Trial Outcomes & Findings for Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B (NCT NCT03855280)
NCT ID: NCT03855280
Last Updated: 2024-05-16
Results Overview
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.
COMPLETED
PHASE3
21 participants
Exposure Day 1 up to 50 exposure days (approximately 6 months)
2024-05-16
Participant Flow
Pediatric patients (less than 11.5 years of age at first dose) with severe to moderately severe hemophilia B. Participants had a minimum of 50 exposure days of factor IX (FIX) replacement therapy prior to enrollment.
Participant milestones
| Measure |
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).
|
|---|---|
|
PK Phase
STARTED
|
21
|
|
PK Phase
PK Phase
|
21
|
|
PK Phase
COMPLETED
|
21
|
|
PK Phase
NOT COMPLETED
|
0
|
|
Treatment Phase
STARTED
|
21
|
|
Treatment Phase
COMPLETED
|
19
|
|
Treatment Phase
NOT COMPLETED
|
2
|
|
Continuation Phase
STARTED
|
19
|
|
Continuation Phase
COMPLETED
|
16
|
|
Continuation Phase
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
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).
|
|---|---|
|
Treatment Phase
Adverse Event
|
1
|
|
Treatment Phase
Withdrawal by Subject
|
1
|
|
Continuation Phase
Protocol Violation
|
1
|
|
Continuation Phase
Adverse Event
|
1
|
|
Continuation Phase
Sponsor Concluded Study
|
1
|
Baseline Characteristics
There was no quantifiable retrospective data to report for this table.
Baseline characteristics by cohort
| Measure |
APVO101 Prophylaxis - Safety Population
n=21 Participants
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).
|
|---|---|
|
Age, Customized
Age Group: < 6
|
10 Participants
n=21 Participants
|
|
Age, Customized
Age Group: 6 to < 12
|
11 Participants
n=21 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
21 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
18 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=21 Participants
|
|
Region of Enrollment
Turkey
|
4 Participants
n=21 Participants
|
|
Region of Enrollment
Ukraine
|
8 Participants
n=21 Participants
|
|
Region of Enrollment
Brazil
|
3 Participants
n=21 Participants
|
|
Region of Enrollment
South Africa
|
3 Participants
n=21 Participants
|
|
Region of Enrollment
Moldova
|
1 Participants
n=21 Participants
|
|
Region of Enrollment
Georgia
|
2 Participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Exposure Day 1 up to 50 exposure days (approximately 6 months)Population: 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.
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.
Outcome measures
| Measure |
APVO101 - Safety Population
n=21 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Annualized Bleeding Rate (ABR)
|
2.93 bleeding episodes per year
Standard Deviation 4.59
|
—
|
PRIMARY outcome
Timeframe: Exposure Day 1 through study completion (up to 2.5 years)Population: 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.
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.
Outcome measures
| Measure |
APVO101 - Safety Population
n=21 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Annualized Bleeding Rate (ABR) Overall
|
2.34 bleeding episodes per year
Standard Deviation 4.23
|
—
|
SECONDARY outcome
Timeframe: Pre-infusion to 50 hours post-infusionPopulation: 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.
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.
Outcome measures
| Measure |
APVO101 - Safety Population
n=20 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Concentration (Cmax)
|
60.1 IU/dL
Standard Deviation 12.2
|
—
|
SECONDARY outcome
Timeframe: Pre-infusion to 50 hours post-infusionPopulation: 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.
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.
Outcome measures
| Measure |
APVO101 - Safety Population
n=21 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Area Under the Curve (0-inf)
|
1170 IU/dL/hr
Standard Deviation 231
|
—
|
SECONDARY outcome
Timeframe: Pre-infusion to 50 hours post-infusionPopulation: 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.
MRT is the average time the molecules of drug reside in the body before elimination.
Outcome measures
| Measure |
APVO101 - Safety Population
n=11 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Mean Residence Time (MRT)
|
20 hours
Standard Deviation 2.53
|
—
|
SECONDARY outcome
Timeframe: Pre-infusion to 50 hours post-infusionPopulation: 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.
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.
Outcome measures
| Measure |
APVO101 - Safety Population
n=12 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Terminal Half-Life (t 1/2)
|
16.3 hours
Standard Deviation 2.2
|
—
|
SECONDARY outcome
Timeframe: Pre-infusion to 50 hours post-infusionPopulation: 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.
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.
Outcome measures
| Measure |
APVO101 - Safety Population
n=11 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Clearance (CL)
|
6.8 mL/(kg*hr)
Standard Deviation 1.5
|
—
|
SECONDARY outcome
Timeframe: Pre-infusion to 50 hours post-infusionPopulation: 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.
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.
Outcome measures
| Measure |
APVO101 - Safety Population
n=11 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Volume of Distribution at Steady-State (Vdss)
|
134 mL/kg
Standard Deviation 30.6
|
—
|
SECONDARY outcome
Timeframe: Pre-infusion to 50 hours post-infusionPopulation: 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.
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.
Outcome measures
| Measure |
APVO101 - Safety Population
n=20 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Incremental Recovery (IR)
|
0.790 IU/dL per IU/kg
Standard Deviation 0.156
|
—
|
SECONDARY outcome
Timeframe: Exposure Day 1 up to 50 exposure days (approximately 6 months)Population: 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.
Subjects rated APVO101 efficacy for each bleeding episode based on a four-point scale: 1. Excellent: a dramatic response with abrupt pain relief and clear reduction in joint or hemorrhage site size 2. Good: pain relief or reduction in hemorrhage site size that may have required an additional infusion for resolution; 3. Fair: probable or slight beneficial response usually requiring one of more additional infusions for resolution; 4. Poor: no improvement or condition worsens.
Outcome measures
| Measure |
APVO101 - Safety Population
n=24 bleeding episodes
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Treatment Phase)
Excellent
|
10 bleeding episodes
|
—
|
|
Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Treatment Phase)
Good
|
5 bleeding episodes
|
—
|
|
Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Treatment Phase)
Fair
|
0.0 bleeding episodes
|
—
|
|
Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Treatment Phase)
No infusions required to treat bleeding episode
|
7 bleeding episodes
|
—
|
|
Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Treatment Phase)
Missing
|
2 bleeding episodes
|
—
|
|
Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Treatment Phase)
Poor
|
0.0 bleeding episodes
|
—
|
SECONDARY outcome
Timeframe: Exposure Day 1 through study completion (up to 2.5 years)Population: 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.
Subjects rated APVO101 efficacy for each bleeding episode based on a four-point scale: 1. Excellent: a dramatic response with abrupt pain relief and clear reduction in joint or hemorrhage site size 2. Good: pain relief or reduction in hemorrhage site size that may have required an additional infusion for resolution; 3. Fair: probable or slight beneficial response usually requiring one of more additional infusions for resolution; 4. Poor: no improvement or condition worsens.
Outcome measures
| Measure |
APVO101 - Safety Population
n=52 bleeding episodes
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Overall)
Excellent
|
28 bleeding episodes
|
—
|
|
Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Overall)
Good
|
13 bleeding episodes
|
—
|
|
Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Overall)
Fair
|
0.0 bleeding episodes
|
—
|
|
Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Overall)
Poor
|
0.0 bleeding episodes
|
—
|
|
Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Overall)
No infusions required to treat bleeding episode
|
9 bleeding episodes
|
—
|
|
Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Overall)
Missing
|
2 bleeding episodes
|
—
|
SECONDARY outcome
Timeframe: Exposure Day 1 up to 50 exposure days (approximately 6 months)Population: 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.
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'.
Outcome measures
| Measure |
APVO101 - Safety Population
n=81 prophylactic efficacy assessment
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Investigator Rating of APVO101 Prophylaxis Efficacy (Treatment Phase)
Effective
|
80 prophylactic efficacy assessment
|
—
|
|
Investigator Rating of APVO101 Prophylaxis Efficacy (Treatment Phase)
Partially Effective
|
1 prophylactic efficacy assessment
|
—
|
|
Investigator Rating of APVO101 Prophylaxis Efficacy (Treatment Phase)
Not Effective
|
0 prophylactic efficacy assessment
|
—
|
SECONDARY outcome
Timeframe: Exposure Day 1 through study completion (up to 2.5 years)Population: 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.
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'.
Outcome measures
| Measure |
APVO101 - Safety Population
n=170 prophylactic efficacy assessment
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Investigator Rating of APVO101 Prophylaxis Efficacy (Overall)
Not Effective
|
0 prophylactic efficacy assessment
|
—
|
|
Investigator Rating of APVO101 Prophylaxis Efficacy (Overall)
Effective
|
167 prophylactic efficacy assessment
|
—
|
|
Investigator Rating of APVO101 Prophylaxis Efficacy (Overall)
Partially Effective
|
3 prophylactic efficacy assessment
|
—
|
SECONDARY outcome
Timeframe: Exposure Day 1 up to 50 exposure days (approximately 6 months)Population: 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.
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'.
Outcome measures
| Measure |
APVO101 - Safety Population
n=16 efficacy for control of bleeding episode
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Investigator Rating of APVO101 Efficacy for Control and Management of Bleeding Episodes (Treatment Phase)
Not effective
|
0 efficacy for control of bleeding episode
|
—
|
|
Investigator Rating of APVO101 Efficacy for Control and Management of Bleeding Episodes (Treatment Phase)
Effective
|
16 efficacy for control of bleeding episode
|
—
|
|
Investigator Rating of APVO101 Efficacy for Control and Management of Bleeding Episodes (Treatment Phase)
Partially effective
|
0 efficacy for control of bleeding episode
|
—
|
SECONDARY outcome
Timeframe: Exposure Day 1 through study completion (up to 2.5 years)Population: 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.
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'.
Outcome measures
| Measure |
APVO101 - Safety Population
n=35 efficacy for control of bleeding episode
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Investigator Rating of APVO101 Efficacy for Control and Management of Bleeding Episodes (Overall)
Effective
|
35 efficacy for control of bleeding episode
|
—
|
|
Investigator Rating of APVO101 Efficacy for Control and Management of Bleeding Episodes (Overall)
Not effective
|
0 efficacy for control of bleeding episode
|
—
|
|
Investigator Rating of APVO101 Efficacy for Control and Management of Bleeding Episodes (Overall)
Partially effective
|
0 efficacy for control of bleeding episode
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Exposure Day 1 up to 50 exposure days (approximately 6 months)Population: 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.
Includes annualized bleeding rate for spontaneous bleeding (i.e. bleeds that occur without obvious cause).
Outcome measures
| Measure |
APVO101 - Safety Population
n=21 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Spontaneous Annualized Bleeding Rate (Treatment Phase)
|
0.81 spontaneous bleeding episodes
Standard Deviation 1.93
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Exposure Day 1 through study completion (up to 2.5 years)Population: 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.
Includes annualized bleeding rate for spontaneous bleeding (i.e. bleeds that occur without obvious cause).
Outcome measures
| Measure |
APVO101 - Safety Population
n=21 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Spontaneous Annualized Bleeding Rate (Overall)
|
0.63 spontaneous bleeding episodes
Standard Deviation 1.26
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Exposure Day 1 through study completion (up to 2.5 years)Population: 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.
Incidence of APVO101 immunogenicity response (development of inhibitory, non-inhibitory factor IX binding antibodies and incidence of antibodies to Chinese hamster ovary cell proteins \[CHOP\])
Outcome measures
| Measure |
APVO101 - Safety Population
n=21 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Occurrence of Inhibitory Factor IX Antibodies (Overall)
|
0 Participants
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Exposure Day 1 through study completion (up to 2.5 years)Population: 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.
Incidence of APVO101 immunogenicity response (development of inhibitory, non-inhibitory factor IX binding antibodies and incidence of antibodies to Chinese hamster ovary cell proteins \[CHOP\])
Outcome measures
| Measure |
APVO101 - Safety Population
n=21 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Occurrence of Non-Inhibitory Factor IX Antibodies (Overall)
|
3 Participants
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Exposure Day 1 through study completion (up to 2.5 years)Population: 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.
Incidence of APVO101 immunogenicity response (development of inhibitory, non-inhibitory factor IX binding antibodies and incidence of antibodies to Chinese hamster ovary cell proteins \[CHOP\])
Outcome measures
| Measure |
APVO101 - Safety Population
n=21 Participants
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).
|
Change From Baseline
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Occurrence of Anti-CHOP Antibodies (Overall)
|
3 Participants
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 1 (pre-dose), 15-30 minutes, 4-6 hours and 24-26 hours post-infusionPopulation: 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.
This study included testing of thrombogenic markers at the PK stage to evaluate for thrombotic risk.
Outcome measures
| Measure |
APVO101 - Safety Population
n=13 Participants
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).
|
Change From Baseline
n=13 Participants
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Thrombogenicity Assessment - D-Dimer
4 to 6 hours post-infusion
|
0.452 mg/L
Standard Deviation 0.2955
|
0.045 mg/L
Standard Deviation 0.2051
|
|
Thrombogenicity Assessment - D-Dimer
24 to 26 hours post-infusion
|
0.578 mg/L
Standard Deviation 0.2766
|
0.114 mg/L
Standard Deviation 0.2586
|
|
Thrombogenicity Assessment - D-Dimer
Day 1, pre-infusion
|
0.432 mg/L
Standard Deviation 0.2320
|
—
|
|
Thrombogenicity Assessment - D-Dimer
15 to 30 minutes post-infusion
|
0.422 mg/L
Standard Deviation 0.1999
|
0.006 mg/L
Standard Deviation 0.1071
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 1 (pre-dose), 15-30 minutes, 4-6 hours and 24-26 hours post-infusionPopulation: 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.
This study included testing of thrombogenic markers at the PK stage to evaluate for thrombotic risk.
Outcome measures
| Measure |
APVO101 - Safety Population
n=13 Participants
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).
|
Change From Baseline
n=13 Participants
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Thrombogenicity Assessment - Thrombin/Antithrombin (TAT) Complex
4 to 6 hours post-infusion
|
12.17 ug/L
Standard Deviation 15.986
|
6.63 ug/L
Standard Deviation 17.399
|
|
Thrombogenicity Assessment - Thrombin/Antithrombin (TAT) Complex
24 to 26 hours post-infusion
|
12.45 ug/L
Standard Deviation 16.459
|
6.78 ug/L
Standard Deviation 17.081
|
|
Thrombogenicity Assessment - Thrombin/Antithrombin (TAT) Complex
15 to 30 minutes post-infusion
|
17.92 ug/L
Standard Deviation 22.971
|
12.71 ug/L
Standard Deviation 24.252
|
|
Thrombogenicity Assessment - Thrombin/Antithrombin (TAT) Complex
Day 1, pre-infusion
|
5.92 ug/L
Standard Deviation 2.579
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 1 (pre-dose), 15-30 minutes, 4-6 hours and 24-26 hours post-infusionPopulation: 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.
This study included testing of thrombogenic markers at the PK stage to evaluate for thrombotic risk.
Outcome measures
| Measure |
APVO101 - Safety Population
n=13 Participants
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).
|
Change From Baseline
n=13 Participants
Change from Baseline in values during the prespecified collection timepoints
|
|---|---|---|
|
Thrombogenicity Assessment - Prothrombin Fragment 1+2
Day 1, pre-infusion
|
99.9 pmol/L
Standard Deviation 41.59
|
—
|
|
Thrombogenicity Assessment - Prothrombin Fragment 1+2
15 to 30 minutes post-infusion
|
318.0 pmol/L
Standard Deviation 433.64
|
218.1 pmol/L
Standard Deviation 439.48
|
|
Thrombogenicity Assessment - Prothrombin Fragment 1+2
4 to 6 hours post-infusion
|
238.6 pmol/L
Standard Deviation 330.63
|
139.5 pmol/L
Standard Deviation 303.66
|
|
Thrombogenicity Assessment - Prothrombin Fragment 1+2
24 to 26 hours post-infusion
|
223.0 pmol/L
Standard Deviation 246.46
|
123.1 pmol/L
Standard Deviation 251.48
|
Adverse Events
APVO101 Prophylaxis - Safety Population
Serious adverse events
| Measure |
APVO101 Prophylaxis - Safety Population
n=21 participants at risk
All subjects in the Safety Population received study treatment.
|
|---|---|
|
Injury, poisoning and procedural complications
Spinal Compression Fracture
|
4.8%
1/21 • Number of events 1 • Adverse events were monitored after the first dose of APVO101 through study completion (up to 2.5 years) with a minimum follow-up period of 30 days.
Collection method: All adverse events were reported by investigators assessments and subjects and/or their parent or legal guardian. Any clinically significant changes (as determined by the investigator) in laboratory test results, vital signs or physical exam were captured as adverse events. Additionally, subjects and/or their parent or legal guardian recorded AEs in the subject diary and the information was reviewed by the investigator during each scheduled visit.
|
|
Renal and urinary disorders
Hematuria
|
4.8%
1/21 • Number of events 1 • Adverse events were monitored after the first dose of APVO101 through study completion (up to 2.5 years) with a minimum follow-up period of 30 days.
Collection method: All adverse events were reported by investigators assessments and subjects and/or their parent or legal guardian. Any clinically significant changes (as determined by the investigator) in laboratory test results, vital signs or physical exam were captured as adverse events. Additionally, subjects and/or their parent or legal guardian recorded AEs in the subject diary and the information was reviewed by the investigator during each scheduled visit.
|
Other adverse events
| Measure |
APVO101 Prophylaxis - Safety Population
n=21 participants at risk
All subjects in the Safety Population received study treatment.
|
|---|---|
|
Infections and infestations
Bronchitis
|
57.1%
12/21 • Number of events 31 • Adverse events were monitored after the first dose of APVO101 through study completion (up to 2.5 years) with a minimum follow-up period of 30 days.
Collection method: All adverse events were reported by investigators assessments and subjects and/or their parent or legal guardian. Any clinically significant changes (as determined by the investigator) in laboratory test results, vital signs or physical exam were captured as adverse events. Additionally, subjects and/or their parent or legal guardian recorded AEs in the subject diary and the information was reviewed by the investigator during each scheduled visit.
|
|
Infections and infestations
Influenza
|
9.5%
2/21 • Number of events 2 • Adverse events were monitored after the first dose of APVO101 through study completion (up to 2.5 years) with a minimum follow-up period of 30 days.
Collection method: All adverse events were reported by investigators assessments and subjects and/or their parent or legal guardian. Any clinically significant changes (as determined by the investigator) in laboratory test results, vital signs or physical exam were captured as adverse events. Additionally, subjects and/or their parent or legal guardian recorded AEs in the subject diary and the information was reviewed by the investigator during each scheduled visit.
|
|
Infections and infestations
Nasopharyngitis
|
23.8%
5/21 • Number of events 7 • Adverse events were monitored after the first dose of APVO101 through study completion (up to 2.5 years) with a minimum follow-up period of 30 days.
Collection method: All adverse events were reported by investigators assessments and subjects and/or their parent or legal guardian. Any clinically significant changes (as determined by the investigator) in laboratory test results, vital signs or physical exam were captured as adverse events. Additionally, subjects and/or their parent or legal guardian recorded AEs in the subject diary and the information was reviewed by the investigator during each scheduled visit.
|
|
Infections and infestations
Respiratory tract infection
|
9.5%
2/21 • Number of events 4 • Adverse events were monitored after the first dose of APVO101 through study completion (up to 2.5 years) with a minimum follow-up period of 30 days.
Collection method: All adverse events were reported by investigators assessments and subjects and/or their parent or legal guardian. Any clinically significant changes (as determined by the investigator) in laboratory test results, vital signs or physical exam were captured as adverse events. Additionally, subjects and/or their parent or legal guardian recorded AEs in the subject diary and the information was reviewed by the investigator during each scheduled visit.
|
|
Infections and infestations
Respiratory tract infection viral
|
9.5%
2/21 • Number of events 2 • Adverse events were monitored after the first dose of APVO101 through study completion (up to 2.5 years) with a minimum follow-up period of 30 days.
Collection method: All adverse events were reported by investigators assessments and subjects and/or their parent or legal guardian. Any clinically significant changes (as determined by the investigator) in laboratory test results, vital signs or physical exam were captured as adverse events. Additionally, subjects and/or their parent or legal guardian recorded AEs in the subject diary and the information was reviewed by the investigator during each scheduled visit.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal Pain
|
9.5%
2/21 • Number of events 2 • Adverse events were monitored after the first dose of APVO101 through study completion (up to 2.5 years) with a minimum follow-up period of 30 days.
Collection method: All adverse events were reported by investigators assessments and subjects and/or their parent or legal guardian. Any clinically significant changes (as determined by the investigator) in laboratory test results, vital signs or physical exam were captured as adverse events. Additionally, subjects and/or their parent or legal guardian recorded AEs in the subject diary and the information was reviewed by the investigator during each scheduled visit.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
9.5%
2/21 • Number of events 3 • Adverse events were monitored after the first dose of APVO101 through study completion (up to 2.5 years) with a minimum follow-up period of 30 days.
Collection method: All adverse events were reported by investigators assessments and subjects and/or their parent or legal guardian. Any clinically significant changes (as determined by the investigator) in laboratory test results, vital signs or physical exam were captured as adverse events. Additionally, subjects and/or their parent or legal guardian recorded AEs in the subject diary and the information was reviewed by the investigator during each scheduled visit.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place