Amyotrophic Lateral SclerosisPhase 1/2de-ALSNCT06598202
Exploring Nasal Drop Therapy With Small Extracellular Vesicles for ALS
A Phase 1/2 study testing exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop in people with Amyotrophic Lateral Sclerosis, run by Xuanwu Hospital, Beijing at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 38 participants
- Where
- 1 siteChina
- Study dates
- 1 Dec 2024to 30 May 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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%;
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- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Xuanwu Hospital ,Capital Medical University
Beijing, Beijing Municipality, 100053
Gaoting Ma, MD, 01083198082, demo_doctor@163.com
About this study
Official title: An Exploratory Study on the Use of Intranasal Administration of Small Extracellular Vesicles for the Treatment of Amyotrophic Lateral Sclerosis
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.
Read the detailed description
This is a multicenter, randomized, double-blind, placebo-controlled, dose-escalation trial. The study will consist of two parts: Part 1 will be a dose-escalation study, and Part 2 will be an expanded safety study based on the findings from Part 1.
A traditional 3+3 dose-escalation design will be implemented in Part 1. Cohort 1 will receive low-dose; Cohort 2 will receive middle-dose; and Cohort 3 will receive high-dose. (Cohort 1 to Cohort 3 will receive a dose of 1 mL per nostril, administered once daily, twice a week, for a total of two weeks.) If no dose-limiting toxicities (DLTs) are observed for 2 weeks after the administration of the first nasal drop, a new cohort will be enrolled at the next planned dose level. If DLTs are observed in one participant in the cohort, an additional three participants will be treated at the same dose level. Dose escalation will be stopped if DLTs are observed in more than 33% of the participants.
In Part 2, 20 subjects will be randomized in a 1:1 ratio [exosome (n=10) or exosome placebo (n=10)]. The dose level will be determined by the primary researcher based on the findings from Part 1.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop, a placebo of exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop
- Keywords
- Neurodegenerative diseases, Inflammation, Motor neuron degeneration, Exosomes, Small extracellular vesicles
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Neurodegenerative Diseases, Inflammation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal dropDrug
Also called hUC-MSC-sEV-001
Exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop (administered once daily, twice a week, for a total of two weeks, based on the recommended dose during the dose-escalation phase).
a placebo of exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal dropDrug
Also called hUC-MSC-sEV-001 placebo
Exosomes placebo
| Study group | Role | What they receive |
|---|---|---|
| Exosomes group | Experimental | Patients in this arm will receive exosomes derived from human umbilical cord blood mesenchymal stem cells as a nasal drop, administered once daily, twice a week, for a total of two weeks. Receives: Drug: exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop |
| Exosomes placebo group | Placebo comparator | Patients in this arm will receive a placebo nasal drop mimicking exosomes derived from human umbilical cord blood mesenchymal stem cells, administered once daily, twice a week, for a total of two weeks. Receives: Drug: a placebo of exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants who experienced dose-limiting Toxicities (DLTs)
DLTs related to hUC-MSC-sEV-001 include adverse events of grade 3 or higher (including significant clinical laboratory findings) that are possibly, likely, or definitely related to the study drug, accompanied by clinical symptoms and requiring medical treatment within 14 days of administration. Adverse events are graded according to the Common Terminology Criteria for Adverse Events Version 5.0 (CTCAE 5.0).
Measured: 24 hours, 4±1 Weeks
Secondary outcomes (10)
Incidence of severe adverse events
The proportion of patients who experienced severe adverse events.
Measured: 4±1 Weeks
Change from baseline in ALS functional rating scale - revised
The ALSFRS-R is rating scale (ratings 0 = can't do, to 4 = normal ability) used to determine participants' assessment of their capability and independence in 12 functional activities.
This is a validated scale, both in person and by phone, which provides a total score from four sub-scores which assess speech and swallowing (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function).
Total scores range from 0 (most impaired) to 48 (normal ability).
Measured: 4±1 Weeks, 12±1 Weeks, 24±1 Weeks
Change from baseline in forced vital capacity to predicted value ratio (FVC% pred)
Spirometry is a physiological test that measures how an individual inhales or exhales volumes of air as a function of time. The primary signal measured in spirometry may be volume or flow. The forced vital capacity (FVC) is the maximal volume of air exhaled with maximally forced effort from a maximal inspiration.
Measured: 4±1 Weeks, 24±1 Weeks
Time to event (death, tracheostomy, and permanent assisted mechanical ventilation)
The number of time-to-event outcomes.
Measured: up to 24 Weeks
Change from baseline in the Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40).
ALSAQ-40 score ranges from 0 to 160 points, and higher scores indicate better quality of life.
Measured: 4±1 Weeks, 12±1 Weeks, 24±1 Weeks
Change from baseline in the Edinburgh Cognitive and Behavioral ALS Screen (ECAS).
ECAS determine cognitive and behavioral changes of patients suffering from Amyotrophic Lateral Sclerosis.
With ECAS, ALS-specific (fluency, executive functions and social cognition, language; minimum score = 0, maximum score = 100) and ALS-nonspecific (memory, visuospatial functions; minimum score = 0, maximum score = 36) functions can be analyzed to enable the distinction from other diseases with cognitive and behavioral impairments.
Measured: 12±1 Weeks, 24±1 Weeks
Change from baseline in the Neurophysiological Index (NI) and Compound Muscle Action Potential (CMAP) score.
The NI is derived from the CMAP, the DML and the F-wave frequency (CMAP amplitude/DML) x F frequency %), representing aspects of the effects of denervation and reinnervation, of degeneration of the terminal part of the motor axons, and of the excitability of anterior horn cells.
The negative amplitudes of the CMAP for motor nerve conduction in the median and ulnar nerves in the upper extremities as well as the peroneal and tibial nerves in the lower extremities of ALS patients were recorded (median nerve-wrist, ulnar-wrist, peroneal-ankle, tibial-ankle). These nerves were scored based on the decrease in the amplitudes of each nerve CMAP- 0 (CMAP> =[X-2s]), 1 (50% [X-2s] < CMAP < [X-2s]), 2 (30% [X-2s] < CMAP < 50% [X-2s]), 3 (CMAP < =30% [X-2s]), respectively. The values of X and S for different nerves and different patient age groups, which were applied in the electrophysiology examination room.
Measured: 12±1 weeks
Change from baseline in the Rasch Overall ALS Disability Scale (ROADS).
ROADS score ranges from 0 to 56 points, and higher scores indicate better basic daily function.
Measured: 4±1 Weeks, 12±1 Weeks, 24±1 Weeks
Change from baseline in the Medical Research Council (MRC) Scale.
MRC ranges from 0 to V grades, and higher grades indicate higher muscle strength.
Measured: 4±1 Weeks, 12±1 Weeks, 24±1 Weeks
Change from baseline in the the Lower Motor Neuro Scale(LMNS).
LMNS was calculated using the score proposed by Devine et al. that assign a separate LMN score for each limb, ranging from 0 (no involvement) to 3 (significant, severe involvement), based on the Medical Research Council and muscle wasting.
Measured: 4±1 Weeks, 12±1 Weeks, 24±1 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start1 Dec 2024 (estimated)
- Primary completion30 May 2026 (estimated)
- Study completion30 May 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xuanwu Hospital, Beijing
- Sponsor type
- University, hospital or other
- Collaborators
- Shengqi Medical Technology (Guangzhou) Co., Ltd., Viyun (Xiamen) Biomedical Research Institute Co., Ltd.
- Responsible party
- Principal investigator, Junwei Hao, MD, Xuanwu Hospital, Beijing
- Investigators
- Junwei Hao, MD; PhD, Principal investigator, Xuanwu Hospital, Beijing
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06598202
- Sponsor's protocol ID
- XMEC-2024-267-002
- Acronym
- de-ALS
- Status last verified
- Oct 2024
- First submitted
- 10 Sep 2024
- Last updated
- 31 Oct 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The study will not share individual participant data to other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 31 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Study on the Use of Intranasal Administration of Small Extracellular Vesicles for the Treatment of Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT06598202
- Acronym / sponsor ID
- de-ALS / XMEC-2024-267-002
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Neurodegenerative diseases, Inflammation, Motor neuron degeneration, Exosomes, Small extracellular vesicles
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 38 participants (estimated)
- Lead sponsor
- Xuanwu Hospital, BeijingUniversity, hospital or other
- Collaborators
- Shengqi Medical Technology (Guangzhou) Co., Ltd., Viyun (Xiamen) Biomedical Research Institute Co., Ltd.
- Study start
- 1 Dec 2024 (estimated)
- Primary completion
- 30 May 2026 (estimated)
- Study completion
- 30 May 2026 (estimated)
- First posted
- 19 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a multicenter, randomized, double-blind, placebo-controlled, dose-escalation trial. The study will consist of two parts: Part 1 will be a dose-escalation study, and Part 2 will be an expanded safety study based on the findings from Part 1.
A traditional 3+3 dose-escalation design will be implemented in Part 1. Cohort 1 will receive low-dose; Cohort 2 will receive middle-dose; and Cohort 3 will receive high-dose. (Cohort 1 to Cohort 3 will receive a dose of 1 mL per nostril, administered once daily, twice a week, for a total of two weeks.) If no dose-limiting toxicities (DLTs) are observed for 2 weeks after the administration of the first nasal drop, a new cohort will be enrolled at the next planned dose level. If DLTs are observed in one participant in the cohort, an additional three participants will be treated at the same dose level. Dose escalation will be stopped if DLTs are observed in more than 33% of the participants.
In Part 2, 20 subjects will be randomized in a 1:1 ratio [exosome (n=10) or exosome placebo (n=10)]. The dose level will be determined by the primary researcher based on the findings from Part 1.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Exosomes group | Experimental | Drug: exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal dropPatients in this arm will receive exosomes derived from human umbilical cord blood mesenchymal stem cells as a nasal drop, administered once daily, twice a week, for a total of two weeks. |
| Exosomes placebo group | Placebo comparator | Drug: a placebo of exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal dropPatients in this arm will receive a placebo nasal drop mimicking exosomes derived from human umbilical cord blood mesenchymal stem cells, administered once daily, twice a week, for a total of two weeks. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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;
Key exclusion criteria (16 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal dropAlso: hUC-MSC-sEV-001Exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop (administered once daily, twice a week, for a total of two weeks, based on the recommended dose during the dose-escalation phase). | Drug | Exosomes group |
| a placebo of exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal dropAlso: hUC-MSC-sEV-001 placeboExosomes placebo | Drug | Exosomes placebo group |
Primary outcome
Number of participants who experienced dose-limiting Toxicities (DLTs)
Time frame: 24 hours, 4±1 Weeks
DLTs related to hUC-MSC-sEV-001 include adverse events of grade 3 or higher (including significant clinical laboratory findings) that are possibly, likely, or definitely related to the study drug, accompanied by clinical symptoms and requiring medical treatment within 14 days of administration. Adverse events are graded according to the Common Terminology Criteria for Adverse Events Version 5.0 (CTCAE 5.0).
10 secondary outcomes
Incidence of severe adverse events
Time frame: 4±1 Weeks
Change from baseline in ALS functional rating scale - revised
Time frame: 4±1 Weeks, 12±1 Weeks, 24±1 Weeks
Change from baseline in forced vital capacity to predicted value ratio (FVC% pred)
Time frame: 4±1 Weeks, 24±1 Weeks
Time to event (death, tracheostomy, and permanent assisted mechanical ventilation)
Time frame: up to 24 Weeks
Change from baseline in the Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40).
Time frame: 4±1 Weeks, 12±1 Weeks, 24±1 Weeks
Change from baseline in the Edinburgh Cognitive and Behavioral ALS Screen (ECAS).
Time frame: 12±1 Weeks, 24±1 Weeks
Change from baseline in the Neurophysiological Index (NI) and Compound Muscle Action Potential (CMAP) score.
Time frame: 12±1 weeks
Change from baseline in the Rasch Overall ALS Disability Scale (ROADS).
Time frame: 4±1 Weeks, 12±1 Weeks, 24±1 Weeks
Change from baseline in the Medical Research Council (MRC) Scale.
Time frame: 4±1 Weeks, 12±1 Weeks, 24±1 Weeks
Change from baseline in the the Lower Motor Neuro Scale(LMNS).
Time frame: 4±1 Weeks, 12±1 Weeks, 24±1 Weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Oct 2024.
- Individual participant dataSharing planned: No. The study will not share individual participant data to other researchers.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Junwei Hao, MD; PhD principal investigator
Xuanwu Hospital, Beijing
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop in people with Amyotrophic Lateral Sclerosis, run by Xuanwu Hospital, Beijing at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 31 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Junwei Hao, MD; PhD (010 8319 8277, haojunwei@vip.163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants who experienced dose-limiting Toxicities (DLTs) (time frame: 24 hours, 4±1 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Oct 2024.