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)

NCT05162066 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: BCX9930-2112020-005855-19
• Study Type: interventional
• Target: 2
• Locations: 4 countries
• Sites: 11

Brief Summary

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

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

Outcome Measures

Primary Outcomes (2)

• Percent Change From Baseline in 24-hour uPCR at Week 12

  Urinary protein/creatinine ratio (mg/mmol) =urine protein concentration (mg/dL)/ urine creatinine concentration (mmol/dL). Decrease of urinary protein/creatinine ratio means improvement of renal disease.

  Baseline, Week 12

• Percent Change From Baseline in 24-hour uPCR at Week 24

  Urinary protein/creatinine ratio (mg/mmol) =urine protein concentration (mg/dL)/ urine creatinine concentration (mmol/dL). Decrease of urinary protein/creatinine ratio means improvement of renal disease.

  Baseline, Week 24

Secondary Outcomes (7)

• Percent Change From Baseline in 24-hour Urinary Protein Excretion

  Baseline, Weeks 12, 24,

• Change From Baseline in Estimated Glomerular Filtration Rate

  Baseline, Weeks 12, 24

• Number of Participants With a Treatment-emergent Adverse Event (TEAE)

  From first dose up to safety follow-up period (Week 28)

• Number of Participants Who Discontinued Due to a TEAE

  From first dose up to safety follow-up period (Week 28)

• Number of Participants Who Experienced a Treatment-emergent Serious Adverse Event (TESAE)

  From first dose up to safety follow-up period (Week 28)

Study Arms (3)

C3G cohort

EXPERIMENTAL

Approximately 14 eligible participants with C3G will be enrolled.

Drug: BCX9930

IgAN cohort

EXPERIMENTAL

Approximately 14 eligible participants with IgAN will be enrolled.

Drug: BCX9930

PMN cohort

EXPERIMENTAL

Approximately 14 eligible participants with PMN will be enrolled.

Drug: BCX9930

Interventions

BCX9930 DRUG

Administered orally at a dose of 200 mg twice daily for the first 2 weeks, then 400 mg twice daily

C3G cohort; IgAN cohort; PMN cohort

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• BioCryst Pharmaceuticals: lead

Study Sites (11)

Investigative Site

Poitiers, France

Location

Investigative Site

Toulouse, France

Location

Investigative Site

Bari, Italy

Location

Investigative Site

Bergamo, Italy

Location

Investigative Site

Brescia, Italy

Location

Investigative Site

Turin, Italy

Location

Investigative Site #1

Barcelona, Spain

Location

Investigative Site #2

Barcelona, Spain

Location

Investigative Site #1

Madrid, Spain

Location

Investigative Site #2

Madrid, Spain

Location

Investigative Site

Oxford, United Kingdom

Location

Related Publications (1)

• Lin J, Radhakrishnan J. What Are Baskets, Umbrellas, and Platforms Doing in Nephrology Clinical Trials? J Am Soc Nephrol. 2025 Feb 3;36(8):1652-1654. doi: 10.1681/ASN.0000000648. No abstract available.

  PMID: 39899371 DERIVED

MeSH Terms

Conditions

Glomerulonephritis, Membranoproliferative; Glomerulonephritis, IGA; Glomerulonephritis, Membranous

Condition Hierarchy (Ancestors)

Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Immune System Diseases; Autoimmune Diseases

Limitations and Caveats

The sponsor decided to prematurely terminate the study due to changes in the competitive landscape. Due to low enrolment in the study (N=2) and early termination of both participants, very limited data were available for analyses. Therefore, only key efficacy endpoints (24-hour uPCR, 24-hour urine protein excretion, and eGFR) and safety were analyzed and reported.

Results Point of Contact

• Title: Study Director
• Organization: BioCryst Pharmaceuticals Inc

clinicaltrials@biocryst.com

+1 919-859-1302

Study Officials

• Carla M Nester, MD, MSA, FASN

  University of Iowa Stead Family Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Three parallel treatment cohorts based on diagnosis of C3G, IgAN, or PMN. All eligible participants will receive open-label BCX9930.

Study Record Dates

• First Submitted: November 26, 2021
• First Posted: December 17, 2021
• Study Start: February 18, 2022
• Primary Completion: September 23, 2022
• Study Completion: September 23, 2022
• Last Updated: May 2, 2024
• Results First Posted: May 2, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1); FR (2); IT (4); ES (4)