NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

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

Active Not RecruitingPhase 2, Phase 3

Sponsor: UCB Biopharma SRL

Conditions: Generalized Myasthenia Gravis

Interventions: rozanolixizumab

Countries: Italy, Japan, Poland, Turkey (Türkiye)

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 overview

Sex: ALL

Age: 2 Years to 17 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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

Locations (9)

Bologna, Italy
Milan, Italy
Naples, Italy
Fuchu-shi, Japan
Ōbu, Japan
Sagamihara, Japan
Lodz, Poland
Warsaw, Poland
Yenimahalle, Turkey (Türkiye)