Clinical trial NCT05162066: Study to Evaluate the Safety, Tolerability of BCX9930 in Participants With Either Complement 3 Glomerulopathy (C3G), Immunoglobulin A Nephropathy (IgAN), or Primary Membranous Nephropathy (PMN)
Official title: An Open-Label, Safety, Tolerability, and Proof-of-Concept Study of Oral BCX9930 Therapy in Subjects With Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, or Primary Membranous Nephropathy
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Complement 3 Glomerulopathy
Immunoglobulin A Nephropathy
Membranous Nephropathy
Keywords: BCX9930
factor D inhibitor
oral therapy
Complement 3 Glomerulopathy
Immunoglobulin A Nephropathy
Primary Membranous Nephropathy
Lead sponsor: BioCryst Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2021-12-17
Enrollment: 2
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Three parallel treatment cohorts based on diagnosis of C3G, IgAN, or PMN. All eligible participants will receive open-label BCX9930.
Primary purpose: TREATMENT

Brief summary: The objective of this study was to determine the safety and therapeutic potential of BCX9930 in participants with C3G, IgAN, or PMN.


Eligibility criteria: Inclusion Criteria:

* Body weight ≥ 40 kilograms (kg)
* Primary diagnosis of C3G, IgAN, or PMN confirmed by central pathology review
* An estimated glomerular filtration rate (eGFR) ≥ 50 milliter per minute per 1.73 meter square (mL/min/1.73 m\^2) (or ≥ 30 mL/min/1.73 m\^2 after Data Monitoring Committee \[DMC\] recommendation)
* Receiving treatment with a stable, maximum recommended or maximum tolerated dose of an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) for at least 60 days prior to the Day 1 Visit
* Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willingness to start vaccination series

Exclusion Criteria:

* Known congenital deficiency of C1s, C1r, C1q, C2, or C4
* History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation
* Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition
* History of malignancy within 5 years prior to the screening visit
* Active serious bacterial, viral, or fungal infection or any other serious infection within 14 days of screening
* Treatment with any systemic immunosuppressive or immunomodulatory therapy within 90 days OR anti-CD20 antibody therapies (eg, rituximab) within 180 days prior to the screening visit
* Treatment with renin inhibitors (eg, aliskiren) or sodium-glucose-cotransporter 2 (SGLT2) inhibitors within 60 days prior to Day 1

Primary outcomes:
measure: Percent Change From Baseline in 24-hour uPCR at Week 12
timeFrame: Baseline, Week 12
measure: Percent Change From Baseline in 24-hour uPCR at Week 24
timeFrame: Baseline, Week 24
Secondary outcomes:
measure: Percent Change From Baseline in 24-hour Urinary Protein Excretion
timeFrame: Baseline, Weeks 12, 24,
measure: Change From Baseline in Estimated Glomerular Filtration Rate
timeFrame: Baseline, Weeks 12, 24
measure: Number of Participants With a Treatment-emergent Adverse Event (TEAE)
timeFrame: From first dose up to safety follow-up period (Week 28)
measure: Number of Participants Who Discontinued Due to a TEAE
timeFrame: From first dose up to safety follow-up period (Week 28)
measure: Number of Participants Who Experienced a Treatment-emergent Serious Adverse Event (TESAE)
timeFrame: From first dose up to safety follow-up period (Week 28)
measure: Number of Participants Who Experienced a Common Terminology Criteria for Adverse Events (CTCAE) Grade 3 or 4 TEAE
timeFrame: From first dose up to safety follow-up period (Week 28)
measure: Number of Participants Who Experienced a CTCAE Treatment-emergent Grade 3 or 4 Laboratory Abnormality
timeFrame: From first dose up to safety follow-up period (Week 28)
Other outcomes:
Locations:
city: Poitiers
country: France
facility: Investigative Site
city: Toulouse
country: France
facility: Investigative Site
city: Bari
country: Italy
facility: Investigative Site
city: Bergamo
country: Italy
facility: Investigative Site
city: Brescia
country: Italy
facility: Investigative Site
city: Turin
country: Italy
facility: Investigative Site
city: Barcelona
country: Spain
facility: Investigative Site #1
city: Barcelona
country: Spain
facility: Investigative Site #2
city: Madrid
country: Spain
facility: Investigative Site #1
city: Madrid
country: Spain
facility: Investigative Site #2
city: Oxford
country: United Kingdom
facility: Investigative Site
Study officials:
affiliation: University of Iowa Stead Family Children's Hospital
name: Carla M Nester, MD, MSA, FASN
role: PRINCIPAL_INVESTIGATOR
References: