What is LentiRed studying for transfusion-dependent beta-thalassemia?

LentiRed is being studied to evaluate its safety and effectiveness as a gene therapy for people with transfusion-dependent beta-thalassemia. Researchers want to see if it can reduce or stop the need for regular blood transfusions by modifying the patient's own stem cells. This study is sponsored by the First Affiliated Hospital of Guangxi Medical University.

Who may be eligible for this transfusion-dependent beta-thalassemia clinical trial?

People aged 5 to 35 years with a confirmed diagnosis of transfusion-dependent beta-thalassemia may be eligible. Both males and females can participate. Key factors include having severe anemia requiring regular transfusions, no prior stem cell transplant, and no active infections like hepatitis or HIV. Please review eligibility criteria for more details.

Is there any cost to participate in this transfusion-dependent beta-thalassemia clinical trial?

Study-related care, including the gene therapy and all trial visits, is provided at no cost. Personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly for any additional costs.

Actively Recruiting

Early Phase 1

Age: 5Years - 35Years

All Genders

ID05762510

Study of Gene Therapy Using LentiRed Stem Cells for Treating Transfusion-Dependent Beta-Thalassemia in Patients Aged 5 to 35

Led by First Affiliated Hospital of Guangxi Medical University · Updated on 2023-05-09

Participants Needed

Research Sites

134 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and effectiveness of a gene therapy called LentiRed using autologous CD34 human hematopoietic stem cells for people with transfusion-dependent beta-thalassemia TDT. This open-label, single-dose study focuses on patients aged 5 to 35 years who have severe anemia requiring regular blood transfusions and standard iron removal therapy. The study is early phase 1 and sponsored by the First Affiliated Hospital of Guangxi Medical University. Participants receive the LentiRed drug product through an intravenous infusion after undergoing myeloablative conditioning with busulfan. The study involves only one treatment group receiving this gene therapy. The treatment aims to reduce or eliminate the need for red blood cell transfusions by modifying the patients own stem cells. Participants will be followed for 5 years with regular monitoring to assess transfusion independence, hemoglobin levels, therapeutic globin expression, and any side effects or adverse events. Blood and bone marrow samples will be collected to measure the presence and activity of the modified cells. Researchers will also evaluate survival rates and other safety outcomes. The primary outcomes focus on transfusion independence and reduction in transfusion needs over 24 months post-infusion.

CONDITIONS

Brief Title

A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]

Who Can Participate

Age: 5Years - 35Years

All Genders

Eligibility Criteria

You may qualify if you...

The subject or legal guardian fully understands the study and voluntarily signs informed consent.
Ages 5 to 35 years, any gender.
Clinical diagnosis of transfusion-dependent beta-thalassemia with specified genotypes.
Severe anemia with hemoglobin persistently below 70 g/L, requiring regular red blood cell transfusions and iron removal therapy.
Karnofsky or Lansky performance score of 70 or higher.
Determined to undergo autologous hematopoietic stem cell transplantation by the principal investigator.
Treated and followed for at least 2 years in a specialized center with detailed medical records including transfusion history.

You will not qualify if you...

Positive for hepatitis B, hepatitis C, HIV, syphilis, or tuberculosis infection.
White blood cell count below 3 x 10^9/L or platelet count below 100 x 10^9/L; previous splenectomy.
Uncured bleeding disorders.
Current or past malignancy, myeloproliferative disease, or immune deficiency.
Immediate family member with known or suspected familial cancer syndrome.
Previous hematopoietic stem cell transplantation.
Advanced liver disease or significant liver abnormalities.
Estimated glomerular filtration rate below 70 mL/min/1.73 m2.
Uncontrolled seizure disorder.
Lung diffusion capacity below 50% predicted.
Cardiac T2* value below 20 ms by MRI.
Severe iron overload as determined by physician.
Clinically significant pulmonary hypertension.
Participation in another investigational drug study within 30 days.
Failure to obtain informed consent.
Any condition making the subject ineligible for stem cell transplantation.
Contraindications to conditioning regimen.
Prior genetic stem cell therapy.
Significant psychiatric disorder impeding participation.
Pregnancy, breastfeeding, or inadequate contraception.
Live vaccines within 6 weeks before screening.
Known hypersensitivity to trial ingredients.
Assessed inability to comply with study procedures.

Research Team

Yongrong Lai, PhD

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