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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)

Must not have

Other active infectious disease that is a contraindication for immunosuppressive therapy

Life expectancy severely limited by disease(s) other than hemophilia A

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through study completion, an average of 4 years

Awards & highlights

All Individual Drugs Already Approved

No Placebo-Only Group

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have any active infections that would prevent me from receiving treatments that weaken my immune system.

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My life expectancy is not majorly shortened by conditions other than hemophilia A.

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I have a planned surgery during the study period.

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I am a man who can father children and agree to use birth control for a year after treatment.

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I cannot undergo certain treatments due to health risks.

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I have a blood clotting disorder.

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My organs work well enough for me to undergo treatment.

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I have a known bone marrow disorder or abnormal bone marrow genetics.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Secondary study objectives

Incidence of toxicity from gene therapy

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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 / 14Eligibility criteria met