Self-Administered Rozanolixizumab for Myasthenia Gravis

Recruiting at 28 trial locations

Overseen By UCB Cares

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: UCB Biopharma SRL

Must be taking: Rozanolixizumab

Must not be taking: Live vaccines

Disqualifiers: Tuberculosis, Severe infection, Myasthenic crisis, 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 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

62 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial aims to see if patients with generalized Myasthenia Gravis can successfully learn to inject themselves with rozanolixizumab, a medication that helps reduce muscle weakness by targeting harmful antibodies. Rozanolixizumab is being tested to see how well it works and how safe it is for treating generalized myasthenia gravis.

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

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

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

Rozanolixizumab has been approved in the USA for treating generalized myasthenia gravis in adults who are positive for specific antibodies, based on its ability to block a receptor involved in the disease. Clinical trials have shown it to be effective and safe for this condition, offering a new option for patients who may not respond well to traditional treatments. ^{1 2 3 4 5}

What is known about the safety of Rozanolixizumab for human use?

Rozanolixizumab has been studied for safety in patients with generalized myasthenia gravis, showing an acceptable safety profile in clinical trials. It is a treatment option for autoimmune diseases and has been approved in the USA for specific types of myasthenia gravis. ^{1 2 3 6 7}

How is the drug Rozanolixizumab unique for treating 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. ^{1 2 3 6 8}

Research Team

UCB Cares

Principal Investigator

001 844 599 2273

Eligibility Criteria

This trial is for individuals with generalized Myasthenia Gravis (gMG) who are capable of self-administering medication and weigh at least 35 kg. Participants must be willing to use a syringe driver or manual push method after training. Those with severe muscle weakness, hypersensitivity to similar drugs, active infections, recent live vaccinations, or certain medical histories cannot join.

Inclusion Criteria

I am willing and able to administer my own treatment at home.

My doctor thinks I should try the rozanolixizumab treatment mentioned in this study.

I have been diagnosed with generalized Myasthenia Gravis.

Exclusion Criteria

I have not had a live vaccine recently and do not plan to during the study.

I haven't had a serious infection or been hospitalized for one in the last 6 weeks.

I am allergic to certain medication ingredients, including polysorbate 80 and L-proline.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Training

Participants are trained in the self-administration technique using the syringe driver and manual push methods

6 weeks

Weekly visits

Self-administration Period 1

Participants self-administer rozanolixizumab using the syringe driver method

6 weeks

Weekly visits

Self-administration Period 2

Participants self-administer rozanolixizumab using the manual push method

6 weeks

Weekly visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

Treatment Details

Interventions

• Rozanolixizumab

Trial Overview The study tests if patients with gMG can successfully self-administer the drug Rozanolixizumab using a syringe driver or by hand. The focus is on patient independence in managing their treatment after receiving proper instruction.

Participant Groups

Group I: Rozanolixizumab Sequence 2: Manual Push - Syringe Driver Experimental Treatment 1 Intervention

Study participants will receive predefined weekly doses of rozanolixizumab for 18 weeks.

Group II: Rozanolixizumab Sequence 1: Syringe Driver - Manual Push Experimental Treatment 1 Intervention

Study participants will receive predefined weekly doses of rozanolixizumab for 18 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

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]

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]

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]

Zilucoplan, a C5 inhibitor, has been approved in Japan, the USA, and the EU for treating generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive and have not responded adequately to other treatments.

This medication represents a significant advancement in gMG treatment options, particularly for patients who do not respond well to steroids or immunosuppressants, and is currently under review in Australia and Canada.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Zilucoplan: First Approval. Shirley, M.[2023]

References

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

Rozanolixizumab: First Approval. [2023]

2. 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]

3. 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]

4. Netherlands pubmed.ncbi.nlm.nih.gov

Efficacy of Rituximab in Refractory Generalized anti-AChR Myasthenia Gravis. [2018]

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

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

6. 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]

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

Rituximab Therapy in the Treatment of Juvenile Myasthenia Gravis: The French Experience. [2022]

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

Zilucoplan: First Approval. [2023]