A Phase 2 interventional study of Danicopan and Placebo in C3 Glomerulopathy, C3 Glomerulonephritis and Dense Deposit Disease, sponsored by Alexion Pharmaceuticals, Inc.. Completed at 7 sites in 2 countries. Open to participants aged 17 Years to 65 Years. Per ClinicalTrials.gov, last updated 2022-10-14.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 2, Interventional, and Treatment
Among 15 studies in Glomerulonephritis, Membranoproliferative with a site in United States — 1 open to participants now, 2 under way, 2 of them phase 3. Glomerulonephritis, Membranoproliferative · United States.
Among the 15 studies in Glomerulonephritis, Membranoproliferative with a site in United States, this one is the 8th largest by enrollment, is one of 3 that started in 2018, is one of 7 in phase 2, and was the 9th registered.
33 studies on the registry are indexed under Glomerulonephritis, Membranoproliferative; 10 are open to participants now.
Browse Glomerulonephritis, Membranoproliferative studies →1 of the 15 studies in Glomerulonephritis, Membranoproliferative with a site in United States is open now. Recruiting first, the newest first:
Novartis Pharmaceuticals · Sites in United States, Argentina, Brazil and 14 more countries
Ages 12–100
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 9 Glomerulonephritis, Membranoproliferative studies recruiting now, 5 set no upper age limit and none stop at 75 or younger; 5 take children under 18; 1 takes healthy volunteers.
This study: 17 Years to 65 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
15 studies; the count open now beside each. This study's row is marked.
First in 1999; this study’s year is marked. 3 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 13 is below the median of 22 across 26 interventional studies indexed under Glomerulonephritis, Membranoproliferative.
Median enrollment 13 across the 15 interventional studies. Of the 11 that completed or were terminated, 8 (73%) have results posted.
This tab as a report to print or save as PDF → With an account.
9 of 15 have an industry lead sponsor. The 10 most frequent:
This study is registered under Alexion Pharmaceuticals, Inc., one of the names counted as AstraZeneca. Under that name alone:
Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 23 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 75 (74%) have results posted.
AstraZeneca has 4 studies filed under Glomerulonephritis, Membranoproliferative — fewer than ten, so there is no comparison to draw.
1 of the studies in Glomerulonephritis, Membranoproliferative with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05809531Phase 3Apellis Pharmaceuticals, Inc.An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis | 2027-07 |
Add it to your calendar (.ics) → With an account.
Among the 15 studies in Glomerulonephritis, Membranoproliferative with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
The primary purpose of this proof-of-concept clinical study was to evaluate the efficacy and safety of the study drug, ACH-0144471 (also known as danicopan and ALXN2040), in participants with C3G who also had significant proteinuria attributable to C3G.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Danicopan was administered at a starting dose of 100 milligrams (mg) 3 times daily (TID) for the first 2 weeks, then dosage was to be increased to 200 mg TID for the remainder of the 6-month treatment period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and were to receive danicopan 200 mg TID.
Drug: Danicopan
Placebo was administered TID during the 6-month treatment period. All participants who completed the double-blind treatment period were enrolled in the open-label extension period and were to receive danicopan 200 mg TID.
Drug: Danicopan · Drug: Placebo
Danicopan was administered as an oral tablet.
Also known as: ACH-4471, ACH4471, 4471, ACH-0144471 (formerly), ALXN2040
Matching placebo was administered as an oral tablet.
Change From Baseline In Composite Biopsy Score At Week 28
The composite biopsy score was based on a score incorporating changes in the activity index, glomerular C3c staining, and glomerular macrophage infiltration at the end of 6 months of treatment. The composite renal biopsy index scoring system ranged from 0 to 21, with higher scores indicating worse outcomes.
Time frame: Baseline, Week 28
Participants With Reduction In Proteinuria At Week 28
Proteinuria reduction was defined as ≥ 30% decrease from baseline based on 24-hour urine protein (mg/day).
Time frame: Week 28
Change From Baseline In Proteinuria At Week 28
Proteinuria was assessed based on 24-hour urine collections at baseline and Week 28.
Time frame: Baseline, Week 28
Percent Change From Baseline In Proteinuria At Week 28
Proteinuria was assessed based on 24-hour urine collections at baseline and Week 28.
Time frame: Baseline, Week 28
Slope Of Estimated Glomerular Filtration Rate (eGFR) From Baseline To 6 Months
Slope of eGFR was estimated using a simple linear regression for each participant, including all data values from baseline until the end of the 6-month blinded treatment period, with eGFR as the dependent variable and time as the independent variable.
Time frame: 6 months
Slope Of Estimated Glomerular Filtration Rate (eGFR) After Open-label Danicopan Treatment
Slope of eGFR was estimated using a simple linear regression for each participant, including all data values during the open-label extension period with eGFR as the dependent variable and time as the independent variable.
Time frame: 12 months
Change From Baseline In eGFR At Week 28
Change from baseline in eGFR at Week 28 is presented.
Time frame: Baseline, Week 28
Participants With Significant Improvement In eGFR Relative To Baseline At Week 28
Significant improvement relative to baseline was defined as a ≥ 20% increase from baseline in eGFR.
Time frame: Baseline, Week 28
Participants With Significant Improvement In eGFR Relative To Baseline At Week 52
Significant improvement relative to baseline was defined as a ≥ 20% increase from baseline in eGFR.
Time frame: Baseline, Week 52
To enroll in the study, participants were required to have a biopsy confirmed diagnosis of C3 Glomerulopathy (C3G), with initial diagnosis made at least 3 months prior to dosing and significant proteinuria.
| Milestone | Danicopan (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period) | Placebo (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period) |
|---|---|---|
| Started | 6 | 7 |
| Received at least 1 dose of study drug | 6 | 7 |
| Completed | 6 | 6 |
| Not completed | 0 | 1 |
| Withdrew: Accidental unblinding | 0 | 1 |
| Milestone | Danicopan (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period) | Placebo (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period) |
|---|---|---|
| Started | 6 | 6 |
| Received at least 1 dose of study drug | 6 | 6 |
| Completed | 1 | 0 |
| Not completed | 5 | 6 |
| Withdrew: Lack of efficacy | 2 | 1 |
| Withdrew: Adverse event | 0 | 1 |
| Withdrew: Sponsor decision to close study | 3 | 4 |
The composite biopsy score was based on a score incorporating changes in the activity index, glomerular C3c staining, and glomerular macrophage infiltration at the end of 6 months of treatment. The composite renal biopsy index scoring system ranged from 0 to 21, with higher scores indicating worse outcomes.
| score on a scale | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) |
|---|---|---|
| Baseline | 11.7 ± 4.23 | 9.3 ± 3.50 |
| Week 28 | 9.2 ± 4.87 | 10.7 ± 3.39 |
| Change from Baseline | -2.0 ± 1.87 | 1.3 ± 2.88 |
Proteinuria reduction was defined as ≥ 30% decrease from baseline based on 24-hour urine protein (mg/day).
| participants | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) |
|---|---|---|
| Participants With Reduction In Proteinuria At Week 28 | 0 | 1 |
Proteinuria was assessed based on 24-hour urine collections at baseline and Week 28.
| mg/day | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) |
|---|---|---|
| Baseline | 6137.67 ± 2904.359 | 4274.47 ± 2992.819 |
| Week 28 | 5301.75 ± 2984.445 | 5186.80 ± 4069.279 |
| Change from Baseline | 302.00 ± 764.211 | 182.20 ± 994.558 |
Proteinuria was assessed based on 24-hour urine collections at baseline and Week 28.
| percent change | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) |
|---|---|---|
| Percent Change From Baseline In Proteinuria At Week 28 | 12.5 ± 31.11 | -10.4 ± 31.35 |
Slope of eGFR was estimated using a simple linear regression for each participant, including all data values from baseline until the end of the 6-month blinded treatment period, with eGFR as the dependent variable and time as the independent variable.
| mL/min/1.73 m^2 per month | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) |
|---|---|---|
| Slope Of Estimated Glomerular Filtration Rate (eGFR) From Baseline To 6 Months | -2.26840 ± 1.790814 | -1.45421 ± 2.228395 |
Slope of eGFR was estimated using a simple linear regression for each participant, including all data values during the open-label extension period with eGFR as the dependent variable and time as the independent variable.
| mL/min/1.73 m^2 per month | Danicopan (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period | Placebo (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period) | Total |
|---|---|---|---|
| Slope Of Estimated Glomerular Filtration Rate (eGFR) After Open-label Danicopan Treatment | -1.02556 ± 1.389128 | -1.84935 ± 3.506810 | -1.39169 ± 2.401039 |
Change from baseline in eGFR at Week 28 is presented.
| mL/min/1.73 m^2 | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) |
|---|---|---|
| Baseline | 79.890 ± 44.4571 | 68.381 ± 36.7249 |
| Week 28 | 67.543 ± 47.8062 | 50.874 ± 15.0517 |
| Change from Baseline | -12.347 ± 10.8063 | -8.700 ± 16.0990 |
Significant improvement relative to baseline was defined as a ≥ 20% increase from baseline in eGFR.
| participants | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) |
|---|---|---|
| Participants With Significant Improvement In eGFR Relative To Baseline At Week 28 | 0 | 0 |
Significant improvement relative to baseline was defined as a ≥ 20% increase from baseline in eGFR.
| participants | Danicopan (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period) | Placebo (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period) |
|---|---|---|
| Participants With Significant Improvement In eGFR Relative To Baseline At Week 52 | 0 | 0 |
Collected over Day 1 (after dosing) through Week 28 (treatment period and follow-up) and Week 104 (open-label extension and follow-up). The median study duration was 548.0 days.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Danicopan (Double-blind Treatment Period) | 0/6 (0%) | 0/6 (0%) | 5/6 (83.3%) |
| Placebo (Double-blind Treatment Period) | 0/7 (0%) | 0/7 (0%) | 5/7 (71.4%) |
| Danicopan (Open-label Extension Period) | 0/12 (0%) | 1/12 (8.3%) | 8/12 (66.7%) |
| Event | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) | Danicopan (Open-label Extension Period) |
|---|---|---|---|
| Acute kidney injuryRenal and urinary disorders | 0/6 | 0/7 | 1/12 |
| Event | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) | Danicopan (Open-label Extension Period) |
|---|---|---|---|
| Nasal congestionRespiratory, thoracic and mediastinal disorders | 2/6 | 0/7 | 0/12 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 2/6 | 0/7 | 0/12 |
| ContusionInjury, poisoning and procedural complications | 0/6 | 2/7 | 0/12 |
| NauseaGastrointestinal disorders | 1/6 | 2/7 | 1/12 |
| VomitingGastrointestinal disorders | 1/6 | 2/7 | 1/12 |
| Orthostatic hypotensionVascular disorders | 1/6 | 0/7 | 0/12 |
| Pregnancy of partnerSocial circumstances | 1/6 | 0/7 | 0/12 |
| Paranasal sinus discomfortRespiratory, thoracic and mediastinal disorders | 1/6 | 0/7 | 0/12 |
| PyrexiaGeneral disorders | 1/6 | 0/7 | 0/12 |
| PainGeneral disorders | 1/6 | 0/7 | 0/12 |
All participants who received at least 1 dose of study drug during the double-blind treatment period.
| Age, Continuous(years) | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) | Total |
|---|---|---|---|
| Mean | 25.5 ± 10.54 | 24.9 ± 4.85 | 25.2 ± 7.63 |
| Sex: Female, Male(Participants) | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) | Total |
|---|---|---|---|
| Female | 2 | 2 | 4 |
| Male | 4 | 5 | 9 |
| Race/Ethnicity, Customized(Participants) | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) | Total |
|---|---|---|---|
| Asian | 0 | 2 | 2 |
| White | 5 | 4 | 9 |
| Other | 1 | 1 | 2 |
| Hispanic or Latino | 3 | 1 | 4 |
| Not Hispanic or Latino | 3 | 6 | 9 |
| Region of Enrollment(participants) | Danicopan (Double-blind Treatment Period) | Placebo (Double-blind Treatment Period) | Total |
|---|---|---|---|
| United States | 6 | 6 | 12 |
| United Kingdom | 0 | 1 | 1 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Jun 2018 to primary completion in Dec 2019, 17 months.
Results were first posted in Nov 2021, 1 year 10 months after primary completion. A results delay was requested in May 2021.
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Studies of the drug tested here: Danicopan
Glomerulonephritis, Membranoproliferative →
Alexion Pharmaceuticals, Inc. →