Neuromyelitis Optica Spectrum Disorder (NMOSD)Phase 2NCT07809724
Efficacy and Safety of Low-Dose IL-2 in Neuromyelitis Optica Spectrum Disorder
A Phase 2 study testing IL-2 (interleukin 2) in people with Neuromyelitis Optica Spectrum Disorder (NMOSD), run by Peking University People's Hospital at 1 site in China.
Trial summary
Interleukin-2 (IL-2) is an essential cytokine for T-cell proliferation. Low-dose IL-2 has been shown to increase the proportion of regulatory T cells (Tregs), negatively regulate effector T cells such as Th17 and Tfh cells, and improve the peripheral Treg/Th17 balance, thus restoring immune homeostasis. Read more about this study
- Who is it for?
- Ages 18 to 74 years
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorder (NMOSD)
- What is being tested?
- IL-2 (interleukin 2)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteChina
- Study dates
- 30 Aug 2026to 30 Dec 2028
- Phase
- Phase 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 74 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age: 18 to 74 years, inclusive at the time of informed consent.
- Diagnosed with NMOSD according to the 2015 international consensus diagnostic criteria for NMOSD.
- Expanded Disability Status Scale (EDSS) score between 0 and 6.5.
- For women of childbearing potential, contraception must be used for more than 2 weeks after meeting the inclusion criteria, and HCG must be negative at the time of enrollment.
- Able to provide written informed consent and demonstrate the ability and willingness to comply with the study protocol requirements.
Who cannot join
- Any prior history of receiving whole-body irradiation or bone marrow transplantation.
- Any treatment with investigational drugs within 3 months prior to baseline.
- Pregnancy or breastfeeding.
- Any surgical procedure within 4 weeks prior to baseline (except minor surgeries).
- Evidence of other demyelinating diseases or progressive multifocal leukoencephalopathy (PML).
- Evidence of severe uncontrolled comorbidities that may hinder participation.
Show all 14Show fewer
- Other neurological disorders, hematological / hematopoietic disorders, congenital/acquired severe immunodeficiencies.
- Heart failure (≥NYHA Class III), renal insufficiency, liver insufficiency, or respiratory failure.
- White blood cell count <3000/ml, lymphocyte count <1000/ml, platelet count <150,000/ml.
- Evidence of chronic active hepatitis B or C.
- Substance abuse or alcoholism history within 1 year prior to baseline.
- Evidence of active tuberculosis (excluding participants undergoing chemoprophylaxis for latent tuberculosis infection).
- Intolerance to IL-2: hypersensitivity reactions to the active substance or any of its excipients (e.g. shock, allergic reactions).
- Active suicidal ideation within the past 6 months before screening, or a history of suicide attempts within the past 3 years.
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
Department of Rheumatology and Immunology, Peking University People's Hospital
Beijing, Beijing Municipality, 100044
Jing He, MD and PhD, +86 010 88326452, hejing1105@gmail.com
Xue Li, +86 18810902920, lixue7976@163.com
About this study
Official title: Efficacy and Safety of Low-Dose IL-2 in Neuromyelitis Optica Spectrum Disorder: A Randomized, Double-Blind, Placebo-Controlled Phase II Trial
Interleukin-2 (IL-2) is an essential cytokine for T-cell proliferation. Low-dose IL-2 has been shown to increase the proportion of regulatory T cells (Tregs), negatively regulate effector T cells such as Th17 and Tfh cells, and improve the peripheral Treg/Th17 balance, thus restoring immune homeostasis. Studies have demonstrated a positive correlation between the Th17/Treg ratio and the severity of various autoimmune diseases. Clinically, low-dose IL-2 has been successfully used in the treatment of diseases such as systemic lupus erythematosus and Sjögren's syndrome. However, the efficacy and safety of IL-2 in neuromyelitis optica spectrum disorder (NMOSD) remain unknown. This clinical trial aims to investigate the safety and biological efficacy of IL-2 in treating NMOSD.
Read the detailed description
In the NMOSD-IL2 trial, 60 patients with NMOSD will participate in a randomized, double-blind, placebo-controlled clinical study. The participants will be allocated in a 1:1 ratio to the intervention and placebo groups, with 30 patients in each group. The intervention group will receive subcutaneous injections of IL-2 (1 MIU) every other day for 4 weeks, followed by twice-weekly injections for an additional 20 weeks. The placebo group will receive parallel dosing.
The primary efficacy criteria will be the percentage change in Treg levels relative to baseline at week 4, indicating the biological response to IL-2.
Secondary efficacy endpoints will include: (i) the maintenance of regulatory T cells during the 24 weeks treatment period with low-dose IL-2 compared to placebo, and (ii) disease activity and relapse rates, as assessed by clinical scores and MRI evaluations, in the IL-2 treatment group compared to the placebo group.
Expected impact: This trial will determine whether NMOSD responds to IL-2 therapy.
- Being tested
- Placebo, IL-2 (interleukin 2)
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
placebo s.c. injection every other day for the first 4 weeks and then twice a week for an additional 20 weeks. IL-2 (1 MIU) : placebo = 1:1
IL-2 (interleukin 2)Drug
Also called Human recombinant IL-2
IL-2 (1 MIU) s.c. injection every other day for the first 4 weeks and then twice a week for an additional 20 weeks. IL-2 (1 MIU) : placebo = 1:1
| Study group | Role | What they receive |
|---|---|---|
| Placebo Comparator | Placebo comparator | placebo s.c. injection every other day for the first 4 weeks and then twice a week for an additional 20 weeks Receives: Drug: Placebo |
| Active Comparator | Experimental | IL-2 (1 MIU) s.c. injection every other day for the first 4 weeks and then twice a week for an additional 20 weeks Receives: Drug: IL-2 (interleukin 2) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in the percentage of Tregs at week 4 compared to baseline (week 0)
expressed as a percentage of the total CD4+ cells
Measured: Week 4
Secondary outcomes (10)
Change in the percentage of Tregs at week 2 compared to baseline (week 0)
expressed as a percentage of the total CD4+ cells
Measured: Week 2
Change in the percentage of Tregs at week 24 compared to baseline (week 0)
expressed as a percentage of the total CD4+ cells
Measured: Week 8, 12, 16, 20, 24
Change of Th1, Th2, and Th17 cells to low-dose IL-2 compared to baseline (week 0)
expressed as a percentage of the total CD4+ cells
Measured: Week 2, 4, 8, 12, 16, 20, 24
Cumulative number of active MRI lesions and/or changes in orbital optic nerve MRI lesions
Measure the number of new gadolinium-enhancing lesions and new or enlarged T2 lesions using MRI.
Measured: Week 24
Change of cumulative total activity (CUA)
Cumulative number of new Gd-enhanced T1-weighted lesions and new or enlarged T2-weighted lesions, without repeat counting
Measured: Week 24
Change in the Expanded Disability Status Scale (EDSS) compared to baseline (week 0)
The Expanded Disability Status Scale (EDSS) ranges from 0 to 10, with higher scores indicating greater neurological disability and a worse outcome. A score of 0 indicates a normal neurological examination, whereas a score of 10 indicates death due to neurological disease. Change from baseline will be calculated as the score at each follow-up visit minus the baseline score; a negative change indicates improvement and a positive change indicates worsening.
Measured: Week 4, 8, 16, 24
Change in the Modified Rankin Scale (mRS) score compared to baseline (week 0)
The Modified Rankin Scale (mRS) ranges from 0 to 6, with higher scores indicating greater disability and a worse outcome. A score of 0 indicates no symptoms, whereas a score of 6 indicates death. Change from baseline will be calculated as the score at each follow-up visit minus the baseline score; a negative change indicates improvement and a positive change indicates worsening.
Measured: Week 4, 8, 16, 24
Percentage of patients with relapses
Measured: Week 24
Percentage of disease free patients, defined as those with no clinical symptoms and no active lesions on MRI
Measured: Week 24
Safety endpoints: IL-2-related adverse events
Assessment of IL-2-related adverse events (AEs) and recording of the AE incidence.
Measured: Week 2, 4, 8, 12, 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2026
- Study start30 Aug 2026 (estimated)
- Primary completion15 Aug 2028 (estimated)
- Study completion30 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis Optica Spectrum Disorder (NMOSD)Studies of the same condition, open and under way.
- More trials run by Peking University People's 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Peking University First Hospital
- Responsible party
- Principal investigator, Jing He, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07809724
- Sponsor's protocol ID
- NMOSD-IL2
- Status last verified
- Aug 2026
- First submitted
- 23 Jan 2025
- Last updated
- 9 Sep 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Low-Dose IL-2 in Neuromyelitis Optica Spectrum Disorder: A Randomized, Double-Blind, Placebo-Controlled Phase II Trial
- ClinicalTrials.gov ID
- NCT07809724
- Acronym / sponsor ID
- NMOSD-IL2
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Collaborators
- Peking University First Hospital
- Study start
- 30 Aug 2026 (estimated)
- Primary completion
- 15 Aug 2028 (estimated)
- Study completion
- 30 Dec 2028 (estimated)
- First posted
- 9 Sep 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.
Interleukin-2 (IL-2) is an essential cytokine for T-cell proliferation. Low-dose IL-2 has been shown to increase the proportion of regulatory T cells (Tregs), negatively regulate effector T cells such as Th17 and Tfh cells, and improve the peripheral Treg/Th17 balance, thus restoring immune homeostasis. Studies have demonstrated a positive correlation between the Th17/Treg ratio and the severity of various autoimmune diseases. Clinically, low-dose IL-2 has been successfully used in the treatment of diseases such as systemic lupus erythematosus and Sjögren's syndrome. However, the efficacy and safety of IL-2 in neuromyelitis optica spectrum disorder (NMOSD) remain unknown. This clinical trial aims to investigate the safety and biological efficacy of IL-2 in treating NMOSD.
Detailed description
In the NMOSD-IL2 trial, 60 patients with NMOSD will participate in a randomized, double-blind, placebo-controlled clinical study. The participants will be allocated in a 1:1 ratio to the intervention and placebo groups, with 30 patients in each group. The intervention group will receive subcutaneous injections of IL-2 (1 MIU) every other day for 4 weeks, followed by twice-weekly injections for an additional 20 weeks. The placebo group will receive parallel dosing.
The primary efficacy criteria will be the percentage change in Treg levels relative to baseline at week 4, indicating the biological response to IL-2.
Secondary efficacy endpoints will include: (i) the maintenance of regulatory T cells during the 24 weeks treatment period with low-dose IL-2 compared to placebo, and (ii) disease activity and relapse rates, as assessed by clinical scores and MRI evaluations, in the IL-2 treatment group compared to the placebo group.
Expected impact: This trial will determine whether NMOSD responds to IL-2 therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo Comparator | Placebo comparator | Drug: Placeboplacebo s.c. injection every other day for the first 4 weeks and then twice a week for an additional 20 weeks |
| Active Comparator | Experimental | Drug: IL-2 (interleukin 2)IL-2 (1 MIU) s.c. injection every other day for the first 4 weeks and then twice a week for an additional 20 weeks |
Who may qualify
- Ages
- 18 to 74 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age: 18 to 74 years, inclusive at the time of informed consent.
- Diagnosed with NMOSD according to the 2015 international consensus diagnostic criteria for NMOSD.
- Expanded Disability Status Scale (EDSS) score between 0 and 6.5.
- For women of childbearing potential, contraception must be used for more than 2 weeks after meeting the inclusion criteria, and HCG must be negative at the time of enrollment.
- Able to provide written informed consent and demonstrate the ability and willingness to comply with the study protocol requirements.
Key exclusion criteria (14 listed)
- Any prior history of receiving whole-body irradiation or bone marrow transplantation.
- Any treatment with investigational drugs within 3 months prior to baseline.
- Pregnancy or breastfeeding.
- Any surgical procedure within 4 weeks prior to baseline (except minor surgeries).
- Evidence of other demyelinating diseases or progressive multifocal leukoencephalopathy (PML).
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 |
|---|---|---|
| Placeboplacebo s.c. injection every other day for the first 4 weeks and then twice a week for an additional 20 weeks. IL-2 (1 MIU) : placebo = 1:1 | Drug | Placebo Comparator |
| IL-2 (interleukin 2)Also: Human recombinant IL-2IL-2 (1 MIU) s.c. injection every other day for the first 4 weeks and then twice a week for an additional 20 weeks. IL-2 (1 MIU) : placebo = 1:1 | Drug | Active Comparator |
Primary outcome
Change in the percentage of Tregs at week 4 compared to baseline (week 0)
Time frame: Week 4
expressed as a percentage of the total CD4+ cells
10 secondary outcomes
Change in the percentage of Tregs at week 2 compared to baseline (week 0)
Time frame: Week 2
Change in the percentage of Tregs at week 24 compared to baseline (week 0)
Time frame: Week 8, 12, 16, 20, 24
Change of Th1, Th2, and Th17 cells to low-dose IL-2 compared to baseline (week 0)
Time frame: Week 2, 4, 8, 12, 16, 20, 24
Cumulative number of active MRI lesions and/or changes in orbital optic nerve MRI lesions
Time frame: Week 24
Change of cumulative total activity (CUA)
Time frame: Week 24
Change in the Expanded Disability Status Scale (EDSS) compared to baseline (week 0)
Time frame: Week 4, 8, 16, 24
Change in the Modified Rankin Scale (mRS) score compared to baseline (week 0)
Time frame: Week 4, 8, 16, 24
Percentage of patients with relapses
Time frame: Week 24
Percentage of disease free patients, defined as those with no clinical symptoms and no active lesions on MRI
Time frame: Week 24
Safety endpoints: IL-2-related adverse events
Time frame: Week 2, 4, 8, 12, 24
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 9 Sep 2026.
Related trials for Neuromyelitis Optica Spectrum Disorder (NMOSD)
Recruiting
Impact of a Structured Wellness Behavioral Intervention on Quality of Life in NMOSD
Neuromyelitis Optica Spectrum Disorder (NMOSD)
RecruitingObservational study
Observational Safety Study in Women With Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® During Pregnancy
Neuromyelitis Optica Spectrum Disorder, Pregnancy Related
RecruitingPhase 2
Efgartigimod for the Treatment of Acute Optic Neuritis
Optic Neuritis
Not yet recruitingPhase 1/2
A Safety and Efficacy Study Evaluating CTX112 in Subjects With Refractory Neurologic Autoimmune Disease
Progressive Multiple Sclerosis (PMS), Neuromyelitis Optica
RecruitingObservational study
Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies
Neuromyelitis Optica Spectrum Disorder, NMOSD
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 2 study testing IL-2 (interleukin 2) in people with Neuromyelitis Optica Spectrum Disorder (NMOSD), run by Peking University People's 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 14 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: opening soon, as of the sponsor's last update on 9 Sep 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. 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 phase mean?
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 Jing He (+86 010 88326452, hejing1105@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in the percentage of Tregs at week 4 compared to baseline (week 0) (time frame: Week 4). 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 9 Sep 2026.