Peking University Third Hospital — Biomedical Watch V2

Traitements12programmes

Essais6liés

Publications9liées

SourceDBlocale

Traitements

12

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
anti-CD19 CAR T cell therapyAutoimmune diseases refer to a common category of diseases caused by the immune system reacting to self-antigens, leading to tissue damage. Autoimmune diseases encompass a wide variety of conditions, such as systemic lupus erythematosus（SLE）, Sjögren's syndrome (SS), systemic sclerosis (SSc), inflammatory myopathies (IM), ANCA-associated vasculitis (AAV), and antiphospholipid syndrome (APS). They affect the quality of life, while in severe cases, they can be life-threatening. Additionally, they impose a heavy economic burden on society. Current treatments for autoimmune diseases include glucocorticoid, immunosuppressants, and biologics. B cell-driven humoral immune abnormalities are a central pathogenic mechanism in many autoimmune diseases. When autoreactive B cells are excessively activated, they produce large amounts of autoantibodies and immune complexes. These antibodies and immune complexes can cause damage to various tissues and organs, leading to the development of multiple autoimmune diseases. Therefore, targeting B cells to treat autoimmune diseases is an attractive therapeutic strategy. Chimeric Antigen Receptor (CAR)-T cells targeting the B cell surface molecule CD19 have achieved significant clinical progress in acute lymphoblastic leukemia and B cell non-Hodgkin lymphoma, with several CD19 CAR-T therapies approved for marketing worldwide. Increasingly, clinical studies are exploring the use of CD19 CAR-T cells for the treatment of autoimmune diseases, and their therapeutic efficacy has been demonstrated. In this study, the investigators used γδ T cells as carrier cells to investigate the safety and efficacy of universal CAR-γδ T cells in the treatment of autoimmune diseases.	SEP	Phase 1/2	Immunomodulation + Thérapie cellulaire	China	À vérifier
BCT301This study primarily involves the use of BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells, for the treatment of patients with refractory autoimmune diseases, aiming to evaluate its safety, tolerability, and dose-limiting toxicities(DLT), and to determine the recommended therapeutic dose for further investigation. Additionally, the study assesses the efficacy of BCT301 cell injection in refractory autoimmune diseases, as well as the pharmacokinetic (PK) and pharmacodynamic (PD) characteristics in study participants.	Lupus	Phase 1	Immunomodulation + Thérapie cellulaire	China	À vérifier
IM19 CAR-T cellsThe study is a single-arm, open-label clinical trial intended to recruit 6 refractory SLE subjects who meet the inclusion and exclusion criteria. The subjects will receive IM19 CAR-T cell infusion therapy at a dose of 1×10\^6/kg or 1×10\^8 CAR-T cells . The primary endpoint is to evaluate the improvement in SLE disease activity (SLEDAI-2K) at 90 days and the occurrence of adverse events related to IM19 CAR-T cell infusion within 28 days post-infusion. Additionally, the long-term efficacy was evaluated, including the improvement of SLEDAI-2K score and achieving lupus low disease activity (LLDAS) at day 180 and day 360, and renal response at day 180 and day 360. The persistence and duration of IM19 CAR-T cells in the peripheral blood of subjects will also be evaluated. The study plan includes five phases: screening phase, cell collection phase, lymphodepletion pretreatment phase, cell infusion phase, and follow-up phase.	Lupus	Non applicable	Thérapie cellulaire + Immunomodulation	China	À vérifier
Natural History and Clinical Features of Amyotrophic Lateral Sclerosis (ALS)Description non affichée : incohérence de maladie détectée. Consultez l’essai clinique officiel associé.	Sclérose latérale amyotrophique	À vérifier	À vérifier	China	À vérifier
Peripheral Neurofilament Levels and Amyotrophic Lateral SclerosisDescription non affichée : incohérence de maladie détectée. Consultez l’essai clinique officiel associé.	Sclérose latérale amyotrophique	À vérifier	Neuroprotection	China	À vérifier
Transcranial Magnetic StimulationThis study is a randomized, crossover, controlled, double-blind clinical trial. Patients (n=45) were randomly divided into Group A and Group B. Patients in Group A will receive 2 weeks (10800 Hz daily, 5 days×2) of Transcranial magnetic stimulation（TMS） treatment, while patients in Group B will receive sham stimulation with the same frequency. After a 4-week washout period, the two groups cross over. Patients in Group A will receive sham stimulation, and patients in Group B will receive TMS treatment for 2 weeks (10800 Hz daily, 5 days×2). Clinical functional scales, imaging evaluations, and gait analysis will be conducted at baseline, after 2 weeks of treatment, before crossover treatment, and after 2 weeks of crossover treatment. The therapist, patients, and assessors will be all blinded throughout the study.	Sclérose latérale amyotrophique	Phase 1	À vérifier		À vérifier
Natural History and Clinical Features of Amyotrophic Lateral Sclerosis (ALS)Description non affichée : incohérence de maladie détectée. Consultez l’essai clinique officiel associé.	Sclérose latérale amyotrophique	À vérifier	À vérifier	China	À vérifier
Peripheral Neurofilament Levels and Amyotrophic Lateral SclerosisDescription non affichée : incohérence de maladie détectée. Consultez l’essai clinique officiel associé.	Sclérose latérale amyotrophique	À vérifier	Neuroprotection	China	À vérifier
Transcranial Magnetic StimulationThis study is a randomized, crossover, controlled, double-blind clinical trial. Patients (n=45) were randomly divided into Group A and Group B. Patients in Group A will receive 2 weeks (10800 Hz daily, 5 days×2) of Transcranial magnetic stimulation（TMS） treatment, while patients in Group B will receive sham stimulation with the same frequency. After a 4-week washout period, the two groups cross over. Patients in Group A will receive sham stimulation, and patients in Group B will receive TMS treatment for 2 weeks (10800 Hz daily, 5 days×2). Clinical functional scales, imaging evaluations, and gait analysis will be conducted at baseline, after 2 weeks of treatment, before crossover treatment, and after 2 weeks of crossover treatment. The therapist, patients, and assessors will be all blinded throughout the study.	Sclérose latérale amyotrophique	Phase 1	À vérifier		À vérifier
IM19 CAR-T cellsThe study is a single-arm, open-label clinical trial intended to recruit 6 refractory SLE subjects who meet the inclusion and exclusion criteria. The subjects will receive IM19 CAR-T cell infusion therapy at a dose of 1×10\^6/kg or 1×10\^8 CAR-T cells . The primary endpoint is to evaluate the improvement in SLE disease activity (SLEDAI-2K) at 90 days and the occurrence of adverse events related to IM19 CAR-T cell infusion within 28 days post-infusion. Additionally, the long-term efficacy was evaluated, including the improvement of SLEDAI-2K score and achieving lupus low disease activity (LLDAS) at day 180 and day 360, and renal response at day 180 and day 360. The persistence and duration of IM19 CAR-T cells in the peripheral blood of subjects will also be evaluated. The study plan includes five phases: screening phase, cell collection phase, lymphodepletion pretreatment phase, cell infusion phase, and follow-up phase.	Lupus	Non applicable	Thérapie cellulaire	China	À vérifier
BCT301This study primarily involves the use of BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells, for the treatment of patients with refractory autoimmune diseases, aiming to evaluate its safety, tolerability, and dose-limiting toxicities(DLT), and to determine the recommended therapeutic dose for further investigation. Additionally, the study assesses the efficacy of BCT301 cell injection in refractory autoimmune diseases, as well as the pharmacokinetic (PK) and pharmacodynamic (PD) characteristics in study participants.	Lupus	Phase 1	Immunomodulation + Thérapie cellulaire	China	À vérifier
anti-CD19 CAR T cell therapyAutoimmune diseases refer to a common category of diseases caused by the immune system reacting to self-antigens, leading to tissue damage. Autoimmune diseases encompass a wide variety of conditions, such as systemic lupus erythematosus（SLE）, Sjögren's syndrome (SS), systemic sclerosis (SSc), inflammatory myopathies (IM), ANCA-associated vasculitis (AAV), and antiphospholipid syndrome (APS). They affect the quality of life, while in severe cases, they can be life-threatening. Additionally, they impose a heavy economic burden on society. Current treatments for autoimmune diseases include glucocorticoid, immunosuppressants, and biologics. B cell-driven humoral immune abnormalities are a central pathogenic mechanism in many autoimmune diseases. When autoreactive B cells are excessively activated, they produce large amounts of autoantibodies and immune complexes. These antibodies and immune complexes can cause damage to various tissues and organs, leading to the development of multiple autoimmune diseases. Therefore, targeting B cells to treat autoimmune diseases is an attractive therapeutic strategy. Chimeric Antigen Receptor (CAR)-T cells targeting the B cell surface molecule CD19 have achieved significant clinical progress in acute lymphoblastic leukemia and B cell non-Hodgkin lymphoma, with several CD19 CAR-T therapies approved for marketing worldwide. Increasingly, clinical studies are exploring the use of CD19 CAR-T cells for the treatment of autoimmune diseases, and their therapeutic efficacy has been demonstrated. In this study, the investigators used γδ T cells as carrier cells to investigate the safety and efficacy of universal CAR-γδ T cells in the treatment of autoimmune diseases.	SEP	Phase 1/2	Immunomodulation + Thérapie cellulaire	China	À vérifier

Essais cliniques

6

Molécule	Indication / population	Phase	NCT	Titre	Statut
anti-CD19 CAR T cell therapy	SEP	Phase 1/2	NCT06828042	Safety and Efficacy of Universal CD19-targeting CAR-γδT Cells in Refractory Autoimmune Diseases	RECRUITING
BCT301	Lupus	Phase 1	NCT07301164	Clinical Study of BCT301 Cell Injection Therapy for Refractory Autoimmune Diseases	RECRUITING
IM19 CAR-T cells	Lupus	Non applicable	NCT06513429	IM19 CAR-T Cell Therapy in Refractory Systemic Lupus Erythematosus (SLE)	RECRUITING
Transcranial Magnetic Stimulation	Sclérose latérale amyotrophique	Phase 1	NCT06819358	iFIRST-ALS — Individualized Functional Imaging-Guided Repetitive Transcranial Magnetic Stimulation (rTMS) for Treating Postural Gait Disorders in Patients with Amyotrophic Lateral Sclerosis (ALS): a Randomized, Crossover, Controlled, Double-Blind Clinical Study	NOT_YET_RECRUITING
Peripheral Neurofilament Levels and Amyotrophic Lateral Sclerosis	Sclérose latérale amyotrophique	À vérifier	NCT04455542	Peripheral Neurofilament Levels and Amyotrophic Lateral Sclerosis	COMPLETED
Natural History and Clinical Features of Amyotrophic Lateral Sclerosis (ALS)	Sclérose latérale amyotrophique	À vérifier	NCT04454892	Natural History and Clinical Features of Amyotrophic Lateral Sclerosis (ALS)	RECRUITING

Publications

9

Molécule	Indication / population	Titre	Journal	Date
Transcranial Magnetic Stimulation		Treatment of posttraumatic stress disorder (PTSD).	Dialogues in clinical neuroscience
Transcranial Magnetic Stimulation		A randomized double-blind sham-controlled preliminary study of a 5-week protocol of repetitive transcranial magnetic stimulation (rTMS) combined with cognitive behavioral therapy (CBT) for gambling disorder in Indonesia.	Addictive behaviors reports
Transcranial Magnetic Stimulation		Assessing the correlation between sensorimotor evoked potential and cognitive impairment in post-stroke patients.	Annals of human biology
Peripheral Neurofilament Levels and Amyotrophic Lateral Sclerosis		Neutrophil-Secreted Enzymes and ALS Risk: Exploring a Potential Mechanistic Link.	Annals of neurology
Natural History and Clinical Features of Amyotrophic Lateral Sclerosis (ALS)		Preferences for Healthcare Delivery in Amyotrophic Lateral Sclerosis (ALS): A Survey of Patients and Caregivers in the United States.	The patient
Peripheral Neurofilament Levels and Amyotrophic Lateral Sclerosis		Silent Damage, Delayed Symptoms: A Case of Breast Cancer Radiation-Induced Lumbosacral Plexopathy.	Reports (MDPI)
Peripheral Neurofilament Levels and Amyotrophic Lateral Sclerosis		Investigating the role of serum NfL, FGF21, NCAM1 and GDF15 as disease biomarkers for Charcot-Marie-Tooth type 2A.	Scientific reports
Natural History and Clinical Features of Amyotrophic Lateral Sclerosis (ALS)		Prospective Validation of the New PLS Diagnostic Criteria From PLS Natural History Study: EMG and Neurofilament Analyses.	Muscle & nerve
Natural History and Clinical Features of Amyotrophic Lateral Sclerosis (ALS)		From Environment to Symptoms: Mapping Premorbid Risk Factors onto Clinical Features of ALS in a Patient-Reported Database in China.	Neurology and therapy