A research study looking at long-term treatment with Mim8 in people with haemophilia A (FRONTIER 4)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novo Nordisk A/S

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

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

Trial duration

4 May 2023 → ongoing

Decision date (initial)

2023-04-18

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Novo Nordisk A/S

External identifiers

EU CT number

2022-502215-10-00

WHO UTN

U1111-1274-4426

ClinicalTrials.gov

NCT05685238

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Prophylaxis, Safety, Efficacy

To investigate long-term safety of Mim8 prophylaxis in participants with haemophilia A with or without FVIII inhibitors.
France, Germany, Italy, the Netherlands, Poland, Spain: For these countries the study will include an Arm 3 (infant) which will investigate the safety, efficacy and exposure of
Mim8 prophylaxis in previously untreated patients and minimally treated patients: 1-5 exposure days, <1 year of age with haemophilia A (intrinsic FVIII level <1%) with or
without FVIII inhibitors.

Secondary objectives 2

1. To evaluate efficacy of Mim8 XXX prophylaxis, and long term efficacy of XXX, XXX and XXX prophylaxis, in participants with haemophilia A with or without inhibitors.
2. To evaluate the device handling experience for administration of Mim8 using the DV3407- C1 pen-injector.

Conditions and MedDRA coding

Haemophilia A (with or without inhibitors)

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, National Medical Products Administration, Food And Drug Administration, Health Products Regulatory Authority, Pharmaceuticals And Medical Devices Agency, Paul Ehrlich Institute, Health Canada

EMA paediatric investigation plan (PIP)

EMEA-002762-PIP02-20

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

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 based on medical records.
3. Ongoing participation in study 4513, 4514, 4516, or 4728 at the time of transfer. Participant should qualify either of the following criteria: a. Participant from study 4513, who has participated in the extension part of the study for at least 12 weeks prior to enrolment in study 4532, or, b. Participant has completed the end of treatment visit for study 4514,4516, or 4728. Participants that are younger than 1 year old at screening are not joining from another study.
4. Participant and/or participant’s parent(s)/participant’s legally acceptable representative (LAR) willingness and ability to comply with scheduled visits and study procedures, including the completion of diary.

Exclusion criteria 8

1. Any disorder, except for conditions associated with haemophilia, which in the investigator’s opinion might jeopardise participant’s safety or compliance with the protocol.
2. Participant who has discontinued or been withdrawn from studies 4513, 4514, 4516, or 4728.
3. Previous participation in this study. Participation is defined as signed informed consent.
4. Female who is pregnant, breast-feeding or intends to become pregnant.
5. Female of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures as defined in protocol or as required by local regulation or practice).
6. Participation (i.e., signed informed consent) in any other interventional clinical study (except from study 4513, 4514, 4516, or 4728) of an approved or non-approved investigational medicinal product.
7. Any planned major surgery, during part 1 of the study.
8. Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Number of treatment‑emergent adverse events. From visit 1 (week 0) until end of study (up to 283 weeks).

Secondary endpoints 9

1. Number of injection site reactions. From visit 1 (week 0) until end of treatment (up to 262 weeks).
2. Occurrence of anti Mim8 antibodies. From visit 1 (week 0) until end of treatment (up to 262 weeks).
3. Number of treated bleeding episodes. From visit 1 (week 0) until end of treatment (up to 262 weeks).
4. Number of treated spontaneous bleeding episodes. From visit 1 (week 0) until end of treatment (up to 262 weeks).
5. Number of treated traumatic bleeding episodes. From visit 1 (week 0) until end of treatment (up to 262 weeks).
6. Number of treated joint bleeding episodes. From visit 1 (week 0) until end of treatment (up to 262 weeks).
7. Number of treated target joint bleeding episodes (arm 2). From visit 1 (week 0) until end of treatment (up to 262 weeks).
8. Mim8 plasma concentration. From visit 1 (week 0) until end of treatment (up to 262 weeks).
9. Device handling experience using Haemophilia Device Assessment Tool (HDAT) (applicable for participants in Arm 2 only).

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 5

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novo Nordisk A/S

Sponsor organisation

Novo Nordisk A/S

Address

Novo Alle 1

City

Bagsvaerd

Postcode

2880

Country

Denmark

Scientific contact point

Organisation

Novo Nordisk A/S

Contact name

EU Submission Hub

Public contact point

Organisation

Novo Nordisk A/S

Contact name

EU Submission Hub

Third parties 7

Locations

16 EU/EEA countries · 45 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 270 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

27 submissions — initial application plus substantial / non-substantial modifications