Exploring Nasal Drop Therapy With Small Extracellular Vesicles for ALS
Recruiting now · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Amyotrophic Lateral Sclerosis
In brief
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.
Key facts
- Study ID
- NCT06598202
- Run by
- Xuanwu Hospital, Beijing
- People needed
- 38
- Starts
- 2024-12-01
- Expected to finish
- 2026-05-30
- Last updated by the study team
- 2024-10-31
Who can join
Age: 18 and older, up to 80. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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%;
- Allowed concomitant treatments: Oral administration of riluzole/edaravone at standard doses for ≥30 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.
You may not qualify if…
- 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;
- 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.
Where it is running
- Xuanwu Hospital ,Capital Medical University — Beijing, Beijing Municipality, China (enrolling)
Full record on ClinicalTrials.gov
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