Satralizumab for Neuromyelitis Optica Spectrum Disorder

(SAkuraSun Trial)

Recruiting at 17 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

Trial Summary

What is the purpose of this trial?

This study will primarily evaluate the pharmacokinetics of satralizumab in pediatric patients aged 2-11 years with anti-aquaporin-4 (AQP4) antibody seropositive neuromyelitis optica spectrum disorder (NMOSD). Efficacy, safety, tolerability, and pharmacodynamics will be evaluated in a descriptive manner, given the small number of patients who will be enrolled in this study.

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.

What data supports the effectiveness of the drug Satralizumab for treating neuromyelitis optica spectrum disorder?

Satralizumab has been shown to reduce the risk of relapse in patients with neuromyelitis optica spectrum disorder (NMOSD) in several studies, including the SAkuraSky and SAkuraStar trials. It is approved for use in patients who are aquaporin-4 immunoglobulin G seropositive, and it has been well tolerated with common side effects like infection and headache. ^{1 2 3 4 5}

Is satralizumab safe for humans?

Satralizumab has been generally well tolerated in clinical trials for neuromyelitis optica spectrum disorder, with common side effects including infections, headaches, joint pain, low white blood cell count, high cholesterol, and reactions at the injection site. ^{1 2 3 4 5}

What makes the drug Satralizumab unique for treating neuromyelitis optica spectrum disorder?

Satralizumab is unique because it is the first IL-6 receptor blocker approved for treating AQP4-IgG-seropositive patients with NMOSD, offering the convenience of subcutaneous administration and being the only targeted treatment approved for adolescent patients. ^{2 3 4 6 7}

Research Team

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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 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 (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]

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, 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]

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]

References

1. England 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. [2021]

2. New Zealand pubmed.ncbi.nlm.nih.gov

Satralizumab: First Approval. [2021]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Satralizumab: A Review in Neuromyelitis Optica Spectrum Disorder. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Satralizumab for the Treatment of Neuromyelitis Optica Spectrum Disorders. [2021]

5. United States pubmed.ncbi.nlm.nih.gov

Long-term Efficacy of Satralizumab in AQP4-IgG-Seropositive Neuromyelitis Optica Spectrum Disorder From SAkuraSky and SAkuraStar. [2023]

6. United States pubmed.ncbi.nlm.nih.gov

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

7. England pubmed.ncbi.nlm.nih.gov

Satralizumab in the treatment of neuromyelitis optica spectrum disorder. [2021]