Clinical Trial Page

Summary
EudraCT Number:2020-001048-24
Sponsor's Protocol Code Number:NN7769-4514
National Competent Authority:Ireland - HPRA
Clinical Trial Type:EEA CTA
Trial Status:Ongoing
Date on which this record was first entered in the EudraCT database:2020-11-26
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
A. Protocol Information
A.1Member State ConcernedIreland - HPRA
A.2EudraCT number2020-001048-24
A.3Full title of the trial
A multinational, open-label, randomised, controlled trial to investigate efficacy and safety of NNC0365-3769 (Mim8) in adults and adolescents with haemophilia A with or without inhibitors.
A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
A research study investigating Mim8 in adults and adolescents with haemophilia A with or without inhibitors
A.4.1Sponsor's protocol code numberNN7769-4514
A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1249-4378
A.7Trial is part of a Paediatric Investigation Plan No
A.8EMA Decision number of Paediatric Investigation Plan
B. Sponsor Information
B.Sponsor: 1
B.1.1Name of SponsorNovo Nordisk A/S
B.1.3.4CountryDenmark
B.3.1 and B.3.2Status of the sponsorCommercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1Name of organisation providing supportNovo Nordisk A/S
B.4.2CountryDenmark
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1Name of organisationNovo Nordisk A/S
B.5.2Functional name of contact pointClinical Transparency (1452)
B.5.3 Address:
B.5.3.1Street AddressNovo Allé
B.5.3.2Town/ cityBagsværd
B.5.3.3Post code2880
B.5.3.4CountryDenmark
B.5.6E-mailclinicaltrials@novonordisk.com
D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3IMP RoleTest
D.2 Status of the IMP to be used in the clinical trial
D.2.1IMP to be used in the trial has a marketing authorisation No
D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
D.2.5.1Orphan drug designation number
D.3 Description of the IMP
D.3.2Product code NNC0365-3769 A 10mg/mL
D.3.4Pharmaceutical form Solution for injection
D.3.4.1Specific paediatric formulation No
D.3.7Routes of administration for this IMPSubcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8INN - Proposed INNN/A
D.3.9.1CAS number *MASKED*
D.3.9.2Current sponsor codeNNC0365-3769
D.3.9.4EV Substance CodeSUB198393
D.3.10 Strength
D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
D.3.10.2Concentration typerange
D.3.10.3Concentration number10 to 100
D.3.11 The IMP contains an:
D.3.11.1Active substance of chemical origin No
D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
The IMP is a:
D.3.11.3Advanced Therapy IMP (ATIMP) No
D.3.11.3.1Somatic cell therapy medicinal product No
D.3.11.3.2Gene therapy medical product No
D.3.11.3.3Tissue Engineered Product No
D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
D.3.11.5Radiopharmaceutical medicinal product No
D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
D.3.11.7Plasma derived medicinal product No
D.3.11.8Extractive medicinal product No
D.3.11.9Recombinant medicinal product Yes
D.3.11.10Medicinal product containing genetically modified organisms No
D.3.11.11Herbal medicinal product No
D.3.11.12Homeopathic medicinal product No
D.3.11.13Another 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.1.1Medical condition(s) being investigated
Haemophilia A
Haemophilia A with inhibitors
E.1.1.1Medical condition in easily understood language
Haemophilia A
Haemophilia A with inhibitors
E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
MedDRA Classification
E.1.2 Medical condition or disease under investigation
E.1.2Version 20.0
E.1.2Level LLT
E.1.2Classification code 10018938
E.1.2Term Haemophilia A (Factor VIII)
E.1.2System Organ Class 100000004850
E.1.2 Medical condition or disease under investigation
E.1.2Version 20.0
E.1.2Level LLT
E.1.2Classification code 10053751
E.1.2Term Hemophilia A with anti factor VIII
E.1.2System Organ Class 100000004850
E.1.3Condition being studied is a rare disease Yes
E.2 Objective of the trial
E.2.1Main objective of the trial
To confirm the haemostatic effect of Mim8 as treatment prophylaxis for adult and adolescent patients with haemophilia A with or without inhibitors.
E.2.2Secondary objectives of the trial
1. To investigate safety of Mim8 prophylaxis in adults and adolescents with haemophilia A with or without FVIII inhibitors.
2. To evaluate the consumption of factor product per bleed treatment (number of injections) after Mim8 prophylaxis in adults and adolescents with haemophilia A with or without inhibitors.
3. To evaluate the development of anti-Mim8 antibodies after Mim8 prophylaxis in adults and adolescents with haemophilia A with or without inhibitors.
4. To evaluate treatment burden after Mim8 prophylaxis in adults and adolescents with haemophilia A with or without inhibitors.
5. To evaluate aspects of patient’s physical functioning after Mim8 prophylaxis in adults and adolescents with haemophilia A with or without inhibitors.
6. To evaluate joint pain intensity after Mim8 prophylaxis in adults and adolescents with haemophilia A with or without inhibitors.
E.2.3Trial contains a sub-study No
E.3Principal inclusion criteria
- Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial
- Male or female with diagnosis of congenital haemophilia A of any severity based on medical records
- Patient has been prescribed, or in need of, treatment with factor VIII or bypassing agent in the last 26 weeks prior to screening
- Age above or equal to 12 years at the time of signing informed consent. Germany, Japan and Taiwan: Local requirements apply
- Body weight above or equal to 30 kg
- Applicable to patients on emicizumab prophylaxis: patient is willing to discontinue emicizumab at the time of screening
- Applicable to patients treated with no prophylaxis prior to enrolment: 5 or more bleeds in the last 26 weeks prior to screening visit
- Applicable to patients with FVIII activity above 1% who are on prophylactic treatment: 1 or more bleeds in the last 26 weeks prior to screening visit
- Willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires
E.4Principal exclusion criteria
- Previous participation in this trial. Participation is defined as signed informed consent
- Participation in any clinical trial of an approved or non-approved investigational medicinal product, within 30 days (or 5 half-lives of the investigational medicinal product, whichever is greater) before screening
- Female who is pregnant, breast-feeding or intends to become pregnant or is of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures as defined in the protocol or as required by local regulation or practice). Breast feeding is allowed only during the run-in period
- Any disorder, except for conditions associated with haemophilia A, which in the investigator’s opinion might jeopardise subject’s safety or compliance with the protocol
- Known or suspected hypersensitivity to trial product(s), any constituents of the product or to related products
- Receipt of gene therapy at any given time point
- Ongoing or planned immune tolerance induction (ITI) therapy
- Major surgery planned at the time of screening
- Known congenital or acquired coagulation disorders other than haemophilia A
- Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) above 3 times the upper limit combined with total bilirubin above 1.5 times the upper limit measured at screening
- Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) below or equal to 30 ml/min/1.73 m^2 for serum creatinine measured at screening
- Previous or current thromboembolic disease or events (includes arterial and venous thrombosis including myocardial infarction, thrombotic microangiography (TMA), pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion) (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or risk of thromboembolic disease, as evaluated by investigator
- Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation
- Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis as evaluated by the investigator
E.5 End points
E.5.1Primary end point(s)
Number of treated bleeds
E.5.1.1Timepoint(s) of evaluation of this end point
- No prophylaxis treatment (Arms 1 and 2): From randomisation (week 0) to end of main (week 26)
- Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (week 26)
E.5.2Secondary end point(s)
1. Number of injection site reactions
2. Occurrence of antiMim8 antibodies
3. Number of treated spontaneous bleeds
4. Number of treated joint bleeds
5. Number of treated traumatic bleeds
6. Number of target joint bleeds
7. Consumption of factor product per bleed treatment (number of injections)
8. Change in physical function domain of paediatric quality of life inventory (PEDS-QL)
9. Change in patient’s treatment burden using the haemophilia treatment experience measure (Hemo-TEM)
10. Change in patient’s joint pain score using Joint Pain Rating Scale
E.5.2.1Timepoint(s) of evaluation of this end point
1. All subjects receiving Mim8 (Arms 2, 3 and 4): From randomisation (week 0) to end of main (week 26)
2. All subjects receiving Mim8 (Arms 2, 3 and 4): From randomisation (week 0) to end of extension (week 52)
3. – 7.:
- No prophylaxis treatment (Arms 1 and 2): From randomisation (week 0) to end of main (week 26)
- Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (week 26)
8. – 10.:
All subjects (Arms 1, 2, 3 and 4): From randomisation (week 0) to the end of the main part (week 26)
E.6 and E.7 Scope of the trial
E.6Scope of the trial
E.6.1Diagnosis No
E.6.2Prophylaxis No
E.6.3Therapy No
E.6.4Safety Yes
E.6.5Efficacy Yes
E.6.6Pharmacokinetic No
E.6.7Pharmacodynamic No
E.6.8Bioequivalence No
E.6.9Dose response No
E.6.10Pharmacogenetic No
E.6.11Pharmacogenomic No
E.6.12Pharmacoeconomic No
E.6.13Others No
E.7Trial type and phase
E.7.1Human pharmacology (Phase I) No
E.7.1.1First administration to humans No
E.7.1.2Bioequivalence study No
E.7.1.3Other No
E.7.1.3.1Other trial type description
E.7.2Therapeutic exploratory (Phase II) No
E.7.3Therapeutic confirmatory (Phase III) Yes
E.7.4Therapeutic use (Phase IV) No
E.8 Design of the trial
E.8.1Controlled Yes
E.8.1.1Randomised Yes
E.8.1.2Open Yes
E.8.1.3Single blind No
E.8.1.4Double blind No
E.8.1.5Parallel group No
E.8.1.6Cross over No
E.8.1.7Other Yes
E.8.1.7.1Other trial design description
Intra-individual (within-patient) comparison
E.8.2 Comparator of controlled trial
E.8.2.1Other medicinal product(s) No
E.8.2.2Placebo No
E.8.2.3Other Yes
E.8.2.3.1Comparator description
Different treatment regimen of Mim8
E.8.2.4Number of treatment arms in the trial4
E.8.3 The trial involves single site in the Member State concerned No
E.8.4 The trial involves multiple sites in the Member State concerned Yes
E.8.4.1Number of sites anticipated in Member State concerned2
E.8.5The trial involves multiple Member States Yes
E.8.5.1Number of sites anticipated in the EEA53
E.8.6 Trial involving sites outside the EEA
E.8.6.1Trial being conducted both within and outside the EEA Yes
E.8.6.2Trial being conducted completely outside of the EEA No
E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
Canada
China
European Union
India
Israel
Japan
Korea, Republic of
Russian Federation
Saudi Arabia
Serbia
South Africa
Switzerland
Taiwan
Turkey
United Kingdom
United States
E.8.7Trial 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.8.9.1In the Member State concerned years2
E.8.9.1In the Member State concerned months3
E.8.9.1In the Member State concerned days4
E.8.9.2In all countries concerned by the trial years2
E.8.9.2In all countries concerned by the trial months3
E.8.9.2In all countries concerned by the trial days4
F. Population of Trial Subjects
F.1 Age Range
F.1.1Trial has subjects under 18 Yes
F.1.1Number of subjects for this age range: 30
F.1.1.1In Utero No
F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
F.1.1.3Newborns (0-27 days) No
F.1.1.4Infants and toddlers (28 days-23 months) No
F.1.1.5Children (2-11years) No
F.1.1.6Adolescents (12-17 years) Yes
F.1.1.6.1Number of subjects for this age range: 30
F.1.2Adults (18-64 years) Yes
F.1.2.1Number of subjects for this age range: 190
F.1.3Elderly (>=65 years) Yes
F.1.3.1Number of subjects for this age range: 10
F.2 Gender
F.2.1Female Yes
F.2.2Male Yes
F.3 Group of trial subjects
F.3.1Healthy volunteers No
F.3.2Patients Yes
F.3.3Specific vulnerable populations Yes
F.3.3.1Women of childbearing potential not using contraception No
F.3.3.2Women of child-bearing potential using contraception Yes
F.3.3.3Pregnant women No
F.3.3.4Nursing women No
F.3.3.5Emergency situation No
F.3.3.6Subjects incapable of giving consent personally No
F.3.3.7Others No
F.4 Planned number of subjects to be included
F.4.1In the member state2
F.4.2 For a multinational trial
F.4.2.1In the EEA 99
F.4.2.2In the whole clinical trial 230
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
N.Competent Authority Decision Authorised
N.Date of Competent Authority Decision2021-01-08
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2021-05-18
P. End of Trial
P.End of Trial StatusOngoing

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