Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

Part of paid clinical trials in Orange, California.

Sponsor
CSL Behring
Study ID
NCT07080905
Phase
PHASE3
Status
Recruiting
Apply to this trial

Conditions

  • Hemophilia B

Eligibility Criteria

Sex
MALE
Age
138 Months - 206 Months
Healthy Volunteers
Not accepted

Interventions

  • CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua) — GENETIC
    Administered as a single IV infusion.

Study Details

This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately severe hemophilia B.

Key Dates

First listed
Jul 23, 2025
Start date
Jul 28, 2025
Status verified
Aug 2026
Primary completion
Oct 24, 2033
Completion
Oct 24, 2033

Study Design

Enrollment
20 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: CSL222
    Participants will receive CSL222 as a single intravenous (IV) infusion of 2 × 10\^13 genome copies per kilogram of body weight (gc/kg).

Primary Outcome Measure

Annualized Bleeding Rate (ABR) [ Time Frame: For the Lead-In Period (at least 6 months) and for the 52 weeks after stable FIX expression (months 7 to 18 after treatment with CSL222) ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
Center for Inherited Blood DisordersOrangeCalifornia92868-
University of FloridaGainesvilleFlorida32611
Trial Contact
[email protected]
352-294-8846
Arthur M. Blank Hospital - Children's Healthcare of AtlantaAtlantaGeorgia30329-
University of Michigan Medical CenterAnn ArborMichigan48109
Trial Contact
[email protected]
734-232-9335
St. Jude Children's Research HospitalMemphisTennessee38105-

Find similar trials in Orange, CA

By research site
Center for Inherited Blood Disorders· Orange, CAUniversity of Florida· Gainesville, FLArthur M. Blank Hospital - Children's Healthcare of Atlanta· Atlanta, GAUniversity of Michigan Medical Center· Ann Arbor, MISt. Jude Children's Research Hospital· Memphis, TN

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