Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)

CSL Behring

Status: Invitation-only

Conditions: Hemophilia B

Treatments: Genetic: AAV5-hFIXco-Padua

Study type: Observational

Funder types: Industry

Identifiers

NCT05962398

CSL222_3003

Details and patient eligibility

About

The primary purpose of this study is to assess the long-term safety in male adults with hemophilia B who were treated with CSL222 in studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).

Enrollment: 56 estimated patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Treatment with CSL222 in Study CSL222_2001 or Study CSL222_3001.
• Provided written informed consent.
• Willing and able to adhere to all protocol requirements.

Exclusion criteria

Trial design

56 participants in 1 patient group

CSL222

Description:

Participants who received CSL222 and completed either the study CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891) will be followed up from Year 5 after administration of CSL222 in parent study up to Year 15.

Treatment:

Genetic: AAV5-hFIXco-Padua