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Trial record 1 of 1 for:    NCT01992549
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Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

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ClinicalTrials.gov Identifier: NCT01992549
Recruitment Status : Completed
First Posted : November 25, 2013
Results First Posted : December 17, 2019
Last Update Posted : January 19, 2021
Sponsor:
Information provided by (Responsible Party):
Octapharma

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Severe Hemophilia A
Intervention Biological: Human-cl rhFVIII
Enrollment 48
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Human-cl rhFVIII
Hide Arm/Group Description Of the total number of patients that started in the study, all those in the safety (SAF) population received at least 1 infusion of Human-cl rhFVIII. Patients who had data collected post-treatment were included in the intent-to-treat (ITT) population. Prophylactic treatment dose given to all patients in the PROPH population (all patients who received at least 1 administration of Human-cl rhFVIII with prophylaxis documented as the reason for treatment): >20 IU FVIII/kg body weight (BW). On-demand treatment of bleeding episodes (BEs) dose given to the BLEED population (all patients with bleeding episodes treated with Human-cl rhFVIII): 20-30 IU FVIII/kg BW (minor haemorrhage), 30-40 IU FVIII/kg BW (moderate to major haemorrhage) or 50-80 IU FVIII/kg BW (major to life-threatening haemorrhage). Surgical prophylaxis dose was given to the SURG population (all patients with surgeries treated with Human-cl rhFVIII): 25-30 IU FVIII/kg BW (minor surgeries); >50 IU FVIII/kg BW (major surgeries).
Period Title: Overall Study
Started 48
SAF Population [1] 48
ITT Population [2] 47
PROPH Population [3] 47
BLEED Population [4] 29
SURG Population [5] 3
Completed 44
Not Completed 4
Reason Not Completed
Lost to Follow-up             2
Serious Adverse Event             1
Product Switch to Extend Half Life             1
[1]
SAF population = study population of patients in safety analysis
[2]
ITT population = intent-to-treat population
[3]
PROPH population = study population of patients receiving prophylaxis
[4]
BLEED population = study population of Bleeding Events (BEs)
[5]
SURG population = study population of patients undergoing surgery treated with Human-cl rhFVIII
Arm/Group Title Human-cl rhFVIII
Hide Arm/Group Description The safety (SAF) population consist of all patients who received at least one infusion of Human-cl rhFVIII (n=48).
Overall Number of Baseline Participants 48
Hide Baseline Analysis Population Description
The safety (SAF) population consist of all patients who received at least one infusion of Human-cl rhFVIII (n=48).
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 48 participants
3.47  (2.13)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 48 participants
Female
0
   0.0%
Male
48
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 48 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
48
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 48 participants
American Indian or Alaska Native
1
   2.1%
Asian
9
  18.8%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
36
  75.0%
More than one race
2
   4.2%
Unknown or Not Reported
0
   0.0%
Body Mass Index (BMI) at screening  
Mean (Full Range)
Unit of measure:  Kg/m^2
Number Analyzed 48 participants
16.46
(12.4 to 22.3)
Height at screening  
Mean (Full Range)
Unit of measure:  Centimeters (cm)
Number Analyzed 48 participants
97.44
(78 to 138)
Weight at screening  
Mean (Full Range)
Unit of measure:  Kilograms (kg)
Number Analyzed 48 participants
15.84
(9.1 to 33.0)
Family history of inhibitors  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 48 participants
Yes
3
   6.3%
No
45
  93.8%
1.Primary Outcome
Title Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors
Hide Description The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII. The definition threshold for a "positive" inhibitor was if the modified Bethesda assay resulted in a titre ≥0.6 BU/mL at any time point during the observation period.
Time Frame Maximum two years
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis was performed for the safety (SAF) population which includes all patients who received at least 1 infusion of Human-cl rhFVIII (N=48)
Arm/Group Title Human-cl rhFVIII
Hide Arm/Group Description:
The safety (SAF) population consist of all patients who received at least one infusion of Human-cl rhFVIII
Overall Number of Participants Analyzed 48
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: participants
0
(0.000 to 7.549)
2.Secondary Outcome
Title Frequency of Spontaneous Break-through Bleeds
Hide Description The annualized bleeding rate (ABR) was calculated during the time of prophylactic treatment with Human-cl rhFVIII for spontaneous bleeding events (BEs).
Time Frame Maximum 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population includes all patients in PROPH population who received at least one prophylactic treatment with Human-cl rhFVIII (n=47).
Arm/Group Title ITT/PROPH Population
Hide Arm/Group Description:
All patients who had data collected post-treatment with Human-cl rhFVIII and at least one prophylactic treatment with Human-cl rhFVIII (n=47).
Overall Number of Participants Analyzed 47
Mean (95% Confidence Interval)
Unit of Measure: Events/year (ABR)
0.283
(0.153 to 0.527)
3.Secondary Outcome
Title Efficacy of Human-cl rhFVIII for the Treatment of Bleeds
Hide Description A personal efficacy assessment (final outcome) to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes (BEs) at the end of a BE. Efficacy was assessed using a four-point scale (excellent, good, moderate, none) by the patient's parent(s)/legal guardian(s) together with the investigator in case of on site treatment.
Time Frame Maximum 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population included all patients who received on-demand treatment with Human-cl rhFVIII for bleeding episodes (BLEED population; n=29).
Arm/Group Title BLEED Population
Hide Arm/Group Description:
All patients that experienced at least one bleeding episode treated with Human-cl rhFVIII (n=29)
Overall Number of Participants Analyzed 29
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding episodes
111
Count of Units
Unit of Measure: Bleeding episodes
Excellent
58
  52.3%
Good
28
  25.2%
Moderate
21
  18.9%
None
3
   2.7%
Not done
1
   0.9%
4.Secondary Outcome
Title Efficacy of Human-cl rhFVIII for Surgical Prophylaxis
Hide Description An overall efficacy assessment to assess the efficacy of human-cl rhFVIII in surgical prophylaxis of minor and major surgeries. The efficacy assessment was analyzed using a four-point scale (excellent, good, moderate, none). If surgeries could not be assessed due to limited data available or having taken place outside the study site, the results were classified as "not done".
Time Frame Maximum 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population includes 3 patients that received Human-cl rhFVIII for surgical prophylaxis during a total of 4 surgeries (SURG population). Of these, 1 patient had two minor surgeries and 2 patients had one major surgery each.
Arm/Group Title Minor Surgeries Major Surgeries SURG Population
Hide Arm/Group Description:
All patients that had minor surgeries performed under Human-cl rhFVIII treatment (n=1)
All patients that had major surgeries performed under Human-cl rhFVIII treatment (n=2)
All patients that had surgeries performed under Human-cl rhFVIII treatment (n=3)
Overall Number of Participants Analyzed 1 2 3
Overall Number of Units Analyzed
Type of Units Analyzed: Surgeries
2 2 4
Count of Units
Unit of Measure: Surgeries
Excellent
0
   0.0%
2
 100.0%
2
  50.0%
Good
0
   0.0%
0
   0.0%
0
   0.0%
Moderate
0
   0.0%
0
   0.0%
0
   0.0%
None
0
   0.0%
0
   0.0%
0
   0.0%
Not done
2
 100.0%
0
   0.0%
2
  50.0%
5.Secondary Outcome
Title The Occurrence of Any Adverse Event (AE)
Hide Description The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.
Time Frame Maximum 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis was performed for the safety (SAF) population which includes all patients who received at least 1 infusion of Human-cl rhFVIII (n=48).
Arm/Group Title SAF Population
Hide Arm/Group Description:
The safety (SAF) population consist of all patients who received at least one infusion of Human-cl rhFVIII (n=48).
Overall Number of Participants Analyzed 48
Measure Type: Count of Participants
Unit of Measure: Participants
Adverse event
29
  60.4%
Serious adverse event (SAE)
5
  10.4%
Severe AE
3
   6.3%
Temporally related adverse event
20
  41.7%
Death
0
   0.0%
AE leading to permanent discontinuation
1
   2.1%
Time Frame Maximum 2 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title SAF Population
Hide Arm/Group Description The safety (SAF) population consist of all patients who received at least one infusion of Human-cl rhFVIII (n=48).
All-Cause Mortality
SAF Population
Affected / at Risk (%)
Total   0/48 (0.00%) 
Hide Serious Adverse Events
SAF Population
Affected / at Risk (%)
Total   5/48 (10.42%) 
Gastrointestinal disorders   
Gastritis   1/48 (2.08%) 
Infections and infestations   
Pneumonia   2/48 (4.17%) 
Varicella   1/48 (2.08%) 
Cellulitis   1/48 (2.08%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Neuroblastoma   1/48 (2.08%) 
Respiratory, thoracic and mediastinal disorders   
Epistaxis   1/48 (2.08%) 
Vascular disorders   
Haemorrhage   1/48 (2.08%) 
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2.08%
SAF Population
Affected / at Risk (%)
Total   29/48 (60.42%) 
Blood and lymphatic system disorders   
Anaemia   4/48 (8.33%) 
Gastrointestinal disorders   
Diarrhoea   2/48 (4.17%) 
Vomiting   2/48 (4.17%) 
General disorders   
Pyrexia   10/48 (20.83%) 
Infections and infestations   
Nasopharyngitis   10/48 (20.83%) 
Varicella   5/48 (10.42%) 
Bronchitis   4/48 (8.33%) 
Ear infection   4/48 (8.33%) 
Pharyngitis   4/48 (8.33%) 
Tonsillitis   3/48 (6.25%) 
Otitis media   2/48 (4.17%) 
Pneumonia   2/48 (4.17%) 
Pulpitis dental   2/48 (4.17%) 
Respiratory tract infection viral   2/48 (4.17%) 
Tracheitis   2/48 (4.17%) 
Respiratory, thoracic and mediastinal disorders   
Cough   7/48 (14.58%) 
Rhinorrhoea   2/48 (4.17%) 
Indicates events were collected by systematic assessment
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.
Octapharma agreements may vary with individual investigators, but will not prohibit any investigator from publishing. Octapharma supports the publication of results from all centers of a multi-center trial but requests that reports based on single-site data not precede the primary publication of the entire clinical trial. Octapharma also reserves the right to review data prior to publishing and provide comments/changes within a certain time period.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Sylvia Werner
Organization: Octapharma
Phone: 415 260-9577
EMail: sylvia.werner@octapharma.com
Layout table for additonal information
Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT01992549    
Other Study ID Numbers: GENA-15
First Submitted: November 19, 2013
First Posted: November 25, 2013
Results First Submitted: November 27, 2019
Results First Posted: December 17, 2019
Last Update Posted: January 19, 2021