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NCT06003387 · ClinicalTrials.gov registry record · Phase 3
Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
A Phase 3 study of Hemophilia B, sponsored by CSL Behring.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 35
- Enrollment target
- 20
- Study locations
NCT06003387: Recruiting Phase 3 study of Hemophilia B, sponsored by CSL Behring.
NCT06003387 is a Phase 3 study of Hemophilia B that is actively recruiting participants, run by CSL Behring. The registered enrollment target is 35 participants, below the 95-participant average among 20 other Hemophilia B trials with a reported enrollment target (63% lower). The trial reports 20 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06003387, a Phase 3 study of Hemophilia B, is actively recruiting participants, sponsored by CSL Behring.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 35 participants
- Enrollment target
- 20
- Study locations
Study Summary
The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.
Primary Outcome
The total bleeding episodes will be analyzed. ABR is calculated as the total bleeding episodes divided by the total time at risk.
Conditions Studied
Interventions
- GENETIC CSL222 (AAV5-hFIXco-Padua)
Study Locations (20)
Other
- Queen Mary Hospital - Hong Kong
- Prince of Wales Hospital Chinese University of Hong Kong - Shatin
- Sheba Medical Center - Tel Litwinsky
- King Faisal Specialist Hospital and Research Center - Riyadh
- National University Hospital - Singapore
- Singapore General Hospital - Singapore
- Haemophilia Comprehensive Care Centre - Johannesburg
- Kyungpook National University Hospital - Daegu
- Kyung Hee University Hospital at Gangdong - Seoul
- Severance Hospital, Yonsei University Health System - Seoul
California
- University of California, San Diego (UCSD) - San Diego
Michigan
- University of Michigan - Ann Arbor
New South Wales
- Royal Prince Alfred Hospital - Camperdown
Queensland
- Royal Brisbane Hospital - Herston
South Australia
- Royal Adelaide Hospital - Adelaide
Victoria
- The Alfred Hospital - Melbourne
Ontario
- McMaster University - Hamilton - Hamilton
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 35 participants |
| Start Date | 2024-01-30 |
| Est. Completion | 2032-04-02 |
| Phase | Phase 3 |
What NCT06003387 shows while recruiting
NCT06003387 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 35 participants, a relatively small participant target, below the 95-participant average among 20 other Hemophilia B trials with a reported enrollment target (63% lower).
The record links to 1 condition, with Hemophilia B appearing as the primary indexed condition, and to 1 intervention - of which CSL222 (AAV5-hFIXco-Padua) is the first listed.
NCT06003387 names 20 study sites across 11 states, led by Other, California, Michigan.
Frequently Asked Questions
What is clinical trial NCT06003387 about?
NCT06003387 is a clinical study titled "Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)". The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.
What is the current status of trial NCT06003387?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 35 participants. The study started on 2024-01-30. Estimated completion is 2032-04-02.
What conditions does trial NCT06003387 study?
This clinical trial studies the following conditions: Hemophilia B.
What interventions are being tested in trial NCT06003387?
The interventions under investigation include: CSL222 (AAV5-hFIXco-Padua) (GENETIC).
Who is sponsoring clinical trial NCT06003387?
This trial is sponsored by CSL Behring, which has 71 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06003387 being conducted?
This trial has 20 study locations across California, Michigan, New South Wales, Queensland, South Australia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Hemophilia B
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06003387's enrollment target sits among peer trials
35 11th of 20 higher than 10 of 20 other Hemophilia B trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Hemophilia B trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
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
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-
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
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-
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
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Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
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A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
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A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
COMPLETED · Phase 3
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