Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A

  • End date
    May 1, 2033
  • participants needed
  • sponsor
    Medical College of Wisconsin
Updated on 31 October 2021
renal function
total bilirubin
coagulation factor
severe haemophilia a
antihemophilic factor
factor viii inhibitor


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.


This is an open label, nonrandomized, single center, phase I cohort study, involving reduced intensity conditioning, followed by a single infusion of autologous CD34+PBSC, transduced with a lentiviral vector (-889ITGA2B-BDDFVIII-WPTS (MUT6)(VSVg)) also known as (Pleightlet(MUT6)) encoding the B domain deleted from of human coagulation factor VIII (BDDFVIII) in up to five hemophilia A patients with a history of FVIII inhibitors (0.6BU) .

Condition Hemophilia A, Hemophilia, hema
Treatment Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII
Clinical Study IdentifierNCT03818763
SponsorMedical College of Wisconsin
Last Modified on31 October 2021


Yes No Not Sure

Inclusion Criteria

Study population will include: adult males >18 years of age with a diagnosis
of severe hemophilia A and currently active or a history of FVIII inhibitors
Subject may be prescribed prophylactic therapy with factor VIII bypassing agents or factor VIII mimetics prior to referral for inclusion in the study
(0.6 BU). Females will be excluded because hemophilia A is an X-linked
Subjects who are treated on demand using factor VIII bypassing agents must have a history of four or more bleeding episodes requiring treatment in the six-month period prior to referral for inclusion in the study
disorder that is extremely rare in females
Confirmed diagnosis of severe hemophilia A by undetectable plasma factor VIII:C by a one-stage PTT-based assay and coatest chromogenic factor VIII assay. Subjects with currently active or a history of positive FVIII inhibitor titers (0.6 BU) irrespective of their titer or current inhibitor status will be included for enrollment
Adequate bone marrow reserve as demonstrated by ANC >1.5/; Hemoglobin >9g/dL; Platelets >100,000/microliter
Adequate renal function, defined as creatinine clearance>60 ml/min (Cockroft-Gault formula)
Adequate liver function, defined as defined as total bilirubin 1.5 times the upper limit of normal (ULN) (excluding Gilbert's syndrome), both AST and ALT 3 times ULN at the time of screening, and no clinical signs or known laboratory/radiographic evidence consistent with cirrhosis
Subject must sign an informed consent after explanation of the study and having questions answered
Subject must be willing and able to document type of bleeding episodes and treatment in a paper or electronic diary during the study
Subject must be willing to return for regular follow-up visits during the 15-year study

Exclusion Criteria

A potential subject who meets any of the following exclusion criteria is ineligible to participate in the study
Therapy with factor VIII with the intent of immune tolerance induction within 30 days prior to inclusion within the study
Enrollment in another interventional clinical trial within 60 days prior to study inclusion
Medical contraindication to PBSC cytokine mobilization, use of GCSF, PBSC apheresis procedure or conditioning regimen
Medically significant organ dysfunction that would prevent compliance with conditioning or would severely limit the probability of survival based on clinical status
Those with a known co-existing clinically significant thrombophilic disorder, or as determined by the presence of any of the below identified on screening laboratory assessments
FV Leiden
Protein S deficiency
Protein C deficiency
Prothrombin mutation (G20210A)
D-dimer >3 x the upper limit of normal (ULN) at Screening All known patients with the above and any patient with a personal or significant family history of thrombotic events (DVT, PE, arterial clots) as deemed by the principal investigator will be screened for the above disorders. 6. Active invasive malignancy (Non-melanoma skin cancers and carcinoma in situ are not excluded). 7. Known bone marrow disorders or abnormal bone marrow cytogenetics. 8. Fertile males who are unwilling to use contraceptive techniques during and for the twelve months following treatment. 9. Life expectancy severely limited by disease(s) other than hemophilia A. 10. Patients with HIV, hepatitis B, hepatitis C (with an AST/ALT > 3 times the upper limit of normal). 11. Other active infectious disease that is a contraindicat ion for immunosuppressive therapy. 12. Patients who have elective surgery scheduled during the study period
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact



Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider


Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 



Reply by • Private

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note