Trial · NCT03124368
A Proof-of-Mechanism Study to Determine the Effect of Danicopan on C3 Levels in Participants With C3G or IC-MPGN
NCT03124368, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2021-07-13
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. A Proof-of-Mechanism Study to Determine the Effect of Danicopan on C3 Levels in Participants With C3G or IC-MPGN. Trials, record NCT03124368. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03124368
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Proof-of-Mechanism Study to Determine the Effect of Danicopan on C3 Levels in Participants With C3G or IC-MPGN
- NCT number
- NCT03124368
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 6 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- C3 Glomerulonephritis, Dense Deposit Disease, C3 Glomerulopathy, Immune Complex Mediated Membranoproliferative Glomerulonephritis, Membranoproliferative Glomerulonephritis Types I, II, and III
- Interventions
- Danicopan
- First posted
- 2017-04-21
- Last updated
- 2021-11-04
- Other registries' IDs
- eudract:2016-003525-42
- Results first posted
- 2021-07-13
- Limitations and caveats
The sample size of the study was based on very limited clinical cases of C3G and IC-MPGN and the exploratory nature of this study to evaluate the effectiveness of danicopan.~Plasma intact C3 values should be regarded with caution. Plasma intact C3 values were generated using a new multiplex assay method with limited historical experience, and internal studies have suggested this new assay is subject to unexplained experimental variation arising during sample preparation and handling.
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15 | Baseline, Day 15 | Serum C3 levels were measured by conventional Roche immunoturbidimetric assay method. Change from Baseline = Serum C3 levels on Day 15 - Baseline Serum C3 levels |
| Primary | Change From Baseline In Plasma Intact C3 Level On Day 15 | Baseline, Day 15 | Plasma Intact C3 level were measured by a novel multiplex assay method. Change from Baseline = Plasma Intact C3 levels on Day 15 - Baseline Plasma Intact C3 levels |
| Secondary | Change From Baseline In Total Complement Classical Pathway (CP) Activity On Day 14 | Baseline, Day 14 | CP activity was measured in serum by the DiaSorin Complement Activation Enzyme (CAE) functional immunoassay method, which measures terminal complement complex formation following activation. Results are expressed in CAE units which are calculated relative to previously established CAE activity of a positive control serum. Change from Baseline = Total Complement CP Activity on Day 14 - Baseline Total Complement CP Activity |
| Secondary | Change From Baseline In Total Complement Alternative Pathway (AP) Functional Activity (AP Wieslab) On Day 15 | Baseline, Day 15 | AP functional activity was measured in serum by the Wieslab functional immunoassay method, which measures terminal complement complex (TCC) formation following AP-specific activation. Results are expressed as percent TCC production relative to a positive control serum. Change from Baseline = Total Complement AP Functional Activity on Day 15 - Baseline Total Complement AP Functional Activity |
| Secondary | Time To Achieving Peak Serum C3 Levels | From The First Day Of Dosing through Day 14 | Serial serum samples were collected on Days 1, 7, and 14. |
| Secondary | Number Of Participants With Serious Adverse Events (SAEs), Grade 3 And Grade 4 Treatment-emergent Adverse Events (TEAEs), And Adverse Events (AEs) Leading To Discontinuation | Up to Day 49 | An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of SAEs and other non-serious AEs regardless of causality is located in the Reported Adverse Events module. |
| Secondary | Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve From Time Of Administration To 8 Hours Postdose (AUC0-8) | Days 1 and 7 | Serial blood samples were collected at 0, 1, 1.5, 2, 2.5, 3, 4, 6, and 8 hours post-dose on Days 1 and 7. |
| Secondary | PK: Maximum Plasma Concentration (Cmax) | Days 1 and 7 | Serial blood samples were collected at 0, 1, 1.5, 2, 2.5, 3, 4, 6, and 8 hours post-dose on Days 1 and 7. |
| Secondary | PK: Time To Maximum Concentration (Tmax) | Days 1 and 7 | Serial blood samples were collected at 0, 1, 1.5, 2, 2.5, 3, 4, 6, and 8 hours post-dose on Days 1 and 7. |
| Secondary | Change From Baseline In Bb Fragment Of Complement Factor B (Bb) At Day 15 | Baseline, Day 15 | Plasma Bb was measured by enzyme-linked immunosorbent assay (ELISA). Change from Baseline = Complement Bb on Day 15 - Baseline |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15 | Baseline, Day 15 | All participants who received at least 1 dose of danicopan and who had a baseline measurement and at least 1 measurement during the treatment period. | g/L | MEAN |
| PRIMARY | Change From Baseline In Plasma Intact C3 Level On Day 15 | Baseline, Day 15 | All participants who received at least 1 dose of danicopan and who had a baseline measurement and at least 1 measurement during the treatment period. | μg/mL | MEAN |
| SECONDARY | Change From Baseline In Total Complement Classical Pathway (CP) Activity On Day 14 | Baseline, Day 14 | All participants who received at least 1 dose of danicopan and who had a baseline measurement and at least 1 measurement during the treatment period. | CAE unit | MEAN |
| SECONDARY | Change From Baseline In Total Complement Alternative Pathway (AP) Functional Activity (AP Wieslab) On Day 15 | Baseline, Day 15 | All participants who received at least 1 dose of danicopan and who had a baseline measurement and at least 1 measurement during the treatment period. | percentage of activity | MEAN |
| SECONDARY | Time To Achieving Peak Serum C3 Levels | From The First Day Of Dosing through Day 14 | All participants who received at least 1 dose of danicopan and who had a baseline measurement and at least 1 measurement during the treatment period. | days | MEAN |
| SECONDARY | Number Of Participants With Serious Adverse Events (SAEs), Grade 3 And Grade 4 Treatment-emergent Adverse Events (TEAEs), And Adverse Events (AEs) Leading To Discontinuation | Up to Day 49 | All participants who received at least 1 dose of danicopan and who had a baseline measurement and at least 1 measurement during the treatment period. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve From Time Of Administration To 8 Hours Postdose (AUC0-8) | Days 1 and 7 | All participants receiving at least 1 dose of danicopan who had a baseline measurement and at least 1 measurement during the treatment period. | h*ng/mL | GEOMETRIC_MEAN |
| SECONDARY | PK: Maximum Plasma Concentration (Cmax) | Days 1 and 7 | All participants receiving at least 1 dose of danicopan who had a baseline measurement and at least 1 measurement during the treatment period. | ng/mL | GEOMETRIC_MEAN |
| SECONDARY | PK: Time To Maximum Concentration (Tmax) | Days 1 and 7 | All participants receiving at least 1 dose of danicopan who had a baseline measurement and at least 1 measurement during the treatment period. | hours | MEDIAN |
| SECONDARY | Change From Baseline In Bb Fragment Of Complement Factor B (Bb) At Day 15 | Baseline, Day 15 | All participants who received at least 1 dose of danicopan and who had a baseline measurement and at least 1 measurement during the treatment period. | µg/mL | MEAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group87
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15 | Group 1: Danicopan 100 mg TID (Sentinel) | Baseline | 0.32 | Standard Deviation 0.060 | g/L |
| Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15 | Group 2: Danicopan up to 200 mg TID | Baseline | 0.56 | Standard Deviation 0.230 | g/L |
| Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15 | Total | Baseline | 0.48 | Standard Deviation 0.220 | g/L |
| Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15 | Group 1: Danicopan 100 mg TID (Sentinel) | Day 15 | 0.35 | Standard Deviation 0.060 | g/L |
| Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15 | Group 2: Danicopan up to 200 mg TID | Day 15 | 0.70 | Standard Deviation 0.350 | g/L |
| Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15 | Total | Day 15 | 0.58 | Standard Deviation 0.330 | g/L |
| Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15 | Group 1: Danicopan 100 mg TID (Sentinel) | Change from Baseline | 0.03 | Standard Deviation 0.000 | g/L |
| Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15 | Group 2: Danicopan up to 200 mg TID | Change from Baseline | 0.14 | Standard Deviation 0.140 | g/L |
| Change From Baseline In Serum C3 Complement Protein (C3) Levels On Day 15 | Total | Change from Baseline | 0.11 | Standard Deviation 0.120 | g/L |
| Change From Baseline In Plasma Intact C3 Level On Day 15 | Group 1: Danicopan 100 mg TID (Sentinel) | Baseline | 39.50 | Standard Deviation 0.710 | μg/mL |
1 to 10 of 87Source ClinicalTrials.gov
See all 87 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Group 1: Danicopan 100 mg TID (Sentinel) | 2 | |
| Overall Study | STARTED | Group 2: Danicopan up to 200 mg TID | 4 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Group 1: Danicopan 100 mg TID (Sentinel) | 2 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Group 2: Danicopan up to 200 mg TID | 4 | |
| Overall Study | COMPLETED | Group 1: Danicopan 100 mg TID (Sentinel) | 2 | |
| Overall Study | COMPLETED | Group 2: Danicopan up to 200 mg TID | 4 | |
| Overall Study | NOT COMPLETED | Group 1: Danicopan 100 mg TID (Sentinel) | 0 | |
| Overall Study | NOT COMPLETED | Group 2: Danicopan up to 200 mg TID | 0 |
Source ClinicalTrials.gov
Adverse events24
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Group 1: Danicopan 100 mg TID (Sentinel) | 1 | 2 | |
| other | Anaemia | Blood and lymphatic system disorders | Group 2: Danicopan up to 200 mg TID | 1 | 4 | |
| other | Dizziness | Nervous system disorders | Group 1: Danicopan 100 mg TID (Sentinel) | 1 | 2 | |
| other | Dizziness | Nervous system disorders | Group 2: Danicopan up to 200 mg TID | 0 | 4 | |
| other | Upper respiratory tract infection | Infections and infestations | Group 1: Danicopan 100 mg TID (Sentinel) | 1 | 2 | |
| other | Upper respiratory tract infection | Infections and infestations | Group 2: Danicopan up to 200 mg TID | 0 | 4 | |
| other | Hypokalaemia | Metabolism and nutrition disorders | Group 1: Danicopan 100 mg TID (Sentinel) | 1 | 2 | |
| other | Hypokalaemia | Metabolism and nutrition disorders | Group 2: Danicopan up to 200 mg TID | 0 | 4 | |
| other | Diarrhoea | Gastrointestinal disorders | Group 1: Danicopan 100 mg TID (Sentinel) | 1 | 2 | |
| other | Diarrhoea | Gastrointestinal disorders | Group 2: Danicopan up to 200 mg TID | 0 | 4 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Group 1: Danicopan 100 mg TID (Sentinel) | 0 | 2 |
| Total, all-cause mortality | Group 2: Danicopan up to 200 mg TID | 0 | 4 |
| Total, other adverse events | Group 1: Danicopan 100 mg TID (Sentinel) | 2 | 2 |
| Total, other adverse events | Group 2: Danicopan up to 200 mg TID | 3 | 4 |
| Total, serious adverse events | Group 1: Danicopan 100 mg TID (Sentinel) | 0 | 2 |
| Total, serious adverse events | Group 2: Danicopan up to 200 mg TID | 1 | 4 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Group 1: Danicopan 100 mg TID (Sentinel) vs Group 2: Danicopan up to 200 mg TID: deaths from any cause | Trial comparisons |
| Group 1: Danicopan 100 mg TID (Sentinel) vs Group 2: Danicopan up to 200 mg TID: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03124368
GET https://conductscience.org/api/trials/v1/NCT03124368/results?page=2