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A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors

Sponsor
Novo Nordisk A/S (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05053139
Collaborator
(none)
244
Enrollment
86
Locations
4
Arms
40.3
Anticipated Duration (Months)
2.8
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study is investigating how Mim8 works compared to other medicines in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used for prevention of bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII).

When and how often participants will receive Mim8 is dependent on their previous treatment - but is otherwise decided by chance. Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.

The study will last 54-124 weeks (12-29 months) depending on how long participants will be followed in run-in before they start treatment and if they continue in the follow period or transfer to an open label extension study. Participants will have 12-17 clinic visits.

Condition or Disease Intervention/Treatment Phase
  • Drug: NNC0365-3769 (Mim8)
 Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
244 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Multinational, Open-label, Randomised, Controlled Study to Investigate Efficacy and Safety of NNC0365-3769 (Mim8) in Adults and Adolescents With Haemophilia A With or Without Inhibitors
Actual Study Start Date :
Dec 2, 2021
Anticipated Primary Completion Date :
May 24, 2024
Anticipated Study Completion Date :
Apr 11, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: no PPX- no PPX - Mim8 PPXQW

Participants not receiving prophylaxis will not enter the run-in period. In arm 1, participants will be randomised to continue no prophylaxis (on-demand treatment with their Standard of Care FVIII concentrates) in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) on once-weekly Mim8 prophylaxis regimen

Drug: NNC0365-3769 (Mim8)
Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.
Experimental: no PPX - Mim8 PPXQW - Mim8 PPXQW

Participants not receiving prophylaxis will not enter the run-in period. In arm 2, participants will be randomised to Mim8 once-weekly prophylaxis in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) on once-weekly Mim8 prophylaxis regimen

Drug: NNC0365-3769 (Mim8)
Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.
Experimental: PPX - Mim8 PPXQW

Participants on coagulation factor prophylaxis prior to enrolment will preferably continue the same product type and dosing frequency in the run-in period for at least 26 weeks before they can be randomised into the main part of the study. These participants will only be allowed to receive coagulation factor prophylaxis. In arm 3, participants will be randomised to once-weekly Mim8 prophylaxis regimen in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) on once-weekly Mim8 prophylaxis regimen.

Drug: NNC0365-3769 (Mim8)
Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.
Experimental: PPX- Mim8 PPXQM

Participants on coagulation factor prophylaxis prior to enrolment will preferably continue the same product type and dosing frequency in the run-in period for at least 26 weeks before they can be randomised into the main part of the study. These participants will only be allowed to receive coagulation factor prophylaxis. In arm 4, participants will be randomised to once-monthly Mim8 prophylaxis regimen in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) on once-monthly Mim8 prophylaxis regimen.

Drug: NNC0365-3769 (Mim8)
Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.

Outcome Measures

Primary Outcome Measures

  1. Number of treated bleeds [No prophylaxis treatment (Arms 1 and 2): From randomisation (week 0) to end of main (Week 26)]

    Count

  2. Number of treated bleeds [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)]

    Count

Secondary Outcome Measures

  1. Number of injection site reactions [All participants receiving Mim8 (Arms 2, 3 and 4): From randomisation (week 0) to end of main (week 26)]

    Count

  2. Occurrence of anti-Mim8 antibodies [All participants receiving Mim8 (Arms 2, 3 and 4): From randomisation (week 0) to end of extension (week 52)]

    Count

  3. Number of treated spontaneous bleeds [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)]

    Count

  4. Number of treated joint bleeds [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)]

    Count

  5. Number of treated traumatic bleeds [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)]

    Count

  6. Number of target joint bleeds [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)]

    Count

  7. Consumption of factor product per bleed treatment (number of injections) [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)]

    Count

  8. Change in physical function domain of PEDS-QL (pediatric quality of life inventory) [All participants (Arms 1, 2, 3 and 4): From randomisation (week 0) to the end of the main part (week 26)]

    Score points Minimum score per question (best) = 0 Maximum score per question (worst) = 4 Total score for 13 questions: 0 (best) to 92 (worst)

  9. Change in patient's treatment burden using the Hemo-TEM (haemophilia treatment experience measure) [All participants (Arms 1, 2, 3 and 4): From randomisation (week 0) to the end of the main part (week 26)]

    Score points Ranges from 0 (best) - 4 (worst) representing answers ranging: 'Not at all difficult' - 'Extremely difficult' 'Never' - 'always' 'Not at all bothered' - 'Extremely bothered' 'Not at all interfering' - 'Extremely interfering' 'Not at all burdened' - 'Extremely burdened'

  10. Change in patient's joint pain score using Joint Pain Rating Scale [All participants (Arms 1, 2, 3 and 4): From randomisation (week 0) to the end of the main part (week 26)]

    Score points ranges from 0 = 'not at all' (best) to 4 = 'extremely' (worst)

Eligibility Criteria

Criteria

Ages Eligible for Study:
12 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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 of any severity based on medical records

  3. Participants has been prescribed treatment with factor VIII concentrates or bypassing agent in the last 26 weeks prior to screening

  4. Age above or equal to 12 years at the time of signing informed consent. (Local requirements for Japan, South Korea and Taiwan.)

  5. Body weight above or equal to 30 kg

  6. Applicable to participants treated with on-demand/no prophylaxis prior to enrolment: at least 5 bleeds in the last 26 weeks prior to screening visit, for which factor VIII concentrates or bypassing agent has been prescribed

  7. Applicable to participants with FVIII activity above or equal to 1% who are on prophylactic treatment: at least 1 bleed in the last 26 weeks prior to screening visit, for which factor VIII concentrates or bypassing agent has been prescribed

  8. Willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires

Exclusion Criteria:

  1. Previous participation in this study. Participation is defined as signed informed consent

  2. Participation in any clinical study 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

  3. Exposure to non-factor haemostatic products for bleeding prophylaxis within 6 months (or 5 half-lives of the medicinal product, whichever is shorter) prior to planned first dose, for participants not included in the run-in.

  4. 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. Breast feeding is allowed only during the run-in period

  5. 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

  6. Known or suspected hypersensitivity to study product(s), any constituents of the product or to related products

  7. Receipt of gene therapy at any given time point

  8. Ongoing or planned immune tolerance induction (ITI) therapy

  9. Major surgery planned at the time of screening.

  10. Known congenital or acquired coagulation disorders other than haemophilia A

  11. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) above 3 times the upper limit combined with total bilirubin above1.5 times the upper limit measured at screening

  12. 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

  13. Previous or current thromboembolic disease or events (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

  14. Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation

  15. Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis as evaluated by the investigator

Contacts and Locations

Locations

Site City State Country Postal Code
1 Novo Nordisk Investigational Site Phoenix Arizona United States 85016-7710
2 Novo Nordisk Investigational Site Los Angeles California United States 90007
3 Novo Nordisk Investigational Site Los Angeles California United States 90027
4 Novo Nordisk Investigational Site Aurora Colorado United States 80045
5 Novo Nordisk Investigational Site Miami Florida United States 33136
6 Novo Nordisk Investigational Site Tampa Florida United States 33607
7 Novo Nordisk Investigational Site Atlanta Georgia United States 30329
8 Novo Nordisk Investigational Site Savannah Georgia United States 31404
9 Novo Nordisk Investigational Site Indianapolis Indiana United States 46260
10 Novo Nordisk Investigational Site Iowa City Iowa United States 52242
11 Novo Nordisk Investigational Site Cleveland Ohio United States 44106
12 Novo Nordisk Investigational Site Columbus Ohio United States 43205
13 Novo Nordisk Investigational Site Dayton Ohio United States 45404
14 Novo Nordisk Investigational Site Hershey Pennsylvania United States 17033-2360
15 Novo Nordisk Investigational Site Milwaukee Wisconsin United States 53226
16 Novo Nordisk Investigational Site Innsbruck Austria A 6020
17 Novo Nordisk Investigational Site Wien Austria 1090
18 Novo Nordisk Investigational Site Bruxelles Belgium 1200
19 Novo Nordisk Investigational Site Edegem Belgium 2650
20 Novo Nordisk Investigational Site Leuven Belgium 3000
21 Novo Nordisk Investigational Site Chengdu Sichuan China 610000
22 Novo Nordisk Investigational Site Tianjing Tianjin China 300020
23 Novo Nordisk Investigational Site Kunming Yunnan China 650101
24 Novo Nordisk Investigational Site Praha 2 Czechia 12000
25 Novo Nordisk Investigational Site København Ø Denmark 2100
26 Novo Nordisk Investigational Site Bron Cedex France 69677
27 Novo Nordisk Investigational Site Le Kremlin Bicetre France 94270
28 Novo Nordisk Investigational Site Nantes Cedex 1 France 44093
29 Novo Nordisk Investigational Site Paris France 75015
30 Novo Nordisk Investigational Site Rennes France 35033
31 Novo Nordisk Investigational Site Berlin Germany 10249
32 Novo Nordisk Investigational Site Dublin Ireland D08 A978
33 Novo Nordisk Investigational Site Dublin Ireland D12 N512
34 Novo Nordisk Investigational Site Tel-Hashomer Israel 52621
35 Novo Nordisk Investigational Site Firenze Italy 50134
36 Novo Nordisk Investigational Site Milano Italy 20089
37 Novo Nordisk Investigational Site Aichi Japan 466-8560
38 Novo Nordisk Investigational Site Kitakyusyu-shi, Fukuoka Japan 807 8555
39 Novo Nordisk Investigational Site Okinawa Japan 901-1193
40 Novo Nordisk Investigational Site Saitama Japan 330-8777
41 Novo Nordisk Investigational Site Tochigi Japan 329-0498
42 Novo Nordisk Investigational Site Tokyo Japan 157-8535
43 Novo Nordisk Investigational Site Tokyo Japan 160-0023
44 Novo Nordisk Investigational Site Tokyo Japan 167-0035
45 Novo Nordisk Investigational Site Daejeon Korea, Republic of 35233
46 Novo Nordisk Investigational Site Seoul Korea, Republic of 03722
47 Novo Nordisk Investigational Site Seoul Korea, Republic of 05278
48 Novo Nordisk Investigational Site Riga Latvia LV-1002
49 Novo Nordisk Investigational Site Kaunas Lithuania LT-50161
50 Novo Nordisk Investigational Site Vilnius Lithuania 08406
51 Novo Nordisk Investigational Site Vilnius Lithuania LT-08661
52 Novo Nordisk Investigational Site Amsterdam Netherlands 1105 AZ
53 Novo Nordisk Investigational Site Utrecht Netherlands 3584 CX
54 Novo Nordisk Investigational Site Lublin Poland 20-093
55 Novo Nordisk Investigational Site Wroclaw Poland 50-556
56 Novo Nordisk Investigational Site Coimbra Portugal 3000-561
57 Novo Nordisk Investigational Site Porto Portugal 4099-001
58 Novo Nordisk Investigational Site Porto Portugal 4200-319
59 Novo Nordisk Investigational Site Moscow Russian Federation 119049
60 Novo Nordisk Investigational Site Moscow Russian Federation 125167
61 Novo Nordisk Investigational Site Petrozavodsk Russian Federation 185019
62 Novo Nordisk Investigational Site Saint-Petersburg Russian Federation 191186
63 Novo Nordisk Investigational Site Riyadh Saudi Arabia 12713
64 Novo Nordisk Investigational Site Belgrade Serbia 11000
65 Novo Nordisk Investigational Site Novi Sad Serbia 21000
66 Novo Nordisk Investigational Site Martin Slovakia 036 59
67 Novo Nordisk Investigational Site Vranov nad Toplou Slovakia 09327
68 Novo Nordisk Investigational Site Johannesburg Gauteng South Africa 2013
69 Novo Nordisk Investigational Site Parktown, Johannesburg Gauteng South Africa 2193
70 Novo Nordisk Investigational Site Barcelona Spain 08035
71 Novo Nordisk Investigational Site Madrid Spain 28046
72 Novo Nordisk Investigational Site Málaga Spain 29009
73 Novo Nordisk Investigational Site Bern Switzerland 3010
74 Novo Nordisk Investigational Site St. Gallen Switzerland 9001
75 Novo Nordisk Investigational Site Zürich Switzerland 8091
76 Novo Nordisk Investigational Site Taichung City Taiwan 407
77 Novo Nordisk Investigational Site Taipei Taiwan 100
78 Novo Nordisk Investigational Site Adana Turkey 01130
79 Novo Nordisk Investigational Site Ankara Turkey 06500
80 Novo Nordisk Investigational Site Antalya Turkey 01010
81 Novo Nordisk Investigational Site Bornova-IZMIR Turkey 35100
82 Novo Nordisk Investigational Site Capa-ISTANBUL Turkey 35100
83 Novo Nordisk Investigational Site Cardiff United Kingdom CF14 4XW
84 Novo Nordisk Investigational Site London United Kingdom NW3 2QG
85 Novo Nordisk Investigational Site London United Kingdom SE1 7EH
86 Novo Nordisk Investigational Site Oxford United Kingdom OX3 7LJ

Sponsors and Collaborators

  • Novo Nordisk A/S

Investigators

  • Study Director: Clinical Transparency (dept. 1452), Novo Nordisk A/S

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT05053139
Other Study ID Numbers:
  • NN7769-4514
  • U1111-1249-4378
  • 2020-001048-24
First Posted:
Sep 22, 2021
Last Update Posted:
Mar 24, 2022
Last Verified:
Mar 1, 2022
Individual Participant Data (IPD) Sharing Statement:
Yes
Plan to Share IPD:
Yes
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Hemophilia A

Study Results

No Results Posted as of Mar 24, 2022