A study to investigate long-term follow-up in subjects who received BMN 270 in a prior BioMarin Clinical Trial with hemophilia A

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Biomarin Pharmaceutical Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male

Therapeutic area

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

Trial duration

16 Aug 2024 → ongoing

Decision date (initial)

2024-06-18

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

BioMarin Pharmaceutical Inc.

External identifiers

EU CT number

2023-507749-27-00

WHO UTN

U1111-1297-8403

ClinicalTrials.gov

NCT05768386

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

Evaluate the long-term safety of BMN 270

Secondary objectives 3

1. Evaluate the long-term effects of BMN 270 in participants with hemophilia A previously treated in a BioMarin clinical trial
2. Evaluate the use of hemostatic agents (eg, emicizumab, FVIII replacement therapy, efanesoctocog, and all approved hemostatic agents)
3. Evaluate the long-term impact of BMN 270 on HRQoL

Conditions and MedDRA coding

Hemophilia A

Study design 3 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Participants must have completed the End of Study Visit in their primary treatment study (Study 270-201, 270-203, 270-205, 270-301, 270-302, or 270-303) or be currently enrolled in one of these studies and have completed a minimum of 24 months of post-infusion follow-up in that study at the time of closure by the Sponsor. Participants may enroll in 270-401 even if they have restarted FVIII prophylaxis or other hemophilia A treatment.
2. Participants must be capable of giving signed informed consent as described in Appendix 10.1.3 of the protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

Exclusion criteria 3

1. Participants who do not directly enroll in 270-401 at the time of the study completion visit in their primary treatment study should enroll in 270-401 within 4 months of the date of that study completion visit. If a participant wishes to enroll in 270-401 after 4 months, critical data (in particular FVIII activity, bleeding episodes, FVIII use, AEs, SAEs, and the use of hemophilia medications) must be available from the extended duration between studies, and, in the opinion of the Investigator or Medical Monitor, any missing data would not impact or interfere with evaluation and interpretation of the study.
2. Participants must be overtly healthy and not have any condition that, in the opinion of the Investigator or Medical Monitor, would prevent the participant from fully complying with the requirements of the study and/or would impact or interfere with evaluation and interpretation of the study data (including, if applicable, advanced HIV disease).
3. Where applicable, per country regulation, the participant must not currently be committed to an institution by virtue of an order issued either by judicial or administrative authorities.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Occurrence of adverse drug reactions, serious adverse events, and events of special interest, including: a) Hepatotoxicity; b) Thromboembolic events; c) Development of FVIII inhibitors; d) Transmission to third parties; e) Integration with theoretical risk of tumorigenesis

Secondary endpoints 4

1. Changes in annualized bleeding rate (ABR) (treated bleeds and all bleeds)
2. FVIII activity measured over time (CSA and OSA)
3. Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate)
4. Changes in Haemo-QoL-A

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Biomarin Pharmaceutical Inc.

Sponsor organisation

Biomarin Pharmaceutical Inc.

Address

105 Digital Drive

City

Novato

Postcode

94949-8703

Country

United States

Scientific contact point

Organisation

Biomarin Pharmaceutical Inc.

Contact name

Project Manager

Public contact point

Organisation

Biomarin Pharmaceutical Inc.

Contact name

Project Manager

Third parties 6

Locations

5 EU/EEA countries · 8 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 44 files

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

Application history

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