Study of Ravulizumab in Pediatric Participants With Primary IgAN
A Phase 3, Open-Label, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Ravulizumab in Pediatric Participants (2 to < 18 Years of Age) With Primary Immunoglobulin A Nephropathy (IgAN)
2 other identifiers
interventional
24
7 countries
14
Brief Summary
The primary objectives of this study are to characterize ravulizumab pharmacokinetics (PK) and pharmacodynamics (PD), and to evaluate safety and efficacy following ravulizumab IV dosing in pediatric participants with IgAN or IgAVN.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Jun 2025
Typical duration for phase_3
14 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 9, 2025
CompletedStudy Start
First participant enrolled
June 14, 2025
CompletedFirst Posted
Study publicly available on registry
June 17, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 12, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
November 27, 2029
March 17, 2026
March 1, 2026
3.1 years
June 9, 2025
March 16, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change from Baseline in Proteinuria Based on Urine Protein to Creatinine Ratio (UPCR) at Week 34
Baseline, Week 34
Secondary Outcomes (11)
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
Change from Baseline in proteinuria based on Urine Protein to Creatinine Ratio (UPCR) at Week 10
Baseline, Week 10
Change from Baseline in Albuminuria based on Urine Albumin to Creatinine Ratio (UACR) at Week 34
Baseline, Week 34
- +6 more secondary outcomes
Study Arms (1)
Ravulizumab
EXPERIMENTALAll 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.
Interventions
Eligibility Criteria
You may qualify if:
- 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 ≥ 1.0 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
- Participants who are receiving SGLT2i, DEARA (eg, sparsentan), MRA, ERA, or GLP-1 agonists must be on a stable and maximum allowed or tolerated dose for ≥ 3 months prior to Screening with no planned change in dose through Week 34.
- Established diagnosis of primary IgAN diagnosis based on kidney biopsy within 3 years prior to Screening or during the Screening Period
You may not qualify if:
- 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
- Clinical remission of IgAN/IgAVN or clinically significant improvement in proteinuria within the last 6 months.
- 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 within≤ 6 months prior to Screening
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (14)
Research Site
Palo Alto, California, 94304, United States
Research Site
Aurora, Colorado, 80045, United States
Research Site
Beijing, 100045, China
Research Site
Shanghai, 201102, China
Research Site
Genova, 16147, Italy
Research Site
Roma, 00165, Italy
Research Site
Torino, 10126, Italy
Research Site
Wakayama, 641-8510, Japan
Research Site
Seoul, 03080, South Korea
Research Site
Barcelona, 08035, Spain
Research Site
Barcelona, 8950, Spain
Research Site
Seville, 41013, Spain
Research Site
Taipei, 10002, Taiwan
Research Site
Taoyuan District, 333, Taiwan
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 9, 2025
First Posted
June 17, 2025
Study Start
June 14, 2025
Primary Completion (Estimated)
July 12, 2028
Study Completion (Estimated)
November 27, 2029
Last Updated
March 17, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, CSR
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.