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Gene Therapy

Gene Therapy for Thalassemia

Phase 1

Waitlist Available

Led By Farid Boulad, MD

Research Sponsored by San Rocco Therapeutics

Eligibility Criteria Checklist

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

Must have

Subjects with a determination of renal function based on: serum creatinine < than or = to 1.5 mg/dL or if serum creatinine is outside the normal range, then CrCl > 60-ml/min/1.73 m2

Subjects must have adequate hepatic function based on: < 3 x ULN ALT and < 2.0 total serum bilirubin (unless secondary to hemolysis)

Must not have

Active infections including Hepatitis B and Hepatitis C

Active infections including HTLV 1 and 2, and HIV 1 and 2

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing whether it is safe to give patients with thalassemia major treated stem cells that have had the genes with mistakes removed and have been given the corrected gene for making normal hemoglobin.

Who is the study for?

This trial is for adults with ß-thalassemia major who are part of a hypertransfusion program and have no HLA-matched sibling donor. They should not have severe liver or heart issues, active infections like Hepatitis B/C or HIV, diabetes, uncontrolled seizures, or be pregnant/breastfeeding. Adequate organ function and off certain treatments for three months prior to the study are required.

What is being tested?

The trial tests if stem cells with corrected genes can safely produce normal red blood cells in patients with ß-thalassemia major. Patients' own stem cells will be modified to correct the genetic defect and then re-injected after pre-treatment with low-dose busulfan to help their body accept the new cells.

What are the potential side effects?

Potential side effects may include reactions related to gene transfer such as immune responses against the modified cells, complications from bone marrow suppression due to busulfan pre-treatment, and typical risks associated with stem cell transplantation.

Eligibility Criteria

Inclusion Criteria

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

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My kidney function is normal, based on my creatinine levels or clearance rate.

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My liver functions are within the required range.

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I am 18 years old or older.

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I have ß-thalassemia major and get regular blood transfusions.

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I do not have a sibling who is an exact genetic match to me.

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I am mostly able to care for myself and carry out normal activities.

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My heart pumps well and my heart MRI results are normal.

Exclusion Criteria

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

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I do not have active infections like Hepatitis B or C.

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I do not have active infections like HTLV or HIV.

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I am currently pregnant or breastfeeding.

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I have been treated for HIV or HTLV.

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My bone marrow has abnormal cells or chromosomes.

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I have diabetes.

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I have seizures that are not controlled by medication.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 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

safety

tolerability

Secondary study objectives

The frequency of post transplant palliative transfusions

the level of engraftment

Awards & Highlights

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+ cells transduced with TNS9.3.55Experimental Treatment1 Intervention

An open label study using a non-myeloablative conditioning regimen of busulfan and 1 or several infusions of autologous hematopoietic stem cells transduced with a lentiviral vector encoding the human ß-globin gene.

0 / 14Eligibility criteria met