Rozanolixizumab for Myasthenia Gravis

(roMyG Trial)

Recruiting at 14 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called rozanolixizumab (Rystiggo) for children and teens with generalized Myasthenia Gravis (gMG), a condition that causes muscle weakness. The goal is to determine if this treatment is safe and well-tolerated when administered under the skin. It targets those who haven't found sufficient relief from their current gMG medications. Participants must have a confirmed gMG diagnosis and require additional support in managing their symptoms. As a Phase 2, Phase 3 trial, this research measures the treatment's effectiveness in an initial, smaller group and represents the final step before FDA approval, offering hope for improved symptom management.

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.

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

Research has shown that rozanolixizumab is generally safe and well-tolerated. In studies with this treatment, no deaths occurred. Participants experienced significant symptom improvements during and after treatment. Another study found that weekly doses of rozanolixizumab were well-tolerated over an extended period, with patients seeing meaningful improvements in their condition. This suggests that the treatment might be safe for people with myasthenia gravis, based on current evidence. ^{1 2 3 4 5}

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

Unlike the standard treatments for myasthenia gravis, such as acetylcholinesterase inhibitors, immunosuppressants, and monoclonal antibodies, rozanolixizumab offers a fresh approach by targeting the neonatal Fc receptor (FcRn). This mechanism helps reduce harmful antibodies that attack the neuromuscular junction, which is a key issue in myasthenia gravis. Researchers are excited because rozanolixizumab could provide a more targeted and potentially faster-acting option with fewer side effects, offering hope for patients who may not respond well to existing therapies.

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

Research shows that rozanolixizumab, which participants in this trial will receive, may help treat generalized Myasthenia Gravis (gMG). In earlier studies, patients with gMG experienced significant improvements after receiving six weekly under-the-skin infusions of this treatment. Rozanolixizumab lowers the levels of harmful IgG autoantibodies, proteins in the blood that mistakenly attack the body's own cells. This mechanism is similar to plasma exchange, a known effective treatment. One study found it reduced these antibodies by up to 78%, easing gMG symptoms. This evidence suggests that rozanolixizumab could be a useful option for managing this condition. ^{2 4 6 7 8}

Who Is on the Research Team?

UCB Cares

Principal Investigator

001 844 599 2273

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

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]

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]

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]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11923026/

the randomized open-label extension MG0004 study In the Phase 3 MycarinG study (NCT03971422), six once-weekly subcutaneous infusions of rozanolixizumab significantly improved myasthenia gravis ...

2. rystiggohcp.com rystiggohcp.com/clinical-trial

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 ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03052751

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 ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12045563/

Rozanolixizumab for Myasthenia Gravis - PubMed Central - NIH This results in a rapid reduction of up to 78% in circulating pathogenic IgG autoantibodies, offering efficacy comparable to plasma exchange. It ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT04650854

NCT04650854 | A Study to Evaluate Rozanolixizumab in ... The purpose of this study is to assess the safety, tolerability and efficacy of additional 6-week treatment cycles with rozanolixizumab in study participants ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37059507/

Safety and efficacy of rozanolixizumab in patients ... - PubMed No deaths occurred. Interpretation: Rozanolixizumab showed clinically meaningful improvements in patient-reported and investigator-assessed outcomes in patients ...

7. ucbcompass.com ucbcompass.com/sites/default/files/2024-11/ANA%20Long-term%20safety%20of%20rozanolixizumab_FINAL.pdf

Long-term safety outcomes of rozanolixizumab treatment in ... Data pool consisted of all rozanolixizumab-treated patients who have undergone ≥1 treatment cycle with an ≤8-week follow-up period starting from the last ...

8. rystiggo.com rystiggo.com/results

Clinical Trial Results For RYSTIGGO® (rozanolixizumab-noli) RYSTIGGO was shown to significantly improve MG-ADL scores by the end of the 6-week treatment period. Improvements were seen in some participants as early as 1 ...