Study of Ravulizumab in Pediatric Participants With Primary IgAN

Phase 3

Not yet recruiting

• Conditions: IgAN; IgAVN; Immunoglobulin A Nephropathy; Immunoglobulin A Vasculitis Associated Nephritis; Henoch-schonlein Purpura Nephritis; IgA Vasculitis
• Interventions: Drug: Ravulizumab

• Registration Number: NCT07024563
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

The primary objective of this study is to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of treatment with ravulizumab IV in pediatric participants to support the extrapolation of efficacy from the adult population.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-06
• Study First Submitted: 2025-06-09
• Study First Submitted QC: 2025-06-09
• Study First Posted: 2025-06-17
• Last Update Submitted: 2025-06-20
• Last Update Posted: 2025-06-22
• Study Start: 2025-09-26
• Primary Completion: 2027-10-20
• Study Completion: 2029-03-08

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

• Participant must be 2 to < 18 years of age at the time of signing the informed consent or assent.
• Stable and maximum allowed or tolerated RASI (ACEI and/or ARB) dose for ≥ 3 months prior to Screening with no planned change during Screening through Week 106.
• UPCR ≥ 0.5 g/g from the mean of 3 first morning voids (FMV) collected within 1 week during the Screening Period
• Estimated GFR ≥ 30 mL/min/1.73 m2 during Screening
• Meningococcal infection vaccine
• Haemophilus influenzae type b and Streptococcus pneumoniae vaccine

Exclusion Criteria

• Diagnosis of rapidly progressive glomerulonephritis
• Secondary forms of IgAN not in the context of primary IgAN or IgAV
• Concomitant clinically significant renal disease other than IgAN or IgAVN
• Uncontrolled diabetes mellitus with HbA1c > 8.5%
• History of kidney transplant or planned kidney transplant during the Primary Evaluation Period.
• History of other solid organ (heart, lung, small bowel, pancreas, or liver) or bone marrow transplant
• Splenectomy or functional asplenia
• Participants with nephrotic syndrome receiving albumin infusions or with acute kidney injury requiring dialysis within the last 6 months prior to Screening.
• Hemolytic uremic syndrome diagnosed any time prior to Screening.
• Planned urological surgery expected to influence kidney function within the study time frame.
• Congenital immunodeficiency
• Active systemic bacterial, viral, or fungal infection within 14 days prior to enrollment
• Received biologics for the treatment of IgAN or IgAVN ≤ 6 months prior to Screening

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ravulizumab	Ravulizumab	All participants will receive a weight-based loading dose of ravulizumab on Day 1, followed by weight-based maintenance treatment with ravulizumab on Day 15 and either once every 8 weeks (q8w) thereafter or once every 4 weeks (q4w) depending on weight.

Primary Outcome Measures

Name	Time	Method
Maximum Observed Plasma Concentration (Cmax) of Ravulizumab	Baseline up to Week 34
Trough Serum Concentration (Ctrough) of Ravulizumab	Baseline up to Week 34
Change From Baseline in Serum Free Complement Component 5 (C5) Concentration	Baseline up to Week 34

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Proteinuria Based on Urine Protein to Creatinine Ratio (UPCR)	Baseline, Weeks 10 and 34
Change From Baseline in Albuminuria Based on Urine Albumin to Creatinine Ratio (UACR)	Baseline, Week 34
Number of Participants With Partial Remission	Week 34
Annualized Total Estimated Glomerular Filtration Rate (eGFR)	Baseline up to Week 106
Change from Baseline in eGFR	Baseline, Weeks 50 and 106
Number of Participants With UPCR < 0.5 grams of protein per gram of creatinine	Week 34
Number of Participants With Treatment Emergent Adverse Events, Treatment Emergent Serious Adverse Events and Adverse Events of Special Interest	Baseline up to Week 106
Number of Participants With Antidrug Antibodies to Ravulizumab and Neutralizing Antibodies	Baseline up to Week 106

Trial Locations

Locations (1)

Research Site; 🇨🇳 Taoyuan City, Taiwan