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NCT03489291 · ClinicalTrials.gov registry record · Phase 2
Dose Confirmation Trial of AAV5-hFIXco-Padua
A Phase 2 study, sponsored by CSL Behring.
- Completed
- Registry status
- Phase 2
- Development phase
- 3
- Enrollment target
NCT03489291: Completed Phase 2 study, sponsored by CSL Behring.
NCT03489291 is a Phase 2 study that has completed, run by CSL Behring. The registered enrollment target is 3 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (98% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03489291, a Phase 2 study, has completed, sponsored by CSL Behring.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 3 participants
- Enrollment target
Study Summary
This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase. The IMP AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The IMP is identified as AAV5-hFIXco-Padua (AMT- 061). The pharmaceutical form of AMT-061 is a solution for intravenous infusion. The administered dose of AMT-061 will be 2 x 10\^13 gc/kg.
Primary Outcome
To confirm that a single dose of 2x10\^13 gc/kg AMT-061 (CSL222) resulted in factor IX activity levels of ≥5% at 6 weeks after dosing measured by the one-stage (activated partial thromboplastin \[aPTT\]-based) assay.
Interventions
- GENETIC AAV5-hFIXco-Padua (AMT-061)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 3 participants |
| Start Date | 2018-07-24 |
| Est. Completion | 2023-09-21 |
| Phase | Phase 2 |
What the finished NCT03489291 record still lists
NCT03489291 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 3 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (98% lower).
The record links to 0 conditions, and to 1 intervention - of which AAV5-hFIXco-Padua (AMT-061) is the first listed.
NCT03489291 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03489291 about?
NCT03489291 is a clinical study titled "Dose Confirmation Trial of AAV5-hFIXco-Padua". This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase. The IMP AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized ...
What is the current status of trial NCT03489291?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 3 participants. The study started on 2018-07-24. Estimated completion is 2023-09-21.
What interventions are being tested in trial NCT03489291?
The interventions under investigation include: AAV5-hFIXco-Padua (AMT-061) (GENETIC).
Who is sponsoring clinical trial NCT03489291?
This trial is sponsored by CSL Behring, which has 71 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03489291's enrollment target sits among peer trials
3 2001st of 2000 the lowest of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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
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