The clinical trial NCT06971094 is a Phase 3 study evaluating the efficacy and safety of sibeprenlimab, an investigational anti-APRIL monoclonal antibody, for treating immunoglobulin A nephropathy (IgAN). This randomized, double-blind, placebo-controlled trial involves approximately 530 adult participants receiving standard-of-care therapy. Interim results indicate that sibeprenlimab significantly reduces proteinuria, suggesting potential as a disease-modifying treatment. The study continues to assess long-term kidney function outcomes, with final results expected in early 2026. These findings could pave the way for accelerated regulatory approval, offering hope for patients with this progressive kidney disease.
The purpose of this trial is to evaluate safety and efficacy of gene therapy drug GC101 in SMA 3 patients. Open-label, dose-escalation clinical trials of GC101 will be conducted in multiple centers in China.
GC101 will be administrated intrathecally. Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will. Patients will be tested at baseline and followed up at various time points.
The primary analysis for efficacy will be assessed at 12 months after treatment with GC101 on the changes from baseline HFMSE (Hammersmith Functional Motor Scale Expanded) and RULM（Revised Upper Limb Module) scores for patients of age ≥ 6 years old.
The purpose of this trial is to evaluate safety and efficacy of gene therapy drug GC101 in SMA 2 patients. Open-label, dose-escalation clinical trial of GC101 will be conducted in multiple centers in China.
GC101 will be administrated intrathecally. Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will. Patients will be tested at baseline and followed up on various time points.
The primary analysis for efficacy will be assessed at 12 months after treatment with GC101 on the motor milestone of stand unassisted for at least 3 seconds for patients of age between 6 and 24 months, or changes from baseline HFMSE scores for patients of age between 24 and 60 months.
The purpose of this trial is to evaluate safety and efficacy of gene therapy drug GC101 in SMA 1 patients. Open-label, dose-escalation clinical trial of GC101 will be conducted in multiple centers in China.
GC101 will be administrated intrathecally. Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will. Patients will be tested at baseline and followed up on various time points.
The primary analysis for efficacy will be assessed when all patients reach 18 months of age on the motor milestone of sit unassisted for at least 10 seconds.
FT-003 is a gene therapy product developed for the treatment of central involvement diabetic macular edema (CI-DME). Diabetic retinopathy is one of the most common microvascular complications of diabetes mellitus, and diabetic macular edema is the main cause of vision loss in patients with diabetic retinopathy. In the latest guidelines, anti-VEGF therapy is preferred for CI-DME.
Administration of FT-003 has the potential to treat CI-DME by providing intraocular protein that is durable and expressed at therapeutic levels. FT-003 is designed to reduce the current treatment burden, which often results in undertreatment and vision loss in patients with CI-DME receiving anti-VEGF therapy in clinical practice.
FT-003 is a gene therapy product developed for the treatment of neovascular age-related macular degeneration (nAMD). Neovascular AMD is the main cause of blindness among elderly individuals. The available therapies for treating nAMD require life-long intravitreal (IVT) injections every 4-12 weeks to maintain efficacy.
Administration of FT-003 has the potential to treat nAMD by providing durable expression of therapeutic levels of intraocular protein and maintaining the vision of patients. FT-003 is designed to reduce the current treatment burden which often results in undertreatment and vision loss in patients with nAMD receiving anti-VEGF therapy in clinical practice.
An Open-label, Single-center, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intraocular Administration of FT-002 in Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa.
The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.
This study is a multi-center, open-label, phase I/II clinical study to evaluate the safety, tolerability, efficacy, immunogenicity, and in vivo biodistribution characteristics of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy. Assessments will include visual acuity, vector shedding, immunogenicity and adverse events. Participants will be monitored for 5 years after treatment.
The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation phase) and Phase II (dose expansion phase).
Exploring the clinical significance of hsa_circ_0001642, hsa-miR-193a-5p, and SPHK1 for colorectal cancer diagnosis and treatment.
Inclusion Criteria:
1) Age >=18 years, confirmed diagnosis of colorectal adenocarcinoma (based on postoperative pathology or biopsy pathology), clinical stage I-IV;
2) Prospective study (2025.01-2027.12) requires signed informed consent and voluntary provision of tumor tissue, paracancerous tissue and peripheral blood samples;
3) Retrospective cohort (2019.01-2023.12) requires complete clinical data and follow-up records (including date of diagnosis, treatment regimen, survival status, etc.);
4) Have not received radiotherapy or targeted therapy (preoperative neoadjuvant therapy patients can be included);
5) Can cooperate to complete long-term follow-up (for retrospective analysis).