Clinical Research Directory

A Prospective, Single-center, Open-label Study of Inhaled BMD003 (CFTR mRNA) in Chinese Adults With Cystic Fibrosis

The study uses a prospective, single-center, open-label clinical trial design to evaluate the safety and efficacy of inhaled BMD003 (CFTR mRNA) in adult Chinese patients with cystic fibrosis. This is a single-dose escalation study of nebulized inhaled BMD003 in patients with cystic fibrosis. The trial adopts sequential dose-escalation cohorts with flexible subject enrollment. Continuous safety monitoring will be performed throughout the study. If treatment-related adverse events meeting specified grade criteria occur during the trial, additional subjects will be enrolled at the current dose level for further safety observation. Subsequent dose escalation will proceed following a comprehensive safety evaluation of the prior cohort. The trial consists of three phases: a 2-week screening period, a 1-day drug administration period, and a long-term follow-up period of approximately 13 months. After successful screening and meeting eligibility criteria, subjects will be admitted on Day 1 to receive nebulized inhalation of the investigational product. Comprehensive safety observations will be completed on Day 2 prior to hospital discharge. Pharmacokinetic sampling and systematic safety assessments will be conducted at predefined study visits. All subjects will complete protocol-specified follow-up procedures after single-dose administration. Upon completion of this single-dose escalation phase, a multiple-dose study of BMD003 will be initiated. The detailed multiple-dose regimen will be defined based on preclinical findings and clinical data from this single-dose study, and implemented after ethical review and approval.

A Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma

The goal of this clinical trial is to evaluate the safety, tolerability, and recommended Phase 2 Dose (RP2D) of KLN-1010 in patients with relapsed or refractory multiple myeloma.

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector. Subjects will be enrolled over 3 years and be followed for 2 years post-infusion on this protocol, then followed long-term on a separate long-term follow-up protocol. Enrollment of subjects will be agreed upon by representatives of both sites. Data will be collected uniformly from both sites through an electronic capture system and key laboratory studies will be centralized. Harvest, cellular manufacturing and infusion will occur at each site using the same SOPs. Key aspects of cellular product characterization will be centralized

CIBMTR Research Database

The primary purpose of the Research Database is to have a comprehensive source of observational data that can be used to study HSC transplantation and cellular therapies. A secondary purpose of the Research Database is to have a comprehensive source of data to study marrow toxic injuries. Objectives: To learn more about what makes stem cell transplants and cellular therapies work well such as: * Determine how well recipients recover from their transplants or cellular therapy; * Determine how recovery after a transplant or cellular therapy can be improved; * Determine how a donor's or recipient's genetics impact recipient recovery after a transplant or cellular therapy; * Determine how access to transplant or cellular therapy for different groups of patients can be improved; * Determine how well donors recover from the collection procedures.

Gene Therapy for Chinese Hemophilia A

IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with \<1 IU/dl residual FVIII levels.

Perceptual Evaluation and Rehabilitation System Development for Congenital Hearing Loss

This study aims to establish a novel multimodal evaluation and precision rehabilitation system for patients with congenital hearing loss who have received hearing aids, cochlear implantation, or emerging gene therapy. The proposed system will integrate age-stratified difficulty levels, combined with a gamified interaction platform or software designed to assess and train three core perceptual domains: auditory speech perception, music perception, and spatial hearing capabilities.

A Clinical Study of the Safety and Efficacy of Chemogenetics Therapy in the Treatment of Parkinson's Disease

The investigators propose a gene therapy strategy using chemical genetic inhibition to intervene in the abnormal activity of the subthalamic nucleus in Parkinson's disease. The investigators design and construct a highly efficient therapeutic injection STP-001 (first drug), through the efficient adeno-associated virus capsid (AAV), neuronal promoter (hSyn), and chemical genetic effector element (hM4Di), and accurately inject the drug into the bilateral subthalamic nucleus, the core pathological nucleus of Parkinson's disease, through stereotactic technology. Combined with a very low dose of clozapine (the second drug), the abnormal activity of the subthalamic nucleus is precisely intervened to improve the core motor symptoms of Parkinson's disease.

Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration. The goal of this clinical trial is to evaluate whether a gene therapy can safely treat children with SPG47, a rare genetic condition that causes progressive spasticity and developmental delays. The main questions it aims to answer are: * Is the gene therapy safe and well tolerated? * Does the gene therapy improve motor function and developmental outcomes? Participants will: * Undergo screening assessments to confirm eligibility * Receive a single dose of the gene therapy vector * Attend follow-up visits for safety monitoring and developmental assessments over the course of five years

Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT children with lifelong supportive therapy and healthy children.