Clinical Trials Register

Summary
EudraCT Number:	2020-003467-26
Sponsor's Protocol Code Number:	NN7769-4516
National Competent Authority:	Lithuania - SMCA
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2022-09-27
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Lithuania - SMCA

A.2

EudraCT number

2020-003467-26

A.3

Full title of the trial

Safety, efficacy and exposure of subcutaneously administered NNC0365-3769
(Mim8) prophylaxis in children with haemophilia A with or without FVIII
inhibitors

Po oda profilaktiškai vartojamo NNC0365-3769 (Mim8) saugumas, veiksmingumas ir poveikis vaikams, kuriems nustatyta A hemofilija su FVIII inhibitoriais ar be jų

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A research study investigating Mim8 in children with haemophilia A with or without inhibitors

Klinikinis tyrimas nustatyti po oda profilaktiškai vartojamo „Mim8“ saugumą vaikams, kuriems nustatyta A hemofilija su FVIII inhibitoriais ar be jų.

A.3.2

Name or abbreviated title of the trial where available

Frontier 3

A.4.1

Sponsor's protocol code number

NN7769-4516

A.5.3

WHO Universal Trial Reference Number (UTRN)

U1111-1255-1540

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/450/2020

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Novo Nordisk A/S
B.1.3.4	Country	Denmark
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Novo Nordisk A/S
B.4.2	Country	Denmark
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Novo Nordisk A/S
B.5.2	Functional name of contact point	Clinical Transparency (2834)
B.5.3	Address:
B.5.3.1	Street Address	Novo Allé
B.5.3.2	Town/ city	Bagsværd
B.5.3.3	Post code	2880
B.5.3.4	Country	Denmark
B.5.6	E-mail	clinicaltrials@novonordisk.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	NNC0365-3769 B 2.0 mg/mL
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	N/A
D.3.9.3	Other descriptive name	Mim8
D.3.9.4	EV Substance Code	SUB254554
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	NNC0365-3769 B 5.0 mg/mL
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	N/A
D.3.9.3	Other descriptive name	Mim8
D.3.9.4	EV Substance Code	SUB254554
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 3
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	NNC0365-3769 B 11.3 mg/mL
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	N/A
D.3.9.3	Other descriptive name	Mim8
D.3.9.4	EV Substance Code	SUB254554
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	11.3
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 4
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	NNC0365-3769 B 25.0 mg/mL
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	N/A
D.3.9.3	Other descriptive name	Mim8
D.3.9.4	EV Substance Code	SUB254554
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	25
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 5
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	NNC0365-3769 B 57.5 mg/mL
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	N/A
D.3.9.3	Other descriptive name	Mim8
D.3.9.4	EV Substance Code	SUB254554
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	57.5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Haemophilia A

Haemophilia A with inhibitors

E.1.1.1

Medical condition in easily understood language

Bleeding disorder, inherited deficiency in clotting factor VIII

Bleeding disorder, inherited deficiency in clotting factor VIII with antibodies to clotting factor replacement therapy

E.1.1.2

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10018938
E.1.2	Term	Haemophilia A (Factor VIII)
E.1.2	System Organ Class	100000004850

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10053751
E.1.2	Term	Hemophilia A with anti factor VIII
E.1.2	System Organ Class	100000004850

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To investigate the safety of Mim8 prophylaxis in children with haemophilia A with or without
FVIII inhibitors.

Nustatyti po oda profilaktiškai vartojamo „Mim8“ saugumą vaikams, kuriems nustatyta A hemofilija su FVIII inhibitoriais ar be jų.

E.2.2

Secondary objectives of the trial

Investigate the efficacy of Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.
Evaluate the consumption of coagulation factor replacement product per bleed treatment (number of injections) with Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.
Evaluate the development of anti-Mim8 antibodies with Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.
Investigate exposure of Mim8 after once-weekly and once-monthly subcutaneous dosing in children with haemophilia A with or without FVIII inhibitors.
Evaluate treatment burden with Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors, as reported by their caregivers.
Investigate treatment preference among caregivers of previously treated children with haemophilia A with or without FVIII inhibitors.
Evaluate aspects of physical functioning with Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.

Nustatyti po oda profilaktiškai vartojamo „Mim8“ veiksmingumą vaikams, kuriems nustatyta A hemofilija su FVIII inhibitoriais ar be jų.Įvertinti kraujo krešėjimo faktoriaus pakaitinio produkto suvartojimą vienam kraujavimui gydyti (injekcijų skaičius) „Mim8“ profilaktiškai naudojant vaikams, kuriems nustatyta A hemofilija su FVIII inhibitoriais ar be jų.Įvertinti anti „Mim8“ antikūnų susidarymą profilaktiškai „Mim8“ naudojant vaikams, kuriems nustatyta A hemofilija su FVIII inhibitoriais ar be jų.Nustatyti „Mim8“ poveikį po oda vartojamos dozės kartą per savaitę ir kartą per mėnesį vaikams, kuriems nustatyta A hemofilija su FVIII inhibitoriais ar be jų.Įvertinti gydymo sunkumus profilaktiškai naudojant „Mim8“ vaikams, kuriems nustatyta A hemofilija su FVIII inhibitoriais ar be jų, kaip nurodyta jų globėjų įrašuose.Ištirti anksčiau gydytų vaikų, sergančių hemofilija A su FVIII inhibitoriais arba be jų, globėjų pasirenkamo gydymo pirmenybę.

E.2.3

Trial contains a sub-study

E.3

Principal 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 and female participants with the diagnosis of congenital haemophilia A of any severity based on medical records.
3. Aged 1−11 years (both inclusive) at the time of signing informed consent.
4. For previously treated participants:
a. Participant has been prescribed treatment with FVIII concentrate or bypassing agent in the last 26 weeks prior to screening.
b. Participants with endogenous FVIII activity ≥1%, based on medical records, must have at least 1 treated bleed during the previous 26 weeks before screening for which factor VIII concentrate or bypassing agent has been prescribed (No requirements for participants with FVIII activity <1%).
5. For previously untreated participants:
a. Diagnosis of severe haemophilia A (endogenous FVIII activity < 1%) based on medical records.
6. Child and parent/caregiver willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires.

1. Informuoto asmens sutikimas gautas prieš atliekant bet kokias su tyrimu susijusias procedūras. Su tyrimu susijusi veikla yra visos procedūros, atliekamos kaip tyrimo dalis, įskaitant paciento tinkamumo dalyvauti tyrime nustatymą.
2. Vyriškos ir moteriškos lyties pacientai, kuriems remiantis medicininiais įrašais diagnozuota įgimta A hemofilija.
3. Amžius: 1−11 metai (įskaitant abi vertes) informuoto asmens sutikimo pasirašymo momentu.
4. Pacientams, anksčiau taikant gydymą FVIII:
a. Pacientui buvo paskirtas ar buvo reikalingas gydymas naudojant FVIII ar apeinančio veikimo preparatu per pastarąsias 26 savaičių iki atrankos.
b. Pacientams, kurių endogeninis FVIII aktyvumas, remiantis medicininiais įrašais, yra ≥1 %, turi būti taikytas bent 1 kraujavimo gydymas per pastarąsias 26 savaičių iki atrankos (reikalavimai netaikomi pacientams, kurių FVIII aktyvumas yra <1 %).
5. Pacientams, kuriems anksčiau netaikytas gydymas FVIII:
a. Sunkios A hemofilijos diagnozė (endogeninis FVIII aktyvumas yra < 1 %), remiantis medicininiais įrašais.
6. Vaiko ir motinos arba tėvo / globėjo sutikimas ir gebėjimas laikytis numatytų vizitų ir tyrimų procedūrų, įskaitant dienoraščio ir paciento nurodytų rezultatų klausimynų pildymą.a
a: Skirta žemyninei Kinijai; vertinama tyrėjo nuožiūra, jei nenurodyta kitaip.

E.4

Principal exclusion criteria

1. Known or suspected hypersensitivity to trial product or related products.
2. Previous participation in this study. Participation is defined as signed informed consent.
3. Participation (i.e., signed informed consent) in any interventional clinical study with receipt of last dose within 6 months (or 5 half-lives of the investigational medicinal product, whichever is shorter) before planned randomisation.
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 randomisation, for participants not included in the run-in.
5. Known congenital or acquired coagulation disorders other than haemophilia A.
6. Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis, as evaluated by the investigator.
7. 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.
8. Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate understanding and cooperation.
9. Lack of adequate parental/caregiver support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary.
10. 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.
11. Major surgery planned to take place after screening. For definition of major surgery, see Table ‎6-7.
12. Immune tolerance induction planned to take place after treatment initiation.
13. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) >3 times the upper limit of normal combined with total bilirubin >1.5 times the upper limit of normal measured at screening.
14. Serum creatinine above 1.5 x upper limit of normal (ULN), measured at screening.
15. Pregnancy (female participants).

1. Žinomas ar įtariamas padidėjęs jautrumas tiriamam produktui ar susijusiems produktams.a
2. Ankstesnis dalyvavimas šiame tyrime. Dalyvavimas apibrėžiamas kaip pasirašytas informuoto asmens sutikimas.
3. Dalyvavimas (t.y. pasirašytas informuotas sutikimas) bet kuriame intervenciniame tyrime, gavus paskutinę dozę per 6 mėnesius (arba 5 vaistinio preparato pusinės eliminacijos periodus, atsižvelgiant į tai, kuris laikotarpis yra trumpesnis) iki planuojamos randomizacijos.
4. Poveikio hemostazei neturinčių preparatų poveikis kraujavimo profilaktikai per 6 mėnesius (arba 5 vaistinio preparato pusinės eliminacijos periodus, atsižvelgiant į tai, kuris laikotarpis yra trumpesnis), prieš planuojamą randomizaciją, pacientams, kuriems netaikomas parengimas.
5. Žinomas įgimtas arba įgytas kraujo krešėjimo sutrikimas, išskyrus A hemofiliją.
6. Tyrėjo įvertintos kitos būklės (pvz., autoimuninė liga) ar laboratorinių tyrimų rezultatų pakitimai, galintys padidinti kraujavimo ar trombozės riziką.a
7. Bet koks sutrikimas, išskyrus su A hemofilija susijusias būkles, kuris, tyrėjo nuomone, galėtų kelti pavojų paciento saugumui arba pažeistų protokolo laikymąsi.a
8. Protinis neveiksnumas, nenorėjimas bendradarbiauti arba kalbos barjeras, neleidžiantys tinkamai suprasti tyrimo ir bendradarbiauti.a
9. Trūksta tinkamos tėvų / globėjo paramos, kad būtų galima tiksliai ir laiku įrašyti informaciją apie gydymą ir kraujavimo epizodus į (elektroninį) paciento dienoraštį.a
10. Ankstesnis ar esamas tromboembolinės ligos gydymas (išskyrus ankstesnę kateterio sukeltą trombozę, kuriai antitrombozinis gydymas šiuo metu nėra taikomas) arba tromboembolinės ligos požymiai.
11. Po atrankos planuojama didelės apimties chirurginė operacija.a Didelės apimties chirurginės operacijos apibrėžimą žiūrėti Lentelėje 6-7.
12. Planuojama imuninės tolerancijos indukcija pradėjus gydymą.a
13. Kepenų funkcijos sutrikimas, toks kaip aspartato aminotransferazė (AST) ir (arba) alanino aminotransferazė (ALT), daugiau kaip 3 kartus viršijanti viršutinę normos ribą, kartu su bendru bilirubino kiekiu, 1,5 karto viršijanti viršutinę normos ribą, nustatytą atrankos metu.
14. Kreatinino koncentracija serume 1,5 karto viršijo viršutinę normos ribą (angl. ULN), nustatytą atrankos metu.
15. Nėštumas (moteriškos lyties dalyviai).
a: Skirta žemyninei Kinijai; vertinama tyrėjo nuožiūra, jei nenurodyta kitaip.
b: Vertinama tyrėjo nuožiūra, jei įtariamas galimas nėštumas.

E.5 End points

E.5.1

Primary end point(s)

1. Number of treatment emergent adverse events

Gydant pasireiškusių nepageidaujamų reiškinių skaičius

E.5.1.1

Timepoint(s) of evaluation of this end point

1. From treatment initiation to follow up visit (week 0 to week 72)

Nuo gydymo pradžios iki sekimo vizito (0–72 savaitės)

E.5.2

Secondary end point(s)

1. Number of treated bleeds
2. Number of treated spontaneous bleeds
3. Number of treated traumatic bleeds
4. Number of treated joint bleeds
5. Number of treated target joint bleeds
6. Number of injection site reactions
7. Consumption of factor product per bleed treatment
8. Occurrence of anti-Mim8 antibodies
9. Mim8 plasma concentration
10. Change in physical function domain of Paediatric Quality of Life inventory (PEDS-QL) Generic Core Scales
11. Treatment preference for Mim8 versus previous treatment using Caregiver Haemophilia Patient Preference Questionnaire (Caregiver H-PPQ)
12. Change in participants’ treatment burden using the Haemophilia treatment experience measure (Hemo TEM)

Gydytų kraujavimų skaičius, Gydytų savaiminių kraujavimų skaičius
Gydytų kraujavimų dėl traumos skaičius
Gydytų sąnarių kraujavimų skaičius
Gydytų tikslinių sąnarių kraujavimų skaičius
Reakcijos injekcijos vietoje skaičius
Faktoriaus produkto suvartojimas vieno kraujavimo gydymui (injekcijų skaičius)
Anti „Mim8“ antikūnų susidarymas
„Mim8“ koncentracija plazmoje
PEDS QL fizinio poveikio srities bendrosios pagrindinės skalės
Gydymui „Mim8“ teikiama pirmenybė, palyginti su ankstesniu gydymu naudojant „Caregiver H PPQ“
Paciento gydymo sunkumų pokytis naudojant „Hemo TEM“

E.5.2.1

Timepoint(s) of evaluation of this end point

1-6 . From treatment initiation to end of treatment (week 0 to week 52)
7. From run-in initiation to end of treatment (week -26 to week 52)
8-10. From treatment initiation to end of treatment (week 0 to week 52)
11. Once during treatment (week 26)
12. From treatment initiation to end of treatment (week 0 to week 52)

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Canada

China

India

Israel

Japan

Korea, Republic of

South Africa

Taiwan

United States

European Union

Switzerland

Russian Federation

United Kingdom

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

Yes

F.1.1.4.1

Number of subjects for this age range:

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

none

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2023-03-22

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2023-02-27

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2024-11-13

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Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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