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Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Acquired Hemophilia A

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ClinicalTrials.gov Identifier: NCT01178294
Recruitment Status : Completed
First Posted : August 10, 2010
Results First Posted : December 21, 2015
Last Update Posted : June 17, 2019
Sponsor:
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Acquired Hemophilia A
Intervention Biological: OBI-1
Enrollment 29
Recruitment Details Enrollment was conducted at 12 clinical sites in 4 countries (USA, Canada, UK, India). Eight sites enrolled subjects under Protocol OBI-1-301 only. Two US sites enrolled subjects under the expanded access protocol OBI-1-301a and subsequently under protocol OBI-1-301. Another 2 US sites enrolled subjects under OBI-1-301a only.
Pre-assignment Details 29 subjects were enrolled and all were treated with OBI-1. Data from the expanded access subjects (Protocol 301a, n= 4) are included with the data from subjects enrolled under Protocol OBI-1-301 (n=25). 10 subjects discontinued prematurely and 18 completed the study. For one subject, the completion status could not be verified.
Arm/Group Title OBI-1
Hide Arm/Group Description Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Period Title: Overall Study
Started 29 [1]
Completed 18
Not Completed 11
Reason Not Completed
Adverse Event             4
Lost to Follow-up             2
Lack of Efficacy             1
Death             1
Development of inhibitors to OBI-1             1
Subject status is terminal             1
Completion status could not be verified             1
[1]
25 subjects enrolled under OBI-1-301 + 4 subjects enrolled under expanded access OBI-1-301a.
Arm/Group Title OBI-1
Hide Arm/Group Description Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Baseline Participants 29
Hide Baseline Analysis Population Description
The baseline analysis population comprises all 29 treated subjects (=safety analysis set).
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 29 participants
69.8  (13.28)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
Female
10
  34.5%
Male
19
  65.5%
Region of Enrollment   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 29 participants
United States 16
Canada 5
United Kingdom 4
India 4
[1]
Measure Description: Of 16 subjects enrolled in the US, 12 were enrolled under Protocol 301 and 4 were enrolled under the expanded access Protocol 301a.
1.Primary Outcome
Title Percentage of Serious Bleeding Episodes Responsive to OBI-1
Hide Description The initial serious ("qualifying") bleeding episode for each subject was analyzed for the primary efficacy outcome measure. A 'positive response' is defined as 'effective' (bleeding stopped with clinical control and FVIII levels of 50% or higher ) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' is defined as 'bleeding slightly reduced or unchanged and FVIII levels of less than 50%'. 'Not effective' is defined as 'bleeding worsening and FVIII levels of less than 50%'.
Time Frame 24 hours after initiation of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat (ITT) population = 29 subjects with initial serious bleeding episodes
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 29
Overall Number of Units Analyzed
Type of Units Analyzed: Initial Serious Bleeding Episodes
29
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of serious bleeding episodes
100
(88.1 to 100)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection OBI-1
Comments Summary statistics for the percentage of participants with serious bleeding episodes responsive at 24 hours after the initiation of treatment are presented, along with the 95% confidence interval (two-sided 95% Clopper-Pearson confidence interval).
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method one-sided binomial exact test
Comments [Not Specified]
2.Secondary Outcome
Title Overall Percentage of Serious Bleeding Episodes Successfully Controlled With OBI-1 Therapy, as Assessed by the Investigator
Hide Description Treatment success was defined as control of qualifying bleeding episode at the time of final treatment dosing. A serious bleeding episode was considered 'successfully controlled' if the investigator had checked 'completed OBI-1 therapy as treatment success' on the eCRF.
Time Frame At the time of final treatment dosing (varied from participant to participant depending on bleeding episodes)
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population = 29 subjects with initial serious bleeding episodes (BEs)
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 29
Overall Number of Units Analyzed
Type of Units Analyzed: Initial Serious Bleeding Episodes
29
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of serious bleeding episodes
86.2
(68.3 to 96.1)
3.Secondary Outcome
Title Percentage of Serious Bleeding Episodes Responsive to OBI-1 Therapy at Designated Assessment Time Points After the Initiation of Therapy, as Assessed by the Investigator
Hide Description A 'positive response' is defined as 'effective' (bleeding stopped with clinical control and FVIII levels of 50% or higher ) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' is defined as 'bleeding slightly reduced or unchanged and FVIII levels of less than 50%'. 'Not effective' is defined as 'bleeding worsening and FVIII levels of less than 50%'.
Time Frame 8 hours
Hide Outcome Measure Data
Hide Analysis Population Description
21 subjects of the ITT population (n=29) had responses available at 8 hours after initial infusion of OBI-1.
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 21
Overall Number of Units Analyzed
Type of Units Analyzed: Responses Available at 8 hrs
21
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of serious bleeding episodes
95.2
(76.2 to 99.9)
4.Secondary Outcome
Title Percentage of Serious Bleeding Episodes Responsive to OBI-1 Therapy at Designated Assessment Time Points After the Initiation of Therapy, as Assessed by the Investigator
Hide Description A 'positive response' is defined as 'effective' (bleeding stopped with clinical control and FVIII levels of 50% or higher ) or 'partially effective' (bleeding reduced with clinical stabilization and FVIII levels of 20% or higher) control of bleeding, as determined by the investigator using a 4-point rating scale (effective - partially effective - poorly effective - not effective). 'Poorly effective' is defined as 'bleeding slightly reduced or unchanged and FVIII levels of less than 50%'. 'Not effective' is defined as 'bleeding worsening and FVIII levels of less than 50%'.
Time Frame 16 hours
Hide Outcome Measure Data
Hide Analysis Population Description
19 subjects of the ITT population (n=29) had responses available at 16 hours after initial infusion of OBI-1.
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 19
Overall Number of Units Analyzed
Type of Units Analyzed: Responses Available at 16 hrs
19
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of serious bleeding episodes
100
(82.4 to 100)
5.Secondary Outcome
Title Frequency of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes
Hide Description 'Frequency of infusions' was calculated as the 'average number of infusions per day'. 'Qualifying bleeding episode' was defined as the 'initial, serious bleeding episode'.
Time Frame Time of successful control of qualifying bleeding episode (varied from participant to participant)
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis was performed in 25 of 29 participants in the ITT population whose 'qualifying' bleeding episode was controlled successfully.
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 25
Mean (Standard Deviation)
Unit of Measure: average number of infusions per day
2.10  (1.109)
6.Secondary Outcome
Title Total Dose of OBI-1 Required to Successfully Control 'Qualifying' Bleeding Episodes
Hide Description 'Qualifying bleeding episode' was defined as the 'initial, serious bleeding episode'.
Time Frame Time of successful control of qualifying bleeding episode (varied from participant to participant)
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis was performed in 25 of 29 participants in the ITT population whose 'qualifying' bleeding episode was controlled successfully.
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 25
Mean (Standard Deviation)
Unit of Measure: dose in U/kg
2683.2  (2928.61)
7.Secondary Outcome
Title Total Number of Infusions of OBI-1 Required to Successfully Control 'Qualifying' Bleeding Episodes
Hide Description 'Qualifying bleeding episode' was defined as the 'initial, serious bleeding episode'. A serious bleeding episode was considered 'successfully controlled' if the investigator had checked 'completed OBI-1 therapy as treatment success' on the eCRF.
Time Frame Time of successful control of qualifying bleeding episode (varied from participant to participant)
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis was performed in 25 of 29 participants in the ITT population whose 'qualifying' bleeding episode was controlled successfully.
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 25
Mean (Standard Deviation)
Unit of Measure: infusions per participant
15.4  (12.64)
8.Secondary Outcome
Title Correlation Between Positive Response to OBI-1 Therapy at 8 Hours and Eventual Control of Serious Bleeding Episodes at 24 Hours
Hide Description [Not Specified]
Time Frame 24 hours
Hide Outcome Measure Data
Hide Analysis Population Description
Of 21 subjects in the ITT population (n=29) with responses available at 8 hours after initial infusion of OBI-1, 20 had a positive response.
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 21
Measure Type: Number
Unit of Measure: subjects with eventual bleed control
17
9.Secondary Outcome
Title Correlation Between Response to OBI-1 Therapy at 16 Hours and Eventual Control of Serious Bleeding Episodes at 24 Hours
Hide Description [Not Specified]
Time Frame 24 hours
Hide Outcome Measure Data
Hide Analysis Population Description
All 19 subjects in the ITT population (n=29) who had responses available at 16 hours after initial infusion of OBI-1 had a positive response.
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 19
Measure Type: Number
Unit of Measure: subjects with eventual bleed control
17
10.Secondary Outcome
Title Correlation Between Response to OBI-1 Therapy at Specified Time Points and Eventual Control of Serious Bleeding Episodes at 24 Hours
Hide Description [Not Specified]
Time Frame 24 hours
Hide Outcome Measure Data
Hide Analysis Population Description
29 subjects (ITT population) had responses available at 24 hours after initial infusion of OBI-1.
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 29
Measure Type: Number
Unit of Measure: participants with bleeds controlled
25
11.Secondary Outcome
Title Correlation Between the Pre-infusion Anti-OBI-1 Antibody Titers, the Total Dose of OBI-1, the Outcome at 24 Hours and the Eventual Control of the Bleeding Episode
Hide Description [Not Specified]
Time Frame Through 90 days ± 7 days following final OBI-1 dose
Hide Outcome Measure Data
Hide Analysis Population Description
Because of expected sparseness of positive anti-OBI-1 antibody titers, formal statistical analyses of correlation were not performed.
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
12.Secondary Outcome
Title Pharmacokinetics (PK) Analysis- Plasma Clearance
Hide Description Participation in the PK sampling was optional. PK parameters obtained from the non-bleeding state were summarized with descriptive statistics.
Time Frame Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours
Hide Outcome Measure Data
Hide Analysis Population Description
PK population (= all subjects in the ITT population who consent to PK draws and have factor VIII levels measured at the central reference laboratory)
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: U/(percent activity*hours)
Chromogenic FVIII activity assay 11.80  (13.44)
One-stage FVIII activity assay 18.07  (21.78)
13.Secondary Outcome
Title PK Analysis- Volume of Distribution (Vd) at Steady State
Hide Description Participation in the PK sampling was optional. PK parameters obtained from the non-bleeding state were summarized with descriptive statistics.
Time Frame Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours
Hide Outcome Measure Data
Hide Analysis Population Description
PK population
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: U/percent activity
Chromogenic FVIII activity assay 53.8  (52.9)
One-stage FVIII activity assay 65.1  (45.1)
14.Secondary Outcome
Title PK Analysis- Area Under the Concentration-time Curve (AUC) From Time 0 to the Last Measurable Concentration
Hide Description Participation in the PK sampling was optional. PK parameters obtained from the non-bleeding state were summarized with descriptive statistics. AUC was calculated as area under the percent activity-time curve.
Time Frame Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours
Hide Outcome Measure Data
Hide Analysis Population Description
PK population
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: percent activity*hours
Chromogenic FVIII activity assay 599  (459)
One-stage FVIII activity assay 423  (340)
15.Secondary Outcome
Title PK Analysis- Terminal Half-life
Hide Description Participation in the PK sampling was optional. PK parameters obtained from the non-bleeding state were summarized with descriptive statistics. Half-life was calculated as the time it took to reduce percent activity by half.
Time Frame Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours
Hide Outcome Measure Data
Hide Analysis Population Description
PK population
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: hours
Chromogenic FVIII activity assay 3.3  (0.4)
One-stage FVIII activity assay 3.5  (1.0)
16.Secondary Outcome
Title Number of Participants Who Developed de Novo Anti-OBI-1 Antibody Titers
Hide Description [Not Specified]
Time Frame Through 90 days ± 7 days following final OBI-1 dose
Hide Outcome Measure Data
Hide Analysis Population Description
28 eligible subjects with acquired hemophilia A in the ITT population (n=29), of whom 18 had no detectable anti-porcine FVIII inhibitor titers at baseline (<0.6 BU) and 10 had detectable anti-porcine FVIII antibody titers at baseline (>=0.6 BU)
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 28
Measure Type: Number
Unit of Measure: participants
5
17.Secondary Outcome
Title Number of Participants Who Developed an Anti-host Cell Protein Baby Hamster Kidney (BHK) Antibody Titer
Hide Description [Not Specified]
Time Frame Through 90 days ± 7 days following final OBI-1 dose
Hide Outcome Measure Data
Hide Analysis Population Description
21 subjects in the ITT population (n=29) with available baseline and follow-up test results
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 21
Measure Type: Number
Unit of Measure: participants
0
18.Other Pre-specified Outcome
Title Anti-human Factor VIII Antibody Titer
Hide Description [Not Specified]
Time Frame Through 90 days ± 7 days following final OBI-1 dose
Hide Outcome Measure Data
Hide Analysis Population Description
Anti-human factor VIII antibody titer data were presented in subject data listings. No statistical test was planned for anti-human factor VIII antibody titer.
Arm/Group Title OBI-1
Hide Arm/Group Description:
Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame Through 90 days ± 7 days following final OBI-1 dose
Adverse Event Reporting Description Subjects were monitored for adverse events from the time the subject presented with the initial bleeding episode until the end of the follow-up period.
 
Arm/Group Title OBI-1
Hide Arm/Group Description Initial dose: 200 U/kg - additional doses at the discretion of the investigator based on FVIII activity level and clinical assessment of response to treatment (upper limit: 400 U/kg every 2 hours)
All-Cause Mortality
OBI-1
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
OBI-1
Affected / at Risk (%) # Events
Total   13/29 (44.83%)    
Cardiac disorders   
Atrial fibrillation * 1  1/29 (3.45%)  1
Gastrointestinal disorders   
Esophagitis * 1  1/29 (3.45%)  1
Gastrointestinal hemorrhage * 1  1/29 (3.45%)  1
Intestinal hemorrhage * 1  1/29 (3.45%)  1
General disorders   
Asthenia * 1 [1]  1/29 (3.45%)  1
Hepatobiliary disorders   
Cholangitis * 1  1/29 (3.45%)  1
Immune system disorders   
Anaphylactic reaction * 1  1/29 (3.45%)  1
Infections and infestations   
Pneumonia * 1  3/29 (10.34%)  3
Sepsis * 1  2/29 (6.90%)  2
Systemic mycosis * 1  1/29 (3.45%)  1
Urinary tract infection * 1  1/29 (3.45%)  1
Injury, poisoning and procedural complications   
Fall * 1  1/29 (3.45%)  1
Tracheostomy malfunction * 1  1/29 (3.45%)  1
Vascular pseudoaneurysm * 1  1/29 (3.45%)  1
Musculoskeletal and connective tissue disorders   
Arthralgia * 1  1/29 (3.45%)  3
Joint swelling * 1  1/29 (3.45%)  2
Hemarthrosis * 1  1/29 (3.45%)  1
Nervous system disorders   
Dizziness * 1  1/29 (3.45%)  1
Brain edema * 1  1/29 (3.45%)  1
Intracranial hemorrhage * 1  2/29 (6.90%)  2
Transient ischemic attack * 1  1/29 (3.45%)  1
Grand mal convulsion * 1  1/29 (3.45%)  1
Renal and urinary disorders   
Renal failure * 1  1/29 (3.45%)  1
Respiratory, thoracic and mediastinal disorders   
Respiratory failure * 1  1/29 (3.45%)  1
Vascular disorders   
Hematoma * 1  1/29 (3.45%)  2
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (13.1)
[1]
Generalized weakness
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
OBI-1
Affected / at Risk (%) # Events
Total   27/29 (93.10%)    
Blood and lymphatic system disorders   
Anemia * 1  6/29 (20.69%)  6
Cardiac disorders   
Cardiac failure congestive * 1  2/29 (6.90%)  2
Gastrointestinal disorders   
Abdominal pain * 1  2/29 (6.90%)  3
Abdominal pain upper * 1  2/29 (6.90%)  3
Constipation * 1  12/29 (41.38%)  12
Diarrhea * 1  7/29 (24.14%)  7
Mouth ulceration * 1  2/29 (6.90%)  2
Nausea * 1  4/29 (13.79%)  5
Vomiting * 1  2/29 (6.90%)  2
General disorders   
Chest pain * 1  2/29 (6.90%)  2
Fatigue * 1  2/29 (6.90%)  2
Peripheral edema * 1  6/29 (20.69%)  7
Pyrexia * 1  3/29 (10.34%)  5
Infections and infestations   
Bacteremia * 1  2/29 (6.90%)  2
Bacteriuria * 1  2/29 (6.90%)  2
Candidiasis * 1  3/29 (10.34%)  3
Sepsis * 1  3/29 (10.34%)  3
Oral candidiasis * 1  3/29 (10.34%)  3
Urinary tract infection * 1  2/29 (6.90%)  2
Investigations   
Antibody test positive * 1 [1]  2/29 (6.90%)  2
Troponin I increased * 1  2/29 (6.90%)  2
Metabolism and nutrition disorders   
Decreased appetite * 1  3/29 (10.34%)  3
Hypoglycemia * 1  2/29 (6.90%)  2
Hypokalemia * 1  7/29 (24.14%)  11
Hypomagnesemia * 1  2/29 (6.90%)  2
Hypophosphatemia * 1  2/29 (6.90%)  2
Musculoskeletal and connective tissue disorders   
Muscle hemorrhage * 1  2/29 (6.90%)  3
Pain in extremity * 1  2/29 (6.90%)  2
Psychiatric disorders   
Depression * 1  2/29 (6.90%)  2
Insomnia * 1  4/29 (13.79%)  6
Respiratory, thoracic and mediastinal disorders   
Wheezing * 1  2/29 (6.90%)  3
Skin and subcutaneous tissue disorders   
Ecchymosis * 1  3/29 (10.34%)  3
Pruritus * 1  2/29 (6.90%)  2
Vascular disorders   
Hypertension * 1  3/29 (10.34%)  4
Hypotension * 1  2/29 (6.90%)  2
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (13.1)
[1]
Positive OBI-1 (anti-porcine FVIII) inhibitor test result
Due to the low number of subjects available for evaluation, statistical tests could only be performed for the primary outcome measure. The results of all other outcome measures are descriptive.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
For this study, PIs are restricted from independently publishing results before completion of a single multicenter publication or one year after study completion, whichever occurs first. Baxter requires a review of results communications (eg, for confidential information) ≥30 days prior to submission or communication. Baxter may request an additional delay of ≤60 days (eg, if a patentable invention is disclosed).
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Shire
Phone: +1 866 842 5335
EMail: ClinicalTransparency@shire.com
Layout table for additonal information
Responsible Party: Shire ( Baxalta now part of Shire )
ClinicalTrials.gov Identifier: NCT01178294     History of Changes
Other Study ID Numbers: OBI-1-301
First Submitted: August 6, 2010
First Posted: August 10, 2010
Results First Submitted: April 28, 2015
Results First Posted: December 21, 2015
Last Update Posted: June 17, 2019