Hemophilia B Gene Therapy With AAV8 Vector
Public ClinicalTrials.gov record NCT01620801. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1 Safety Study in Subjects With Severe Hemophilia B (Factor IX Deficiency) Using a Single-Stranded, Adeno-Associated Pseudotype 8 Viral Vector to Deliver the Gene for Human Factor IX
Study identification
- NCT ID
- NCT01620801
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Enrollment
- 4 participants
Conditions and interventions
Conditions
Interventions
- AAV8-hFIX19 Biological
Biological
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 30, 2012
- Primary completion
- Feb 29, 2016
- Completion
- Feb 29, 2016
- Last update posted
- Mar 11, 2019
2012 – 2016
United States locations
- U.S. sites
- 2
- U.S. states
- 1
- U.S. cities
- 2
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Pittsburgh | Pittsburgh | Pennsylvania | 15213 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01620801, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 11, 2019 · Synced Jul 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01620801 live on ClinicalTrials.gov.