Gene therapy for haemophilia B

Gene therapy for haemophilia B: Insights from early adopters in the Netherlands - Gene therapy for haemophilia B:Insights from early adoptersin the Netherlands

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON58404

Enrollment

Registered

2025-12-18

Start date

2026-03-02

Completion date

Unknown

Last updated

2026-04-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Haemophilia Haemophilia

Interventions

AMT-060 used in clinical trials. This product is now licensed as etranacogene dezaparvovec (Hemgenix)

Eligibility

Age

18 Years to 64 Years

Inclusion criteria

Inclusion criteria: Male (self-identified), aged =18 years.Diagnosed with haemophilia B (any severity).Received systemic gene therapy for haemophilia with AMT-060 at least 6 months prior to interview (to allow stabilisation of early effects).Able to give informed consent and participate in a 60-minute interview in the study language.

Exclusion criteria

Exclusion criteria: Received gene therapy with any other product.Cognitive or severe communication impairment precluding meaningful participation (unless supported participation is possible and ethically approved).Non-Native speakers only if translation resources are not available.

Design outcomes

Primary

Measure	Time frame
To understand the impact of gene therapy on adult males with haemophilia B. This will give in depth information about the day-to-day impact beyond factor levels and bleeds.	—

Countries

Netherlands

Contacts

Public ContactK Khair

Haemnet Ltd

Kate@Haemnet.com447515900812

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP) · Data processed: May 1, 2026