A Phase 3 safety study of BAY 94-9027 in children 7 to <12 years of age with severe hemophilia A

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Bayer Consumer Care AG, Bayer AG

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

26 Sep 2022 → 25 Jun 2025

Decision date (initial)

2024-03-18

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Bayer AG, D-51368, Leverkusen, Germany

External identifiers

EU CT number

2023-504388-18-00

EudraCT number

2021-004858-30

ClinicalTrials.gov

NCT05147662

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Safety, Prophylaxis

To assess safety and tolerability of BAY 94-9027 replacement therapy in previously treated patients 7 to <12 years of age with severe hemophilia A

Secondary objectives 1

1. To describe clinical efficacy of BAY 94-9027

Conditions and MedDRA coding

Severe hemophilia A (<1% FVIII:C)

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Participant must be 7 to <12 years of age at the time of parent / guardian signing the informed consent.
2. Participants with known medical history of severe hemophilia A (participant's own FVIII activity [FVIII:C] <1%. FVIII:C) based on reliable prior documentation in clinical records of the participants. If no reliable documentation is available, FVIII activity must be measured at the time of screening after a 48-72 hours wash-out period (depending on his previous product).
3. Male
4. Participants must be previously treated with FVIII concentrate(s) (plasma derived or recombinant) for a minimum of 50 exposure days (EDs) at the time of signing the informed consent.
5. Participant has understood the study and if appropriate for his age, has signed an informed assent. The parent(s) or guardian(s) is capable of giving signed informed consent and are able to comply with the requirements and restrictions listed in the informed consent form (ICF) and the protocol.
6. Willingness and ability of participants and/or parents /caregivers to complete training in the use of the electronic patient diary (EPD) and to document infusions during the study.

Exclusion criteria 13

1. "History of FVIII inhibitors. Inhibitor to FVIII is defined as a titer >0.6 BU/mL or clinical history suggestive of inhibitor requiring modification of treatment. Participants with a maximum historical titer of <1.0 BU on no more than 1 occasion with the classical Bethesda assay but at least 3 subsequent negative results [<0.6 BU] are eligible."
2. Current evidence of inhibitor to FVIII measured using the Nijmegen modified Bethesda assay (>0.6 BU/mL) at the time of screening (central laboratory). Participants should not receive FVIII within 48 h prior to the collection of screening samples and should have FVIII administered within the prior 2-3 weeks
3. Any other inherited or acquired bleeding disorder in addition to hemophilia A (e.g., von Willebrand disease, hemophilia B)
4. Platelet count <100,000 cells/μL
5. Serum creatinine > 2x upper limit of normal
6. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 5x upper limit of normal
7. Known hypersensitivity or allergic reaction to drug substance, excipients or mouse or hamster protein
8. Any other significant medical condition that the investigator feels would be a risk to the participant or would impede the study
9. Requires any pre-medication to tolerate FVIII treatment (e.g. antihistamines)
10. Planned major surgery during the study
11. Any individual who is currently receiving or received chemotherapy, immune modulatory drugs other than anti-retroviral chemotherapy, or chronic use of oral or intravenous (IV) corticosteroids (> 14 days) within the last 3 months
12. Any individual who received commercially available subcutaneous factor substitution therapy (emicizumab) within the last 6 months
13. The participant is currently participating in another investigational drug study or has participated in a clinical study involving an investigational drug within 30 days of study entry or previous participation in a clinical study with BAY 94-9027.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. AESI (hypersensitivity and LOE) associated with the first 4 EDs leading to discontinuation

Secondary endpoints 6

1. Adverse drug reactions (ADRs)
2. Anti-drug antibody (ADA) development
3. Inhibitor development
4. Annualized bleeding rate (ABR)
5. BAY94-9027 consumption
6. Number of infusions/month and year (Annualized Infusion Rate)

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Bayer Consumer Care AG

Sponsor organisation

Bayer Consumer Care AG

Address

Peter Merian-Strasse 84

City

Basel

Postcode

4052

Country

Switzerland

Scientific contact point

Organisation

Bayer AG

Contact name

Therapeutic Area Head

Public contact point

Organisation

Bayer AG

Contact name

Therapeutic Area Head

Third parties 4

Bayer AG

Sponsor organisation

Bayer AG

Address

-

City

Leverkusen

Postcode

51368

Country

Germany

Sponsor responsibilities

Article 77 compliance

Bayer Consumer Care AG

Article 77 implementation

Bayer Consumer Care AG

Locations

2 EU/EEA countries · 2 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

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 14 files

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

Application history

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