunction defined as AST and/or ALT greater than (\>) 3 times the upper limit of normal (ULN) combined with total bilirubin \>1.5 times the ULN. 12. Serum creatinine above 1.5 times the ULN.

Interventions

DRUG

Mim8

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Conditions

Haemophilia AHaemophilia A With Inhibitors

Locations

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California 90027

United States

UC Denver Hemoph & Thrombo Ctr

Aurora, Colorado 80045

United States

Univ of Miami/SCCC

Miami, Florida 33136

United States

St Joseph's Hospital Foundation

Tampa, Florida 33607

United States

Children's Healthcare Atlanta

Atlanta, Georgia 30329

United States

Rush University Med. Cntr

Chicago, Illinois 60612

United States

Rush University Medical Center

Chicago, Illinois 60612

United States

Indiana Hemophilia-Thromb Ctr

Indianapolis, Indiana 46260

United States

University of Iowa_Iowa City

Iowa City, Iowa 52242

United States

Central Michigan University

Detroit, Michigan 48201

United States

Univ Hosp Cleveland Med Ctr

Cleveland, Ohio 44106

United States

Dayton Children Hemostati Ctr

Dayton, Ohio 45404

United States

Penn State MS Hershey Med Ctr

Hershey, Pennsylvania 17033-2360

United States

St Christopher Hosp for Child

Philadelphia, Pennsylvania 19134

United States

Vanderbilt U Med Ctr_Nashville

Nashville, Tennessee 37212

United States

Universitätsklinik für Innere Medizin V

Innsbruck, 6020

Austria

Universitätsklinik für Innere Medizin V

Innsbruck, A 6020

Austria

AKH - Klin. Abt. f. Haematologie u. Haemostaseologie

Vienna, 1090

Austria

AKH - Klin. Abt. f. Haematologie u. Haemostaseologie

Vienna, 1090

Austria

Cliniques universitaires Saint-Luc - Service Hématologie

Brussels, 1200

Belgium

UZ Leuven - Kindergeneeskunde

Leuven, 3000

Belgium

SHAT of Haematological Diseases EAD, Clinic of Paediatric Clinical Haematology

Sofia, 1797

Bulgaria

McMaster University

Hamilton, Ontario L8N 3Z5

Canada

The Hospital for Sick Children

Toronto, Ontario M5G 1X8

Canada

Beijing Children's Hospital, Capital Medical University

Beijing, Beijing Municipality 100045

China

Beijing Children's Hospital,Capital Medical University

Beijing, Beijing Municipality 100045

China

Haemotology, Nanfang Hospital, Southern Medical University

Guangzhou, Guangdong 510515

China

Nanfang Hospital, Southern Medical University-Haematology

Guangzhou, Guangdong 510515

China

Tongji Hospital, Tongji Medical College of HUST-Hematology

Wuhan, Hubei 430030

China

Tongji Hospital, Tongji Medical College of HUST

Wuhan, Hubei 430030

China

Xiangya Hospital Central-South University

Changsha, Hunan 410008

China

Jinan Central Hospital Affiliated to Shandong University

Jinan, Shandong 250062

China

Jinan Central Hospital

Jinan, Shandong 250062

China

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan 610074

China

Institute of Hematology and Blood Diseases Hospital, Tianjin-Hematology

Tianjin, Tianjin Municipality 300020

China

Institute of hematology and Blood Diseases Hospital, Tianjin-Hematology

Tianjin, Tianjin Municipality 300020

China

Children's Hospital, Zhejiang University School of Medicine-Hematology

Huzhou, Zhejiang 313200

China

Jinan Central Hospital

Jinan, 250062

China

Rigshospitalet - Department of Haematology, 2081

København Ø, 2100

Denmark

Hospices Civils de Lyon- Hopital Louis Pradel

Bron, 69500

France

Ap-Hp-Hopital de Bicetre-1

Le Kremlin-Bicêtre, 94275

France

Ap-Hp-Hopital de Bicetre

Le Kremlin-Bicêtre, 94275

France

Centre Hospitalier Universitaire de Lille-Institut Coeur Poumon

Lille, 59037

France

INSTITUT COEUR POUMON_Service d'hémostase et transfusion

Lille, 59037

France

Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu

Nantes, 44000

France

Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu

Nantes, Nantes

France

Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain

Berlin, 10249

Germany

Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie

Bonn, 53127

Germany

HZRM Haemophilie-Zentrum Rhein Main GmbH

Frankfurt am Main, 60596

Germany

Nirmal Hospital Pvt. Ltd.

Surat, Gujarat 395002

India

Nirmal Hospital Pvt. Ltd.

Surat, Gujarat 395002

India

Seth GS medical college and KEM Hospital

Mumbai, Maharashtra 400012

India

Sahyadri Clinical Research And Development Center

Pune, Maharashtra 411004

India

Sahyadri Clinical Research And Development Center

Pune, Maharashtra 411004

India

Christian Medical College and Hospital

Ludhiana, Punjab 141008

India

Christian Medical College and Hospital

Ludhiana, Punjab 141008

India

SMS Medical College & Hospital

Jaipur, Rajasthan 302004

India

CMCV

Ranipet, Tamil Nadu 632517

India

Post Graduate Institute of Child Health

Noida, Uttar Pradesh 201303

India

Seth GS medical college and KEM Hospital

Mumbai, 400012

India

St James's CRF

Dublin, Leinster D08 A978

Ireland

St James's CRF

Dublin, Leinster D08 A978

Ireland

Sheba MC - The Israeli National Hemophilia Center

Tel Litwinsky, 52621

Israel

A.O.U. Policlinico Umberto I

Rome, Lazio 00161

Italy

AOU Careggi Firenze

Florence, 50134

Italy

IRCCS Humanitas Research Hospital - Centro Trombosi e Malattie Emorragiche

Milan, 20089

Italy

Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122

Italy

Azienda Ospedaliera di Rilievo Nazionale Santobono Pausilipon

Naples, 80122

Italy

Azienda Ospedaliera Santobono Pausilipon - U.S.D. Centro Regionale Pediatrico Malattie della Coagulazione

Naples, 80122

Italy

Ospedale Pediatrico Bambino Ges

Roma, 00165

Italy

Ospedale Pediatrico Bambino Ges

Roma, 00165

Italy

A.O.U. Policlinico Umberto I

Rome, 00161

Italy

AOU Città della Salute e della Scienza di Torino - Ospedale Regina Margherita

Torino, 10126

Italy

Nagoya University Hospital_Blood Transfusion

Aichi, 466-8560

Japan

Ota Memorial Hospital_Pediatrics

Gunma, 373-8585

Japan

Nara Medical University Hospital_Pediatrics

Nara, 634-8522

Japan

Saitama Children's Med Centre_Hematology-Oncology

Saitama, 330-8777

Japan

Jichi Medical University Hospital_Hematology

Tochigi, 329-0498

Japan

Jichi Medical University Hospital_Pediatrics

Tochigi, 329-0498

Japan

National Center for Child Health and Development_Hematology

Tokyo, 157-8535

Japan

Tokyo Medical Univ. Hospital_Laboratory Medicine

Tokyo, 160-0023

Japan

Ogikubo Hospital_Pediatries & Blood

Tokyo, 167-0035

Japan

Stradini Clinic of Oncology

Riga, 1002

Latvia

Children University Clinical Hospital

Riga, 1004

Latvia

Vilnius University hospital Santaros klinikos

Vilnius, 08661

Lithuania

Centre of Oncology and Hematology, Vilnius University

Vilnius, LT-08406

Lithuania

Hospital Queen Elizabeth 1

Kota Kinabalu, Sabah 88586

Malaysia

Hospital Ampang

Ampang, Selangor, Selangor 68000

Malaysia

Hospital Ampang

Ampang, Selangor, Selangor 68000

Malaysia

Centro Multidisciplinario Para El Desarrollo Especializado De La Investigación Clínica En Yucatán S.C.P. (CEMDEICY S.C.P.)

Mérida, 97130

Mexico

AmsterdamUMC AMC

Amsterdam, 1105 AZ

Netherlands

UMC Utrecht, Van Creveldkliniek

Utrecht, 3584 CX

Netherlands

Uniwersytecki Szpital Kliniczny W Poznaniu

Poznan, Greater Poland Voivodeship 60-569

Poland

Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego

Wroclaw, Lower Silesian Voivodeship 50-556

Poland

Instytut Hematologii i Transfuzjologii

Warsaw, Masovian Voivodeship 02-776

Poland

Intytut Hematologii i Transfuzjologii

Warsaw, Masovian Voivodeship 02-776

Poland

Szpital Uniwersytecki, Oddzial Kliniczny Hematologii

Krakow, 30-688

Poland

Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa

Lublin, 20-093

Poland

Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa

Lublin, 20-093

Poland

Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego

Wroclaw, 50-556

Poland

Unidade De Coimbra, E.P.E. - Hospital da Universidade de Coimbra

Coimbra, 3000-459

Portugal

ULS De São José, E.P.E.- Hospital D. Estefânia

Lisbon, 1169-045

Portugal

ULS De Santo António, E.P.E. _H.Santo António_ Imunohemoterapia

Porto, 4050-342

Portugal

ULS São João, E.P.E._H.São João_Imunohemoterapia

Porto, 4200-319

Portugal

Institut Oncologic "Prof. Dr. Ion Chiricuta" Cluj Napoca

Cluj-Napoca, 400015

Romania

King Faisal Specialist Hospital & Research Centre, Riyadh

Riyadh, 12713

Saudi Arabia

Clinical Centre of Serbia, Institute for Haematology

Belgrade, 11000

Serbia

Clinical Centre of Vojvodina

Novi Sad, 21000

Serbia

Univerzitna Nemocnica Martin

Martin, 036 59

Slovakia

Vseobecna nemocnica Rimavska Sobota

Rimavská Sobota, 979 01

Slovakia

Vranovska nemocnica, a.s.

Vranov nad Topľou, 09327

Slovakia

Wits Bara Clinical Trial Site

Johannesburg, Gauteng 2013

South Africa

Charlotte Maxeke Johannesburg Academic Hospital

Parktown, Johannesburg, Gauteng 2193

South Africa

Daejeon Eulji Medical Center, Eulji University

Daejeon, 35233

South Korea

Daejeon Eulji Medical Center, Eulji University

Daejeon, 35233

South Korea

Severance Hospital, Yonsei University Health System

Seoul, 03722

South Korea

Severance Hospital, Yonsei University Health System

Seoul, 03722

South Korea

Gangdong Kyung Hee University Hospital

Seoul, 05278

South Korea

Kyung Hee University Hospital at Gangdong

Seoul, 05278

South Korea

Hospital Vall d'Hebron

Barcelona, 08035

Spain

Hospital Sant Joan de Déu

Esplugues Llobregat, 08950

Spain

Hospital Universitario La Paz

Madrid, 28046

Spain

Hospital Carlos Haya

Málaga, 29010

Spain

Hospital Regional Universitario de Málaga

Málaga, 29010

Spain

Universitätsklinik für Hämatologie

Bern, 3010

Switzerland

Pädiatrische Onkologie-Hämatologie

Lucerne, 6000

Switzerland

Zentrum für Labormedizin

Sankt Gallen, 9008

Switzerland

Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie

Zurich, 8091

Switzerland

Chung Shan Medical University Hospital

Taichung, 402

Taiwan

Taichung Veterans General Hospital

Taichung, 407

Taiwan

National Taiwan University Hospital_main

Taipei, 100

Taiwan

National Taiwan University Hospital

Taipei, 100

Taiwan

Gazi University

Ankara, Beşevler/Ankara 06500

Turkey (Türkiye)

Gazi Üniversitesi Hastanesi- Hematoloji

Ankara, Beşevler/Ankara 06500

Turkey (Türkiye)

Istanbul University Oncology Institute

Capa-ISTANBUL, Capa-ISTANBUL 34093

Turkey (Türkiye)

İstanbul Üniversitesi İstanbul Tıp Fakültesi Hastanesi- Onkoloji Enstitüsü

Capa-ISTANBUL, Capa-ISTANBUL 34093

Turkey (Türkiye)

Akdeniz Üniversitesi Hastanesi- Hematoloji

Antalya, Konyaaltı/ Antalya 01010

Turkey (Türkiye)

Acıbadem Adana Hastanesi-Hematoloji

Adana, 01130

Turkey (Türkiye)

Hacettepe Universitesi

Ankara, 06100

Turkey (Türkiye)

Hacettepe Üniversitesi Hastanesi- Hematoloji

Ankara, 06100

Turkey (Türkiye)

Akdeniz Universitesi

Antalya, 01010

Turkey (Türkiye)

Ege Universitesi Tip Fakultesi

Bornova-IZMIR, 35100

Turkey (Türkiye)

Ege Üniversitesi Hastanesi- Hematoloji

Bornova-IZMIR, 35100

Turkey (Türkiye)

Trakya University

Edirne, 22030

Turkey (Türkiye)

Trakya Üniversitesi Tıp Fakültesi Hastanesi-Hematoloji

Edirne, 22030

Turkey (Türkiye)

Belfast City Hospital

Belfast, BT9 78B

United Kingdom

University Hospital of Wales - Haemophilia

Cardiff, CF14 4XW

United Kingdom

Royal Free Haemophilia Comprehensive Care Center

London, NW3 2QG

United Kingdom

Royal Free Hospital - Haemophilia

London, NW3 2QG

United Kingdom

Evelina London Children's Hospital - Haemophilia

London, SE1 7EH

United Kingdom

St Thomas' Hospital - Haemostasis and Thrombosis Centre

London, SE1 7EH

United Kingdom

Royal Hallamshire Hospital

Sheffield, S10 2JF

United Kingdom