Rozanolixizumab for Healthy Subjects

No longer recruiting at 1 trial location

Overseen By UCB Cares

Age: 18+

Sex: Female

Trial Phase: Phase 1

Sponsor: UCB Biopharma SRL

Must not be taking: Biologics, Live vaccines

Disqualifiers: Breast surgery, Infections, others

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

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

Compensation: Varies

Number of Visits: 2

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to understand the effects of rozanolixizumab, a medication, on breast milk when administered as a single dose to lactating women. Researchers seek to determine the amount of the medicine that enters breast milk, which could inform future treatments. The trial is specifically for women who are at least six weeks postpartum, have chosen to stop breast milk feeding, and are willing to temporarily cease breastfeeding for eight weeks after taking the medication. Participants should not have had breast surgeries or recent infections. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants a chance to contribute to groundbreaking research.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are taking biologic agents (like monoclonal antibodies), you must not have used them within 3 months or 5 half-lives before the study.

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

Research has shown that rozanolixizumab was generally well-tolerated in past studies, which included participants with conditions like myasthenia gravis, an autoimmune disease. Some participants experienced side effects, such as headaches and injection site reactions, but these were usually mild to moderate. Importantly, the FDA has already approved rozanolixizumab for treating certain conditions, which supports its safety profile. While this trial involves healthy volunteers and only one dose, existing safety data suggest it is unlikely to cause serious harm. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Rozanolixizumab is unique because it targets the neonatal Fc receptor (FcRn), which plays a key role in the regulation of antibodies in the body. This mechanism is different from traditional treatments that often focus on suppressing the immune system more broadly. By specifically targeting FcRn, rozanolixizumab has the potential to reduce harmful antibodies without broadly dampening immune function, potentially leading to fewer side effects. Researchers are excited about its subcutaneous administration, which could offer a more convenient and less invasive option compared to intravenous treatments.

What evidence suggests that rozanolixizumab might be an effective treatment?

Research has shown that rozanolixizumab effectively treats generalized myasthenia gravis (gMG), a condition that causes muscle weakness. One study found that patients taking rozanolixizumab experienced significant improvements, with 60.9% in the lower dose group and 74.2% in the higher dose group responding well to the treatment. Another study confirmed its safety and effectiveness over a 6-week period, helping patients manage their symptoms. These findings suggest that rozanolixizumab can be a good treatment option for certain autoimmune conditions like gMG, by reducing symptoms and improving patient health. Participants in this trial will receive a single dose of subcutaneous rozanolixizumab to further evaluate its effects in healthy subjects. ^{2 3 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 healthy lactating women who are at least 18 years old and a minimum of 6 weeks postpartum. Participants must agree to stop breastfeeding from Day 1 and not resume or donate breast milk for 8 weeks after receiving rozanolixizumab. Women must follow contraceptive guidance or be incapable of becoming pregnant due to medical reasons.

Inclusion Criteria

I am breastfeeding and will be 6 weeks postpartum when the study starts.

I have chosen to stop breastfeeding before joining the study and agree not to breastfeed or donate milk for 8 weeks after getting rozanolixizumab.

* A woman of childbearing potential (WOCBP) who agrees to follow the contraceptive guidance during the Sampling Period and for at least 1 week after the last dose of study treatment OR * Not a WOCBP (ie, premenopausal female with documented hysterectomy, documented bilateral salpingectomy, or documented bilateral oophorectomy)

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive a single dose of subcutaneous rozanolixizumab

1 day

1 visit (in-person)

Sampling Period

Breast milk samples are collected to assess the concentration of rozanolixizumab

7 days

Daily visits for sample collection

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Rozanolixizumab

Trial Overview

The study is testing the presence of rozanolixizumab in mature breast milk after administering a single dose to healthy lactating women. The goal is to understand how much, if any, of the drug passes into breast milk.

How Is the Trial Designed?

Group I: Rozanolixizumab arm Experimental Treatment 1 Intervention

Study participants enrolled in this arm will receive a single dose of subcutaneous rozanolixizumab

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

A 72-year-old woman developed severe anterior uveitis after receiving tocilizumab infusions for rheumatoid arthritis, suggesting a potential adverse effect of this anti-IL-6 receptor therapy.

The patient's ocular inflammation was successfully managed with corticosteroids, indicating that while tocilizumab can effectively treat rheumatoid arthritis, it may also lead to serious eye complications like uveitis after treatment cessation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Acute anterior uveitis after discontinuation of tocilizumab in a patient with rheumatoid arthritis. Sato, T., Minakuchi, S., Mochizuki, M., et al.[2021]

In a phase IIIb trial involving 288 patients with moderate-to-severe rheumatoid arthritis (RA), weekly subcutaneous tocilizumab (TCZ-SC) significantly reduced disease activity as measured by the Clinical Disease Activity Index (CDAI) within just 2 weeks, with sustained improvements observed up to 52 weeks.

TCZ-SC demonstrated low immunogenicity and no unexpected toxicities, making it a safe option for patients, and the study suggests that home administration of the treatment is feasible after a median of just 2 doses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Subcutaneous tocilizumab alone or with a csDMARD in rheumatoid arthritis patients: subanalysis of Italian data from a multicenter phase IIIb/IV trial. Bazzichi, L., Nacci, F., Sinigaglia, L., et al.[2020]

Tocilizumab, a humanized monoclonal antibody targeting sIL-6R, shows promise as a treatment for rheumatoid arthritis, with safety data from randomized controlled trials indicating no increased risk of opportunistic infections or cancers.

However, there are concerns about elevated liver function tests and altered lipid profiles, which need to be further investigated in larger studies with longer follow-up to understand their significance.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Interleukin-6 inhibition--tolerability profile and clinical implications. Strand, V., Yazici, Y.[2016]

Tocilizumab, an anti-IL-6 receptor monoclonal antibody, effectively blocks IL-6 receptors and normalizes C-reactive protein levels in patients with rheumatoid arthritis and systemic juvenile idiopathic arthritis, with biweekly doses of 8 mg/kg for patients ≥ 30 kg and 12 mg/kg for those < 30 kg.

The pharmacokinetic parameters, including serum concentrations and area under the curve, were similar across both dosing groups, indicating consistent efficacy and safety in achieving clinical endpoints regardless of the dose.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical pharmacology of tocilizumab for the treatment of systemic juvenile idiopathic arthritis. Zhang, X., Morcos, PN., Saito, T., et al.[2016]

In a 12-week Phase IIb study involving 221 patients with moderate-to-severe rheumatoid arthritis who had previously failed TNF inhibitor therapy, olokizumab (OKZ) significantly reduced disease activity (measured by DAS28(CRP)) compared to placebo, indicating its efficacy as a treatment option.

OKZ demonstrated similar efficacy to tocilizumab (TCZ) across multiple endpoints, with most adverse events being mild or moderate, suggesting a favorable safety profile consistent with other drugs in its class.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of olokizumab in patients with rheumatoid arthritis with an inadequate response to TNF inhibitor therapy: outcomes of a randomised Phase IIb study. Genovese, MC., Fleischmann, R., Furst, D., et al.[2023]

Citations

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ucb-usa.com

ucb-usa.com/stories-media/UCB-U-S-News/detail/article/ucb-to-unveil-new-data-for-rystiggo-rozanolixizumab-noli-and-and-zilbrysq-zilucoplan-for-gmg-at-the-2025-aanem-annual-meeting-and-mgfa-scientific-session

UCB to unveil new data for RYSTIGGO® (rozanolixizumab ...

These presentations will cover data on rozanolixizumab and zilucoplan, including new insights on corticosteroid-sparing strategies and quality ...

2.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/40034001/

Long-term safety of cyclical rozanolixizumab in patients ...

The Phase 3 MycarinG study demonstrated the efficacy and safety of one 6-week cycle of weekly rozanolixizumab in adult patients with gMG.

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 ...

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ucb.com

ucb.com/innovation/clinical-studies/clinical-studies-index/Rozanolixizumab-UCB7665

Rozanolixizumab (Rystiggo®)

A Study to Assess Long-term Safety, Tolerability and Efficacy of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy ...

5.

rystiggohcp.com

rystiggohcp.com/clinical-trial

Clinical Trial Data For RYSTIGGO® (rozanolixizumab-noli)

MGC responder rates from baseline at Week 6 (Day 43) were 60.9% (n=39/64) in the RYSTIGGO 7 mg/kg group and 74.2% (n=46/62) in the RYSTIGGO 10 mg/kg group ...

6.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/label/2023/761286s000lbl.pdf

Rystiggo - accessdata.fda.gov

Following subcutaneous administration of rozanolixizumab-noli, peak plasma levels were achieved after approximately 2 days in healthy subjects. Distribution.

7.

accp1.onlinelibrary.wiley.com

accp1.onlinelibrary.wiley.com/doi/full/10.1002/cpdd.1484

Safety, Tolerability, Pharmacokinetics, and ...

These data support the applicability of safety data from previous clinical studies of rozanolixizumab to individuals of Japanese and Chinese ...

8.

pubmed.ncbi.nlm.nih.gov

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

Safety and efficacy of rozanolixizumab in patients ... - PubMed

We aimed to assess the safety and efficacy of rozanolixizumab for generalised myasthenia gravis. Methods: MycarinG is a randomised, double-blind, placebo- ...