Complement 3 GlomerulopathyPhase 2RENEWNCT05162066
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)
A Phase 2 study testing BCX9930 in people with Complement 3 Glomerulopathy and Immunoglobulin A Nephropathy, run by BioCryst Pharmaceuticals at 11 sites in 4 countries.
Trial summary
The objective of this study was to determine the safety and therapeutic potential of BCX9930 in participants with C3G, IgAN, or PMN. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, Membranous Nephropathy
- What is being tested?
- BCX9930
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 11 sitesin 4 countries
- Study dates
- 18 Feb 2022to 23 Sep 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
11 sites in 4 countries.
11 sites in 4 countries
Italy4 sites
Investigative Site
Bari
Investigative Site
Bergamo
Investigative Site
Brescia
Investigative Site
Turin
Spain4 sites
Investigative Site #1
Barcelona
Investigative Site #2
Barcelona
Investigative Site #1
Madrid
Investigative Site #2
Madrid
About this study
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
The objective of this study was to determine the safety and therapeutic potential of BCX9930 in participants with C3G, IgAN, or PMN.
- Being tested
- BCX9930
- Keywords
- BCX9930, factor D inhibitor, oral therapy, Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, Primary Membranous Nephropathy
- Medical subject headings
- Glomerulonephritis, Membranoproliferative, Glomerulonephritis, IGA, Glomerulonephritis, Membranous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BCX9930Drug
Administered orally at a dose of 200 mg twice daily for the first 2 weeks, then 400 mg twice daily
| Study group | Role | What they receive |
|---|---|---|
| C3G cohort | Experimental | Approximately 14 eligible participants with C3G will be enrolled. Receives: Drug: BCX9930 |
| IgAN cohort | Experimental | Approximately 14 eligible participants with IgAN will be enrolled. Receives: Drug: BCX9930 |
| PMN cohort | Experimental | Approximately 14 eligible participants with PMN will be enrolled. Receives: Drug: BCX9930 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Baseline, Week 24
Secondary outcomes (7)
Percent Change From Baseline in 24-hour Urinary Protein Excretion
Measured: Baseline, Weeks 12, 24,
Change From Baseline in Estimated Glomerular Filtration Rate
eGFR was calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula for participants ≥ 18 years old and by the bedside Schwartz formula for participants ≤ 18 years old. eGFR was reported in milliliter per minute per 1.73 per square meter (mL/min/1.73m^2).
Measured: Baseline, Weeks 12, 24
Number of Participants With a Treatment-emergent Adverse Event (TEAE)
An Adverse event (AE) was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug.
Measured: From first dose up to safety follow-up period (Week 28)
Number of Participants Who Discontinued Due to a TEAE
An AE was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug.
Measured: From first dose up to safety follow-up period (Week 28)
Number of Participants Who Experienced a Treatment-emergent Serious Adverse Event (TESAE)
An AE was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug.
A serious adverse event (SAE) was an adverse event/reaction that results in any of the following outcomes:
- Death
- Is life-threatening (participant was at immediate risk of death at the time of the event; it does not refer to an event that hypothetically might have caused death if it were more severe)
- Requires participant hospitalization (formal admission to a hospital for medical reasons) or prolongation of existing hospitalization
- Results in persistent or significant disability/incapacity (ie, there was a substantial disruption of a person's ability to carry out normal life functions)
- Is a congenital anomaly/birth defect
Measured: From first dose up to safety follow-up period (Week 28)
Number of Participants Who Experienced a Common Terminology Criteria for Adverse Events (CTCAE) Grade 3 or 4 TEAE
An AE was any untoward medical occurrence has no causal relationship with the study drug or with the clinical study itself. It was an unfavorable \& unintended sign, symptom, syndrome, or illness that developed or worsened during clinical study.
TEAEs: AEs that started on or after first dose of treatment through 30 days after the last dose of study drug. All AEs were graded using the CTCAE Grades 1 through 5.
Grade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated
Grade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living (ADL)
Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL
Grade 4: Life-threatening consequences; urgent intervention indicated
Grade 5: Death
Participants with Grades 3 or 4 AEs were reported
Measured: From first dose up to safety follow-up period (Week 28)
Number of Participants Who Experienced a CTCAE Treatment-emergent Grade 3 or 4 Laboratory Abnormality
Treatment-emergent Laboratory Abnormality were defined as an event that started on or after the first dose of study treatment through 30 days after the last dose of study drug.
CTCAE Grades for laboratory abnormalities include:
Grade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated.
Grade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental ADL.
Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL.
Grade 4: Life-threatening consequences; urgent intervention indicated.
Grade 5: Death
Participants with Grades 3 or 4 laboratory abnormality were reported.
Measured: From first dose up to safety follow-up period (Week 28)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2021
- Study start18 Feb 2022
- Primary completion23 Sep 2022
- Study completion23 Sep 2022
Why it stopped: Sponsor Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Complement 3 GlomerulopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- 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)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioCryst Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Carla M Nester, MD, MSA, FASN, Principal investigator, University of Iowa Stead Family Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05162066
- Sponsor's protocol ID
- BCX9930-211
- Other IDs
- 2020-005855-19
- Acronym
- RENEW
- Status last verified
- Apr 2024
- First submitted
- 26 Nov 2021
- Last updated
- 2 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 39899371
Study at a glance
From the registry record, last updated by the sponsor on 2 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05162066
- Acronym / sponsor ID
- RENEW / BCX9930-211
- Keywords
- BCX9930, factor D inhibitor, oral therapy, Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, Primary Membranous Nephropathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor Decision
- Enrolment
- 2 participants (actual)
- Lead sponsor
- BioCryst PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 18 Feb 2022 (actual)
- Primary completion
- 23 Sep 2022 (actual)
- Study completion
- 23 Sep 2022 (actual)
- First posted
- 17 Dec 2021 (actual)
- Results
- Posted 2 May 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study was to determine the safety and therapeutic potential of BCX9930 in participants with C3G, IgAN, or PMN.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Three parallel treatment cohorts based on diagnosis of C3G, IgAN, or PMN. All eligible participants will receive open-label BCX9930.
| Arm | Type | Receives |
|---|---|---|
| C3G cohort | Experimental | Drug: BCX9930Approximately 14 eligible participants with C3G will be enrolled. |
| IgAN cohort | Experimental | Drug: BCX9930Approximately 14 eligible participants with IgAN will be enrolled. |
| PMN cohort | Experimental | Drug: BCX9930Approximately 14 eligible participants with PMN will be enrolled. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BCX9930Administered orally at a dose of 200 mg twice daily for the first 2 weeks, then 400 mg twice daily | Drug | C3G cohort, IgAN cohort, PMN cohort |
Primary outcome
Percent Change From Baseline in 24-hour uPCR at Week 12
Time frame: Baseline, 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.
Percent Change From Baseline in 24-hour uPCR at Week 24
Time frame: Baseline, 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.
7 secondary outcomes
Percent Change From Baseline in 24-hour Urinary Protein Excretion
Time frame: Baseline, Weeks 12, 24,
Change From Baseline in Estimated Glomerular Filtration Rate
Time frame: Baseline, Weeks 12, 24
Number of Participants With a Treatment-emergent Adverse Event (TEAE)
Time frame: From first dose up to safety follow-up period (Week 28)
Number of Participants Who Discontinued Due to a TEAE
Time frame: From first dose up to safety follow-up period (Week 28)
Number of Participants Who Experienced a Treatment-emergent Serious Adverse Event (TESAE)
Time frame: From first dose up to safety follow-up period (Week 28)
Number of Participants Who Experienced a Common Terminology Criteria for Adverse Events (CTCAE) Grade 3 or 4 TEAE
Time frame: From first dose up to safety follow-up period (Week 28)
Number of Participants Who Experienced a CTCAE Treatment-emergent Grade 3 or 4 Laboratory Abnormality
Time frame: From first dose up to safety follow-up period (Week 28)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39899371Lin 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Carla M Nester, MD, MSA, FASN principal investigator
University of Iowa Stead Family Children's Hospital
11 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BCX9930 in people with Complement 3 Glomerulopathy and Immunoglobulin A Nephropathy, run by BioCryst Pharmaceuticals at 11 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 2 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Italy, Spain, France, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 May 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change From Baseline in 24-hour uPCR at Week 12 (time frame: Baseline, Week 12). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05162066, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 May 2024.