NCT06598202
Exploring Nasal Drop Therapy With Small Extracellular Vesicles for ALS
Recruiting · Phase 1, Phase 2 · Xuanwu Hospital, Beijing · registry updated 2024-10-31
Inclusion and exclusion lines below are quoted from ClinicalTrials.gov. No match score is shown, because a score needs a person's age, biomarkers, and treatment dates. Confirm the record with the study team.
This is a multicenter, randomized, double-blind, placebo-controlled, dose-escalation trial. The goal of this clinical trial is to evaluate the safety and preliminary efficacy of nasal drop exosomes derived from human umbilical cord blood mesenchymal stem cells (hUC-MSC-sEV-001) in amyotrophic lateral sclerosis.
Inclusion
- Age: 18-80 years, inclusion of both genders;
- Disease duration: ≥6 months and ≤2 years (counted from the onset of any ALS symptoms);
- Subjects must meet the El Escorial revised criteria (2000) for the diagnosis of ALS, with a diagnosis of Definite ALS, Probable ALS, Probable laboratory-supported ALS, or Possible ALS;
- A score of ≥2 on each item of the revised ALS Functional Rating Scale (ALSFRS-R), with a score of 4 for items related to dyspnea, orthopnea, and respiratory insufficiency;
- BMI: Between 18 and 30 kg/m²;
- Subjects must have a baseline forced vital capacity percentage (%FVC) ≥70%;
Exclusion
- Diagnosed as non-ALS based on clinical presentation and available clinical examinations (e.g., neurophysiological tests, MRI, or other imaging, laboratory tests);
- Abnormal nasal anatomy, nasal cavity damage, severe rhinitis, or nasal disease affecting the administration of the study drug;
- Requires nasal insertion of a gastric tube;
- Peripheral venous hemoglobin (HGB) \< 100 g/L, absolute neutrophil count (NEUT) \< 1.5×10\^9/L, platelet count (PLT) \< 100×10\^9/L, white blood cell count (WBC) \< 4.0×10\^9/L or ≥ 12×10\^9/L, serum albumin \< 30 g/L; alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≥ 3× the upper limit of normal (ULN);
- Severe renal insufficiency: Glomerular Filtration Rate (GFR) \< 30 mL/min (Cockcroft-Gault formula), or other known severe renal diseases;
- Positive for hepatitis B surface antigen, e antigen, e antibody, or core antibody combined with positive hepatitis B virus DNA; positive for hepatitis C virus antibody; positive syphilis serum antibody; or positive for HIV antibody;