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New treatment option for Hemophilia B
Official title Lentiviral FIX Gene Therapy
ClinicalTrials.gov ID: NCT03961243
What this study is testing
What is YUVA-GT-F901?
YUVA-GT-F901 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for hemophilia b.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor IX into patients with hemophilia B, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 65
You may be able to join if
- 1\. Able to provide informed consent and comply with requirements of the study. 2. Males ≥2 years with confirmed diagnosis of hemophilia B...
- 3\. A minimum average of 4 bleeding events per year requiring episodic treatment of factor IX infusions or prophylactic factor IX infusions.
- 4\. No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein.
- 5\. Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences.
You likely can't join if
- 1\. Significant liver dysfunction as defined by abnormal alanine transaminase, bilirubin and alkaline phosphatase.
- 2\. History of inhibitor against factor IX. 3. Evidence of active hepatitis B or C and currently on antiviral therapy. 4. Have serological evidence...
- 5\. Any evidence of active infection or any immunosuppressive disorder. 6. Participated in a gene transfer trial within the last 6 months or in a...
- 7\. Unable or unwilling to comply with study assessments.
The study team makes the final eligibility decision.
Where it's taking place
- Shenzhen, Guangdong, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shenzhen, Guangdong, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.