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Gene Therapy

Gene Therapy for Hemophilia A

Phase 1

Recruiting

Led By Mary Eapen, MD

Research Sponsored by Parameswaran Hari

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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 (≥0.6 BU)

Adequate renal function, defined as creatinine clearance>60 ml/min (Cockroft-Gault formula)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through study completion, an average of 4 years

Awards & highlights

Study Summary

This trial is testing a new gene therapy for hemophilia A. A virus is used to introduce a gene that creates a protein, Factor VIII, which helps the blood to clot. This protein is normally lacking in people with hemophilia A.

Who is the study for?

Adult males over 18 with severe hemophilia A and a history of FVIII inhibitors. They must have had multiple bleeding episodes, adequate bone marrow reserve, renal and liver function, and be willing to document their condition for 15 years. Excluded are those recently in other trials or with certain medical conditions.Check my eligibility

What is being tested?

This Phase I gene therapy trial is testing if genetically modified stem cells can produce platelets that create Factor VIII on their own in patients with severe hemophilia A. Patients' stem cells will be altered using a viral vector and then returned to them.See study design

What are the potential side effects?

Potential side effects may include reactions related to the immune system's response to the viral vector or complications from the genetic modification process. Specific side effects are not listed but monitoring for any adverse events is part of the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am a man over 18 with severe hemophilia A and have or had inhibitors.

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My kidneys work well, with a creatinine clearance over 60 ml/min.

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I have been diagnosed with severe hemophilia A.

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My blood tests show enough white cells, hemoglobin, and platelets.

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I've needed treatment for bleeding more than four times in the last six months.

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My liver functions within normal limits, without signs of severe disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through study completion, an average of 4 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through study completion, an average of 4 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and through study completion, an average of 4 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of enrolled participants with adequate gene transduced hematopoietic stem cells for FVIII gene therapy infusion

Secondary outcome measures

Incidence of toxicity from gene therapy

Trial Design

1Treatment groups

Experimental Treatment

Group I: Autologous CD34+PBSC transduced with a lentiviral vectorExperimental Treatment1 Intervention

Patients will receive a patient specific (autologous) cytokine mobilized CD34+Peripheral Blood Stem Cells (PBSC) transduced ex vivo with a lentiviral vector containing cDNA encoding the human B-domain deleted FVIII protein.

0 / 6Eligibility criteria met

Find a Location

Who is running the clinical trial?

Parameswaran HariLead Sponsor

1 Previous Clinical Trials

38 Total Patients Enrolled

Medical College of WisconsinLead Sponsor

610 Previous Clinical Trials

1,162,708 Total Patients Enrolled

Mary Eapen, MDPrincipal InvestigatorFroedtert Hosptial and Medical College of Wisconsin

1 Previous Clinical Trials

138 Total Patients Enrolled

Media Library

Hemophilia A Research Study Groups: Autologous CD34+PBSC transduced with a lentiviral vector

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has there been precedent for using a lentiviral vector to modify Auto CD34+PBSC with a truncated version of human coagulation factor VIII?

"Presently, 4 studies are analyzing Auto CD34+PBSC transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII; two trials have entered Phase 3. Although many of these trial sites are based in Lanzhou, Gansu Province, 26 medical centres around the world offer testing for this particular intervention."

Answered by AI

Is there space available for participants to join this experiment?

"Affirmative. Clinicaltrials.gov details that this research is currently recruiting, with initial posting on April 29th 2020 and most recent edits occurring October 18th 2021. 5 participants are expected to be registered from a single site."

Answered by AI

How is Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII usually utilized?

"Auto CD34+PBSC, modified with a lentiviral vector encoding the B domain deleted from human coagulation factor VIII, is capable of treating hemophilia A and preventing excessive bleeding during or after surgery related to von Willebrand disease as well as hemorrhagic episodes."

Answered by AI

To what extent can individuals expect security when utilizing Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII?

"According to our team's assessment, the safety rating of Auto CD34+PBSC transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII is 1. This evaluation was based on limited clinical data supporting efficacy and safety for this Phase 1 trial."

Answered by AI

To what extent is the current population being incorporated into this research?

"Affirmative. Information available on clinicaltrials.gov suggests that this research project is actively enrolling patients and has been since April 29th 2020. The study seeks five volunteers from a single medical site, with the data most recently updated on October 18 2021."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

Mary Eapen MBBS MS 2020. "Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03818763.

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