| Primary | Annualized Bleeding Rate (ABR) | The primary efficacy variable was the ABR while on prophylaxis to prevent bleeding episodes. The ABR was defined as the number of bleeding episodes per year. | Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form and received at least one dose of APVO101. | Posted | | Mean | Standard Deviation | bleeding episodes per year | | Exposure Day 1 up to 50 exposure days (approximately 6 months) | | | | ID | Title | Description |
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| OG000 | APVO101 - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Primary | Annualized Bleeding Rate (ABR) Overall | The primary efficacy variable was the ABR while on prophylaxis to prevent bleeding episodes. The ABR was defined as the number of bleeding episodes per year. | Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form and received at least one dose of APVO101. | Posted | | Mean | Standard Deviation | bleeding episodes per year | | Exposure Day 1 through study completion (up to 2.5 years) | | | | ID | Title | Description |
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| OG000 | APVO101 - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Concentration (Cmax) | Maximum post-infusion plasma concentration of FIX activity was measured at the following time points post infusion: 15-30 minutes, 4-6 hours, 24-26 hours and 46-50 hours. | Analysis population included all participants for whom legally acceptable representative had signed informed consent/assent form, were in non-bleeding state, participated in single PK assessment at start of study and for whom an adequate PK profile had been obtained. "Number analyzed" signifies participants evaluable at specified time points. | Posted | | Mean | Standard Deviation | IU/dL | | Pre-infusion to 50 hours post-infusion | | | | ID | Title | Description |
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| OG000 | APVO101 - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Area Under the Curve (0-inf) | Area under plasma concentration curve, FIX activity-time profile from time zero extrapolated to infinity. FIX activity was measured at the following time points post infusion: 15-30 minutes, 4-6 hours, 24-26 hours and 46-50 hours. | Analysis population included all participants for whom legally acceptable representative had signed informed consent/assent form, were in non-bleeding state, participated in single PK assessment at start of study and for whom an adequate PK profile had been obtained. "Number analyzed" signifies participants evaluable at specified time points. | Posted | | Mean | Standard Deviation | IU/dL/hr | | Pre-infusion to 50 hours post-infusion | | | | ID | Title | Description |
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| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Mean Residence Time (MRT) | MRT is the average time the molecules of drug reside in the body before elimination. | Analysis population included all participants for whom legally acceptable representative had signed informed consent/assent form, were in non-bleeding state, participated in single PK assessment at start of study and for whom an adequate PK profile had been obtained. "Number analyzed" signifies participants evaluable at specified time points. | Posted | | Mean | Standard Deviation | hours | | Pre-infusion to 50 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Terminal Half-Life (t 1/2) | Terminal half-life is the length of time required for the concentration of drug to decrease by one half of its starting dose in the body. FIX activity was measured at the following time points post infusion: 15-30 minutes, 4-6 hours, 24-26 hours and 46-50 hours. | Analysis population included all participants for whom legally acceptable representative had signed informed consent/assent form, were in non-bleeding state, participated in single PK assessment at start of study and for whom an adequate PK profile had been obtained. "Number analyzed" signifies participants evaluable at specified time points. | Posted | | Mean | Standard Deviation | hours | | Pre-infusion to 50 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Clearance (CL) | Clearance is a measure of the volume of plasma from which FIX activity is removed per unit time. Weight normalized clearance calculated as CL=Dose/AUC 0-inf. FIX activity was measured at the following time points post infusion: 15-30 minutes, 4-6 hours, 24-26 hours and 46-50 hours. | Analysis population included all participants for whom legally acceptable representative had signed informed consent/assent form, were in non-bleeding state, participated in single PK assessment at start of study and for whom an adequate PK profile had been obtained. "Number analyzed" signifies participants evaluable at specified time points. | Posted | | Mean | Standard Deviation | mL/(kg*hr) | | Pre-infusion to 50 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Volume of Distribution at Steady-State (Vdss) | Volume of distribution is defined as the theoretical volume in which the total amount of FIX would need to be uniformly distributed to produce the observed plasma concentration of FIX. Steady state volume of distribution (Vdss) is the apparent volume of distribution at steady-state. FIX activity was measured at the following time points post infusion: 15-30 minutes, 4-6 hours, 24-26 hours and 46-50 hours. | Analysis population included all participants for whom legally acceptable representative had signed informed consent/assent form, were in non-bleeding state, participated in single PK assessment at start of study and for whom an adequate PK profile had been obtained. "Number analyzed" signifies participants evaluable at specified time points. | Posted | | Mean | Standard Deviation | mL/kg | | Pre-infusion to 50 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Incremental Recovery (IR) | Incremental recovery was the increase in circulating FIX activity for every international unit (IU) of APVO101 administered per kilogram of body weight of participant. FIX activity was measured at the following time points post infusion: 15-30 minutes, 4-6 hours, 24-26 hours and 46-50 hours. | Analysis population included all participants for whom legally acceptable representative had signed informed consent/assent form, were in non-bleeding state, participated in single PK assessment at start of study and for whom an adequate PK profile had been obtained. "Number analyzed" signifies participants evaluable at specified time points. | Posted | | Mean | Standard Deviation | IU/dL per IU/kg | | Pre-infusion to 50 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Treatment Phase) | Subjects rated APVO101 efficacy for each bleeding episode based on a four-point scale:
- Excellent: a dramatic response with abrupt pain relief and clear reduction in joint or hemorrhage site size
- Good: pain relief or reduction in hemorrhage site size that may have required an additional infusion for resolution;
- Fair: probable or slight beneficial response usually requiring one of more additional infusions for resolution;
- Poor: no improvement or condition worsens.
| Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form, received at least one dose of APVO101 and who had experienced at least one bleeding episode. | Posted | | Count of Units | | bleeding episodes | | Exposure Day 1 up to 50 exposure days (approximately 6 months) | bleeding episodes | bleeding episodes | | ID | Title | Description |
|---|
| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Overall) | Subjects rated APVO101 efficacy for each bleeding episode based on a four-point scale:
- Excellent: a dramatic response with abrupt pain relief and clear reduction in joint or hemorrhage site size
- Good: pain relief or reduction in hemorrhage site size that may have required an additional infusion for resolution;
- Fair: probable or slight beneficial response usually requiring one of more additional infusions for resolution;
- Poor: no improvement or condition worsens.
| Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form, received at least one dose of APVO101 and who had experienced at least one bleeding episode. | Posted | | Count of Units | | bleeding episodes | | Exposure Day 1 through study completion (up to 2.5 years) | bleeding episodes | bleeding episodes | | ID | Title | Description |
|---|
| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Investigator Rating of APVO101 Prophylaxis Efficacy (Treatment Phase) | The investigator will indicate the overall assessment of APVO101 prophylaxis efficacy, considering the absence of bleeding episodes, site, severity and types of bleeding episodes treated, and other factors that might influence the therapeutic response. The investigator's efficacy assessment categories for prophylaxis will include: 'effective', 'partially effective' and 'not effective'. | Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form and had received at least one dose of APVO101. | Posted | | Count of Units | | prophylactic efficacy assessment | | Exposure Day 1 up to 50 exposure days (approximately 6 months) | prophylactic efficacy assessment | prophylactic efficacy assessment | | ID | Title | Description |
|---|
| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Investigator Rating of APVO101 Prophylaxis Efficacy (Overall) | The investigator will indicate the overall assessment of APVO101 prophylaxis efficacy, considering the absence of bleeding episodes, site, severity and types of bleeding episodes treated, and other factors that might influence the therapeutic response. The investigator's efficacy assessment categories for prophylaxis will include: 'effective', 'partially effective' and 'not effective'. | Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form and had received at least one dose of APVO101. | Posted | | Count of Units | | prophylactic efficacy assessment | | Exposure Day 1 through study completion (up to 2.5 years) | prophylactic efficacy assessment | prophylactic efficacy assessment | | ID | Title | Description |
|---|
| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Investigator Rating of APVO101 Efficacy for Control and Management of Bleeding Episodes (Treatment Phase) | Of the bleeding episodes requiring treatment, the investigator considered the site, severity and type of the bleeding episode while evaluating efficacy for control and management of the bleeding episode. The investigator's efficacy assessment categories control of bleeding episodes included: 'effective', 'partially effective' and 'not effective'. | Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form, received at least one dose of APVO101 and who had experienced at least one bleeding episode. | Posted | | Count of Units | | efficacy for control of bleeding episode | | Exposure Day 1 up to 50 exposure days (approximately 6 months) | efficacy for control of bleeding episode | efficacy for control of bleeding episode | | ID | Title | Description |
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| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Secondary | Investigator Rating of APVO101 Efficacy for Control and Management of Bleeding Episodes (Overall) | Of the bleeding episodes requiring treatment, the investigator considered the site, severity and type of the bleeding episode while evaluating efficacy for control and management of the bleeding episode. The investigator's efficacy assessment categories control of bleeding episodes included: 'effective', 'partially effective' and 'not effective'. | Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form, received at least one dose of APVO101 and who had experienced at least one bleeding episode. | Posted | | Count of Units | | efficacy for control of bleeding episode | | Exposure Day 1 through study completion (up to 2.5 years) | efficacy for control of bleeding episode | efficacy for control of bleeding episode | | ID | Title | Description |
|---|
| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Other Pre-specified | Spontaneous Annualized Bleeding Rate (Treatment Phase) | Includes annualized bleeding rate for spontaneous bleeding (i.e. bleeds that occur without obvious cause). | Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form and received at least one dose of APVO101. | Posted | | Mean | Standard Deviation | spontaneous bleeding episodes | | Exposure Day 1 up to 50 exposure days (approximately 6 months) | | | | ID | Title | Description |
|---|
| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Other Pre-specified | Spontaneous Annualized Bleeding Rate (Overall) | Includes annualized bleeding rate for spontaneous bleeding (i.e. bleeds that occur without obvious cause). | Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form and received at least one dose of APVO101. | Posted | | Mean | Standard Deviation | spontaneous bleeding episodes | | Exposure Day 1 through study completion (up to 2.5 years) | | | | ID | Title | Description |
|---|
| OG000 | APVO101 Prophylaxis - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Other Pre-specified | Occurrence of Inhibitory Factor IX Antibodies (Overall) | Incidence of APVO101 immunogenicity response (development of inhibitory, non-inhibitory factor IX binding antibodies and incidence of antibodies to Chinese hamster ovary cell proteins [CHOP]) | Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form and received at least one dose of APVO101. | Posted | | Count of Participants | | Participants | | Exposure Day 1 through study completion (up to 2.5 years) | | | | ID | Title | Description |
|---|
| OG000 | APVO101 - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Other Pre-specified | Occurrence of Non-Inhibitory Factor IX Antibodies (Overall) | Incidence of APVO101 immunogenicity response (development of inhibitory, non-inhibitory factor IX binding antibodies and incidence of antibodies to Chinese hamster ovary cell proteins [CHOP]) | Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form and received at least one dose of APVO101. | Posted | | Count of Participants | | Participants | | Exposure Day 1 through study completion (up to 2.5 years) | | | | ID | Title | Description |
|---|
| OG000 | APVO101 - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Other Pre-specified | Occurrence of Anti-CHOP Antibodies (Overall) | Incidence of APVO101 immunogenicity response (development of inhibitory, non-inhibitory factor IX binding antibodies and incidence of antibodies to Chinese hamster ovary cell proteins [CHOP]) | Analysis population included all participants for whom a legally acceptable representative had signed the informed consent/assent form and received at least one dose of APVO101. | Posted | | Count of Participants | | Participants | | Exposure Day 1 through study completion (up to 2.5 years) | | | | ID | Title | Description |
|---|
| OG000 | APVO101 - Safety Population | Following a four day washout period or three half-lives washout of a factor IX product with a prolonged half-life, all study participants entered the PK Phase.
- PK Phase - PK evaluation consisted of administration of a single 75 (±5) IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
- Treatment Phase - after completion of the PK Phase, participants (safety population) received APVO101 prophylaxis (starting prophylaxis dose was to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 to 75 IU/kg, twice weekly or at a frequency determined as appropriate by the Investigator) for 50 exposure days (approximately 6 months).
- Continuation Phase - participants could continue APVO101 prophylaxis (recommended dose range: 35 to 75 IU/kg, twice weekly) for an additional 50 or more exposure days (approximately 6 months).
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| Other Pre-specified | Thrombogenicity Assessment - D-Dimer | This study included testing of thrombogenic markers at the PK stage to evaluate for thrombotic risk. | To accommodate blood volume limitations for participants less than 17 kg, no thrombogenicity assessments were performed during the PK Phase. Participants weighing 17 kg to 19 kg, the 4 to 6 hour post-infusion timepoint was not collected. | Posted | | Mean | Standard Deviation | mg/L | | Day 1 (pre-dose), 15-30 minutes, 4-6 hours and 24-26 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | Actual Value | Prespecified collection timepoint value | | OG001 | Change From Baseline | Change from Baseline in values during the prespecified collection timepoints |
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| Other Pre-specified | Thrombogenicity Assessment - Thrombin/Antithrombin (TAT) Complex | This study included testing of thrombogenic markers at the PK stage to evaluate for thrombotic risk. | To accommodate blood volume limitations for participants less than 17 kg, no thrombogenicity assessments were performed during the PK Phase. Participants weighing 17 kg to 19 kg, the 4 to 6 hour post-infusion timepoint was not collected. | Posted | | Mean | Standard Deviation | ug/L | | Day 1 (pre-dose), 15-30 minutes, 4-6 hours and 24-26 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | Actual Value | Prespecified collection timepoint value | | OG001 | Change From Baseline | Change from Baseline in values during the prespecified collection timepoints |
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| Other Pre-specified | Thrombogenicity Assessment - Prothrombin Fragment 1+2 | This study included testing of thrombogenic markers at the PK stage to evaluate for thrombotic risk. | To accommodate blood volume limitations for participants less than 17 kg, no thrombogenicity assessments were performed during the PK Phase. Participants weighing 17 kg to 19 kg, the 4 to 6 hour post-infusion timepoint was not collected. | Posted | | Mean | Standard Deviation | pmol/L | | Day 1 (pre-dose), 15-30 minutes, 4-6 hours and 24-26 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | Actual Value | Prespecified collection timepoint value | | OG001 | Change From Baseline | Change from Baseline in values during the prespecified collection timepoints |
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