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Clinical Trials/2022-502074-16-00
2022-502074-16-00
Recruiting
Phase 2/3

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

UCB Biopharma5 sites in 2 countries7 target enrollmentStarted: February 1, 2024Last updated:
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Interventions-

Overview

Phase
Phase 2/3
Status
Recruiting
Enrollment
7
Locations
5
Primary Endpoint
- Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit (up to 18 weeks) - Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) 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)
OverviewEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Overview

Brief Summary

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

Ages
0 years to 17 years (0-17 Years)
Accepts Healthy Volunteers
Yes

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 g (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-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

Arms & Interventions

-

Auxiliary

Participants receiving -

Intervention: - (Drug)

Outcomes

Primary Outcomes

- Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit (up to 18 weeks) - Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) 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)

- Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit (up to 18 weeks) - Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) 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)

Secondary Outcomes

  • Percent change in total Immunoglobulin G (IgG) from Baseline at the end of Week 6
  • Absolute change in total IgG from Baseline at the end of Week 6
  • Percent change from Baseline in MG-specific autoantibodies (anti-AChR or anti-MuSK) levels at the end of Week 6
  • Absolute change from Baseline in MG-specific autoantibodies (anti-AChR or anti-MuSK) levels at 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
  • Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6
  • Occurrence of other TEAEs (including headache, nausea, and infusion site reactions) during Treatment Period 1 (TP1) and Observation Period 1(OP1)
  • Evaluation of local tolerability at each scheduled assessment during TP1
  • Plasma concentration of rozanolixizumab at the 6-week treatment cycle
  • Incidence of antidrug antibodies (ADAs) at the end of Week 6

Investigators

Sponsor Class
Pharmaceutical company
Responsible Party
Principal Investigator
Principal Investigator

UCB Cares

Scientific

UCB Biopharma

Study Sites (5)

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