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NCT03818763 · ClinicalTrials.gov registry record · Phase 1
Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A
A Phase 1 study of Hemophilia A, sponsored by Medical College of Wisconsin.
- Active
- Registry status
- Phase 1
- Development phase
- 5
- Enrollment target
- 1
- Study location
NCT03818763 is a Phase 1 study of Hemophilia A that is active but no longer recruiting, run by Medical College of Wisconsin. The registered enrollment target is 5 participants, below the 91-participant average among 42 other Hemophilia A trials with a reported enrollment target (95% lower). The trial reports 1 study location across 1 state.
The verdict
NCT03818763, a Phase 1 study of Hemophilia A, is active but no longer recruiting, sponsored by Medical College of Wisconsin.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 5 participants
- Enrollment target
- 1
- Study location
Study Summary
This is a Phase I study. This research study is being conducted to find new ways to treat severe hemophilia A. This study is a gene therapy study. Gene therapy is an experimental way to introduce, into a person's cells, specific genetic material. A gene can be delivered/introduced into a cell using a carrier known as a "vector." In this study, a virus (lentivirus), the "vector", is used to introduce or deliver a gene that creates and stores a protein Factor VIII (FVIII) in your platelets. These platelets are made from stem cells (mother cells for your bone marrow) that are removed from your blood by a procedure called apheresis. This research study will take some of the patient's own stem cells, from the apheresis procedure, and genetically modify them using the vector in order to make them produce FVIII in platelets that arise from the stem cells. They will then give the genetically modified stem cells back to the patient so that they can possibly create platelets that produce and store Factor VIII on their own.
Conditions Studied
Interventions
- BIOLOGICAL Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII
Study Locations (1)
Wisconsin
- Froedtert Hospital and the Medical College of Wisconsin - Milwaukee
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2020-04-29 |
| Est. Completion | 2033-05-01 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03818763
The ClinicalTrials.gov registry entry for NCT03818763 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 5 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 91-participant average among 42 other Hemophilia A trials with a reported enrollment target (95% lower). The listed sponsor is Medical College of Wisconsin, which has 447 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Hemophilia A appearing as the primary indexed condition, and to 1 intervention - of which Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII is the first listed.
NCT03818763 reports 1 study location spanning 1 distinct geographic area - top geographies include Wisconsin.
Frequently Asked Questions
What is clinical trial NCT03818763 about?
NCT03818763 is a clinical study titled "Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A". This is a Phase I study. This research study is being conducted to find new ways to treat severe hemophilia A. This study is a gene therapy study. Gene therapy is an experimental way to introduce, into a person's cells, specific genetic material. A gene can be delivered/introduced into a cell using ...
What is the current status of trial NCT03818763?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2020-04-29. Estimated completion is 2033-05-01.
What conditions does trial NCT03818763 study?
This clinical trial studies the following conditions: Hemophilia A.
What interventions are being tested in trial NCT03818763?
The interventions under investigation include: Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII (BIOLOGICAL).
Who is sponsoring clinical trial NCT03818763?
This trial is sponsored by Medical College of Wisconsin, which has 447 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03818763 being conducted?
This trial has 1 study location across Wisconsin. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Related
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