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BCX9930 for the Treatment of C3G, IgAN, and PMN (RENEW)

Primary Purpose

Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, Membranous Nephropathy

Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
BCX9930
Sponsored by
BioCryst Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Complement 3 Glomerulopathy focused on measuring BCX9930, factor D inhibitor, oral therapy, Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, Primary Membranous Nephropathy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Male or female, aged ≥ 18 years old
  • Body weight ≥ 40 kg
  • Primary diagnosis of C3G, IgAN, or PMN confirmed by central pathology review
  • An eGFR ≥ 50 mL/min/1.73 m2 (or ≥ 30 mL/min/1.73 m2 after 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 within 180 days for anti-CD20 antibody therapies (eg, rituximab) prior to the screening visit
  • Treatment with renin inhibitors (eg, aliskiren) or sodium-glucose-cotransporter 2 (SGLT2) inhibitor within 60 days prior to Day 1

Sites / Locations

  • Investigative Site
  • Investigative Site
  • Investigative Site
  • Investigative Site
  • Investigative Site
  • Investigative Site
  • Investigative Site #1
  • Investigative Site #2
  • Investigative Site #1
  • Investigative Site #2
  • Investigative Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Other

Other

Other

Arm Label

C3G cohort

IgAN cohort

PMN cohort

Arm Description

Approximately 14 eligible participants with C3G will be enrolled.

Approximately 14 eligible participants with IgAN will be enrolled.

Approximately 14 eligible participants with PMN will be enrolled.

Outcomes

Primary Outcome Measures

Percent change from baseline in 24-hour urine protein-to-creatinine ratio (uPCR)

Secondary Outcome Measures

Proportion of subjects with a uPCR response: ≥ 50% reduction from baseline, ≤ 500 mg/g, or ≤ 200 mg/g
Percent change from baseline in 24-hour urinary protein excretion
Change from baseline in estimated glomerular filtration rate
Change from baseline in serum albumin
Proportion of subjects with protein ≥ 3.5 g in a 24-hour urine collection and serum albumin ≤ 2.5 g/dL
Proportion of subjects with a morphologic response
Treatment-emergent adverse event and serious adverse event

Full Information

First Posted
November 26, 2021
Last Updated
January 20, 2023
Sponsor
BioCryst Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT05162066
Brief Title
BCX9930 for the Treatment of C3G, IgAN, and PMN (RENEW)
Official Title
An Open-Label, Safety, Tolerability, and Proof-of-Concept Study of Oral BCX9930 Therapy in Subjects With C3G, IgAN, or PMN
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Terminated
Why Stopped
Sponsor Decision
Study Start Date
October 29, 2021 (Actual)
Primary Completion Date
September 16, 2022 (Actual)
Study Completion Date
September 23, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
BioCryst Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to determine the safety and therapeutic potential of BCX9930 in participants with C3G, IgAN, or PMN.
Detailed Description
This is an open-label, multicenter, proof-of-concept study to evaluate the safety, tolerability, and therapeutic potential of BCX9930 administered for 52 weeks in adult (≥ 18 years old) participants with either C3G, IgAN, or PMN. All participants will be enrolled into one of the three parallel treatment cohorts based on diagnosis of C3G, IgAN, or PMN and will receive BCX9930 for the 52-week treatment period.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, Membranous Nephropathy
Keywords
BCX9930, factor D inhibitor, oral therapy, Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, Primary Membranous Nephropathy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Three parallel treatment cohorts based on diagnosis of C3G, IgAN, or PMN. All eligible participants will receive open-label BCX9930.
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
2 (Actual)

8. Arms, Groups, and Interventions

Arm Title
C3G cohort
Arm Type
Other
Arm Description
Approximately 14 eligible participants with C3G will be enrolled.
Arm Title
IgAN cohort
Arm Type
Other
Arm Description
Approximately 14 eligible participants with IgAN will be enrolled.
Arm Title
PMN cohort
Arm Type
Other
Arm Description
Approximately 14 eligible participants with PMN will be enrolled.
Intervention Type
Drug
Intervention Name(s)
BCX9930
Intervention Description
Administered orally at a dose of 200 mg twice daily for the first 2 weeks, then 400 mg twice daily
Primary Outcome Measure Information:
Title
Percent change from baseline in 24-hour urine protein-to-creatinine ratio (uPCR)
Time Frame
Week 12, 24, 36, 52
Secondary Outcome Measure Information:
Title
Proportion of subjects with a uPCR response: ≥ 50% reduction from baseline, ≤ 500 mg/g, or ≤ 200 mg/g
Time Frame
Week 12, 24, 36, 52
Title
Percent change from baseline in 24-hour urinary protein excretion
Time Frame
Week 12, 24, 36, 52
Title
Change from baseline in estimated glomerular filtration rate
Time Frame
Week 12, 24, 36, 52
Title
Change from baseline in serum albumin
Time Frame
Week 12, 24, 36, 52
Title
Proportion of subjects with protein ≥ 3.5 g in a 24-hour urine collection and serum albumin ≤ 2.5 g/dL
Time Frame
Week 12, 24, 36, 52
Title
Proportion of subjects with a morphologic response
Time Frame
Week 24
Title
Treatment-emergent adverse event and serious adverse event
Time Frame
through Week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female, aged ≥ 18 years old Body weight ≥ 40 kg Primary diagnosis of C3G, IgAN, or PMN confirmed by central pathology review An eGFR ≥ 50 mL/min/1.73 m2 (or ≥ 30 mL/min/1.73 m2 after 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Carla M Nester, MD, MSA, FASN
Organizational Affiliation
University of Iowa Stead Family Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Investigative Site
City
Poitiers
Country
France
Facility Name
Investigative Site
City
Toulouse
Country
France
Facility Name
Investigative Site
City
Bari
Country
Italy
Facility Name
Investigative Site
City
Bergamo
Country
Italy
Facility Name
Investigative Site
City
Brescia
Country
Italy
Facility Name
Investigative Site
City
Turin
Country
Italy
Facility Name
Investigative Site #1
City
Barcelona
Country
Spain
Facility Name
Investigative Site #2
City
Barcelona
Country
Spain
Facility Name
Investigative Site #1
City
Madrid
Country
Spain
Facility Name
Investigative Site #2
City
Madrid
Country
Spain
Facility Name
Investigative Site
City
Oxford
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

BCX9930 for the Treatment of C3G, IgAN, and PMN (RENEW)

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