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New treatment option for Amyotrophic Lateral Sclerosis
Official title Exploring Nasal Drop Therapy With Small Extracellular Vesicles for ALS
ClinicalTrials.gov ID: NCT06598202
What this study is testing
What is exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop?
exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop is an investigational medicine, given as an once-daily, being studied as a potential treatment for amyotrophic lateral sclerosis.
Also referred to as hUC-MSC-sEV-001.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 80
You may be able to join if
- Age: 18-80 years, inclusion of both genders;
- Disease duration: ≥6 months and ≤2 years (counted from the onset of any ALS symptoms);
- people must meet the El Escorial revised criteria (2000) for the diagnosis of ALS, with a diagnosis of Definite ALS, Probable ALS, Probable...
- 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...
- BMI: Between 18 and 30 kg/m²;
You likely can't join if
- Diagnosed as non-ALS based on clinical presentation and available clinical examinations (e.g., neurophysiological tests, MRI, or other imaging...
- 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...
- 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...
See the full eligibility criteria
- Age: 18-80 years, inclusion of both genders;
- Disease duration: ≥6 months and ≤2 years (counted from the onset of any ALS symptoms);
- people 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;
- people 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...
- people 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.
- 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...
- 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...
- History of acute myocardial infarction or treatment 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.
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, Beijing Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 80 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, Beijing Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.