ALS - Amyotrophic Lateral SclerosisPhase 1NTF-ALSNCT07618585
Safety and Efficacy of Intrathecal NTF001 Injection in ALS
A Phase 1 study testing NTF001 Injection in people with ALS - Amyotrophic Lateral Sclerosis and ALS (Amyotrophic Lateral Sclerosis), run by Ruijin Hospital at 1 site in China.
Trial summary
This is a single-arm, open-label, early-phase clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of intrathecal NTF001 injection, an AAV-mediated human neuron-derived neurotrophic factor gene therapy, in patients with amyotrophic lateral sclerosis (ALS). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- ALS - Amyotrophic Lateral Sclerosis, ALS (Amyotrophic Lateral Sclerosis)
- What is being tested?
- NTF001 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
- 1 Apr 2026to 31 Mar 2028
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Voluntarily participate in this study and sign the informed consent form.
- Agree to comply with study procedures and cooperate with all study-related assessments throughout the study.
- Male or female patients aged 18 to 65 years.
- Meet the diagnostic and exclusion criteria for amyotrophic lateral sclerosis according to the Chinese Expert Consensus on the Diagnosis and Treatment of Amyotrophic Lateral Sclerosis 2022 issued by the Neurology Branch of the Chinese Medical Association.
- Have a history of amyotrophic lateral sclerosis of no more than 5 years.
Who cannot join
- Mini-Mental State Examination (MMSE) score < 24.
- Patient Health Questionnaire-9 (PHQ-9) score ≥ 16.
- Abnormal liver or renal function, defined as AST or ALT > 1.5 × upper limit of normal (ULN), or serum creatinine (Cr) > 1.5 × ULN.
- Abnormal coagulation function or current use of anticoagulants.
- Positive infectious disease screening, including positive HBsAg or HBV-DNA, positive HCV-RNA, positive HIV test, or positive syphilis serology.
- Currently receiving antiviral treatment for hepatitis B or hepatitis C.
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- Unstable or severe systemic diseases, including active tuberculosis, cardiovascular, respiratory, gastrointestinal, urinary, psychiatric or neurological disorders, such as epilepsy, hematological disorders, immune system diseases, or abnormal laboratory findings that, in the opinion of the investigator, make the participant unsuitable for this study.
- Current or previous history of malignant tumor.
- History of severe allergic reactions, allergy to contrast agents, or inability to undergo surgical anesthesia.
- Currently participating in another clinical trial, or participation in another clinical trial within 3 months before screening.
- Previous receipt of gene therapy before screening.
- Receipt of stem cell therapy within 6 months before screening.
- Use of other investigational drugs within 4 weeks before screening or within 5 half-lives of the investigational drug, whichever is longer, or use of any medication that, in the opinion of the investigator, may affect this study.
- Receipt of a live vaccine within 2 months before screening, or any vaccination within 30 days before screening.
- History of alcohol dependence or drug addiction, and inability to stop alcohol consumption as instructed during the study.
- Female participants who are pregnant or breastfeeding.
- Participants considered unsuitable for enrollment by the investigator.
- Patients requiring ventilator-assisted ventilation.
- Patients allergic to the investigational intervention.
- Patients with obvious signs of dementia.
- Patients with other psychiatric disorders that may affect disease assessment.
- Severely obese patients, defined as BMI > 35 kg/m².
Trial locations and contacts
1 site in 1 country.
China1 site
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, 200025
About this study
Official title: A Study of the Safety and Efficacy of Intrathecal NTF001 Injection in the Treatment of Amyotrophic Lateral Sclerosis
This is a single-arm, open-label, early-phase clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of intrathecal NTF001 injection, an AAV-mediated human neuron-derived neurotrophic factor gene therapy, in patients with amyotrophic lateral sclerosis (ALS).
12 patients with ALS will be enrolled. Each participant will receive a single intrathecal administration of NTF001 and will be followed for 52 weeks after treatment. The primary outcome measures include treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). Secondary outcome measures include changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), quality-of-life assessments, and neurological function.
This study aims to provide preliminary clinical evidence regarding the safety and potential efficacy of intrathecal NTF001 injection in patients with ALS.
Read the detailed description
This is a single-arm, open-label, early-phase clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of NTF001 injection in patients with amyotrophic lateral sclerosis (ALS). NTF001 is an investigational AAV-mediated gene therapy product designed to express human neuron-derived neurotrophic factor (NDNF). The investigational product will be administered once by intrathecal injection.
12 patients with ALS will be enrolled in this study. All eligible participants will receive a single intrathecal administration of NTF001 and will undergo scheduled safety and efficacy assessments during a 52-week follow-up period after treatment.
The primary objective of this study is to assess the safety and tolerability of intrathecal NTF001 injection, primarily by evaluating the occurrence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), clinically significant laboratory abnormalities, vital signs, neurological examinations, and other safety-related findings.
The secondary objective is to explore the preliminary efficacy of NTF001 in patients with ALS. Efficacy assessments will include changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), quality-of-life assessments, neurological function, and other exploratory clinical indicators.
This study is expected to provide preliminary clinical evidence regarding the safety, tolerability, and potential therapeutic effects of intrathecal NTF001 injection in patients with ALS, and to support further clinical development of AAV-mediated neurotrophic factor gene therapy for ALS.
- Being tested
- NTF001 Injection
- Keywords
- Neuron-derived Neurotrophic Factor, Amyotrophic Lateral Sclerosis, NTF001, Gene Therapy, Adeno-associated Virus, Intrathecal Injection
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NTF001 InjectionGenetic
NTF001 is an investigational AAV-mediated gene therapy product designed to express human neuron-derived neurotrophic factor (NDNF). It will be administered once by intrathecal injection to patients with amyotrophic lateral sclerosis (ALS). Participants will receive NTF001 according to the assigned dose cohort and will be followed for safety, tolerability, and preliminary efficacy.
| Study group | Role | What they receive |
|---|---|---|
| Low-dose NTF001 | Experimental | Participants in this cohort will receive a single intrathecal administration of low-dose NTF001 （2E+14 vg）injection. Receives: Genetic: NTF001 Injection |
| High-dose NTF001 | Experimental | Participants in this cohort will receive a single intrathecal administration of high-dose NTF001(4-5 E+14 vg) injection after safety evaluation of the low-dose cohort. Receives: Genetic: NTF001 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events and Serious Adverse Events
Safety and tolerability will be assessed by evaluating the incidence, severity, seriousness, and relationship to the investigational product of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). Safety assessments will also include clinical laboratory tests, vital signs, physical examinations, neurological examinations, and other clinically significant safety findings.
Measured: From administration of NTF001 through Week 52
Secondary outcomes (4)
Change From Baseline in ALS Functional Rating Scale-Revised Score
The ALS Functional Rating Scale-Revised (ALSFRS-R) will be used to assess functional status in patients with ALS. The total score ranges from 0 to 48, with higher scores indicating better functional status.
Measured: Baseline through Week 52
Change From Baseline in ALS Quality of Life Assessment
Quality of life will be assessed using an ALS-specific quality-of-life questionnaire. Changes from baseline will be evaluated during the follow-up period, with higher or lower scores interpreted according to the scoring rules of the selected questionnaire.
Measured: Baseline through Week 52
Change From Baseline in ALSAQ-40 Score
The Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) will be used to assess disease-specific quality of life. The questionnaire evaluates multiple domains of daily functioning and well-being in patients with ALS, with higher scores generally indicating worse quality of life.
Measured: Baseline through Week 52
Change From Baseline in Norris Scale Score
The Norris Scale will be used to assess neurological function and disease severity in patients with amyotrophic lateral sclerosis. Changes from baseline in the Norris Scale score will be evaluated during the follow-up period, with lower scores indicating greater functional impairment.
Measured: Baseline through Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2026
- Study start1 Apr 2026
- Primary completion31 Mar 2028 (estimated)
- Study completion31 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALS - Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by Ruijin HospitalThe sponsor's other registered studies.
- 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
- Non-randomised
- Intervention model
- Sequential
- Model description
- Participants will be assigned sequentially to different dose cohorts of NTF001 injection. Each participant will receive a single intrathecal administration and will be followed for 52 weeks after treatment.
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ruijin Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07618585
- Sponsor's protocol ID
- NTF-ALS
- Acronym
- NTF-ALS
- Status last verified
- May 2026
- First submitted
- 25 May 2026
- Last updated
- 1 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of the Safety and Efficacy of Intrathecal NTF001 Injection in the Treatment of Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT07618585
- Acronym / sponsor ID
- NTF-ALS / NTF-ALS
- Keywords
- Neuron-derived Neurotrophic Factor, Amyotrophic Lateral Sclerosis, NTF001, Gene Therapy, Adeno-associated Virus, Intrathecal Injection
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Ruijin HospitalUniversity, hospital or other
- Study start
- 1 Apr 2026 (actual)
- Primary completion
- 31 Mar 2028 (estimated)
- Study completion
- 31 Mar 2028 (estimated)
- First posted
- 1 Jun 2026 (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 single-arm, open-label, early-phase clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of intrathecal NTF001 injection, an AAV-mediated human neuron-derived neurotrophic factor gene therapy, in patients with amyotrophic lateral sclerosis (ALS).
12 patients with ALS will be enrolled. Each participant will receive a single intrathecal administration of NTF001 and will be followed for 52 weeks after treatment. The primary outcome measures include treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). Secondary outcome measures include changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), quality-of-life assessments, and neurological function.
This study aims to provide preliminary clinical evidence regarding the safety and potential efficacy of intrathecal NTF001 injection in patients with ALS.
Detailed description
This is a single-arm, open-label, early-phase clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of NTF001 injection in patients with amyotrophic lateral sclerosis (ALS). NTF001 is an investigational AAV-mediated gene therapy product designed to express human neuron-derived neurotrophic factor (NDNF). The investigational product will be administered once by intrathecal injection.
12 patients with ALS will be enrolled in this study. All eligible participants will receive a single intrathecal administration of NTF001 and will undergo scheduled safety and efficacy assessments during a 52-week follow-up period after treatment.
The primary objective of this study is to assess the safety and tolerability of intrathecal NTF001 injection, primarily by evaluating the occurrence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), clinically significant laboratory abnormalities, vital signs, neurological examinations, and other safety-related findings.
The secondary objective is to explore the preliminary efficacy of NTF001 in patients with ALS. Efficacy assessments will include changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), quality-of-life assessments, neurological function, and other exploratory clinical indicators.
This study is expected to provide preliminary clinical evidence regarding the safety, tolerability, and potential therapeutic effects of intrathecal NTF001 injection in patients with ALS, and to support further clinical development of AAV-mediated neurotrophic factor gene therapy for ALS.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Participants will be assigned sequentially to different dose cohorts of NTF001 injection. Each participant will receive a single intrathecal administration and will be followed for 52 weeks after treatment.
| Arm | Type | Receives |
|---|---|---|
| Low-dose NTF001 | Experimental | Genetic: NTF001 InjectionParticipants in this cohort will receive a single intrathecal administration of low-dose NTF001 （2E+14 vg）injection. |
| High-dose NTF001 | Experimental | Genetic: NTF001 InjectionParticipants in this cohort will receive a single intrathecal administration of high-dose NTF001(4-5 E+14 vg) injection after safety evaluation of the low-dose cohort. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Voluntarily participate in this study and sign the informed consent form.
- Agree to comply with study procedures and cooperate with all study-related assessments throughout the study.
- Male or female patients aged 18 to 65 years.
- Meet the diagnostic and exclusion criteria for amyotrophic lateral sclerosis according to the Chinese Expert Consensus on the Diagnosis and Treatment of Amyotrophic Lateral Sclerosis 2022 issued by the Neurology Branch of the Chinese Medical Association.
- Have a history of amyotrophic lateral sclerosis of no more than 5 years.
Key exclusion criteria (22 listed)
- Mini-Mental State Examination (MMSE) score < 24.
- Patient Health Questionnaire-9 (PHQ-9) score ≥ 16.
- Abnormal liver or renal function, defined as AST or ALT > 1.5 × upper limit of normal (ULN), or serum creatinine (Cr) > 1.5 × ULN.
- Abnormal coagulation function or current use of anticoagulants.
- Positive infectious disease screening, including positive HBsAg or HBV-DNA, positive HCV-RNA, positive HIV test, or positive syphilis serology.
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 |
|---|---|---|
| NTF001 InjectionNTF001 is an investigational AAV-mediated gene therapy product designed to express human neuron-derived neurotrophic factor (NDNF). It will be administered once by intrathecal injection to patients with amyotrophic lateral sclerosis (ALS). Participants will receive NTF001 according to the assigned dose cohort and will be followed for safety, tolerability, and preliminary efficacy. | Genetic | High-dose NTF001, Low-dose NTF001 |
Primary outcome
Incidence of Treatment-Emergent Adverse Events and Serious Adverse Events
Time frame: From administration of NTF001 through Week 52
Safety and tolerability will be assessed by evaluating the incidence, severity, seriousness, and relationship to the investigational product of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). Safety assessments will also include clinical laboratory tests, vital signs, physical examinations, neurological examinations, and other clinically significant safety findings.
4 secondary outcomes
Change From Baseline in ALS Functional Rating Scale-Revised Score
Time frame: Baseline through Week 52
Change From Baseline in ALS Quality of Life Assessment
Time frame: Baseline through Week 52
Change From Baseline in ALSAQ-40 Score
Time frame: Baseline through Week 52
Change From Baseline in Norris Scale Score
Time frame: Baseline through Week 52
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 1 Jun 2026.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
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 NTF001 Injection in people with ALS - Amyotrophic Lateral Sclerosis and ALS (Amyotrophic Lateral Sclerosis), run by Ruijin Hospital 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 5 inclusion and 22 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: under way, not looking for participants, as of the sponsor's last update on 1 Jun 2026. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events and Serious Adverse Events (time frame: From administration of NTF001 through Week 52). 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 1 Jun 2026; AzTrials last refreshed its copy on 8 Nov 2026.