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
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.
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- 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.
- 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