Clinical trial NCT07024563: Study of Ravulizumab in Pediatric Participants With Primary IgAN
Official title: 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)
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE3
Conditions: IgAN
IgAVN
Immunoglobulin A Nephropathy
Immunoglobulin A Vasculitis Associated Nephritis
Henoch-schonlein Purpura Nephritis
IgA Vasculitis
Keywords: ravulizumab
IgAN
IgAVN
pediatric
proteinuria
glomerulonephropathy
Immunoglobulin A Nephropathy
Immunoglobulin A Vasculitis Associated Nephritis
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2025-06-17
Enrollment: 18
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Maximum Observed Plasma Concentration (Cmax) of Ravulizumab
timeFrame: Baseline up to Week 34
measure: Trough Serum Concentration (Ctrough) of Ravulizumab
timeFrame: Baseline up to Week 34
measure: Change From Baseline in Serum Free Complement Component 5 (C5) Concentration
timeFrame: Baseline up to Week 34
Secondary outcomes:
measure: Change From Baseline in Proteinuria Based on Urine Protein to Creatinine Ratio (UPCR)
timeFrame: Baseline, Weeks 10 and 34
measure: Change From Baseline in Albuminuria Based on Urine Albumin to Creatinine Ratio (UACR)
timeFrame: Baseline, Week 34
measure: Number of Participants With Partial Remission
timeFrame: Week 34
measure: Annualized Total Estimated Glomerular Filtration Rate (eGFR)
timeFrame: Baseline up to Week 106
measure: Change from Baseline in eGFR
timeFrame: Baseline, Weeks 50 and 106
measure: Number of Participants With UPCR < 0.5 grams of protein per gram of creatinine
timeFrame: Week 34
measure: Number of Participants With Treatment Emergent Adverse Events, Treatment Emergent Serious Adverse Events and Adverse Events of Special Interest
timeFrame: Baseline up to Week 106
measure: Number of Participants With Antidrug Antibodies to Ravulizumab and Neutralizing Antibodies
timeFrame: Baseline up to Week 106
Other outcomes:
Locations:
city: Aurora
country: United States
facility: Research Site
state: Colorado
city: Beijing
country: China
facility: Research Site
city: Shanghai
country: China
facility: Research Site
city: Genova
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Torino
country: Italy
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Barcelona
country: Spain
facility: Research Site
city: Barcelona
country: Spain
facility: Research Site
city: Sevilla
country: Spain
facility: Research Site
city: Taipei
country: Taiwan
facility: Research Site
city: Taoyuan City
country: Taiwan
facility: Research Site
Study officials:
References: