Actively Recruiting

Phase 3
Age: 2Years - 18Years
All Genders
ID07024563

A Phase 3, Open-Label Study to Evaluate Ravulizumab Pharmacokinetics, Pharmacodynamics, Safety, and Efficacy in Children Aged 2 to Under 18 With Primary Immunoglobulin A Nephropathy (IgAN)

Led by Alexion Pharmaceuticals, Inc. · Updated on 2026-03-17

24

Participants Needed

14

Research Sites

71 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating the pharmacokinetics, pharmacodynamics, safety, and effectiveness of ravulizumab given by intravenous infusion in children aged 2 to under 18 years with primary Immunoglobulin A Nephropathy (IgAN) or Immunoglobulin A Vasculitis Nephritis (IgAVN). This Phase 3, open-label study aims to better understand how this medication behaves in the body and its potential benefits for this condition. All participants will receive a weight-based loading dose of ravulizumab on the first day, followed by weight-based maintenance doses on Day 15 and then either every 4 or 8 weeks depending on their weight. This treatment schedule will continue through the study period, which includes up to 106 weeks for long-term evaluation. Participants will be monitored regularly through urine tests to measure protein levels, blood tests for drug concentration and complement component levels, and kidney function assessments. Researchers will also track adverse events and immune responses to the drug. The main outcome is the change in proteinuria measured by urine protein to creatinine ratio at Week 34, with ongoing safety and kidney function monitoring throughout the study.

CONDITIONS

Brief Title

Study of Ravulizumab in Pediatric Participants With Primary IgAN

Who Can Participate

Age: 2Years - 18Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • 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 63 3 months prior to Screening with no planned change during Screening through Week 106.
  • UPCR 63 1.0 g/g from the mean of 3 first morning voids (FMV) collected within 1 week during the Screening Period.
  • Estimated GFR 63 30 mL/min/1.73 m2 during Screening.
  • Meningococcal infection vaccine.
  • Haemophilus influenzae type b and Streptococcus pneumoniae vaccine.
  • Participants receiving SGLT2i, DEARA (eg, sparsentan), MRA, ERA, or GLP-1 agonists must be on a stable and maximum allowed or tolerated dose for 63 3 months prior to Screening with no planned change in dose through Week 34.
  • Established diagnosis of primary IgAN based on kidney biopsy within 3 years prior to Screening or during the Screening Period.
Not Eligible

You will not qualify if you...

  • 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 64 6 months prior to Screening.

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

Treatment

Duration - Up to 34 weeks

Participants receive weight-based loading and maintenance doses of Ravulizumab via intravenous infusion to treat primary IgAN.

Infusions on Day 1 and Day 15, then once every 4 or 8 weeks depending on weight

Trial Site Locations

Total: 14 locations

1

Research Site

Palo Alto, California, United States, 94304

Not Yet Recruiting

2

Research Site

Aurora, Colorado, United States, 80045

Actively Recruiting

3

Research Site

Beijing, China, 100045

Actively Recruiting

4

Research Site

Shanghai, China, 201102

Actively Recruiting

5

Research Site

Genova, Italy, 16147

Actively Recruiting

6

Research Site

Roma, Italy, 00165

Actively Recruiting

7

Research Site

Torino, Italy, 10126

Actively Recruiting

8

Research Site

Wakayama, Japan, 641-8510

Actively Recruiting

9

Research Site

Seoul, South Korea, 03080

Actively Recruiting

10

Research Site

Barcelona, Spain, 08035

Actively Recruiting

11

Research Site

Barcelona, Spain, 8950

Actively Recruiting

12

Research Site

Seville, Spain, 41013

Actively Recruiting

13

Research Site

Taipei, Taiwan, 10002

Actively Recruiting

14

Research Site

Taoyuan, Taiwan, 333

Actively Recruiting

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Research Team

A

Alexion Pharmaceuticals, Inc. (Sponsor)

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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