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Gene Therapy

Gene Therapy for Thalassemia

Q: Is enrollment for this study still taking place?

<p>Evidently, this trial's last update was on August 25th 2022 and it is no longer recruiting participants. However, there are 59 other trials that offer potential enrolment opportunities for interested patients.</p>

Q: Has the FDA approved Autologous CD34+ cells transduced with TNS9.3.55 for therapeutic use?

<p>Considering limited data surrounding the safety and efficacy of Autologous CD34+ cells transduced with TNS9.3.55, our team at Power has rated its perceived risk as a 1 on their scale from 1 to 3.</p>

Phase 1

Waitlist Available

Led By Farid Boulad, MD

Research Sponsored by Memorial Sloan Kettering Cancer Center

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)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

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

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

Primary outcome measures

safety

tolerability

Secondary outcome measures

The frequency of post transplant palliative transfusions

the level of engraftment

Side effects data

From 2021 Phase 2 trial • 22 Patients • NCT01203020

41%

Death

100%

80%

60%

40%

20%

Study treatment Arm

Allogeneic Hematopoietic Progenitor Cell Transplant

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 / 7Eligibility criteria met

Find a Location

Who is running the clinical trial?

Memorial Sloan Kettering Cancer CenterLead Sponsor

1,933 Previous Clinical Trials

585,610 Total Patients Enrolled

Farid Boulad, MDPrincipal InvestigatorMemorial Sloan Kettering Cancer Center

8 Previous Clinical Trials

213 Total Patients Enrolled

Andromachi Scaradavou, MDPrincipal InvestigatorMemorial Sloan Kettering Cancer Center

Media Library

Autologous CD34+ cells transduced with TNS9.3.55 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT01639690 — Phase 1

Beta Thalassemia Research Study Groups: Autologous CD34+ cells transduced with TNS9.3.55

Beta Thalassemia Clinical Trial 2023: Autologous CD34+ cells transduced with TNS9.3.55 Highlights & Side Effects. Trial Name: NCT01639690 — Phase 1

Autologous CD34+ cells transduced with TNS9.3.55 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01639690 — Phase 1

Frequently Asked Questions

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

Is enrollment for this study still taking place?

"Evidently, this trial's last update was on August 25th 2022 and it is no longer recruiting participants. However, there are 59 other trials that offer potential enrolment opportunities for interested patients."

Answered by AI

Has the FDA approved Autologous CD34+ cells transduced with TNS9.3.55 for therapeutic use?

"Considering limited data surrounding the safety and efficacy of Autologous CD34+ cells transduced with TNS9.3.55, our team at Power has rated its perceived risk as a 1 on their scale from 1 to 3."

Answered by AI