Actively Recruiting
An Open Label Study Evaluating the Safety and Efficacy of Gene Therapy for Transfusion-dependent Beta-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a LentiRed Lentiviral Vector (GMCN-508B Drug Product, Also Called LentiRed)
Led by First Affiliated Hospital of Guangxi Medical University · Updated on 2023-05-09
5
Participants Needed
1
Research Sites
134 weeks
Total Duration
On this page
Sponsors
F
First Affiliated Hospital of Guangxi Medical University
Lead Sponsor
G
Genmedicn Biopharma Ltd.
Collaborating Sponsor
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
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.
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single treatment period with follow-up up to 24 months
Participants receive a myeloablative conditioning with busulfan followed by intravenous infusion of the LentiRed Drug Product.
Multiple visits during treatment and follow-up over 24 months
Duration - Up to 5 years
Participants are monitored for safety, efficacy, and long-term outcomes including transfusion independence and adverse events for up to 5 years after treatment.
Regular visits during the 5-year follow-up period
Trial Site Locations
Total: 1 location
1
The affiliated hospital of guangxi medical university
Nanning, Guangxi, China, 530021
Actively Recruiting
Research Team
Y
Yongrong Lai, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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