A Study of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis

NCT06149559 | Recruiting Phase 2 DMC FDA Drug

• 3 other identifiers: MG00062022-502074-16-00U1111-1285-0787
• Study Type: interventional
• Target: 12
• Locations: 6 countries
• Sites: 13

Brief Summary

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).

Conditions

Generalized Myasthenia Gravis

Keywords

Generalized Myasthenia Gravis; gMG; rozanolixizumab; pediatric

Outcome Measures

Primary Outcomes (3)

• Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit

  Serious TEAEs are any untoward medical incidence in a subject during administered study treatment, whether or not these events are related to study treatment and additionally are emergent untoward medical occurrence that at any dose: * Results in death * Is life-threatening * Requires in patient hospitalisation or prolongation of existing hospitalisation * Results in persistent disability/incapacity * Is a congenital anomaly or birth defect * Important medical events

  From Baseline up to the EOS Visit (up to 18 weeks)

• Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) up to the EOS Visit

  An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

  From Baseline up to the EOS Visit (up to 18 weeks)

• Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit (up to 18 weeks)

  AESMs are: Severe and/or serious headache, suspected aseptic meningitis, severe Gastrointestinal (GI) disorders, and opportunistic infection.

  From Baseline up to the EOS Visit (up to 18 weeks)

Secondary Outcomes (10)

• Percent change in total Immunoglobulin G (IgG) from Baseline at the end of Week 6

  From Baseline to the end of Week 6

• Absolute change in total IgG from Baseline at the end of Week 6

  From Baseline to the end of Week 6

• Percent change from Baseline in myasthenia gravis (MG) autoantibody levels at the end of Week 6

  From Baseline to the end of Week 6

• Absolute change from Baseline in MG-specific autoantibody levels at the end of Week 6

  From Baseline to the end of Week 6

• Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at the end of Week 6

  From Baseline to the end of Week 6

Study Arms (1)

rozanolixizumab

EXPERIMENTAL

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

Drug: rozanolixizumab

Interventions

rozanolixizumab DRUG

rozanolixizumab solution for injection

Also known as: UCB7665

rozanolixizumab

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Study participant must be ≥2 to \<18 years of age inclusive, at the time of signing the informed consent/assent according to local regulation
• Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG) at Screening that includes a record confirming the presence of MG specific autoantibodies to acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) prior to Screening
• Study participant has Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IVa at Screening
• Study participant has received existing conventional treatment(s) for gMG (eg, pyridostigmine, corticosteroids, and/or immune suppressants) prior to Screening
• Study participant has had an unsatisfactory clinical response or worsening of gMG symptoms and is in need of additional therapy (for example, plasma exchange (PEX) or treatment with intravenous immunoglobulin (IVIg))

You may not qualify if:

• Study participant with severe weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis at Screening or Baseline
• Study participant has a known hypersensitivity to any components of the Investigational Medicinal Product (IMP) or other anti-neonatal-Fc receptor (FcRn) medications
• Study participant with any active or untreated thymoma
• Study participant has a history of thymectomy within 6 months prior to Screening
• Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of IMP
• Study participant has received a live vaccination within 4 weeks prior to Baseline or intends to have a live vaccination during the course of the study

Sponsors & Collaborators

• UCB Biopharma SRL: lead

Study Sites (13)

Mg0006 50574

Flower Mound, Texas, 75028, United States

WITHDRAWN

Mg0006 40290

Bologna, Italy

RECRUITING

Mg0006 40144

Milan, Italy

RECRUITING

Mg0006 40733

Naples, Italy

RECRUITING

Mg0006 20340

Fuchu-shi, Japan

RECRUITING

Mg0006 20339

Ōbu, Japan

RECRUITING

Mg0006 20343

Sagamihara, Japan

RECRUITING

Mg0006 40155

Warsaw, Poland

RECRUITING

Mg0006 40734

Warsaw, Poland

RECRUITING

Mg0006 20081

Taipei, Taiwan

RECRUITING

Mg0006 20095

Taipei, Taiwan

RECRUITING

Mg0006 40841

Altindağ/ankara, Turkey (Türkiye)

RECRUITING

Mg0006 40836

Kocaeli, Turkey (Türkiye)

RECRUITING

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

rozanolixizumab

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• UCB Cares

  001 844 599 2273

  STUDY DIRECTOR

Central Study Contacts

UCB Cares

CONTACT

1-844-599-2273; ucbcares@ucb.com

UCB Cares

CONTACT

001 844 599 2273; ucbcares@ucb.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 25, 2023
• First Posted: November 29, 2023
• Study Start: June 14, 2024
• Primary Completion (Estimated): June 8, 2026
• Study Completion (Estimated): August 17, 2026
• Last Updated: April 24, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
• Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed.All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.

Locations

US (1); PL (2); TU (2); IT (3); JP (3); TW (2)