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Gene Therapy of Haemophilia: Patient Experiences 2025

Gene Therapy of Haemophilia: Patient Experiences 2025

Status
Recruiting
Phases
Unknown
Study type
Observational
Source
DRKS
Registry ID
DRKS00039722
Enrollment
3
Registered
2026-03-27
Start date
2026-06-01
Completion date
Unknown
Last updated
2026-08-03

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

Conditions

D67

Interventions

Group 1: Patients who have received factor IX gene therapy will be interviewed by phone.

Sponsors

Hämophiliezentrum/Hämostaseologie, Universitätsklinikum Frankfurt
Lead Sponsor

Eligibility

Sex/Gender
Male
Age
18 Years to No maximum

Inclusion criteria

Inclusion criteria: - patients with haemophilia B - patients who have participated in a clinical study on factor IX gene therapy in Germany, study centres Frankfurt or Berlin

Exclusion criteria

Exclusion criteria: - patients who were recruited in other study centres

Design outcomes

Primary

MeasureTime frame
• Gene therapy: implementation, effectiveness, satisfaction with the gene therapy, reasons for participation in the study • social aspects such as work, family, friends, and social activities • physical aspects such as impairments, mobility, and daily (treatment) routine • emotional aspects such as fears and concerns, expectations • patient care, including prior information about gene therapy and support from the treating physician The data will be collected once ??????? patient interviews.

Secondary

MeasureTime frame
age duration of disease FIX substitution bleeding events

Countries

Germany

Contacts

Public ContactWolfgang Miesbach

Hämophiliezentrum/Hämostaseologie, Universitätsklinikum Frankfurt

Wolfgang.miesbach@unimedizin-ffm.de+496963015051

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: Aug 10, 2026