Clinical trial NCT06149559: A Study of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis
Official title: An Open-label, Single-arm Study Evaluating the Activity, Safety, and Pharmacokinetics of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: Generalized Myasthenia Gravis
Keywords: Generalized Myasthenia Gravis
gMG
rozanolixizumab
pediatric
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2023-11-29
Enrollment: 12
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


Eligibility criteria: Inclusion Criteria:

* 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))

Exclusion Criteria:

* 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

Primary outcomes:
measure: Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit
timeFrame: From Baseline up to the EOS Visit (up to 18 weeks)
measure: Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) up to the EOS Visit
timeFrame: From Baseline up to the EOS Visit (up to 18 weeks)
measure: Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit (up to 18 weeks)
timeFrame: From Baseline up to the EOS Visit (up to 18 weeks)
Secondary outcomes:
measure: Percent change in total Immunoglobulin G (IgG) from Baseline at the end of Week 6
timeFrame: From Baseline to the end of Week 6
measure: Absolute change in total IgG from Baseline at the end of Week 6
timeFrame: From Baseline to the end of Week 6
measure: Percent change from Baseline in myasthenia gravis (MG) autoantibody levels at the end of Week 6
timeFrame: From Baseline to the end of Week 6
measure: Absolute change from Baseline in MG-specific autoantibody levels at the end of Week 6
timeFrame: From Baseline to the end of Week 6
measure: Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at the end of Week 6
timeFrame: From Baseline to the end of Week 6
measure: Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6
timeFrame: From Baseline to the end of Week 6
measure: Occurrence of other TEAEs (including headache, nausea, and infusion site reactions) during Treatment Period 1 (TP1) and Observation Period 1 (OP1)
timeFrame: During TP1 and OP1 (up to 14 weeks)
measure: Evaluation of local tolerability at each scheduled assessment during TP1
timeFrame: At each scheduled assessment during TP1 (Baseline, week 2, 3, 4, 5, up to 6 weeks)
measure: Plasma concentration of rozanolixizumab at the 6-week treatment cycle
timeFrame: At the 6-week treatment cycle
measure: Incidence of antidrug antibodies (ADAs) at the end of Week 6
timeFrame: At the end of Week 6
Other outcomes:
Locations:
city: Denton
country: United States
facility: Mg0006 50574
state: Texas
city: Bologna
country: Italy
facility: Mg0006 40290
city: Milano
country: Italy
facility: Mg0006 40144
city: Napoli
country: Italy
facility: Mg0006 40733
city: Lodz
country: Poland
facility: Mg0006 40734
city: Warszawa
country: Poland
facility: Mg0006 40155
city: Taipei City
country: Taiwan
facility: Mg0006 20095
city: Taipei
country: Taiwan
facility: Mg0006 20081
Study officials:
affiliation: 001 844 599 2273
name: UCB Cares
role: STUDY_DIRECTOR
References: