Study to Evaluate Avacopan in Combination With a Rituximab or Cyclophosphamide-containing Regimen, in Children From 6 Years to < 18 Years of Age With AAV.

Vasculitis Clinical Trial

Official title:

A Phase 3, Open-label, Uncontrolled Single-arm Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Avacopan in Combination With a Rituximab or a Cyclophosphamide-containing Regimen in Children From 6 Years to < 18 Years of Age With Active ANCA-associated Vasculitis (AAV)

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06321601
• Other study ID #: 20230070
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: July 31, 2024
• Est. completion date: April 28, 2030

Study information

• Verified date: May 2024
• Source: Amgen
• Contact: Amgen Call Center
• Phone: 866-572-6436
• Email: medinfo[@]amgen.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The main objective of this study is to explore the efficacy of avacopan in participants affected by AAV.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 20
• Est. completion date: April 28, 2030
• Est. primary completion date: April 28, 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 17 Years

Eligibility

Inclusion Criteria:

• Male and female children and adolescents from 6 to < 18 years of age
• Clinical diagnosis of granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), consistent with Chapel-Hill Consensus Conference definitions (Jennette et al, 2013)
• Newly diagnosed or relapsed AAV with positive test for anti-PR3 or anti-MPO antibodies
• At least 1 PVAS major item, at least 3 PVAS nonmajor items, or at least the 2 renal items of proteinuria and hematuria.
• eGFR > 15 mL/min/1.73 m2 (using modified Schwartz equation per central lab guidelines)
• Participants must have a bodyweight of = 15 kg at day 1.

Exclusion Criteria:

• Any other known multisystem autoimmune disease including eosinophilic granulomatosis with polyangiitis (Churg-Strauss), systemic lupus erythematosus , IgA vasculitis (Henoch-Schönlein), rheumatoid vasculitis, Sjogren's syndrome, anti-glomerular basement membrane disease, or cryoglobulinemic vasculitis
• Alveolar hemorrhage requiring invasive pulmonary ventilation support anticipated to last beyond the screening period of the study
• Any medical condition requiring or expected to require continued use of immunosuppressive treatments, including corticosteroids that may cause confoundment with study assessments and study conclusions.

Related Conditions & MeSH terms

• Vasculitis

Intervention

Drug:
• Avacopan
Oral administration

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Amgen

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of Participants Achieving Disease Remission at Week 26 According to the Pediatric Vasculitis Activities Score (PVAS)		Week 26
Primary	Proportion of Participants With Sustained Disease Remission at Week 52 According to the PVAS		Week 52
Secondary	Plasma Concentrations of Avacopan		Day 1 up to Week 52
Secondary	Number of Participants Experiencing Treatment-emergent Adverse Events (TEAE)	TEAEs are any event that occurred after the participant received study treatment. Any clinically significant changes in vital signs, electrocardiograms, and clinical laboratory tests that occurred after study treatment administration were recorded as TEAEs. A serious TEAE is any untoward medical occurrence in a clinical study participant after first dose irrespective of a causal relationship with the study treatment(s) that resulted in death, was immediately life threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, or another medically important serious event.	Day 1 up to approximately Week 60
Secondary	Proportion of Participants Achieving Disease Remission at Week 26 According to the Birmingham Vasculitis Activity Score (BVAS)		Week 26
Secondary	Proportion of Participants Achieving Disease Remission at Week 52 According to the BVAS		Week 52
Secondary	Proportion of Participants With PVAS of 0 Over Time Through Week 52		Up to Week 52
Secondary	Proportion of Participants With BVAS of 0 Over Time Through Week 52		Up to Week 52
Secondary	Change From Baseline Over 52 Weeks in Urinary Albumin-Creatinine Ratio (UACR)		Baseline up to Week 52
Secondary	Change From Baseline Over 52 Weeks in Estimated Glomerular Filtration Rate (eGFR)		Baseline up to Week 52
Secondary	Change From Baseline Over 52 Weeks In Physician Global Assessment (PGA) of Disease Activity		Baseline up to Week 52
Secondary	Change From Baseline Over 52 Weeks in Pediatric Vasculitis Damage Index (PVDI)		Baseline up to Week 52
Secondary	Number of Glucocorticoid Dosages Administered		Screening up to Week 52
Secondary	Proportion of Participants Across the Taste Score Categories of the TASTY Faces Scale	The TASTY faces scale will be utilized to assess the taste of the oral pediatric formulation. A 7-point version of the scale will be used, where higher scores correspond to faces showing favorable tastes. Upon drug administration, the child will be shown the TASTY scale and asked to rate his/her perception of tastiness by pointing to the appropriate face on the scale.	Day 1 and Week 2
Secondary	Taste and Acceptability Score of Avacopan per TASTY Faces Scale	The TASTY faces scale will be utilized to assess the taste of the oral pediatric formulation. A 7-point version of the scale will be used, where higher scores correspond to faces showing favorable tastes. Upon drug administration, the child will be shown the TASTY scale and asked to rate his/her perception of tastiness by pointing to the appropriate face on the scale.	Day 1 and Week 2

External Links

•   AmgenTrials clinical trials website