Rozanolixizumab for Myasthenia Gravis

(roMyG Trial)

Recruiting at 6 trial locations

Overseen By UCB Cares

Age: < 18

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: UCB Biopharma SRL

Must be taking: Pyridostigmine, Corticosteroids

Must not be taking: Anti-FcRn medications

Disqualifiers: Severe weakness, Thymoma, Infection, others

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

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 6 weeks

12 Participants Needed

Trial Summary

What is the purpose of this trial?

The purpose of the study is to assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥2 to \<18 years with generalized Myasthenia Gravis (gMG).

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications. It mentions that participants should have received existing treatments for Myasthenia Gravis before joining, so you might be able to continue them.

What data supports the effectiveness of the drug Rozanolixizumab for treating myasthenia gravis?

Rozanolixizumab has been shown to be effective in treating generalized myasthenia gravis, as it was approved in the USA for adults with specific antibodies related to the condition. Clinical trials have demonstrated its safety and efficacy, providing a new option for patients who may not respond well to traditional treatments. ^{1 2 3 4 5}

Is Rozanolixizumab safe for humans?

Rozanolixizumab has been studied for safety in patients with generalized myasthenia gravis, and it has been approved in the USA for this condition, indicating it has an acceptable safety profile. However, like many treatments, it may have side effects, and its safety is still being evaluated for other conditions. ^{2 3 4 5 6}

How is the drug Rozanolixizumab different from other treatments for myasthenia gravis?

Rozanolixizumab is unique because it is the first drug approved in the USA for treating generalized myasthenia gravis in adults who are positive for either anti-acetylcholine receptor or anti-muscle-specific kinase antibodies, and it works by blocking the neonatal Fc receptor, which is a novel approach compared to other treatments. ^{2 3 4 6 7}

Research Team

UCB Cares

Principal Investigator

001 844 599 2273

Eligibility Criteria

This trial is for children and teens aged 2 to less than 18 with moderate to severe generalized Myasthenia Gravis (gMG). They should have tried other treatments like pyridostigmine or corticosteroids without enough improvement. Participants must have confirmed gMG with specific autoantibodies.

Inclusion Criteria

My gMG symptoms are getting worse and I need more treatment.

I am between 12 and 17 years old.

My myasthenia gravis is moderate to severe but not requiring a tube for breathing.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive subcutaneous administration of rozanolixizumab for 6 weeks

6 weeks

Weekly visits for administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

up to 12 weeks

Regular visits for safety assessments

Treatment Details

Interventions

• Rozanolixizumab

Trial Overview The study tests the safety of rozanolixizumab, given as a subcutaneous injection, in young patients with gMG. It aims to see how well they tolerate this medication.

Participant Groups

Group I: rozanolixizumab Experimental Treatment 1 Intervention

Study participants will receive pre-defined doses of rozanolixizumab for 6 weeks.

Rozanolixizumab is already approved in United States, European Union, Japan for the following indications:

Approved in United States as Rystiggo for:

• Generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

Approved in European Union as Rystiggo for:

• Generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

Approved in Japan as Rystiggo for:

• Generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

Who Is Running the Clinical Trial?

UCB Biopharma SRL

Lead Sponsor

Trials

118

Recruited

23,200+

Jean-Christophe Tellier

UCB Biopharma SRL

Chief Executive Officer since 2015

MD from University of Reims Champagne-Ardenne, Rheumatology specialization from University of Paris V, Executive business programs at Harvard and INSEAD

Dr. Iris Loew-Friedrich

UCB Biopharma SRL

Chief Medical Officer since 2014

MD from University of Leuven, PhD in Medical Sciences from University of Leuven

Findings from Research

In a study of 77 patients with anti-muscle-specific kinase (MuSK) myasthenia gravis, rituximab treatment led to a significantly higher rate of favorable outcomes (58%) compared to controls (16%) after a median follow-up of over 3.5 years.

Rituximab-treated patients also required lower doses of prednisone (29% on 4.5 mg/day) compared to controls (74% on 13 mg/day), indicating that rituximab may reduce the need for other immunosuppressive therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rituximab as treatment for anti-MuSK myasthenia gravis: Multicenter blinded prospective review. Hehir, MK., Hobson-Webb, LD., Benatar, M., et al.[2018]

Rozanolixizumab is a monoclonal antibody that targets the neonatal Fc receptor (FcRn) and has been approved in the USA for treating generalized myasthenia gravis (gMG) in adults who test positive for specific antibodies (anti-AChR or anti-MuSK).

This approval marks rozanolixizumab as the first treatment option in the USA specifically for both anti-AChR and anti-MuSK antibody-positive gMG, with ongoing clinical development for other autoimmune conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rozanolixizumab: First Approval. Hoy, SM.[2023]

In a phase 2a trial involving 43 patients with generalized myasthenia gravis, rozanolixizumab showed a trend towards clinical benefit, particularly in secondary measures like the MG-Activities of Daily Living and MG-Composite scores, although the primary endpoint (QMG score) did not reach statistical significance.

Rozanolixizumab was generally well tolerated, with headache being the most common adverse event, occurring in 57% of patients receiving the treatment compared to 14% in the placebo group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Rozanolixizumab in Moderate to Severe Generalized Myasthenia Gravis: A Phase 2 Randomized Control Trial. Bril, V., Benatar, M., Andersen, H., et al.[2021]

In a phase 3 study involving 200 patients with generalized myasthenia gravis, rozanolixizumab significantly improved symptoms compared to placebo, with both 7 mg/kg and 10 mg/kg doses showing clinically meaningful reductions in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score.

Rozanolixizumab was generally well tolerated, with a high incidence of treatment-emergent adverse events (TEAEs) similar to placebo, indicating a favorable safety profile for this new treatment option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Bril, V., Drużdż, A., Grosskreutz, J., et al.[2023]

Ravulizumab demonstrated sustained efficacy in treating generalized myasthenia gravis over 60 weeks, with significant improvements in Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores for patients who continued treatment from the randomized controlled period.

The treatment was well tolerated with no reported cases of meningococcal infections, indicating a favorable safety profile for long-term use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term efficacy and safety of ravulizumab in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis: results from the phase 3 CHAMPION MG open-label extension. Meisel, A., Annane, D., Vu, T., et al.[2023]

Eculizumab, administered to 117 patients over a median of 22.7 months, demonstrated a long-term safety profile consistent with previous studies, with no reported cases of meningococcal infection.

The treatment significantly reduced myasthenia gravis exacerbation rates by 75% and maintained improvements in daily activities, muscle strength, and quality of life, with 56% of patients achieving minimal manifestations or pharmacological remission.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety and efficacy of eculizumab in generalized myasthenia gravis. Muppidi, S., Utsugisawa, K., Benatar, M., et al.[2020]

Rituximab (RTX) can effectively treat refractory myasthenia gravis, but in this case, the patient experienced prolonged depletion of CD20+ B cells for over three years after treatment, leading to significant health issues.

The patient developed persistent hypogammaglobulinemia and multiple opportunistic infections following RTX treatment, highlighting the risks associated with long-term B cell depletion, especially in the context of thymoma relapse.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Case report: Persistent hypogammaglobulinemia in thymoma-associated myasthenia gravis: the impact of rituximab or Good's syndrome? Ren, J., Wang, J., Liu, R., et al.[2023]

References

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

Rituximab as treatment for anti-MuSK myasthenia gravis: Multicenter blinded prospective review. [2018]

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

Rozanolixizumab: First Approval. [2023]

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

Efficacy and Safety of Rozanolixizumab in Moderate to Severe Generalized Myasthenia Gravis: A Phase 2 Randomized Control Trial. [2021]

4. England pubmed.ncbi.nlm.nih.gov

Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. [2023]

5. Germany pubmed.ncbi.nlm.nih.gov

Long-term efficacy and safety of ravulizumab in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis: results from the phase 3 CHAMPION MG open-label extension. [2023]

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

Long-term safety and efficacy of eculizumab in generalized myasthenia gravis. [2020]

7. Switzerland pubmed.ncbi.nlm.nih.gov

Case report: Persistent hypogammaglobulinemia in thymoma-associated myasthenia gravis: the impact of rituximab or Good's syndrome? [2023]