Amyotrophic Lateral Sclerosis (ALS)Phase 1NCT06671236
Clinical Study of Regulatory T Cells (Tregs) in the Treatment of Neurodegenerative Diseases
A Phase 1 study testing Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection) in people with Amyotrophic Lateral Sclerosis (ALS), run by Novabio Therapeutics at 1 site in China.
Trial summary
An open, multi- center phase Ⅰ clinical study evaluating the safety and efficacy of autologous human polyclonal regulatory T cell injection (NP001 cell injection) in patients with Neurodegenerative diseases (ALS). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 21 Nov 2024to Dec 2027
- Phase
- Phase 1
- 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- -
- Patients must meet all of the following criteria to be eligible for enrollment in this study:
- Male or female patients aged 18 to 70 years;
- According to current international diagnostic criteria:
- ALS: defined by the Gold Coast Diagnostic Criteria (Shefner, 2020) as having a diagnosis of sporadic or familial amyotrophic lateral sclerosis (ALS), diagnosed as a probable, probable, or definite patient with laboratory support according to the World Federation of Neurology El Escorial criteria;
- If there is a stable dose for more than one month prior to study entry. For example, patients with ALS can continue treatment with riluzole (Rilutek®) and/or edaravone (Radicava®);
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- Patients must have > two weeks after the end of major surgery and after the completion of participation in other research trials;
- Patients must have recovered from clinical toxicity (CTCAE [5th Edition] toxicity values have resolved to < 2);
- Serum creatinine less than or equal to 2.0 mg/dL;
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 3 x upper limit of normal;
- Bilirubin < 1.5 (except Gilbert's disease);
- Lung slow vital capacity (SVC) > 70% of predicted normal;
- No history of abnormal bleeding tendency;
- Informed consent must be obtained prior to performing any study-related procedures that are not part of standard medical care, with the understanding that the participant may withdraw from the study without influence for the future medical care.
Who cannot join
- -
- Subjects with any of the following cannot be enrolled in this study:
- uncontrolled infection;
- < 3 drugs do not adequately control hypertension;
- Documented history of pulmonary embolism within 6 months of enrollment;
- Clinically significant cardiology, defined as: myocardial infarction, NYHA-graded class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmia, or ECG evidence of acute ischemia or abnormal conduction system within 6 months prior to enrollment;
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- Patients with a history of coronary artery bypass grafting or angioplasty will be evaluated by cardiology and considered on a case-by-case basis;
- Seropositive for HIV, hepatitis B or hepatitis C;
- Pregnant or lactating patients;
- Patients of childbearing potential or males with partners of childbearing potential who are unwilling to use contraception;
- Participation in any other interventional study;
- Treatment with another investigational drug, biologic, or device within 30 days or 5 half-lives (whichever is longer) of the screening period. Patient participation in observational/non-interventional clinical studies will be discussed with the Medical Monitor;
- Prior treatment with ALS gene or cell therapy;
- History of clinically significant tumor, liver or kidney disease, or other uncontrolled disease;
- presence of a feeding tube;
- Current use of antipsychotics, antiepileptic drugs (except benzodiazepines, gabapentin, pre-Bahrain) or class 1 (e.g., flecainide) or class 3 (e.g., amiodarone) antiarrhythmic drugs;
- Subjects who, in the opinion of the investigator, are at significant risk of suicide;
- Other conditions that the investigator considers unsuitable for enrollment.
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
The First Affiliated Hospital of Zhengzhou University
Zhengzhou, Henan, 450052
Yi Zhang, MD, +86 371 6629 5320, yizhang@zzu.edu.cn
About this study
Official title: An Open, Single-Arm, Multi-Center Clinical Study to Evaluate the Safety and Tolerability of Regulatory T Cells (Tregs) In the Treatment of Neurodegenerative Diseases
An open, multi- center phase Ⅰ clinical study evaluating the safety and efficacy of autologous human polyclonal regulatory T cell injection (NP001 cell injection) in patients with Neurodegenerative diseases (ALS).
Read the detailed description
This is an open-label, non-randomized, multi-center clinical trial of single-agent NP001 cell injection in patients with with Neurodegenerative diseases (ALS).
After subject consents to the study, an apheresis procedure will be performed to collect cells to manufacture the investigational product, NP001 cell injection. NP001 cell injection are manufactured ex vivo to yield enriched Tregs.
This study is evaluating NP001 cell injection at the dose of 1x E6 cells, 1x E7 cells, and 1x E8 cells/times, with up to 3 times separated by 4 weeks among dosing (intrathecally on Days 1, 29, and 57). Study subjects are then followed for several months to capture safety and efficacy parameters. The total duration of NP001 cell injection and follow-up interval on this protocol is approximately 12-months.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection)
- Keywords
- Treg, regulatory T cells, neurodegenerative diseases, Amyotrophic Lateral Sclerosis
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Neurodegenerative Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection)Biological
Regulatory T cell therapy, intrathecal injection
| Study group | Role | What they receive |
|---|---|---|
| Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection) | Experimental | Regulatory T cell therapy, intrathecal injection Receives: Biological: Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment Related adverse events (AEs)
Incidence of Treatment Related AEs, AEs of special interest and serious adverse events (SAEs)
Measured: 6 months
Identification of Maximum Tolerated Dose (MTD)
Incidence of dose-limiting toxicities (DLTs)
Measured: 28 days
Secondary outcomes (2)
Amyotrophic lateral sclerosis functional rating scale- revised (ALSFRS-R)
The ALSFRS-R is a simple, validated, and reliable tool for evaluating declines in function. It involves self-rating by patients on their ability and independence across 12 functional activities, with each question scored from 0 to 4 (4 means normal). The total score ranges from 0 (worst) to 48 (best), covering aspects of speech, swallowing, fine motor skills, gross motor skills, and respiratory function.
Measured: 1 year
Rasch Overall ALS Disability Scale (ROADS)
ROADS is a patient-reported outcome measure that assesses overall disability level in patients with ALS. The scale contains 28 items, each scored 0, 1, or 2.
The scale was constructed and validated using Rasch analyses - a modern test theory technique that produces linearly-weighted scales, meaning that a 1-point change is a quantifiable, consistent measurement of disability across the scale, and a 2-point change reflects twice the amount of disability.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2024
- Study start21 Nov 2024
- Primary completionDec 2026 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novabio Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06671236
- Sponsor's protocol ID
- NP001-NDD02
- Status last verified
- Sep 2025
- First submitted
- 29 Oct 2024
- Last updated
- 25 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open, Single-Arm, Multi-Center Clinical Study to Evaluate the Safety and Tolerability of Regulatory T Cells (Tregs) In the Treatment of Neurodegenerative Diseases
- ClinicalTrials.gov ID
- NCT06671236
- Acronym / sponsor ID
- NP001-NDD02
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Keywords
- Treg, regulatory T cells, neurodegenerative diseases, Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Novabio TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- The First Affiliated Hospital of Zhengzhou University
- Study start
- 21 Nov 2024 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 4 Nov 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.
An open, multi- center phase Ⅰ clinical study evaluating the safety and efficacy of autologous human polyclonal regulatory T cell injection (NP001 cell injection) in patients with Neurodegenerative diseases (ALS).
Detailed description
This is an open-label, non-randomized, multi-center clinical trial of single-agent NP001 cell injection in patients with with Neurodegenerative diseases (ALS).
After subject consents to the study, an apheresis procedure will be performed to collect cells to manufacture the investigational product, NP001 cell injection. NP001 cell injection are manufactured ex vivo to yield enriched Tregs.
This study is evaluating NP001 cell injection at the dose of 1x E6 cells, 1x E7 cells, and 1x E8 cells/times, with up to 3 times separated by 4 weeks among dosing (intrathecally on Days 1, 29, and 57). Study subjects are then followed for several months to capture safety and efficacy parameters. The total duration of NP001 cell injection and follow-up interval on this protocol is approximately 12-months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection) | Experimental | Biological: Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection)Regulatory T cell therapy, intrathecal injection |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- -
- Patients must meet all of the following criteria to be eligible for enrollment in this study:
- Male or female patients aged 18 to 70 years;
- According to current international diagnostic criteria:
- ALS: defined by the Gold Coast Diagnostic Criteria (Shefner, 2020) as having a diagnosis of sporadic or familial amyotrophic lateral sclerosis (ALS), diagnosed as a probable, probable, or definite patient with laboratory support according to the World Federation of Neurology El Escorial criteria;
Key exclusion criteria (18 listed)
- -
- uncontrolled infection;
- < 3 drugs do not adequately control hypertension;
- Documented history of pulmonary embolism within 6 months of enrollment;
- Clinically significant cardiology, defined as: myocardial infarction, NYHA-graded class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmia, or ECG evidence of acute ischemia or abnormal conduction system within 6 months prior to enrollment;
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 |
|---|---|---|
| Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection)Regulatory T cell therapy, intrathecal injection | Biological | Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection) |
Primary outcome
Incidence of Treatment Related adverse events (AEs)
Time frame: 6 months
Incidence of Treatment Related AEs, AEs of special interest and serious adverse events (SAEs)
Identification of Maximum Tolerated Dose (MTD)
Time frame: 28 days
Incidence of dose-limiting toxicities (DLTs)
2 secondary outcomes
Amyotrophic lateral sclerosis functional rating scale- revised (ALSFRS-R)
Time frame: 1 year
Rasch Overall ALS Disability Scale (ROADS)
Time frame: 1 year
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 25 Sep 2025.
- Individual participant dataSharing planned: Yes
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection) in people with Amyotrophic Lateral Sclerosis (ALS), run by Novabio Therapeutics 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 14 inclusion and 18 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 25 Sep 2025. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
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 Mingqi Lu, MD., PhD (609-716-7786; +86 15800843507, lumingqi@novabiotx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment Related adverse events (AEs) (time frame: 6 months). The study lists 2 primary outcomes in all. 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 25 Sep 2025; AzTrials last refreshed its copy on 8 Nov 2026.