Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)

Generalized Myasthenia Gravis Clinical Trial

Official title:

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 < 18 Years of Age) With Generalized Myasthenia Gravis (gMG)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05644561
• Other study ID #: ALXN1210-MG-319
• Status: Recruiting
• Phase: Phase 3
• Start date: June 24, 2023
• Est. completion date: July 31, 2028

Study information

• Verified date: March 2024
• Source: Alexion Pharmaceuticals, Inc.
• Contact: Alexion Pharmaceuticals, Inc. (Sponsor)
• Phone: 1-855-752-2356
• Email: clinicaltrials[@]alexion.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: July 31, 2028
• Est. primary completion date: July 15, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Diagnosis of gMG confirmed by a positive serologic test for anti-AChR antibodies (Abs) obtained at Screening and/or during Screening Period

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

• Participants receiving treatment must be on a stable dosing regimen of adequate duration prior to Screening and during the Screening Period.

• Eculizumab-experienced participants must have been enrolled and treated with eculizumab in Study ECU-MG-303 for at least 6 months (180 days) and must have been on a stable dose for = 2 months (60 days) prior to Screening.

• All participants must be vaccinated against meningococcal infection

Exclusion Criteria:

Medical Conditions

• Any untreated thymic malignancy, carcinoma, or thymoma.

• Participants with a history of treated benign thymoma

• History of thymectomy, thymomectomy, or any thymic surgery within the 12 months prior to Screening

• History of N meningitidis infection

• Known to be human immunodeficiency virus (HIV) positive

• History of unexplained infections

• Known or suspected history of drug or alcohol abuse or dependence within 1 year prior to the start of the Screening Period

Related Conditions & MeSH terms

• Generalized Myasthenia Gravis
• Muscle Weakness
• Myasthenia Gravis

Intervention

Drug:
• Ravulizumab
Ravulizumab will be administered by intravenous (IV) infusion.

Locations

Country	Name	City	State
France	Research Site	Marseille
France	Research Site	PARIS Cedex 12
Italy	Research Site	Milano
Italy	Research Site	Roma
Italy	Research Site	Torino
Japan	Research Site	Itabashi-ku
Netherlands	Research Site	Leiden
Serbia	Research Site	Belgrade
Serbia	Research Site	Belgrade
Spain	Research Site	Madrid
Spain	Research Site	Malaga
Switzerland	Research Site	Bern
United States	Research Site	Akron	Ohio
United States	Research Site	Chapel Hill	North Carolina
United States	Research Site	Chicago	Illinois
United States	Research Site	Los Angeles	California
United States	Research Site	Philadelphia	Pennsylvania
United States	Research Site	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  France,  Italy,  Japan,  Netherlands,  Serbia,  Spain,  Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Plasma Concentration of Ravulizumab		Day 1 predose through Week 18 predose
Primary	Serum Free C5 Concentration of Ravulizumab		Day 1 predose through Week 18 predose
Secondary	Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18		Baseline, Up to Week 18
Secondary	Change From Baseline in Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score at Up to Week 18		Baseline, Up to Week 18
Secondary	Change From Baseline in Myasthenia Gravis Composite (MGC) Score at Up to Week 18		Baseline, Up to Week 18
Secondary	Change in Status from Week 10 in Myasthenia Gravis Foundation of America Postintervention Status (MGFA-PIS) as Assessed by the Investigator or Neurologist at Up to Week 18		Week 10, Up to Week 18
Secondary	Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at Up to Week 18	Participants =8 years of age will be evaluated.	Baseline, Up to Week 18
Secondary	Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18	Participants <8 years of age will be evaluated.	Baseline, Up to Week 18
Secondary	Number of Participants With =5-point Reduction Compared to Baseline in the QMG Total Score Over Time Through Week 18		Baseline through Week 18
Secondary	Number of Participants With =3 point Reduction Compared to Baseline in the MG-ADL Total Score Over Time Through Week 18		Baseline through Week 18
Secondary	Number of Participants That Improve or Remain Stable in QMG Total Score at Week 18 Compared to Baseline	Stable is defined as a ±5-point change from Baseline.	Baseline through Week 18
Secondary	Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline	Stable is defined as a ±3-point change from baseline.	Baseline through Week 18
Secondary	Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events		Baseline up to Week 126 (8 weeks after last dose of study drug)
Secondary	Number of Participants With Anti-Drug Antibody (ADA) at Week 18		Baseline through Week 18