Self-Administered Rozanolixizumab for Myasthenia Gravis

No longer recruiting at 32 trial locations
UC
Overseen ByUCB Cares
Age: 18+
Sex: Any
Trial Phase: Phase 3
Sponsor: UCB Biopharma SRL
Must be taking: Rozanolixizumab
No Placebo GroupAll 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 DataThis treatment has passed at least one previous human trial
Approved in 3 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called rozanolixizumab for individuals with generalized Myasthenia Gravis (gMG), a condition that causes muscle weakness. The main goal is to determine if participants can successfully self-administer the medication at home using two methods: a syringe driver and a manual push. Participants will receive weekly doses for 18 weeks. This trial suits those with gMG who feel comfortable managing their medication at home. As a Phase 3 trial, it represents the final step before potential FDA approval, offering participants a chance to contribute to the advancement of a promising treatment.

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.

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

Research has shown that rozanolixizumab is generally safe for people with generalized myasthenia gravis (gMG). In studies, patients received weekly treatments for up to a year, and most tolerated it well without major issues. The most common minor side effects included headaches and injection site reactions. Importantly, the FDA has already approved rozanolixizumab for treating gMG, indicating a strong safety record for this condition.12345

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

Rozanolixizumab is unique because it offers a new approach for treating myasthenia gravis by targeting the neonatal Fc receptor (FcRn), which plays a role in the degradation of antibodies. Unlike traditional treatments like cholinesterase inhibitors, corticosteroids, or immunosuppressants, rozanolixizumab specifically aims to reduce the levels of harmful antibodies without broadly suppressing the immune system. Researchers are excited about this treatment because its targeted action could lead to fewer side effects and a more efficient alleviation of symptoms. Additionally, the option for self-administration using syringe drivers or manual push methods could greatly enhance convenience and quality of life for patients.

What evidence suggests that rozanolixizumab might be an effective treatment for Myasthenia Gravis?

In earlier studies, rozanolixizumab showed promising results for treating generalized Myasthenia Gravis (gMG). Research indicates that patients experienced noticeable improvements in their symptoms with this treatment. Clinical trials demonstrate that it works quickly, providing significant symptom relief for many patients. Additionally, its long-term safety and tolerability have been well-documented, suggesting it is a reliable option for ongoing use. Overall, strong clinical evidence supports rozanolixizumab as a hopeful treatment for those with gMG.

In this trial, participants will receive rozanolixizumab through two different administration sequences: one group will use a syringe driver followed by manual push, while the other group will use manual push followed by a syringe driver.45678

Who Is on the Research Team?

UC

UCB Cares

Principal Investigator

001 844 599 2273

Are You a Good Fit for This Trial?

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.
See 2 more

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.
See 3 more

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: Rozanolixizumab Sequence 2: Manual Push - Syringe DriverExperimental Treatment1 Intervention
Group II: Rozanolixizumab Sequence 1: Syringe Driver - Manual PushExperimental Treatment1 Intervention

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

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Approved in United States as Rystiggo for:
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Approved in European Union as Rystiggo for:
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Approved in Japan as Rystiggo for:

Find a Clinic Near You

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

Published Research Related to This Trial

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

Citations

1.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/40105996/
the randomized open-label extension MG0004 studyChronic weekly rozanolixizumab for up to 52 infusions was generally well tolerated, and clinically relevant improvements across MG-specific outcomes were ...
NCT03052751 | Study to Test the Safety, Tolerability and ...The purpose of the study is to evaluate the clinical efficacy of UCB7665 as a chronic-intermittent treatment in subjects with generalized myasthenia gravis ...
Clinical Trial Data For RYSTIGGO® (rozanolixizumab-noli)Rapid efficacy in action for a broad population of adults with gMG1,2*. In a clinical study, RYSTIGGO demonstrated statistically significant improvements vs ...
UCB to unveil new data for RYSTIGGO® (rozanolixizumab ...Data from a further analysis of the Phase 3 MycarinG study explores the long-term safety of cyclic rozanolixizumab treatment in patients with ...
Rozanolixizumab (Rystiggo®)Myasthenia Gravis, A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia ...
Rystiggo - accessdata.fda.govMedian MG-ADL total score was 8, and the median Quantitative Myasthenia Gravis (QMG) total score was 15. The majority of patients, 89.5% (n=179) were positive ...
7.rystiggo.comrystiggo.com/
Discover RYSTIGGO® (rozanolixizumab-noli) For gMGRYSTIGGO is the first FDA-approved treatment for both anti-AChR and anti-MuSK antibody-positive adults with generalized myasthenia gravis (gMG).
the randomized open-label extension MG0004 studyChronic weekly rozanolixizumab for up to 52 infusions was generally well tolerated, and clinically relevant improvements across MG-specific outcomes were ...
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