Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age

• Sponsor: Wyeth is now a wholly owned subsidiary of Pfizer (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT00759655
• Collaborator: (none)
• Enrollment: 1
• Arm: 1
• Duration (Months): 6

Study Details

Study Description

Brief Summary

This study will be investigating the safety and efficacy of Xyntha (moroctocog alfa (AF-CC)) in male patients less than 6 years old. Annualized bleeding rates and physician / caregiver assessments of responses to treatment will be characterized. FVIII inhibitor levels will be assessed throughout the study.

Condition or Disease	Intervention/Treatment	Phase
Hemophilia A	Biological: Moroctocog alfa	Phase 3

Detailed Description

The study was terminated on 22 Sept 2009 due to competition with another Wyeth study for a similar patient population. The decision to terminate the trial was not based on any safety issues.

Study Design

• Study Type: Interventional
• Actual Enrollment: 1 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: An Open-Label Study To Evaluate The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age In Usual Care Settings
• Study Start Date: Jun 1, 2009
• Actual Primary Completion Date: Dec 1, 2009
• Actual Study Completion Date: Dec 1, 2009

Arms and Interventions

Arm	Intervention/Treatment
Other: open label	Biological: Moroctocog alfa; Patients will receive Moroctocog alfa according to their investigator's prescription.

Outcome Measures

Primary Outcome Measures

1. Percentage of Participants With Factor VIII (FVIII) Inhibitor Development [Baseline to 24 months or early withdrawal.]

   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.

2. Percentage of Participants With Less Than Expected Therapeutic Effects (LETE) in the On-Demand Setting [Baseline to 24 months or early withdrawal.]

   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.

3. Percentage of Participants With LETE in the Prophylaxis Setting [Baseline to 24 months or early withdrawal.]

   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.

4. Percentage of Participants With Low Recovery LETE [Baseline to 24 months or early withdrawal.]

   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.

Secondary Outcome Measures

1. Mean Annualized Bleed Rate (ABR) [Baseline to 24 months or early withdrawal.]

   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.

2. Number of Xyntha Infusions Needed to Treat Each New Bleed [Baseline to 24 months or early withdrawal.]

   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.

3. Response to First On-demand Xyntha Treatment for All New Bleeds as Assessed by the Caregiver [Baseline to 24 months or early withdrawal]

   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).

4. Mean Number of Breakthrough (Spontaneous/Non-traumatic) Bleeds [Baseline to 24 months or early withdrawal.]

   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).

Eligibility Criteria

Criteria

• Ages Eligible for Study: 0 Years to 5 Years
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Male patients less than 6 years of age with moderately severe to severe hemophilia A (FVIII less than or equal to 2%).

• Treatment history of less than 50 exposure days to prior recombinant or plasma-derived FVIII replacement products.

• Not receiving treatment for HIV or hepatitis infection, or the patient is on a stable antiviral regimen at the time of enrollment in the study.

Exclusion Criteria:

• Presence of any bleeding disorder in addition to hemophilia A.

• Inhibitor titer of greater than or equal to 5 Bethesda Units (BU) at screening.

• Treated with immunomodulatory therapy during the screening period

• Treatment history of more than 5 exposure days (ED) to Xyntha.

• Known hypersensitivity to hamster protein.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

• Wyeth is now a wholly owned subsidiary of Pfizer

Investigators

• Study Director: Pfizer CT.gov Call Center, Pfizer

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• To obtain contact information for a study center near you, click here.

Publications

None provided.

• Responsible Party: Wyeth is now a wholly owned subsidiary of Pfizer
• ClinicalTrials.gov Identifier: NCT00759655
• Other Study ID Numbers: 3082B2-3315; B1831002; 3082B2-3315-WW
• First Posted: Sep 25, 2008
• Last Update Posted: Jun 15, 2022
• Last Verified: May 1, 2022

• Keywords provided by Wyeth is now a wholly owned subsidiary of Pfizer: hemophilia A; Xyntha; ReFacto; moroctocog alfa; bleeding disorder
• Additional relevant MeSH terms: Hemophilia A; Factor VIII

Study Results

Participant Flow

Recruitment Details	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.

Arm/Group Title	Xyntha
Arm/Group Description	Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
Period Title: Overall Study
STARTED	1
COMPLETED	0
NOT COMPLETED	1

Baseline Characteristics

Arm/Group Title	Xyntha
Arm/Group Description	Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
Overall Participants	1
Age (Month) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Month]	1
Sex: Female, Male (Count of Participants)
Female	0 0%
Male	1 100%

Outcome Measures

1. Primary Outcome

Title	Percentage of Participants With Factor VIII (FVIII) Inhibitor Development
Description	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.
Time Frame	Baseline to 24 months or early withdrawal.

Outcome Measure Data

Analysis Population Description

Intent-to-treat (ITT) population: Participants who had received at least 1 dose of Xyntha.

Arm/Group Title	Xyntha
Arm/Group Description	Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
Measure Participants	1
Number [Percentage of participants]	0 0%

2. Primary Outcome

Title	Percentage of Participants With Less Than Expected Therapeutic Effects (LETE) in the On-Demand Setting
Description	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.
Time Frame	Baseline to 24 months or early withdrawal.

Outcome Measure Data

Analysis Population Description

The study was terminated, analysis was not conducted.

Arm/Group Title	Xyntha
Arm/Group Description	Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
Measure Participants	0

3. Primary Outcome

Title	Percentage of Participants With LETE in the Prophylaxis Setting
Description	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.
Time Frame	Baseline to 24 months or early withdrawal.

Outcome Measure Data

Analysis Population Description

The study was terminated, analysis was not conducted.

Arm/Group Title	Xyntha
Arm/Group Description	Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
Measure Participants	0

4. Primary Outcome

Title	Percentage of Participants With Low Recovery LETE
Description	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.
Time Frame	Baseline to 24 months or early withdrawal.

Outcome Measure Data

Analysis Population Description

The study was terminated, analysis was not conducted.

Arm/Group Title	Xyntha
Arm/Group Description	Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
Measure Participants	0

5. Secondary Outcome

Title	Mean Annualized Bleed Rate (ABR)
Description	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.
Time Frame	Baseline to 24 months or early withdrawal.

Outcome Measure Data

Analysis Population Description

The study was terminated, analysis was not conducted.

Arm/Group Title	Xyntha
Arm/Group Description	Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
Measure Participants	0
Measure Bleeds	0

6. Secondary Outcome

Title	Number of Xyntha Infusions Needed to Treat Each New Bleed
Description	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.
Time Frame	Baseline to 24 months or early withdrawal.

Outcome Measure Data

Analysis Population Description

The study was terminated, analysis was not conducted.

Arm/Group Title	Xyntha
Arm/Group Description	Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
Measure Participants	0
Measure Number of Infusions	0

7. Secondary Outcome

Title	Response to First On-demand Xyntha Treatment for All New Bleeds as Assessed by the Caregiver
Description	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).
Time Frame	Baseline to 24 months or early withdrawal

Outcome Measure Data

Analysis Population Description

The study was terminated, analysis was not conducted.

Arm/Group Title	Xyntha
Arm/Group Description	Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
Measure Participants	0

8. Secondary Outcome

Title	Mean Number of Breakthrough (Spontaneous/Non-traumatic) Bleeds
Description	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).
Time Frame	Baseline to 24 months or early withdrawal.

Outcome Measure Data

Analysis Population Description

The study was terminated, analysis was not conducted.

Arm/Group Title	Xyntha
Arm/Group Description	Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
Measure Participants	0
Measure Bleeds	0

Adverse Events

Time Frame	AEs and SAEs were collected from the signing of the informed consent form to minimum 15 days after the last administered dose of Xyntha.
Adverse Event Reporting Description	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.
Arm/Group Title	Xyntha
Arm/Group Description	Participants received intravenous infusion (s) of Xyntha as per dosage and administration frequency as prescribed by the treating physician.
All Cause Mortality
	Xyntha
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Xyntha
	Affected / at Risk (%)	# Events
Total	1/1 (100%)
Gastrointestinal disorders
Anal fistula	1/1 (100%)	2
Infections and infestations
Anal abscess	1/1 (100%)	1
Other (Not Including Serious) Adverse Events
	Xyntha
	Affected / at Risk (%)	# Events
Total	1/1 (100%)
General disorders
Infusion site extravasation	1/1 (100%)	1
Infections and infestations
Anal abscess	1/1 (100%)	1
Influenza	1/1 (100%)	1
Otitis media	1/1 (100%)	1
Injury, poisoning and procedural complications
Contusion	1/1 (100%)	1
Procedural pain	1/1 (100%)	2
Vascular disorders
Haematoma	1/1 (100%)	1

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

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.