| CTRI Number |
CTRI/2023/01/048708 [Registered on: 03/01/2023] Trial Registered Prospectively |
| Last Modified On: |
03/04/2025 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Single Arm Study |
|
Public Title of Study
|
A research study looking at Mim8 in children
with haemophilia A with or without inhibitors |
|
Scientific Title of Study
|
Safety, efficacy and exposure of subcutaneously administered NNC0365-3769 (Mim8) prophylaxis in children with haemophilia A with or without FVIII inhibitors. |
| Trial Acronym |
NN7769-4516 |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| 2020-003467-26 |
EudraCT |
| NCT05306418 |
ClinicalTrials.gov |
| NN7769-4516 version 7.0 dated 22-Aug-2022 |
Protocol Number |
| U1111-1255-1540 |
UTN |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
|
| Designation |
|
| Affiliation |
|
| Address |
|
| Phone |
|
| Fax |
|
| Email |
|
|
Details of Contact Person
Scientific Query
Modification(s)
|
| Name |
Dr Maya Sharma |
| Designation |
Vice President -Clinical, Medical, Regulatory & Pharmacovigilance |
| Affiliation |
Novo Nordisk India Private Ltd |
| Address |
Novo Nordisk India Private Limited,
Nxt Tower - 2, Floor 1 & 2
Embassy Manyata Business Park,
Nagavara Village, Kasaba Hobli,
Bangalore
KARNATAKA
560045
India |
| Phone |
9911497869 |
| Fax |
080-41123518 |
| Email |
yrms@novonordisk.com |
|
Details of Contact Person
Public Query
Modification(s)
|
| Name |
Dr Maya Sharma |
| Designation |
Vice President -Clinical, Medical, Regulatory & Pharmacovigilance. |
| Affiliation |
Novo Nordisk India Private Ltd |
| Address |
Novo Nordisk India Private Ltd
Nxt Tower - 2, Floor 1 & 2
Embassy Manyata Business Park,
Nagavara Village, Kasaba Hobli,
Bangalore
KARNATAKA
560045
India |
| Phone |
9911497869 |
| Fax |
080-41123518 |
| Email |
yrms@novonordisk.com |
|
Source of Monetary or Material Support
Modification(s)
|
| Novo Nordisk AS Novo Allé, 2880 Bagsvaerd, Denmark |
|
Primary Sponsor
Modification(s)
|
| Name |
Novo Nordisk India Private Limited |
| Address |
Nxt Tower - 2, Floor 1 & 2, Embassy Manyata Business Park, Nagavara Village, Kasaba Hobli, Bangalore - 560045. India |
| Type of Sponsor |
Pharmaceutical industry-Global |
|
|
Details of Secondary Sponsor
|
|
Countries of Recruitment
Modification(s)
|
Canada
China
India
Israel
Italy
Japan
Lithuania
Netherlands
Poland
Portugal
South Africa
Spain
Switzerland
Taiwan
United Kingdom
United States of America
Republic of Korea |
|
Sites of Study
|
| No of Sites = 4 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Kapil Garg |
J K Lon Hospital |
Department of Paediatrics, J K Lon Hospital,
Attached to SMS College, Jaipur-302004, Rajasthan
Jaipur
RAJASTHAN |
9829182888
drkapilgargjkl@gmail.com |
| Dr Nita Radhakrishnan |
Post Graduate Institute of Child Health |
Department of Paediatric Haematology-Oncology, Post Graduate Institute of Child Health, Sector 30, Noida, Gautam Budh Nagar, Uttar Pradesh - 201303, India
Gautam Buddha Nagar
UTTAR PRADESH |
9999041524
nitark@gmail.com |
| Dr Chandrakala S |
Seth GS Medical College and KEM Hospital |
Seth GS Medical College and KEM Hospital, Parel, Mumbai, Maharashtra - 400012
Mumbai
MAHARASHTRA |
9699087654
drchandra_s@rediffmail.com |
| Dr Shashikant Apte |
Shahyadri Super Speciality Hospital |
Shahyadri Super Speciality Hospital, 30 C, Erandawane, Karve Road, Pune, Maharashtra - 411004
Pune
MAHARASHTRA |
9822404983
shashikant.apte@gmail.com |
|
Details of Ethics Committee
Modification(s)
|
| No of Ethics Committees= 4 |
| Name of Committee |
Approval Status |
| Ethics Committee SMS Medical college and Attached Hospital |
Approved |
| Institutional Ethics Committee (IEC)-I, Seth G. S. Medical College & K. E. M Hospital |
Approved |
| Institutional Ethics Committee, Super specialty Pediatric Hospital and PGTI SSPHPGTI |
Approved |
| Sahyadri Hospitals PVT Ltd Ethics Committee |
Approved |
|
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Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: D66||Hereditary factor VIII deficiency, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Intervention |
Mim8 |
For treatment part 1, all participants will start on once-weekly treatment and continue on this regimen until week 26. For treatment part 2, starting at week 26, all participants will be offered the choice to remain on once-weekly or switch to once-monthly dosing.
Mim8 will be injected with a thin needle into the skin |
| Comparator Agent |
NOT APPLICABLE |
NOT APPLICABLE |
|
|
Inclusion Criteria
|
| Age From |
1.00 Year(s) |
| Age To |
11.00 Year(s) |
| Gender |
Both |
| Details |
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 and female participants with the diagnosis of congenital haemophilia A of any severity based on medical records.
Aged 1-11 years (both inclusive) at the time of signing informed consent.
For previously treated participants -
a. Participant has been prescribed treatment with Factor Eight concentrate or bypassing agent in the last 26 weeks prior to screening.
b. Participants with endogenous factor eight activity greater than or equal to 1 percentage, based on medical records, must have at least 1 treated bleed during the previous 26 weeks before screening for which factor eight concentrate or bypassing agent has been prescribed (No requirements for participants with factor right activity below 1 percentage).
For previously untreated participants -
a. Diagnosis of severe haemophilia A (endogenous Factor eight activity below 1 percentage) based on medical records.
Child and parent or caregiver willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires. (For China mainland, assessed at the investigator discretion unless otherwise stated.) |
|
| ExclusionCriteria |
| Details |
Known or suspected hypersensitivity to trial product or related products. (For China mainland, assessed at the investigator discretion unless otherwise stated.)
Previous participation in this study. Participation is defined as signed informed consent.
Participation (that is, 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.
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.
Known congenital or acquired coagulation disorders other than haemophilia A.
Other conditions (eg, autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis, as evaluated by the investigator. (For China mainland, assessed at the investigator discretion unless otherwise stated.)
Any disorder, except for conditions associated with haemophilia A, that in the investigator opinion might jeopardise the participant safety or compliance with the protocol. (For China mainland, assessed at the investigator discretion unless otherwise stated.)
Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate understanding and cooperation. (For China mainland; assessed at the investigator discretion unless otherwise stated.)
Lack of adequate parental or caregiver support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary. (For China mainland, assessed at the investigator discretion unless otherwise stated.)
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.
Major surgery planned to take place after screening. (For China mainland, assessed at the investigator discretion unless otherwise stated.)
Immune tolerance induction planned to take place after treatment initiation. (For China mainland, assessed at the investigator discretion unless otherwise stated.)
Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) greater than 3 times the upper limit of normal combined with total bilirubin greater than 1.5 times the upper limit of normal measured at screening.
Hepatic dysfunction defined as aspartate aminotransferase (AST) and or alanine aminotransferase (ALT) greater than 3 times the upper limit of normal combined with total bilirubin greater than 1.5 times the upper limit of normal measured at screening.
Serum creatinine above 1.5 multiplied by the upper limit of normal (ULN), measured at screening.
Pregnancy (female participants). (Will be assessed at investigator discretion, according to suspicion of pregnancy.) |
|
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Method of Generating Random Sequence
|
Computer generated randomization |
|
Method of Concealment
|
Centralized |
|
Blinding/Masking
|
Open Label |
|
Primary Outcome
|
| Outcome |
TimePoints |
| Number of treatment emergent adverse events |
Time Frame: From treatment initiation to follow up visit (week 0 to week 72)
Count of events |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
| Number of treated bleeds |
Time Frame: From treatment initiation to end of treatment (week 0 to week 52)
Count of bleeds |
| Number of treated spontaneous bleeds |
Time Frame: From treatment initiation to end of treatment (week 0 to week 52
Count of bleeds |
| Number of treated traumatic bleeds |
Time Frame: From treatment initiation to end of treatment (week 0 to week 52)
Count of bleeds |
| Number of treated joint bleeds |
Time Frame: From treatment initiation to end of treatment (week 0 to week 52)
Count of bleeds |
| Number of treated target joint bleeds |
Time Frame: From treatment initiation to end of treatment (week 0 to week 52)
Count of bleeds |
| Number of injection site reactions |
Time Frame: From treatment initiation to end of treatment (week 0 to week 52)
Count of reactions |
| Consumption of factor product per bleed treatment |
Time Frame: From run-in initiation to end of treatment (week -26 to week 52)
Count of injections |
| Occurrence of anti-Mim8 antibodies |
Time Frame: From treatment initiation to end of treatment (week 0 to week 52)
Count of participants |
| Mim8 plasma concentration |
Time Frame: From treatment initiation to end of treatment (week 0 to week 52)
µg/mL |
| Change in physical function domain of PEDS QL (Paediatric Quality of Life inventory) Generic Core Scales |
Time Frame: From treatment initiation to end of treatment (week 0 to week 52)
Score on a scale 0-100 (applies for scale scores and total score). A higher score indicates a better health-related quality of life |
| Treatment preference for Mim8 versus previous treatment using Caregiver H PPQ (Caregiver Haemophilia Patient Preference ) |
Time Frame: Once during treatment (week 26)
Percentage of participants |
| Change in participants treatment burden using the Hemo TEM (Haemophilia treatment experience measure) |
Time Frame: From treatment initiation to end of treatment (week 0 to week 52)
Score on a scale 0-100 (applies for scale scores and total score). A lower score indicates a lower treatment burden. |
|
|
Target Sample Size
|
Total Sample Size="70"
Sample Size from India="10"
Final Enrollment numbers achieved (Total)= "72"
Final Enrollment numbers achieved (India)="9" |
|
Phase of Trial
|
Phase 3 |
Date of First Enrollment (India)
Modification(s)
|
09/05/2023 |
| Date of Study Completion (India) |
07/04/2025 |
| Date of First Enrollment (Global) |
04/04/2022 |
| Date of Study Completion (Global) |
07/04/2025 |
Estimated Duration of Trial
Modification(s)
|
Years="1"
Months="6"
Days="7" |
Recruitment Status of Trial (Global)
Modification(s)
|
Completed |
| Recruitment Status of Trial (India) |
Completed |
Publication Details
Modification(s)
|
NIL |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
|
Brief Summary
|
This study is looking at how Mim8 works compared to other medicines in children with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used for prevention of bleeds. Mim8 will be injected with a thin needle into the skin. The study will last for about 54-98 weeks, from screening to follow-up visit, In case the participant experiences bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. |