| Primary | Percentage of Participants With Active Disease at Baseline Who Achieved Normalization of Serum Albumin and Improvement in Prespecified Clinical Outcomes at Week 24 | Normalization of serum albumin was defined as serum albumin within the normal range at least 70 percent (%) of measurements between weeks 12 and 24, and no single albumin measurement of <2.5 grams per deciliter (g/dL) between weeks 12 and 24, and no requirement for albumin infusion between weeks 12 and 24. Improvement in the following 4 prespecified clinical outcomes that were evaluable for improvement at baseline, without worsening of the others: Daily bowel movement frequency, the presence and severity of facial edema (physician-reported), the presence and severity of peripheral edema (physician-reported), and the participant/caregiver assessment of frequency of problematic abdominal pain. Percentage of participants with active disease at baseline who achieved normalization of serum albumin and improvement in prespecified clinical outcomes at Week 24 were reported. | The FAS included all enrolled participants who received the study drug. | Posted | | Number | 90% Confidence Interval | percentage of participants | | At Week 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Severity of TEAEs | TEAEs are defined as AEs that developed or worsened during the on-treatment period. The on-treatment period is defined as the time from first dose of investigational product up to 21 weeks after the last dose of investigational product. Severity of TEAEs was graded according to the following scale: Mild: Does not interfere in a significant manner with the patient's normal functioning level, Moderate: Produces some impairment of functioning but is not hazardous to health and Severe: Produces significant impairment of functioning or incapacitation and is a definite hazard to the participants health. | The safety analysis set included all enrolled participants who received the study drug. | Posted | | Count of Participants | | Participants | | From start of study drug administration up to approximately 144 weeks | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Number of Participants With Improvement in Most Bothersome Signs and Symptoms at Week 24 | Improvement in most bothersome sign/symptom determined using semi-structured concept elicitation interview, from 'core' clinical endpoints of frequency of bowel movements, peripheral edema, facial edema, abdominal pain frequency, nausea, vomiting, stool consistency. | FAS included all enrolled participants who received the study drug. | Posted | | Count of Participants | | Participants | | At Week 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24 | Daily bowel movements captured by e-diary. The number of bowel movements per day was calculated each week of the study. It was based on a 1-week average and calculated as the sum of the number of bowel movements in a given week divided by the number of days with non-missing bowel movement frequency data. If more than 3 days of bowel movement data was missing in a given week, bowel movement frequency data was considered missing for that week. | FAS included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories. | Posted | | Median | Full Range | Bowel movements per day | | Baseline, Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24 | The number of days per week with >=1 loose/watery bowel movement, is calculated each week of the study as the sum of the number of days with >=1 loose/watery bowel movement in a given week divided by the number of days with non-missing stool consistency data and then multiplied by 7, is presented. If more than 3 days of stool consistency data was missing in a given week, stool consistency data was considered missing for that week. | FAS included all enrolled participants who received any study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories. | Posted | | Median | Full Range | Days per week | | Baseline, Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Number of Participants With Abdominal Ascites at Week 24 | The measurement of abdominal ascites (excess abdominal fluid) was based on abdominal circumference. Abdominal circumference was measured regardless of the physician's assessment of the presence or absence of ascites. | FAS included all enrolled participants who received any study drug. | Posted | | Count of Participants | | Participants | | Baseline up to Week 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Absolute Value of Albumin at Specified Timepoints up to Week 24 | Blood samples were collected from participants at defined time points for the assessment of albumin. Absolute value of albumin at specified timepoints was reported. | FAS included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories. | Posted | | Mean | Standard Deviation | g/dL | | Baseline, Day 2, Weeks 1, 2, 3, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22 and 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Absolute Values of Protein, and Immunoglobulin G (IgG) at Baseline and Week 24 | Blood samples were collected from participants at defined time points for the assessment of protein and IgG. Absolutes values of protein and IgG measured as g/L at baseline and Week 24 was reported. | The safety analysis set included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories. | Posted | | Mean | Standard Deviation | g/L | | Baseline, Week 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Absolute Values of Immunoglobulin (Ig), Immunoglobulin M (IgM), and Immunoglobulin A (IgA) at Baseline and Week 24 | Blood samples were collected from participants at defined time points for the assessment of Ig, IgM and IgA. Absolute value of Ig, IgM and IgA measured as mg/dL at baseline and Week 24 was reported. | The safety analysis set included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories. | Posted | | Mean | Standard Deviation | mg/dL | | Baseline, Week 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Absolute Values of Vitamin B12 at Baseline and Week 24 | Blood samples were collected from participants at defined time points for the assessment of vitamin B12. Absolute values of vitamin B12 at baseline and Week 24 was reported. | The safety analysis set included all enrolled participants who received the study drug. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | picomoles per liter (pmol/L) | | Baseline, Week 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Absolute Values of Iron and Unsaturated Iron Binding Capacity at Baseline and Week 24 | Blood samples were collected from participants at defined time points for the assessment of iron indices. Absolute values of unsaturated iron and unsaturated iron binding capacity measured as micromoles per liter (mcmol/L) at baseline and Week 24 was reported. | The safety analysis set included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories. | Posted | | Mean | Standard Deviation | mcmol/L | | Baseline, Week 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Absolute Values of Vitamin B9 up to Week 24 | Absolute Values of Vitamin B9 - Central Lab | Number of participants based on the total in the safety analysis set (SAF) | Posted | | Mean | Standard Deviation | nanomoles per liter (nmol/L) | | Baseline up to Week 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Absolute Values of Ferritin at Baseline and Week 24 | Blood samples were collected from participants at defined time points for the assessment of iron indices. Absolute values of ferritin was reported. | The safety analysis set included all enrolled participants who received the study drug. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | | Mean | Standard Deviation | microgram per liter (mcg/L) | | Baseline, Week 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Absolute Values of Magnesium, Total Cholesterol, and Triglycerides at Week 24 | Blood samples were collected from participants at defined time points for the assessment of magnesium, total cholesterol, and triglycerides. Absolute values of magnesium, total cholesterol, and triglycerides measured as mmol/L at baseline and Week 24 was reported. | The safety analysis set included all enrolled participants who received the study drug. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure and "number analyzed" signifies those participants who were evaluable for specified timepoints in categories. | Posted | | Mean | Standard Deviation | mmol/L | | Baseline, Week 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Alpha-1 Antitrypsin Levels in Stool at Week 12 and Week 24 | | The FAS included all enrolled participants who received the study drug. | Posted | | Mean | Standard Deviation | milligrams per deciliter (mg/dL) | | Week 12, Week 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Alpha-1 Antitrypsin Levels in Blood at Week 12 and Week 24 | | Number of participants based on the total in the safety analysis set (SAF) | Posted | | Mean | Standard Deviation | mg/dL | | Week 12, Week 24 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Percentage of Participants With Active Disease at Baseline Who Maintained Disease Control | Measured by normalization of serum albumin, no worsening of facial or peripheral edema, increase in bowel movement, or increase in abdominal pain frequency, no increase in dose of permitted concomitant medication for the treatment of PLE at any time as described in the protocol | All enrolled participants who received the study drug. 1 participant was consented to Protocol Amendment 5, and completed the study at week 104. The remaining 9 participants were consented to Protocol Amendment 6 and completed the study at week 144. | Posted | | Number | | percentage of participants | | Weeks 12 to 48; Weeks 12 to 144; Weeks 24 to 48; Weeks 48 to 96; Weeks 96 to 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Physician Assessment of Facial Edema Based on a 5-point Likert Rating Scale | The physician assessment of facial edema is based on a 5-point Likert scale ranging from no edema (1) to very severe edema (5). | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | score on a scale | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Physician Assessment of Peripheral Edema Based on a 5-point Likert Rating Scale | The physician assessment of peripheral edema is based on a 5-point Likert scale ranging from no edema (1) to very severe edema (5). | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | score on a scale | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Food and Drink Limitations as Assessed by the PedsQL™ GI Symptom Scales' Food and Drink Limits Sub-scale | Frequency of limitations is assessed on a 5-point Likert response scale, ranging from never a problem (0) to almost always a problem (4). Items were reverse scored and linearly transformed to a 0 to 100 scale, where lower scores indicate more frequent problems with food and drink limitations. | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at this time point for this outcome measure. | Posted | | Mean | Standard Deviation | score on a scale | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Health-Related Quality of Life (HRQoL) as Assessed by the PedsQL™ Generic Core Scales | Physical functioning, emotional functioning, social functioning, and school/work/studies functioning is assessed using a 5-point Likert scale, ranging from never a problem (0) to almost always a problem (4). Items were reverse-scored and linearly transformed to a 0 to 100 scale, with higher scores indicating better HRQoL. The total scale score is computed as the sum of all the items over the number of items answered on individual scales. Subscale score is calculated as the sum of the items in the scale divided by the number of items answered in the scale. | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at this time point for this outcome measure. | Posted | | Mean | Standard Deviation | score on a scale | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Number of Participants With Albumin Infusion by 24 Week Periods | | All enrolled participants who received the study drug. 1 participant was consented to Protocol Amendment 5, and completed the study at week 104. The remaining 9 participants were consented to Protocol Amendment 6 and completed the study at week 144. | Posted | | Count of Participants | | Participants | | Weeks 0 to 24; Weeks 24 to 48; Weeks 48 to 72; Weeks 72 to 96; Weeks 96 to 120; Weeks 120 to 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Albumin Values | | | Posted | | Mean | Standard Deviation | grams per deciliter (g/dL) | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Percentage Change From Baseline in Albumin Values | | | Posted | | Mean | Standard Deviation | percentage of change | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for Albumin Values | | | Posted | | Median | 95% Confidence Interval | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Protein Values | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at this time point for this outcome measure. | Posted | | Mean | Standard Deviation | grams per liter (g/L) | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for Total Protein | | | Posted | | Median | 95% Confidence Interval | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Immunoglobulin (Ig) Values | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | mg/dL | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for Ig Values | | All enrolled participants with available Ig lab reference ranges. | Posted | | Median | Full Range | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in IgG Values | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | g/L | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for IgG Values | | | Posted | | Median | 95% Confidence Interval | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in IgM Values | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | mg/dL | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for IgM Values | | All enrolled participants who received the study drug who were evaluable for this outcome measure. | Posted | | Median | Full Range | days | | Baseline up to Week 44 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in IgA Values | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | mg/dL | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for IgA Values | | | Posted | | Median | Full Range | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Vitamin B12 Values | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | picomoles per liter (pmol/L) | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for Vitamin B12 Values | | | Posted | | Median | Full Range | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Vitamin B9 (Folate) Values | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | nanomole per liter (nmol/L) | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for Vitamin B9 (Folate) Values | | | Posted | | Median | Full Range | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Iron Values | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | micromole/liter (umol/L) | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for Iron Values | | | Posted | | Median | Full Range | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Unsaturated Iron Binding Capacity | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | umol/L | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for Unsaturated Iron Binding Capacity | | | Posted | | Median | Full Range | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Ferritin Values | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | microgram per liter (ug/L) | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for Ferritin Values | | | Posted | | Median | Full Range | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Magnesium Values | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | millimoles per liter (mmol/L) | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for Magnesium Values | | | Posted | | Median | Full Range | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Fasting Cholesterol Values | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | mmol/L | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for Fasting Cholesterol Values | | | Posted | | Median | Full Range | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Fasting Triglycerides Values | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | mmol/L | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Time to First Normalization for Fasting Triglycerides Values | | | Posted | | Median | Full Range | days | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Number of Participants Who Used Concomitant Medication | | | Posted | | Count of Participants | | Participants | | Baseline up to Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Number of Hospitalization Days by 24 Week Period | | All enrolled participants who received the study drug. 1 participant was consented to Protocol Amendment 5 and completed the study at week 104. The remaining 9 participants were consented to Protocol Amendment 6 and completed the study at week 144. | Posted | | Median | Full Range | days | | Weeks 0 to 24; Weeks 24 to 48; Weeks 48 to 72; Weeks 72 to 96; Weeks 96 to 120; Weeks 120 to 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Body Weight Z-Score | Weight-for-age z-score compares a participant's weight to children of the same age and sex from a healthy reference population. A z-score reflects an individual score as compared to a population mean and is expressed in units of standard deviation above (positive values) and below (negative values). Body weight z-score, regardless of magnitude, represents catch-up growth. | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | z-score | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Height Z-Score | Height-for-age z-score compares a participant's height to children of the same age and sex from a healthy reference population. A z-score reflects an individual score as compared to a population mean and is expressed in units of standard deviation above (positive values) and below (negative values). Any increase in height z-score, regardless of magnitude, represents catch-up growth. | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | z-score | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Change From Baseline in Total Complement Activity Complement Hemolytic Assay (CH50) | | All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure. | Posted | | Mean | Standard Deviation | international units/milliliter (IU/mL) | | Baseline and Week 144 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Concentrations of Total Pozelimab in Serum | | The Pharmacokinetic Analysis Set (PKAS) includes all treated participants who received any amount of study drug and had at least 1 non-missing total pozelimab measurement following the first dose of study drug. The PKAS is based on the actual treatment received (as treated) rather than as randomized. | Posted | | Mean | Standard Deviation | mg/L | | Baseline up to Week 164 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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| Secondary | Number of Participants With Treatment-emergent Anti-drug Antibodies (ADA) to Pozelimab | | The Anti-Drug Antibody Analysis Set (AAS) includes all treated participants who received any amount of study drug and had at least 1 non-missing ADA result following the first dose of study drug. The AAS is based on the actual treatment received (as treated) rather than as randomized. | Posted | | Count of Participants | | Participants | | Baseline up to Week 164 | | | | ID | Title | Description |
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| OG000 | Pozelimab Injection | Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight <10 kg, 200 mg for >=10 kg and <20 kg, 350 mg for >=20 kg and <40 kg, 500 mg for >=40 kg and <60 kg, and 800 mg for >=60 kg over the treatment period. |
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