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NCT03569891 · ClinicalTrials.gov registry record · Phase 3
HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
A Phase 3 study, sponsored by CSL Behring.
- Completed
- Registry status
- Phase 3
- Development phase
- 67
- Enrollment target
NCT03569891: Completed Phase 3 study, sponsored by CSL Behring.
NCT03569891 is a Phase 3 study that has completed, run by CSL Behring. The registered enrollment target is 67 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (91% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03569891, a Phase 3 study, has completed, sponsored by CSL Behring.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 67 participants
- Enrollment target
Study Summary
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). 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 pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10\^13 gc/kg.
Primary Outcome
Adjusted ABR for Lead-in and Post-Treatment period was estimated from a repeated measures generalized estimating equations negative binomial regression model accounting for the paired design of the trial with an offset parameter to account for the differential collection periods. Treatment period was included as a categorical covariate.
Interventions
- BIOLOGICAL Factor IX (FIX)
- GENETIC AAV5-hFIXco-Padua
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 67 participants |
| Start Date | 2018-06-27 |
| Est. Completion | 2025-03-19 |
| Phase | Phase 3 |
What the finished NCT03569891 record still lists
NCT03569891 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 67 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (91% lower).
The record links to 0 conditions, and to 2 interventions - of which Factor IX (FIX) is the first listed.
NCT03569891 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03569891 about?
NCT03569891 is a clinical study titled "HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients". This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containi...
What is the current status of trial NCT03569891?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 67 participants. The study started on 2018-06-27. Estimated completion is 2025-03-19.
What interventions are being tested in trial NCT03569891?
The interventions under investigation include: Factor IX (FIX) (BIOLOGICAL), AAV5-hFIXco-Padua (GENETIC).
Who is sponsoring clinical trial NCT03569891?
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 NCT03569891's enrollment target sits among peer trials
67 1796th of 2000 higher than 204 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 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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