Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2016-003523-34 | EudraCT Number |
Not provided
Not provided
Not provided
After careful consideration, UCB has decided to no longer pursue PNH as a potential indication for zilucoplan.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The purpose of this study is to enable continued access to zilucoplan (RA101495) for patients with paroxysmal nocturnal hemoglobinuria (PNH) after they complete a zilucoplan clinical study.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Zilucoplan (RA101495) | Experimental | Subjects will continue to receive the final maintenance dose they were receiving in the qualifying study |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Zilucoplan (RA101495) | Drug | Subjects will continue to receive the final maintenance dose they were receiving in the qualifying study. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) | TEAEs were defined as an AE that occurs after a participant's initial treatment zilucoplan start for this study (RA101495-01.202) that was not present at the time of treatment start, or an AE that increases in severity after treatment start in this study, if the event was present at the time of treatment start. | From Day 1 until the Final Study Visit (up to Month 49) |
| Percentage of Participants With Serious TEAEs | Serious Adverse event (SAE) was defined as any untoward medical occurrence that:• results in death, • is life-threatening threatening (note that this refers to an event in which the participant was at 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 hospitalization or prolongation of existing hospitalization, • results in persistent or significant disability/incapacity, and • results in a congenital anomaly/birth defect. | From Day 1 until the Final Study Visit (up to Month 49) |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Anti-drug Antibodies (ADA) | Blood samples collection were planned to analyze for the presence/absence of ADAs to zilucoplan for immunogenicity assessments. | At Day 1, Month 1, 2, 3, 6, 9, and 12 |
| Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point |
Not provided
Inclusion Criteria:
Exclusion criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Dr. Anita Hill | St James' Institute of Oncology | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigative Site 4 | Los Angeles | California | 90033 | United States | ||
| Investigative Site 19 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37534517 | Result | Kulasekararaj AG, Lehtinen AE, Forsyth C, Gandhi S, Griffin M, Korper S, Mikala G, Muus P, Overgaard U, Patriquin CJ, Pullon H, Shen YM, Spearing R, Szer J, De la Borderie G, Duda PW, Farzaneh-Far R, Ragunathan S, Sayegh CE, Vadysirisack DD, Schrezenmeier H. Phase II trials of zilucoplan in paroxysmal nocturnal hemoglobinuria. Haematologica. 2024 Mar 1;109(3):929-935. doi: 10.3324/haematol.2022.281780. No abstract available. |
Not provided
Not provided
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
The Participant Flow refers to the All Enrolled Subjects.
The study started to enroll participants in July 2017 and concluded in October 2021.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Zilucoplan-Cohort A (Eculizumab Naïve) | Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 20, 2016 | Sep 7, 2022 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Serum LDH levels were measure of intravascular hemolysis. As high level of LDH in the blood was indicative of hemolysis in participants with PNH. |
| Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49) |
| Change From Baseline in Total Bilirubin Values at Each Time Point | Total Bilirubin was monitored for signs and symptoms of hepatic or biliary dysfunction. | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49) |
| Change From Baseline in Total Hemoglobin Values at Each Time Point | Total Hemoglobin Values were analyzed for hematology assessments. | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49) |
| Change From Baseline in Free Hemoglobin Values at Each Time Point | Free Hemoglobin Values were analyzed for hematology assessments. | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49) |
| Change From Baseline in Haptoglobin Values at Each Time Point | Haptoglobin values were analyzed for hematology assessments. | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49) |
| Change From Baseline in Reticulocytes at Each Time Point | Reticulocytes values were analyzed for hematology assessments. | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49) |
| Change From Baseline in Hemoglobinuria Values at Each Time Point | Hemoglobinuria was assessed using a urine colorimetric scoring system with a score of 1 through 10 where 1 represents no hemoglobinuria and 10 represents maximum hemoglobinuria. | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48 and Final Study Visit (Month 49) |
| Plasma Concentrations of RA101495 and Its Major Metabolite(s) | Blood samples of RA101495 (zilucoplan) and its metabolites (RA102758 and RA103488) were collected for Plasma concentration analysis. | Predose: At Day 1 (Screening), Month 1, 2, 3, 6, 9, 12, and Final Study Visit (Month 49) |
| Maximum Plasma Concentration (Cmax) of RA101495 | Cmax is the maximum plasma concentration. | At Day 1, Month 1, 2, 3, 6, 9, and 12 |
| Time Corresponding to Cmax (Tmax) of RA101495 | tmax is the time to corresponding Cmax. | At Day 1, Month 1, 2, 3, 6, 9, and 12 |
| Area Under the Drug Concentration-time Curve (AUC0-t) of RA101495 | AUC0-t is area under the drug concentration-time curves. | At Day 1, Month 1, 2, 3, 6, 9, and 12 |
| Total Complement (CH50) Levels | Blood samples collection were planned to assess complement (CH50) levels. The planned analysis of CH50 was not performed because the CH50 assay was not able to be validated due to lack of reproducibility of the manufacturer's kits. | At Day 1, Month 1, 2, 3, 6, 9, and 12 |
| Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point | Blood samples were collected for measurement of sRBC lysis for the Classical Complement Pathways. | Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49) |
| Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point | Blood samples were collected for measurement of membrane attack complex (MAC) by Wieslab ELISA for alternative complement pathway. | Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49) |
| Change From Baseline in Complement Component 5 (C5) Values at Each Time Point | Blood samples were collected for measurement of Complement component 5 (C5) levels. | Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49) |
| Dallas |
| Texas |
| 75390 |
| United States |
| Investigative Site 3 | Gosford | Australia |
| Investigative Site 5 | Melbourne | Australia |
| Investigative Site 10 | Toronto | Canada |
| Investigative Site 14 | Helsinki | Finland |
| Investigative Site 9 | Ulm | Germany |
| Investigative Site 17 | Budapest | Hungary |
| Investigative Site 13 | Christchurch | New Zealand |
| Investigative Site 12 | Hamilton | New Zealand |
| Investigative Site 6 | Leeds | United Kingdom |
| Investigative Site 7 | London | United Kingdom |
| FG001 | Zilucoplan-Cohort B (Eculizumab Switch) | Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
| FG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Baseline Characteristics refer to the Safety Population which consisted of all participants who received at least 1 injection of zilucoplan on or after Day 1 of the extension study.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Zilucoplan-Cohort A (Eculizumab Naïve) | Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
| BG001 | Zilucoplan-Cohort B (Eculizumab Switch) | Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
| BG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) | TEAEs were defined as an AE that occurs after a participant's initial treatment zilucoplan start for this study (RA101495-01.202) that was not present at the time of treatment start, or an AE that increases in severity after treatment start in this study, if the event was present at the time of treatment start. | Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. | Posted | Number | percentage of participants | From Day 1 until the Final Study Visit (up to Month 49) |
|
|
| ||||||||||||||||||||||||||||||||
| Primary | Percentage of Participants With Serious TEAEs | Serious Adverse event (SAE) was defined as any untoward medical occurrence that:• results in death, • is life-threatening threatening (note that this refers to an event in which the participant was at 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 hospitalization or prolongation of existing hospitalization, • results in persistent or significant disability/incapacity, and • results in a congenital anomaly/birth defect. | Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. | Posted | Number | percentage of participants | From Day 1 until the Final Study Visit (up to Month 49) |
| ||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Anti-drug Antibodies (ADA) | Blood samples collection were planned to analyze for the presence/absence of ADAs to zilucoplan for immunogenicity assessments. | Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. The planned analysis of immunogenicity (ADA) was not performed as the assay was considered not fit for purpose due to an insufficient level of drug tolerance; therefore, no samples were processed. | Posted | At Day 1, Month 1, 2, 3, 6, 9, and 12 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point | Serum LDH levels were measure of intravascular hemolysis. As high level of LDH in the blood was indicative of hemolysis in participants with PNH. | Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Baseline was generally the final visit of the qualifying study (RA101495-01.201 or RA101495-01.203). Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | Units per litre (U/L) | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49) |
| |||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Total Bilirubin Values at Each Time Point | Total Bilirubin was monitored for signs and symptoms of hepatic or biliary dysfunction. | Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Baseline was generally the final visit of the qualifying study (RA101495-01.201 or RA101495-01.203). Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | micromole per litre (umol/L) | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49) |
| |||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Total Hemoglobin Values at Each Time Point | Total Hemoglobin Values were analyzed for hematology assessments. | Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Baseline was generally the final visit of the qualifying study (RA101495-01.201 or RA101495-01.203). Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | grams per litre (g/L) | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49) |
| |||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Free Hemoglobin Values at Each Time Point | Free Hemoglobin Values were analyzed for hematology assessments. | Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Baseline was generally the final visit of the qualifying study (RA101495-01.201 or RA101495-01.203). Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | milligrams per decilitre (mg/dL) | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49) |
| |||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Haptoglobin Values at Each Time Point | Haptoglobin values were analyzed for hematology assessments. | Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Baseline was generally the final visit of the qualifying study (RA101495-01.201 or RA101495-01.203). Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | g/L | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49) |
| |||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Reticulocytes at Each Time Point | Reticulocytes values were analyzed for hematology assessments. | Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | 10^12 reticulocytes (cells)/L | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49) |
| |||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Hemoglobinuria Values at Each Time Point | Hemoglobinuria was assessed using a urine colorimetric scoring system with a score of 1 through 10 where 1 represents no hemoglobinuria and 10 represents maximum hemoglobinuria. | Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Baseline was generally the final visit of the qualifying study (RA101495-01.201 or RA101495-01.203). Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48 and Final Study Visit (Month 49) |
| |||||||||||||||||||||||||||||||||
| Secondary | Plasma Concentrations of RA101495 and Its Major Metabolite(s) | Blood samples of RA101495 (zilucoplan) and its metabolites (RA102758 and RA103488) were collected for Plasma concentration analysis. | Pharmacokinetic (PK) population included all participants in the Safety population who had at least 1 plasma sample obtained for PK assessment. Here, 'n' (Number analyzed) signifies participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | micrograms per litre (ug/L) | Predose: At Day 1 (Screening), Month 1, 2, 3, 6, 9, 12, and Final Study Visit (Month 49) |
| |||||||||||||||||||||||||||||||||
| Secondary | Maximum Plasma Concentration (Cmax) of RA101495 | Cmax is the maximum plasma concentration. | PK population included all participants in the Safety population who had at least 1 plasma sample obtained for PK assessment. Data was not collected and analyzed for this outcome measure due to change in planned analysis. | Posted | At Day 1, Month 1, 2, 3, 6, 9, and 12 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Time Corresponding to Cmax (Tmax) of RA101495 | tmax is the time to corresponding Cmax. | PK population included all participants in the Safety population who had at least 1 plasma sample obtained for PK assessment. Data was not collected and analyzed for this outcome measure due to change in planned analysis. | Posted | At Day 1, Month 1, 2, 3, 6, 9, and 12 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Area Under the Drug Concentration-time Curve (AUC0-t) of RA101495 | AUC0-t is area under the drug concentration-time curves. | PK population included all participants in the Safety population who had at least 1 plasma sample obtained for PK assessment. As per the change in planned analyses, samples/data were not collected, and AUC0-t not calculated. | Posted | At Day 1, Month 1, 2, 3, 6, 9, and 12 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Total Complement (CH50) Levels | Blood samples collection were planned to assess complement (CH50) levels. The planned analysis of CH50 was not performed because the CH50 assay was not able to be validated due to lack of reproducibility of the manufacturer's kits. | Pharmacodynamic (PD) population included all participants in the Safety population who had at least 1 plasma sample obtained for PD assessment. Data was not collected and analyzed for this outcome measure due to change in planned analysis. | Posted | At Day 1, Month 1, 2, 3, 6, 9, and 12 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point | Blood samples were collected for measurement of sRBC lysis for the Classical Complement Pathways. | PD population included all participants in the Safety population who had at least 1 plasma sample obtained for PD assessment. Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | percent lysis of sheep erythrocytes | Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49) |
| |||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point | Blood samples were collected for measurement of membrane attack complex (MAC) by Wieslab ELISA for alternative complement pathway. | PD population included all participants in the Safety population who had at least 1 plasma sample obtained for PD assessment. Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | percentage of activity | Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49) |
| |||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Complement Component 5 (C5) Values at Each Time Point | Blood samples were collected for measurement of Complement component 5 (C5) levels. | PD population included all participants in the Safety population who had at least 1 plasma sample obtained for PD assessment. Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points. | Posted | Mean | Standard Deviation | ug/mL | Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49) |
|
From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Zilucoplan-Cohort A (Eculizumab Naïve) | Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. | 0 | 10 | 1 | 10 | 9 | 10 |
| EG001 | Zilucoplan-Cohort B (Eculizumab Switch) | Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. | 0 | 6 | 3 | 6 | 6 | 6 |
| EG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. | 0 | 3 | 2 | 3 | 2 | 3 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Tongue haematoma | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Enterocolitis infectious | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Pneumonia pneumococcal | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Rotator cuff syndrome | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Suicide attempt | Psychiatric disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA Version 23.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Haemolysis | Blood and lymphatic system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Aplastic anaemia | Blood and lymphatic system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Pancytopenia | Blood and lymphatic system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Lacrimation increased | Eye disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Large intestine polyp | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Oral discomfort | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Oral pain | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Rectal polyp | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Retching | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Chest pain | General disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Chills | General disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Injection site haematoma | General disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Alloimmunisation | Immune system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Skin bacterial infection | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Post vaccination syndrome | Injury, poisoning and procedural complications | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Animal bite | Injury, poisoning and procedural complications | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Bone contusion | Injury, poisoning and procedural complications | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Eye contusion | Injury, poisoning and procedural complications | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Post-traumatic pain | Injury, poisoning and procedural complications | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Tooth fracture | Injury, poisoning and procedural complications | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Traumatic haematoma | Injury, poisoning and procedural complications | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Grip strength decreased | Investigations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Limb discomfort | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Muscle tightness | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Plantar fasciitis | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Rotator cuff syndrome | Musculoskeletal and connective tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Amnesia | Nervous system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Sensory disturbance | Nervous system disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Paroxysmal nocturnal haemoglobinuria | Renal and urinary disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Chromaturia | Renal and urinary disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Laryngospasm | Respiratory, thoracic and mediastinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Actinic keratosis | Skin and subcutaneous tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Psoriasis | Skin and subcutaneous tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA Version 23.0 | Non-systematic Assessment |
| |
| Lymphoedema | Vascular disorders | MedDRA Version 23.0 | Non-systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| UCB | Cares | 001 844 599 2273 | UCBCares@ucb.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 15, 2021 | Sep 7, 2022 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000719268 | zilucoplan |
Not provided
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
| White |
|
| Not Reported |
|
| Not Reported |
|
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
|
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
|
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
|
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
|
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
|
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
|
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
|
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
|
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
|
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
|
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
|
| OG002 | Zilucoplan (Inadequate Responder to Eculizumab) | Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily. |
|
|