Active — Not RecruitingPhase 1

ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene

Italy10 participantsStarted 2012-07

Plain-language summary

The patient has inherited ß-thalassemia major through the genes. These genes have mistakes in them, so the body cannot make normal red blood cells. Stem cells are made in the bone marrow. They are the earliest form of blood cells. This study is being done to see if the investigators can make the stem cells produce normal red blood cells and hemoglobin. The investigators do this by collecting the stem cells. The genes with mistakes are removed from the cells. These cells are then treated so they have the corrected gene for making normal hemoglobin. These treated cells are given back to the patient through an injection (shot) in the vein. This is also known as gene transfer. In order for the body to accept these cells, the patient will need to receive a low dose of a drug called busulfan. It is a drug that will prepare the body to receive the new stem cells. This study will let the investigators know: * If it is safe to give the patient the treated stem cells * If the treated stem cells will go into the bone marrow without causing side effects. Gene transfer has been used for the past five years. It has been successful in treating many blood disorders. At least 20 patients have received the type of treatment that the patient will get on this study. This treatment for B-thalassemia major was developed at Memorial Sloan Kettering (MSK). It was studied for a long time in the lab before being given to patients.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Subjects must be 18 years or older * Subjects may be of either gender or of any ethnic background * Subjects must have a confirmed diagnosis of ß-thalassemia major and have been enrolled in a hypertransfusion program with a confirmed annual transfusion of ≥100 mL/kg/yr but \< 200 mL/kg/yr, AND ≥ 8 transfusions of blood per year over a minimum of two years. * Patients must NOT have an HLA-matched sibling * Patients must be off hydroxyurea (HU) or erythropoietin (EPO) treatment for at least three months prior to entry onto the study * Subjects must have a performance score of Karnofsky ≥70% at the time of entry into the study. * Subjects must have liver iron value of \< 15 mg/g/dry weight Iron quantitation may be performed by imaging such as T2\*MRI or by biopsy * Subjects must have no evidence of cirrhosis\*\* of the liver. Fibrosis of the liver can be tested by Fibroscan (47, 48, 49), or by liver biopsy. These should be performed within approximately a one year period prior to entry onto the study. * Subjects with an evaluation of cardiac function indicating: * normal function on MUGA scan (Multiple Gated Acquisition scan) or other methodology. And * Patients must have a left ventricular ejection fraction (LVEF) of ≥ 60% and/or T2\*MRI cardiac evaluation with T2\* ≥20 milliseconds * Subjects with asymptomatic pulmonary function based on Lung Diffusion Testing DLCO Test DLCO ≥ 50% of predicted (corrected for hemoglobin) * Subjects with a determination …

What they're measuring

safety

Timeframe: 2 years

tolerability

Timeframe: 2 years

Trial details

NCT IDNCT01639690

SponsorSan Rocco Therapeutics

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-07