๐ฌIn plain words
What the trial is, and whether it could suit you
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.
Sign in to get the eligibility rules as simple questions to take to your doctor.
๐ Eligibility rules (as published)
Inclusion Criteria:
* Age: 18-80 years, inclusion of both genders;
* Disease duration: โฅ6 months and โค2 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 โฅ2 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/mยฒ;
* Subjects must have a baseline forced vital capacity percentage (%FVC) โฅ70%;
* Allowed concomitant treatments: Oral administration of riluzole/edaravone at standard doses for โฅ30 days; regular intravenous edaravone with planned sequential oral treatment. During the trial and follow-up period, the dosage and type of concomitant medications must remain unchanged;
* Subjects of childbearing potential must use appropriate and effective contraception from 2 weeks prior to trial enrollment until the end of the follow-up period;
* The subject or legal representative must be able to sign an informed consent form and comply with the study requirements for medication administration and follow-up.
Exclusion Criteria:
* 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) \< 1.5ร10\^9/L, platelet count (PLT) \< 100ร10\^9/L, white blood cell count (WBC) \< 4.0ร10\^9/L or โฅ 12ร10\^9/L, serum albumin \< 30 g/L; alanine aminotransferase (ALT) and aspartate aminotransferase (AST) โฅ 3ร 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;
* History of acute myocardial infarction or interventional treatment within the last 6 months, or heart failure (classified as NYHA III-IV);
* Presence of severe localized or systemic infection, immunodeficiency, or currently taking immunosuppressants;
* Concurrent severe systemic diseases such as immunodeficiency diseases, coagulation disorders, or malignancies;
* Vaccination within 1 month prior to the first administration or during the study until the end of follow-up;
* Known allergy to the drugs used in this study or similar drugs;
* Participation in another study and administration of an investigational product within the last 3 months;
* Contraindications to MRI (e.g., presence of metal implants) or inability to tolerate MRI (e.g., claustrophobia);
* Pregnant or breastfeeding women, or women of childbearing potential who cannot or are unwilling to use appropriate contraception;
* Unwillingness or inability to comply with the procedures required by the protocol;
* Any other conditions deemed unsuitable for inclusion by the investigators.