Trial Outcomes & Findings for Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age (NCT NCT00759655)
NCT ID: NCT00759655
Last Updated: 2022-06-15
Results Overview
Incidence of inhibitor development was defined as any result determined positive at a central laboratory (Bethesda inhibitor titer of \>=0.6 BU/mL) using Nijmegen modification of the Bethesda assay.
TERMINATED
PHASE3
1 participants
Baseline to 24 months or early withdrawal.
2022-06-15
Participant Flow
The study was planned to be conducted in major hemophilia centers in North America, Latin America, and Asia-Pacific regions. Seven (7) sites were initiated; however, recruitment occurred at only 1 site in United State of America (USA) between June 2009 to December 2009. The study was terminated by the Sponsor.
Participant milestones
| Measure |
Xyntha
Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
|
|---|---|
|
Overall Study
STARTED
|
1
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Xyntha
Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
|
|---|---|
|
Overall Study
Termination of the study by sponsor
|
1
|
Baseline Characteristics
Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age
Baseline characteristics by cohort
| Measure |
Xyntha
n=1 Participants
Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
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|---|---|
|
Age, Continuous
|
1 Month
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to 24 months or early withdrawal.Population: Intent-to-treat (ITT) population: Participants who had received at least 1 dose of Xyntha.
Incidence of inhibitor development was defined as any result determined positive at a central laboratory (Bethesda inhibitor titer of \>=0.6 BU/mL) using Nijmegen modification of the Bethesda assay.
Outcome measures
| Measure |
Xyntha
n=1 Participants
Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
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|---|---|
|
Percentage of Participants With Factor VIII (FVIII) Inhibitor Development
|
0 Percentage of participants
|
PRIMARY outcome
Timeframe: Baseline to 24 months or early withdrawal.Population: The study was terminated, analysis was not conducted.
LETE in the on-demand setting was based on the response to the treatment of a bleeding episode. LETE in the on-demand setting occurred if the participant recorded 2 successive "No Response" ratings (indicated there was no improvement at all between infusions or during the 24 hour interval following an infusion, or condition worsened) after 2 successive Xyntha infusions, respectively. The infusions was to be administered within 24 hours (=\<24 hours) of each other for the treatment of the same bleeding event in the absence of confounding factor.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline to 24 months or early withdrawal.Population: The study was terminated, analysis was not conducted.
The LETE in the prophylaxis setting was the occurrence of a bleed. LETE in the prophylaxis setting occurred if there was a spontaneous bleed within 48 hours (=\<48 hours) after a regularly scheduled prophylactic dose of Xyntha (which was not used to treat a bleed) in the absence of confounding factors.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline to 24 months or early withdrawal.Population: The study was terminated, analysis was not conducted.
The LETE could be considered lower than expected recovery of FVIII in the opinion of the investigator following infusion of Xyntha in the absence of confounding factors.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to 24 months or early withdrawal.Population: The study was terminated, analysis was not conducted.
An annualized bleeding rate (ABR) for each participant was calculated as the number of bleeds requiring administration of FVIII replacement product (taken from the electronic Infusion Log Diary), divided by his total therapy duration (in days), and then multiplied by 365.25.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to 24 months or early withdrawal.Population: The study was terminated, analysis was not conducted.
The data from the electronic Infusion Log Diary plus the Test Article case report form (CRF) was used to determine the number of infusions administered to treat a bleed. This was calculated by adding the initial 'for a new bleed' (on demand) infusion to any subsequent (on demand) infusions for the (same) 'previously treated bleed'. An on-demand infusion for a 'previously treated bleed' was counted toward the bleed with the most recent start time prior to that infusion.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to 24 months or early withdrawalPopulation: The study was terminated, analysis was not conducted.
A 4-point response scale to be completed is as defined as follows: (Excellent: definite pain relief/improvement in signs of bleeding starting within 8 hrs after an infusion, with no additional infusion; Good: definite pain relief/improvement in signs of bleeding starting within 8 hrs or following the infusion; Moderate: probable/slight improvement starting after 8 hours following the infusion; No Response: no improvement at all between infusions).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to 24 months or early withdrawal.Population: The study was terminated, analysis was not conducted.
The number of breakthrough (spontaneous/non-traumatic) bleeds within 48 hours following a prophylaxis dose of Xyntha was summarized. The data from the electronic Infusion Log Diary plus the Test Article CRF was used to determine the number of infusions administered to treat a new bleed, counting only those infusions administered =\<48 hours after an infusion marked as 'prophylaxis' (which had no associated bleed).
Outcome measures
Outcome data not reported
Adverse Events
Xyntha
Serious adverse events
| Measure |
Xyntha
n=1 participants at risk
Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
|
|---|---|
|
Gastrointestinal disorders
Anal fistula
|
100.0%
1/1 • Number of events 2 • AEs and SAEs were collected from the signing of the informed consent form to minimum 15 days after the last administered dose of Xyntha.
Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
|
|
Infections and infestations
Anal abscess
|
100.0%
1/1 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to minimum 15 days after the last administered dose of Xyntha.
Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
|
Other adverse events
| Measure |
Xyntha
n=1 participants at risk
Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
|
|---|---|
|
General disorders
Infusion site extravasation
|
100.0%
1/1 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to minimum 15 days after the last administered dose of Xyntha.
Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
|
|
Infections and infestations
Anal abscess
|
100.0%
1/1 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to minimum 15 days after the last administered dose of Xyntha.
Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
|
|
Infections and infestations
Influenza
|
100.0%
1/1 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to minimum 15 days after the last administered dose of Xyntha.
Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
|
|
Infections and infestations
Otitis media
|
100.0%
1/1 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to minimum 15 days after the last administered dose of Xyntha.
Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
|
|
Injury, poisoning and procedural complications
Contusion
|
100.0%
1/1 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to minimum 15 days after the last administered dose of Xyntha.
Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
100.0%
1/1 • Number of events 2 • AEs and SAEs were collected from the signing of the informed consent form to minimum 15 days after the last administered dose of Xyntha.
Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
|
|
Vascular disorders
Haematoma
|
100.0%
1/1 • Number of events 1 • AEs and SAEs were collected from the signing of the informed consent form to minimum 15 days after the last administered dose of Xyntha.
Safety population = subjects who received at least 1 dose of Xyntha. The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
- Publication restrictions are in place
Restriction type: OTHER