| Primary | OLTP: Number of Participants With Treatment-emergent Adverse Events (TEAEs) | | The safety analysis set included all participants who received any amount of study drug. | Posted | | Count of Participants | | Participants | | Up to Day 225 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| | | Title | Denominators | Categories |
|---|
| | |
| |
| Secondary | OLTP: Percent Change in Lactate Dehydrogenase (LDH) From Pre-treatment to End-of-treatment Period | | The full analysis set (FAS) included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | percent change | | Screening (Day 1) through Day 225 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Percent Change in LDH From Pre-treatment Through Day 29 | | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Mean | Standard Deviation | percent change | | Screening (Day 1) through Day 29 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Percentage of Participants Who Were Transfusion-free From Baseline Through Week 32 | | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Baseline through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Percentage of Participants Who Were Transfusion-free From Week 4 Through Week 32 | | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Week 4 through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Rate of Red Blood Cell (RBC) Transfusions From Baseline Through Week 32 | The rate of units of transfusion for a participant was calculated based on the duration of treatment exposure of the participant. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Number | 95% Confidence Interval | transfusions per person-year | | Baseline through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Rate of RBC Transfusions From Week 4 Through Week 32 | The rate of units of transfusion for a participant was calculated based on the duration of treatment exposure of the participant. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Number | 95% Confidence Interval | transfusions per person-year | | Week 4 through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Number of RBC Units Transfused From Baseline Through Week 32 | | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Mean | Standard Deviation | RBC units | | Baseline through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Number of RBC Units Transfused From Week 4 Through Week 32 | | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Mean | Standard Deviation | RBC units | | Week 4 through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Percentage of Participants With Breakthrough Hemolysis From Baseline Through Week 32 | Breakthrough hemolysis was defined as having an LDH ≥ 2 x upper limit of normal (ULN) and having signs or symptoms. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Baseline through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Percentage of Participants With Breakthrough Hemolysis From Week 4 Through Week 32 | Breakthrough hemolysis was defined as having an LDH ≥ 2 x ULN and having signs or symptoms. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Week 4 through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Percentage of Participants Who Maintained Adequate Control of Hemolysis From Day 1 Through Week 32 | Adequate control was defined as LDH ≤ 1.5 x ULN from Day 1 through Week 32. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Day 1 through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Percentage of Participants Who Maintained Adequate Control of Hemolysis From Week 8 Through Week 32 | Adequate control was defined as LDH ≤ 1.5 x ULN from Week 8 through Week 32. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Week 8 through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Number of Participants With Adequate Control of Hemolysis at Each Visit | Adequate control at a visit was defined as having LDH ≤1.5 x ULN at that visit. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure and "Number analyzed" is the number of participants evaluated at each time point. | Posted | | Count of Participants | | Participants | | Days 1, 8, 15, 29, 43, 57, 71, 85, 113, 141, 169, 197, 225 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Number of Participants With Normalization of Their LDH at Each Visit | Normalization was defined as LDH ≤ 1.0 x ULN at that visit. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure and "Number analyzed" is the number of participants evaluated at each time point. | Posted | | Count of Participants | | Participants | | Days 1, 8, 15, 29, 43, 57, 71, 85, 113, 141, 169, 197, 225 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Average LDH From Baseline Through Week 32 | The trapezoidal rule was used to calculate area under the curve (AUC). Individual mean LDH is defined as AUC divided by (last assessment date of LDH - first assessment date of LDH) of specific period. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Mean | Standard Deviation | Units per Liter (U/L) | | Baseline through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Average LDH From Week 8 Through Week 32 | The trapezoidal rule was used to calculate AUC. Individual mean LDH is defined as AUC divided by (last assessment date of LDH - first assessment date of LDH) of specific period. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | U/L | | Week 8 through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Percentage of Participants With Hemoglobin Stabilization From Baseline Through Week 32 | Hemoglobin stabilization was defined as not receiving an RBC transfusion and having no decrease in hemoglobin level of ≥ 2 grams per deciliter (g/dL). | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Baseline through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Percentage of Participants With Hemoglobin Stabilization From Week 4 Through Week 32 | Hemoglobin stabilization was defined as not receiving an RBC transfusion and having no decrease in hemoglobin level of ≥ 2 g/dL. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Week 4 through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Change From Baseline in Hemoglobin Levels | | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | g/dL | | Baseline, Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Change From Baseline in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Score | The FACIT-Fatigue is a 13-item, self-reported measure assessing an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health-related quality of life (QoL) in participants with cancer and other chronic illnesses. The FACIT-fatigue assesses the level of fatigue using a Likert scale ranging from 0 (not at all) to 4 (very much). Total scores range from 0 to 52, with higher scores indicating a higher quality of life. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | score on a scale | | Baseline, Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Change From Baseline in Health Related Quality of Life (HRQoL) as Measured by the Global Health Status Subscale of the European Organization for Research and Treatment of Cancer (EORTC)- Quality of Life Cancer Patients Questionnaire (QLQ) - 30 Scale | EORTC-QLQ-C30 is a 30-item self-reported questionnaire composed of both multi-item and single scales, including a global health status/quality of life (GHS/QoL) scale. The GHS subscale scores ranged from 0 to 100 with higher scores indicating better quality of life. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | score on a scale | | Baseline, Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Change From Baseline in Physical Function (PF) Scores on the EORTC QLQ-C30 | EORTC-QLQ-C30 is a 30-item participant self-report questionnaire composed of both multi-item and single scales, including a PF scale. PF subscale scores ranged from 0 to 100 with higher scores indicating better quality of life. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | score on a scale | | Baseline, Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Change From Baseline in Total Complement Hemolytic Activity Assay (CH50) | This assay assessed the activity of the classical pathway of complement to measure C5 activity. | The FAS included all enrolled participants who received any amount of study drug and had at least 1 post-baseline assessment. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | International units per mL (IU/mL) | | Baseline through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP and OLEP: Concentration of Total Pozelimab in Serum | | The pharmacokinetic (PK) analysis population included all participants who received any amount of study drug and who had at least 1 non-missing result following the first dose of study drug. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure and "Number analyzed" is the number of participants evaluated at each time point. | Posted | | Mean | Standard Deviation | milligrams per liter (mg/L) | | Pre-dose Days 28, 56, 84, 112, 140, 168, 196, 224, 392, 588; Post-dose Day 28 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Concentration of Total Eculizumab in Serum | | The PK analysis population included all participants who received any amount of study drug and who had at least 1 non-missing result following the first dose of study drug. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure and "Number analyzed" is the number of participants evaluated at each time point. | Posted | | Mean | Standard Deviation | mg/L | | Pre-dose Days 0, 14, 28, 56, 84, 112, 140, 168, 196, 224 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP and OLEP: Concentration of Total Cemdisiran in Plasma | | The PK analysis population included all participants who received any amount of study drug and who had at least 1 non-missing result following the first dose of study drug. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure and "Number analyzed" is the number of participants evaluated at each time point. | Posted | | Mean | Standard Deviation | nanograms per milliliter (ng/mL) | | Pre-dose and Post-dose Days 0, 84, 196, 224, 588 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP and OLEP: Concentration of Total C5 in Plasma | | The PK analysis population included all participants who received any amount of study drug and who had at least 1 non-missing result following the first dose of study drug. Here, "Overall Number of Participants Analyzed" is the number of participants evaluable for this outcome measure and "Number analyzed" is the number of participants evaluated at each time point. | Posted | | Mean | Standard Deviation | mg/L | | Pre-dose Days 0, 7, 14, 28, 56, 84, 112, 140, 168, 196, 224, 392, 588 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Number of Participants With Pozelimab Anti-drug Antibodies (ADA) | | The ADA analysis set included all participants who received study drug and had at least 1 non-missing ADA result following the first study dose. | Posted | | Count of Participants | | Participants | | Baseline through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Number of Participants With Cemdisiran ADA | | The ADA analysis set included all participants who received study drug and had at least 1 non-missing ADA result following the first study dose. | Posted | | Count of Participants | | Participants | | Baseline through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLTP: Number of Participants Who Received Dose Intensification With TEAEs | | No participants required treatment intensification during the study. | Posted | | | | | | Baseline through Week 32 | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLEP: Number of Participants With TEAEs | | The OLEP safety analysis set included all participants who participated in the OLEP who received any amount of study drug in the OLEP. | Posted | | Count of Participants | | Participants | | Day 1 through Week 52 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLEP: Percent Change in LDH From Day 1 Through Week 24 of the OLEP | | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Mean | Standard Deviation | percent change | | Day 1 through Week 24 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLEP: Percent Change in LDH From Day 1 Through Week 52 of the OLEP | | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Mean | Standard Deviation | percent change | | Day 1 through Week 52 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLEP: Percentage of Participants Who Were Transfusion-free From Day 1 Through Week 24 of the OLEP | | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Day 1 through Week 24 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLEP: Percentage of Participants Who Were Transfusion-free From Day 1 Through Week 52 of the OLEP | | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Day 1 through Week 52 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
| |
| Secondary | OLEP: Rate of RBC Transfusions From Day 1 Through Week 24 of the OLEP | The rate of units of transfusion for a participants was calculated based on the duration of treatment exposure of the participant. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Number | 95% Confidence Interval | transfusions per person-year | | Day 1 through Week 24 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Rate of RBC Transfusions From Day 1 Through Week 52 of the OLEP | The rate of units of transfusion for a participants was calculated based on the duration of treatment exposure of the participant. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Number | 95% Confidence Interval | transfusions per person-year | | Day 1 through Week 52 of the OLEP | | | | ID | Title | Description |
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| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Number of RBC Units Transfused From Day 1 Through Week 24 of the OLEP | | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Mean | Standard Deviation | RBC units | | Day 1 through Week 24 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Number of RBC Units Transfused From Day 1 Through Week 52 of the OLEP | | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Mean | Standard Deviation | RBC units | | Day 1 through Week 52 of the OLEP | | | | ID | Title | Description |
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| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Percentage of Participants With Breakthrough Hemolysis From Day 1 Through Week 24 of the OLEP | Breakthrough hemolysis was defined as having an LDH ≥ 2 x ULN and having signs or symptoms. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Day 1 through Week 24 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Percentage of Participants With Breakthrough Hemolysis From Day 1 Through Week 52 of the OLEP | Breakthrough hemolysis was defined as having an LDH ≥ 2 x ULN and having signs or symptoms. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Day 1 through Week 52 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Percentage of Participants Who Maintained Adequate Control of Hemolysis From Day 1 Through Week 24 of the OLEP | Adequate control was defined as LDH ≤ 1.5 x ULN. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Day 1 through Week 24 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Percentage of Participants Who Maintained Adequate Control of Hemolysis From Day 1 Through Week 52 of the OLEP | Adequate control was defined as LDH ≤ 1.5 x ULN. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Day 1 through Week 52 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Number of Participants Who Maintained Adequate Control of Hemolysis at Each Visit in OLEP | Adequate control was defined as LDH ≤ 1.5 x ULN at that visit. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Count of Participants | | Participants | | Baseline, Days 57, 113, 169, 225, 281, 365 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Number of Participants With Normalization of Their LDH at Each Visit in OLEP | Normalization was defined as LDH ≤ 1.0 x ULN at that visit. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Count of Participants | | Participants | | Baseline, Days 57, 113, 169, 225, 281, 365 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Average LDH From Day 1 Through Week 24 of the OLEP | The trapezoidal rule was used to calculate AUC. Individual mean LDH is defined as AUC divided by (last assessment date of LDH - first assessment date of LDH) of specific period. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Mean | Standard Deviation | U/L | | Day 1 through Week 24 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Average LDH From Day 1 Through Week 52 of the OLEP | The trapezoidal rule was used to calculate AUC. Individual mean LDH is defined as AUC divided by (last assessment date of LDH - first assessment date of LDH) of specific period. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Mean | Standard Deviation | U/L | | Day 1 through Week 52 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Percentage of Participants With Hemoglobin Stabilization From Day 1 Through Week 24 of the OLEP | Hemoglobin stabilization was defined as not receiving an RBC transfusion and having no decrease in hemoglobin level of ≥ 2 g/dL. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Day 1 through Week 24 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Percentage of Participants With Hemoglobin Stabilization From Day 1 Through Week 52 of the OLEP | Hemoglobin stabilization was defined as not receiving an RBC transfusion and having no decrease in hemoglobin level of ≥ 2 g/dL. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Number | 95% Confidence Interval | percentage of participants | | Day 1 through Week 52 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Change From Baseline in Hemoglobin Levels | | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Mean | Standard Deviation | g/dL | | Day 1 and Week 24 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Change From Baseline in Hemoglobin Levels | | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Mean | Standard Deviation | g/dL | | Day 1 and Week 52 of the OLEP | | | | ID | Title | Description |
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| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Change From Baseline in FACIT-Fatigue Score | The FACIT-Fatigue is a 13-item, self-reported measure assessing an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health-related quality of life (QoL) in patients with cancer and other chronic illnesses. The FACIT-fatigue assesses the level of fatigue using a Likert scale ranging from 0 (not at all) to 4 (very much). Total scores range from 0 to 52, with higher scores indicating a higher quality of life. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Mean | Standard Deviation | score on a scale | | Day 1 and Week 52 of the OLEP | | | | ID | Title | Description |
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| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Change From Baseline in HRQoL as Measured by the Global Health Status Subscale of the EORTC-QLQ-30 Scale | EORTC-QLQ-C30 is a 30-item self-reported questionnaire composed of both multi-item and single scales, including a global health status/quality of life (GHS/QoL) scale. GHS subscale scores ranged from 0 to 100 with higher scores indicating better quality of life. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Mean | Standard Deviation | score on a scale | | Day 1 and Week 52 of the OLEP | | | | ID | Title | Description |
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| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Change From Baseline in PF Scores on the EORTC QLQ-C30 | EORTC-QLQ-C30 is a 30-item participant self-report questionnaire composed of both multi-item and single scales, including a PF scale. PF subscale scores ranged from 0 to 100 with higher scores indicating better quality of life. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Mean | Standard Deviation | score on a scale | | Day 1 and Week 52 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Change From Baseline in CH50 | This assay assessed the activity of the classical pathway of complement to measure C5 activity. | The OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline assessment in the OLEP. | Posted | | Mean | Standard Deviation | IU/mL | | Baseline, Week 16, Week 32, Week 52 of the OLEP | | | | ID | Title | Description |
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| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Number of Participants With Pozelimab ADA | | The ADA-OLEP analysis set included all participants who received study drug in the OLEP and had at least 1 non-missing ADA result following the first OLEP dose. | Posted | | Count of Participants | | Participants | | Day 1 through Week 52 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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| Secondary | OLEP: Number of Participants With Cemdisiran ADA | | The ADA-OLEP analysis set included all participants who received study drug in the OLEP and had at least 1 non-missing ADA result following the first OLEP dose. | Posted | | Count of Participants | | Participants | | Day 1 through Week 52 of the OLEP | | | | ID | Title | Description |
|---|
| OG000 | Participants With PNH | Participants received cemdisiran by subcutaneous (SC) injection along with their usual dose of eculizumab by intravenous (IV) infusion. After completion of Open Label Treatment Period (OLTP), for participants who willing to continue to receive study treatment, SC pozelimab and cemdisiran Q4W were administered up to 52 weeks during the Open-Label Extension Period (OLEP) |
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