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 Gainesville, Florida.

Sponsor
CSL Behring
Study ID
NCT07080905
Phase
PHASE3
Status
Recruiting
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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

Start date
Jul 28, 2025
Status verified
Jun 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 (2)

FacilityCityStateZIPSite coordinators
University of Florida - 84000399GainesvilleFlorida32611
Trial Contact
[email protected]
352-294-8846
University of Michigan Medical Center - 84000491Ann ArborMichigan48109
Trial Contact
[email protected]
734-232-9335

Find similar trials in Gainesville, FL

By research site
University of Florida - 84000399· Gainesville, FLUniversity of Michigan Medical Center - 84000491· Ann Arbor, MI

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