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A Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER 5)

Sponsor
Novo Nordisk A/S (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05878938
Collaborator
(none)
48
Enrollment
28
Locations
1
Arm
20
Anticipated Duration (Months)
1.7
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant 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.

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

Study Design

Study Type:
Interventional
Anticipated Enrollment :
48 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Open-label Safety Study in Adults and Adolescents With Haemophilia A With and Without FVIII Inhibitors Switching Directly From Emicizumab Prophylaxis to NNC0365-3769 (Mim8) Prophylaxis
Anticipated Study Start Date :
Jun 26, 2023
Anticipated Primary Completion Date :
Sep 9, 2024
Anticipated Study Completion Date :
Feb 24, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: NNC0365-3769 (Mim8) PPX

Participants will receive Mim8 prophylaxis (PPX) subcutaneous (s.c.) injection using a prefilled fixed dose DV3407-C1 pen-injector.

Drug: NNC0365-3769 (Mim8) PPX
Participants will receive Mim8 PPX once-weekly dosing (QW), once every two weeks dosing (Q2W), or once-monthly dosing s.c. injection using a prefilled fixed dose DV3407-C1 pen-injector for 26 weeks.

Outcome Measures

Primary Outcome Measures

  1. Number of treatment-emergent adverse events [From Visit 2 (week 0) until week 26]

    Measured as count of events.

Secondary Outcome Measures

  1. Device handling experience using the Hemophilia Device Handling and Preference Assessment (HDHPA) questionnaire [Visit 8 (after 26 weeks of treatment)]

    Measured as percentage of participants. HDHPA measures device handling experience and device preference. The measure consists of 26 items that are reported individually. it is measures in units: Percentage of participants = the distribution of participant answers within each response category, for each of the 26 individual items.

  2. Change in participants' treatment burden using the Hemophilia treatment experience measure (Hemo-TEM) total score [From Visit 2 (week 0) until end of treatment (up to 26 weeks)]

    Measured as score points. Hemo-TEM measures treatment burden. The measure consists of 26 items yielding 5 domain scores and 1 total score. Domain scores (score range): Injection difficulties (0-100), physical impact (0-100), treatment bother (0-100), interference with daily life (0-100), and emotional impact (0-100). Total score ranges 0-100. Higher scores indicate greater treatment burden.

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. Age 12 years or above at the time of signing the informed consent.

  4. Participants treated with emicizumab once-weekly (QW), once every two weeks (Q2W), or once every four weeks (Q4W) according to the label for at least 8 weeks prior to screening.

  5. Participants choosing to discontinue emicizumab treatment and switch to Mim8 QW, Q2W, or once-monthly (QM) treatment for 26 weeks from start of treatment (Visit 2).

  6. Participant and/or caregiver willingness and ability to comply with scheduled visits and study procedures, including the completion of an electronic diary and patient-reported outcomes (PRO) questionnaires.

Exclusion Criteria:

  1. Participation (i.e., signed informed consent) in any interventional, clinical study, with the exception of emicizumab, with receipt of the last dose within 8 weeks (or 5 half-lives of the investigational medicinal product [IMP], whichever is longer) before screening.

  2. Any disorder, which in the investigator's opinion might jeopardise the participant's compliance with the protocol or safety, including ongoing Adverse Events (AEs) associated with emicizumab.

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

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

  5. 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.

  6. Neutralising antibodies towards emicizumab have been detected or, for patients adherent to emicizumab therapy, are suspected based on clinical and laboratory assessments.

  7. Receipt of FVIII gene therapy at any time.

  8. Ongoing or planned immune tolerance induction therapy.

  9. Minor or major surgery planned to take place after screening and during the 26-week treatment period.

  10. Known or suspected hypersensitivity to study intervention, related products, any constituents of the product or to other monoclonal antibodies.

  11. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) greater than (>) 3 times the upper limit combined with total bilirubin >1.5 times the upper limit measured at screening.

  12. Renal impairment defined as estimated glomerular filtration rate (eGFR) lesser than or equal to (≤) 30 milliliter per minute per 1.73 square meter (mL/min/1.73 m^2) for serum creatinine measured at screening.

  13. Female who is pregnant, breast-feeding or intends to become pregnant or is of childbearing potential and not using highly effective contraceptive method.

  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 Los Angeles California United States 90027
2 Novo Nordisk Investigational Site Aurora Colorado United States 80045
3 Novo Nordisk Investigational Site Tampa Florida United States 33607
4 Novo Nordisk Investigational Site Iowa City Iowa United States 52242
5 Novo Nordisk Investigational Site East Lansing Michigan United States 48824
6 Novo Nordisk Investigational Site Mount Pleasant Michigan United States 48859
7 Novo Nordisk Investigational Site Cleveland Ohio United States 44106
8 Novo Nordisk Investigational Site Hershey Pennsylvania United States 17033-2360
9 Novo Nordisk Investigational Site Nashville Tennessee United States 37212
10 Novo Nordisk Investigational Site Innsbruck Austria A 6020
11 Novo Nordisk Investigational Site Wien Austria 1090
12 Novo Nordisk Investigational Site Bruxelles Belgium 1200
13 Novo Nordisk Investigational Site Hamilton Ontario Canada L8N 3Z5
14 Novo Nordisk Investigational Site Bron Cedex France 69500
15 Novo Nordisk Investigational Site Berlin Germany 10249
16 Novo Nordisk Investigational Site Bonn Germany 53127
17 Novo Nordisk Investigational Site Firenze Italy 50134
18 Novo Nordisk Investigational Site Milano Italy 20122
19 Novo Nordisk Investigational Site Napoli Italy 80131
20 Novo Nordisk Investigational Site Nara Japan 634-8522
21 Novo Nordisk Investigational Site Seoul Korea, Republic of 03722
22 Novo Nordisk Investigational Site Seoul Korea, Republic of 05278
23 Novo Nordisk Investigational Site Parktown, Johannesburg Gauteng South Africa 2193
24 Novo Nordisk Investigational Site Madrid Spain 28046
25 Novo Nordisk Investigational Site Málaga Spain 29010
26 Novo Nordisk Investigational Site Cardiff United Kingdom CF14 4XW
27 Novo Nordisk Investigational Site London United Kingdom NW3 2QG
28 Novo Nordisk Investigational Site Sheffield United Kingdom S10 2JF

Sponsors and Collaborators

  • Novo Nordisk A/S

Investigators

  • Study Director: Clinical Transparency (dept. 2834), Novo Nordisk A/S

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT05878938
Other Study ID Numbers:
  • NN7769-4728
  • U1111-1281-9323
  • 2022-003053-66
First Posted:
May 30, 2023
Last Update Posted:
May 30, 2023
Last Verified:
May 1, 2023
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 May 30, 2023