AMT-061 is currently being studied in the Phase III HOPE-B Pivotal Study in Patients with Hemophilia B.
AMT-061 is an investigational gene therapy being studied in clinical trials by uniQure in the HOPE-B (Health Outcomes with Padua gene; Evaluation in Hemophilia-B) trial for adults with moderately severe or severe hemophilia. The goal of treatment with AMT-061 is to help the liver produce Factor IX in participants with hemophilia B without the complications associated with other hemophilia treatments. If the trial is successful, the study data will be used to seek approval of this new gene therapy for adults with hemophilia B.
- HOPE-B will evaluate whether AMT-061 can create enough active Factor IX to prevent bleeding episodes.
- HOPE-B will compare the effect of this gene therapy on bleeding episodes to Factor IX prophylaxis.
- Safety of AMT-061 will be assessed throughout the trial by monitoring adverse events, physical exams, and laboratory measures.
- One dose of AMT-061 is expected to provide increased Factor IX levels for many years, and we will learn more about the duration of its effect in HOPE-B.
- Participants in the HOPE-B trial will be monitored for other safety markers including:
- Reactions to the gene therapy infusion
- Increase in liver enzymes
- Bleeding events
- Inhibitor formation
- Lack of treatment benefit
You can learn more about the HOPE-B clinical trial on ClinicalTrials.gov (see link below).
If you are a patient interested in more information about the HOPE-B pivotal study of AMT-061 in hemophilia B, please contact us at uniQureHOPE-B@uniqure.com.
If you are a healthcare professional interested in more information on uniQure’s clinical trials, please visit our Clinical Trial Information page or contact us at MedInfo@uniqure.com.
> ClinicalTrials.gov: HOPE-B Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients