Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222) (IX-TEND 3003)

CSL Behring

Status: Invitation-only

Conditions: Hemophilia B

Treatments: Genetic: AAV5-hFIXco-Padua

Study type: Observational

Funder types: Industry

Identifiers

NCT05962398

CSL222_3003

EU CT Number (Other Identifier)

Details and patient eligibility

About

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

Enrollment: 56 estimated patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

Received treatment with CSL222 in Study CSL222_2001 or Study CSL222_3001, and completed participation in Study CSL222_2001 or Study CSL222_3001 or at least 5 years have passed since receiving CSL222.

Exclusion criteria

Not Applicable

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