BridgeMD
Tests treatment safety and results for Transfusion Dependent Beta-Thalassemia
Official title A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]
ClinicalTrials.gov ID: NCT05762510
What this study is testing
What is GMCN-508B (LentiRed)?
GMCN-508B (LentiRed) is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for transfusion dependent beta-thalassemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced with LentiRed Lentivrial vector.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 5 to 35
You may be able to join if
- The subject himself/herself or one legal guardian/agent of the subject is required to fully understand the study and voluntarily sign a written...
- Ages 5 to 35, no gender limitation.
- The clinical diagnosis of TDT includes β0/β0, β+/β0, βE/β0 and β+/β+ genotypes. TDT was defined as severe anemia in patients with thalassemia (Hb...
- Karnofsky Level of Performance (KPS) score ≥70 in adult people and Lansky Level of Performance (LPS) score ≥70 in children people.
- people were determined to undergo autologous hematopoietic stem cell transplantation by the principle investigator.
You likely can't join if
- Hepatitis B virus (HBV) : HbsAg or HbcAb positive, nucleic acid test positive; Hepatitis C virus (HCV) : HCAb positive, nucleic acid test positive...
- A white blood cell (WBC) count \<3×10\^9/L and/or platelet count \<100×10\^9/L, splenectomy was performed before.
- Uncured bleeding abnormalities.
- Any previous or current malignancy, myeloproliferative disease, or immune deficiency disease.
- Immediate family member with a known or suspected Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer...
- Previous hematopoietic stem cell transplantation (HSCT).
See the full eligibility criteria
- The subject himself/herself or one legal guardian/agent of the subject is required to fully understand the study and voluntarily sign a written informed consent.
- Ages 5 to 35, no gender limitation.
- The clinical diagnosis of TDT includes β0/β0, β+/β0, βE/β0 and β+/β+ genotypes. TDT was defined as severe anemia in patients with thalassemia (Hb persistent \<70 g/L), regular RBC transfusion and standard iron removal...
- Karnofsky Level of Performance (KPS) score ≥70 in adult people and Lansky Level of Performance (LPS) score ≥70 in children people.
- people were determined to undergo autologous hematopoietic stem cell transplantation by the principle investigator.
- people must have been treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history.
- Hepatitis B virus (HBV) : HbsAg or HbcAb positive, nucleic acid test positive; Hepatitis C virus (HCV) : HCAb positive, nucleic acid test positive; Positive for Human immunodeficiency virus (HIV) antibody or Treponema...
- A white blood cell (WBC) count \<3×10\^9/L and/or platelet count \<100×10\^9/L, splenectomy was performed before.
- Uncured bleeding abnormalities.
- Any previous or current malignancy, myeloproliferative disease, or immune deficiency disease.
- Immediate family member with a known or suspected Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndromes, hereditary non-polyposis colorectal cancer syndromes and familial...
- Previous hematopoietic stem cell transplantation (HSCT).
- Advanced liver disease, defined as: 1) Baseline alanine aminotransferase (ALT) or direct bilirubin ≥3 normal upper limit (ULN), or 2) Liver biopsy demonstrating cirrhosis, any evidence of bridging fibrosis, or acute...
- Baseline estimated glomerular filtration rate (eGFR) \< 70 mL/min /1.73 m2, as determined using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation for ≥18 years of age, and Besides...
- Uncontrolled seizure disorder.
- Diffusion capacity of Carbon monoxide dispersion (DLco) \<50% of predicted (corrected for hemoglobin and or alveolar ventilation, as clinically indicated ).
- A cardiac T2\ \<20 ms by magnetic resonance imaging (MRI).
- Severe iron overload, which in the opinion of the physician is grounds for exclusion.
- Clinically significant pulmonary hypertension.
- Participation in another clinical study with an investigational drug within 30 days of screening.
- Failure to obtain appropriate informed consent.
- Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician or investigator.
- Contraindications to the conditioning regimen.
- Prior receipt of genetic stem cell therapy.
- Diagnosis of significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study.
- Pregnancy or breastfeeding in a postpartum female or absence of adequate contraception for fertile people. Females of child-bearing potential are required to use effective contraception from the screening period until...
- Live vaccines were administered within 6 weeks prior to screening.
- Known history of hypersensitivity to the ingredients used in the trial.
- An assessment by the investigator that the subject would not comply with the study procedures outlined in the protocol.
The study team makes the final eligibility decision.
Where it's taking place
- Nanning, Guangxi, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 5 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Nanning, Guangxi, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.