臨床研究等提出・公開システム

A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to less than 18 years of age) with Generalized Myasthenia Gravis (gMG)

Phase 3 PK and PD Study of Ravulizumab IV in Pediatric Participants with gMG

Terada Michinori

Alexion Pharma GK

Tamachi Station Tower N, 3-1-1, Shibaura, Minato-ku, Tokyo 108-0023, Japan

JPDept-DevOps-PMCO@alexion.com

Terada Michinori

Alexion Pharma GK

Tamachi Station Tower N, 3-1-1, Shibaura, Minato-ku, Tokyo 108-0023, Japan

+81-3-3457-9559

JPDept-DevOps-PMCO@alexion.com

Not Recruiting

July. 01, 2023

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

Diagnosis of gMG confirmed by a positive serologic test for anti AChr antibodies (Abs) obtained at Screening and / or during Screening Period and at least 1 of the following:
- History of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography or repetitive nerve stimulation
- History of positive anticholinesterase test
- Demonstrated improvement in MG signs on oral AChIs, as assessed by the Investigator

Must have QMG total score as outlined below:
- Complement inhibitor treatment-naive participants 12 to less than 18 years of age must have QMG total score over 12 at Screening and on Day1
- Complement inhibitor treatment-naive participants 6 to less than 12 years of age have no minimum QMG required for inclusion; however, parti must have documented limb weakness in at least 1 limb

Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to Class IV at Screening

Clinical features that, in the opinion of the Investigator, are consistent with MG crisis / exacerbation or Clinical Deterioration during the Screening Period or within:
- under 28 days prior to Screening for complement inhibitor treatment-naive participants
- under 6 months prior to Screening for eculizumab-experienced participants

Both

Generalized Myasthenia Gravis (gMG)

Compound:
Ravulizumab (ALXN1210)

Dosage and administration:
The usual starting dose of Ravulizumab (genetical recombination) is 600-3,000 mg per dose, taking into account the patient's body weight, followed by 600-3,600 mg per dose intravenously 2 weeks after the first dose and 600-3,600 mg per dose every 4 or 8 weeks thereafter.

Dosing period:
The total treatment duration is up to 122 weeks (18-week Primary Treatment Period followed by an Extension Period of up to 104 weeks). For participants in Japan, the total duration is up to 66 weeks.

Various PK and PD of pediatric gMG patients included in the population for PK and PD analysis

- Change from baseline in QMG total score through Week 18
- Change from baseline in MG-ADL total score through Week 18
- Change from baseline in MGC score through Week 18
- MGFA-PIS through Week 18

+81-3-3964-1211

May. 23, 2023

Yes

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

United States of America/France/Germany/Italy/Netherlands/Serbia/Spain/Switzerland