NCT06598202 Exploring Nasal Drop Therapy With Small Extracellular Vesicles for ALS
| NCT ID | NCT06598202 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Xuanwu Hospital, Beijing |
| Condition | Amyotrophic Lateral Sclerosis |
| Study Type | INTERVENTIONAL |
| Enrollment | 38 participants |
| Start Date | 2024-12-01 |
| Primary Completion | 2026-05-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 38 participants in total. It began in 2024-12-01 with a primary completion date of 2026-05-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: * 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. 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) \< 1.5×10\^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.
Contact & Investigator
Junwei Hao, MD; PhD
PRINCIPAL INVESTIGATOR
Xuanwu Hospital, Beijing
Frequently Asked Questions
Who can join the NCT06598202 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 80 Years, studying Amyotrophic Lateral Sclerosis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06598202 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06598202 currently recruiting?
Yes, NCT06598202 is actively recruiting participants. Contact the research team at haojunwei@vip.163.com for enrollment information.
Where is the NCT06598202 trial being conducted?
This trial is being conducted at Beijing, China.
Who is sponsoring the NCT06598202 clinical trial?
NCT06598202 is sponsored by Xuanwu Hospital, Beijing. The principal investigator is Junwei Hao, MD; PhD at Xuanwu Hospital, Beijing. The trial plans to enroll 38 participants.
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