Clinical trial NCT06540144: An Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis
Official title: An Open-label Extension Study to Evaluate Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
Keywords: Generalized Myasthenia Gravis
gMG
rozanolixizumab
pediatric
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2024-08-06
Enrollment: 12
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to assess the long term safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in pediatric participants with generalized Myasthenia Gravis (gMG) aged ≥2 at the time of the Screening Visit of MG0006.


Eligibility criteria: Inclusion Criteria:

Study participant must meet one of the following:

* Study participant completed MG0006 according to the protocol
* Study participant completed the MG0006 Treatment Period and has a worsening of generalized myasthenia gravis (gMG) symptoms in the Observation Period of MG0006

Exclusion Criteria:

* Study participant met any mandatory withdrawal or mandatory permanent investigational medicinal product (IMP) discontinuation criteria in MG0006 or permanently discontinued IMP
* Study participant has a known hypersensitivity to any components of the IMP or other neonatal Fc receptor (FcRn) drugs
* Study participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at Baseline, and could jeopardize or compromise the study participant's ability to participate in this 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 52 weeks)
measure: Occurrence of TEAEs leading to permanent withdrawal of IMP up to the EOS Visit
timeFrame: From Baseline up to the EOS Visit (up to 52 weeks)
measure: Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit
timeFrame: From Baseline up to the EOS Visit (up to 52 weeks)
Secondary outcomes:
measure: Percent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP)
timeFrame: From Baseline to the end of Week 6 of each TP (up to 52 weeks)
measure: Absolute change in total IgG from Baseline to the end of Week 6 of each TP
timeFrame: From Baseline to the end of Week 6 of each TP (up to 52 weeks)
measure: Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG ADL) total score at the end of Week 6 of each TP
timeFrame: From Baseline to the end of Week 6 of each TP (up to 52 weeks)
measure: Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 of each TP
timeFrame: From Baseline to the end of Week 6 of each TP (up to 52 weeks)
Other outcomes:
Locations:
city: Bologna
country: Italy
facility: Mg0008 40290
city: Milano
country: Italy
facility: Mg0008 40144
city: OBU
country: Japan
facility: Mg0008 20339
city: Lodz
country: Poland
facility: Mg0008 40734
city: Warszawa
country: Poland
facility: Mg0008 40155
city: Taipei City
country: Taiwan
facility: Mg0008 20095
city: Taipei
country: Taiwan
facility: Mg0008 20081
Study officials:
affiliation: 001 844 599 2273
name: UCB Cares
role: STUDY_DIRECTOR
References: