Satralizumab for Neuromyelitis Optica Spectrum Disorder

(SAkuraSun Trial)

Recruiting at 22 trial locations

Overseen By Global Medical Information

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Hoffmann-La Roche

Must be taking: Immunosuppressants

Disqualifiers: Pregnancy, Infections, Hepatitis, Tuberculosis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 5 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Ongoing

8 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness and safety of satralizumab, an immunotherapy drug, for children aged 2-11 with neuromyelitis optica spectrum disorder (NMOSD), a condition affecting the nerves and eyes. The drug will be administered in varying doses based on the child's weight to assess its effects. Children who have experienced at least one NMOSD attack in the past year and have stable conditions may qualify for this study. As a Phase 3 trial, this study serves as the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

Do I need to stop my current medications to join the trial?

The trial requires that if you are on a baseline immunosuppressant treatment, you must continue it at a stable dose for at least 4 weeks before starting the trial. The protocol does not specify about other medications.

Is there any evidence suggesting that satralizumab is likely to be safe for humans?

Research has shown that satralizumab, a treatment for neuromyelitis optica spectrum disorder (NMOSD), is generally safe for patients. In past studies, most participants did not experience serious side effects. The most common side effects were mild, such as headaches and joint pain, while serious side effects were rare. However, satralizumab is not recommended for individuals with certain conditions, like an active hepatitis B infection.

This treatment has been tested in adults and already has FDA approval for this condition, indicating confidence in its safety. The current trial aims to explore its effects on younger patients, but existing data provides a reassuring safety record for potential participants. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for NMOSD?

Unlike the standard treatments for Neuromyelitis Optica Spectrum Disorder (NMOSD), which typically include immunosuppressive therapies like rituximab or azathioprine, Satralizumab offers a unique approach by targeting and inhibiting the interleukin-6 (IL-6) receptor. This mechanism helps reduce the inflammation that characterizes NMOSD attacks. Researchers are excited about Satralizumab because it is administered subcutaneously, which could improve convenience and adherence compared to intravenous options. Additionally, its targeted action on the IL-6 pathway may provide a more tailored treatment, potentially leading to better outcomes for patients.

What evidence suggests that satralizumab might be an effective treatment for neuromyelitis optica spectrum disorder?

Research shows that satralizumab helps reduce relapses in people with neuromyelitis optica spectrum disorder (NMOSD). In this trial, participants will receive satralizumab based on their body weight, with different dosing schedules for each cohort. Studies have found that using satralizumab alongside other immune-suppressing treatments significantly lowers the chance of a relapse compared to a placebo. While it does not seem to affect pain or fatigue, it helps prevent relapses, which is crucial for managing the disease. About 70% of people with untreated NMOSD experience relapses within two years, highlighting the need for effective treatments like satralizumab. Long-term studies confirm its safety and effectiveness for NMOSD patients. ^{2 6 7 8 9}

Who Is on the Research Team?

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Are You a Good Fit for This Trial?

This trial is for children aged 2-11 with Neuromyelitis Optica Spectrum Disorder (NMOSD) who are positive for anti-aquaporin-4 antibodies. They must weigh at least 10 kg, have a certain level of disability or less, and be neurologically stable for over a month before the trial. Girls able to have children must avoid pregnancy. Those on immunosuppressants need stable doses for 4 weeks.

Inclusion Criteria

For female patients of childbearing potential (postmenarchal): agreement to either remain completely abstinent (refrain from heterosexual intercourse) or to use a reliable means of contraception

I am between 2 and 11 years old.

I have been on a stable dose of my immunosuppressant for at least 4 weeks.

Exclusion Criteria

I currently have or recently had a serious infection.

I have untreated TB, whether it's showing symptoms or not.

You have ongoing hepatitis B or C.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Satralizumab is administered subcutaneously at Weeks 0, 2, 4, and every 4 weeks thereafter for Cohorts 2 and 3, and every 6 weeks for Cohort 1

Ongoing

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Satralizumab

Trial Overview The study tests Satralizumab in young NMOSD patients to understand how the drug moves through their bodies (pharmacokinetics). It also looks at how well it works (efficacy), its safety, tolerability, and effects on the disease process (pharmacodynamics) in a small patient group.

How Is the Trial Designed?

Group I: Cohort 3 Participants with body weight ≥40kg Experimental Treatment 1 Intervention

Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.

Group II: Cohort 2 Participants with body weight ≥20kg to <40kg Experimental Treatment 1 Intervention

Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.

Group III: Cohort 1: Participants with body weight ≥10kg to <20kg Experimental Treatment 1 Intervention

Satralizumab will be administered SC Q6W in a cohort of at least 2 evaluable patients

Satralizumab is already approved in United States, European Union, Canada, Japan, Switzerland for the following indications:

Approved in United States as Enspryng for:

• Neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive

Approved in European Union as Enspryng for:

• Neuromyelitis optica spectrum disorder (NMOSD)

Approved in Canada as Enspryng for:

• Neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive

Approved in Japan as Enspryng for:

• Neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive

Approved in Switzerland as Enspryng for:

• Neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive

Who Is Running the Clinical Trial?

Hoffmann-La Roche

Lead Sponsor

Trials

2,482

Recruited

1,107,000+

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Avastin, Herceptin, Rituxan, Accu-Chek

Dr. Levi Garraway

Hoffmann-La Roche

Chief Medical Officer since 2019

MD from the University of Basel

Dr. Thomas Schinecker

Hoffmann-La Roche

Chief Executive Officer since 2023

PhD in Molecular Biology from New York University

Published Research Related to This Trial

Satralizumab, an anti-IL-6 receptor monoclonal antibody, received its first global approval in June 2020 for treating neuromyelitis optica spectrum disorder (NMOSD) in patients aged 12 and older who are AQP4-IgG seropositive, based on positive results from two pivotal phase III trials.

The drug has since been approved in multiple countries, including Japan, Switzerland, and the USA, and is currently under review in the EU, highlighting its growing acceptance as a treatment option for NMOSD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Satralizumab: First Approval. Heo, YA.[2021]

Satralizumab, an approved treatment for Neuromyelitis optica spectrum disorder (NMOSD), works by targeting the IL-6 receptor, inhibiting both pro-inflammatory and anti-inflammatory effects of IL-6.

Sgp130FC (Olamkicept) is a new monoclonal antibody that selectively blocks only the pro-inflammatory pathway of IL-6, potentially offering a more targeted and effective approach for treating NMOSD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Olamkicept(sgp130Fc): The missing trial in Neuromyelitis Optica Spectrum Disorder. Magro, G.[2022]

Satralizumab, a monoclonal antibody targeting the IL-6 receptor, has been shown to significantly reduce the risk of relapse in patients with AQP4 seropositive neuromyelitis optica spectrum disorder (NMOSD) based on two Phase III trials.

The treatment demonstrated good tolerability, making it a promising option for managing NMOSD, a condition that previously had no approved medications until 2019.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Satralizumab in the treatment of neuromyelitis optica spectrum disorder. Duchow, A., Bellmann-Strobl, J.[2021]

Satralizumab, a monoclonal antibody targeting the interleukin-6 receptor, has been shown to significantly reduce relapse rates in patients with neuromyelitis optica spectrum disorders (NMOSDs), with a 30% relapse rate compared to 50% for placebo in monotherapy, and 20% compared to 43% for placebo when combined with immunosuppressants.

The treatment is generally well tolerated, with injection-related reactions being the most common side effect, indicating that satralizumab could be a safe and effective option for managing NMOSD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Satralizumab for the Treatment of Neuromyelitis Optica Spectrum Disorders. Gao, Y., Zhang, B., Yang, J.[2021]

Satralizumab (Enspryng®) is an effective treatment for neuromyelitis optica spectrum disorder (NMOSD) in AQP4-IgG seropositive patients, significantly reducing relapse risk in phase III trials compared to placebo.

The treatment is well tolerated, with common side effects including infections and headaches, and it is notable for being the first IL-6 receptor blocker approved in the EU for this condition, including for adolescents.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Satralizumab: A Review in Neuromyelitis Optica Spectrum Disorder. Fung, S., Shirley, M.[2023]

In a phase 3 trial involving 95 participants with neuromyelitis optica spectrum disorder (NMOSD), satralizumab monotherapy significantly reduced the risk of relapse compared to placebo, with a hazard ratio of 0.45, indicating nearly half the relapse rate.

The safety profile of satralizumab was favorable, with similar rates of adverse events between the satralizumab and placebo groups, suggesting it is a safe treatment option for NMOSD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of satralizumab monotherapy in neuromyelitis optica spectrum disorder: a randomised, double-blind, multicentre, placebo-controlled phase 3 trial. Traboulsee, A., Greenberg, BM., Bennett, JL., et al.[2021]

Satralizumab, an interleukin 6 receptor inhibitor, has shown long-term efficacy in preventing relapses in patients with aquaporin-4-immunoglobulin G (AQP4-IgG+) neuromyelitis optica spectrum disorder (NMOSD), with 71% of patients remaining free from protocol-defined relapse after over 3.5 years of treatment.

In a study involving 111 patients, satralizumab demonstrated a consistently low annualized relapse rate (ARR) of 0.12 in one study and 0.08 in another, indicating its effectiveness in maintaining disease stability over time.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term Efficacy of Satralizumab in AQP4-IgG-Seropositive Neuromyelitis Optica Spectrum Disorder From SAkuraSky and SAkuraStar. Kleiter, I., Traboulsee, A., Palace, J., et al.[2023]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT02073279

NCT02073279 | Efficacy and Safety Study of Satralizumab ... The objectives of this study are to evaluate the efficacy, safety, pharmacodynamic, pharmacokinetic and immunogenic profiles of satralizumab in participants ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10978816/

Review of Satralizumab for Neuromyelitis Optica Spectrum ... Approximately 70% of NMOSD patients experienced relapse within two years of disease without effective preventive therapy, and it is a matter of ...

3. nejm.org nejm.org/doi/full/10.1056/NEJMoa1901747

Trial of Satralizumab in Neuromyelitis Optica Spectrum ... Satralizumab added to immunosuppressant treatment led to a lower risk of relapse than placebo but did not differ from placebo in its effect on pain or fatigue.

4. neurology.org neurology.org/doi/10.1212/NXI.0000000000200450

Long-Term Efficacy and Safety of Satralizumab in Patients ... This study provides Class IV evidence that SAT is safe and effective in patients with NMOSD. Introduction. Neuromyelitis optica spectrum ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT02028884

NCT02028884 | Efficacy and Safety Study of Satralizumab ... The objective of this study is to evaluate the efficacy, safety, pharmacodynamic, pharmacokinetic, and immunogenic profiles of satralizumab, compared with ...

6. enspryng-hcp.com enspryng-hcp.com/safety/important-safety-information.html

Important Safety Information | ENSPRYNG® (satralizumab) ENSPRYNG is contraindicated in patients with a known hypersensitivity to satralizumab or any of the inactive ingredients, an active hepatitis B infection, or ...

7. enspryng.com enspryng.com/

ENSPRYNG® (satralizumab) | Neuromyelitis Optica Spectrum ... ENSPRYNG is a prescription medicine used to treat neuromyelitis optica spectrum disorder (NMOSD) in adults who are aquaporin-4 (AQP4) antibody positive.

8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7935419/

Safety and efficacy of satralizumab monotherapy in ... Satralizumab, a humanised monoclonal antibody targeting the interleukin-6 receptor, reduced the risk of relapse in patients with neuromyelitis optica spectrum ...

9. sciencedirect.com sciencedirect.com/science/article/pii/S2211034822005338

Long-term safety of satralizumab in neuromyelitis optica ... This analysis evaluated long-term safety findings from the SAkuraSky and SAkuraStar studies with satralizumab in patients with neuromyelitis optica spectrum ...