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Trial record 2 of 2 for:    LFB USA

A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (PERSEPT2)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02448680
Recruitment Status : Completed
First Posted : May 19, 2015
Results First Posted : August 28, 2020
Last Update Posted : August 28, 2020
Sponsor:
Collaborator:
Laboratoire français de Fractionnement et de Biotechnologies
Information provided by (Responsible Party):
LFB USA, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Hemophilia A With Inhibitors
Hemophilia B With Inhibitors
Intervention Biological: Coagulation FVIIa (Recombinant)
Enrollment 25
Recruitment Details  
Pre-assignment Details  
Arm/Group Title FVIIa: 75 µg/kg First, Then 225 µg/kg FVIIa: 225 µg/kg First, Then 75 µg/kg
Hide Arm/Group Description Coagulation Factor VIIa (Recombinant) : First Intervention (3 months), Second Intervention (3 months), repeat sequence for entirety of study. Coagulation Factor VIIa (Recombinant) : First Intervention (3 months), Second Intervention (3 months), repeat sequence for entirety of study.
Period Title: Overall Study
Started 12 13
Completed 11 10
Not Completed 1 3
Reason Not Completed
Physician Decision             1             1
Withdrawal by Subject             0             2
Arm/Group Title FVIIa: 75 µg/kg First, Then 225 µg/kg FVIIa: 225 µg/kg First, Then 75 µg/kg Total
Hide Arm/Group Description Coagulation Factor VIIa (Recombinant) : First Intervention (3 months), Second Intervention (3 months), repeat sequence for entirety of study. Coagulation Factor VIIa (Recombinant) : First Intervention (3 months), Second Intervention (3 months), repeat sequence for entirety of study. Total of all reporting groups
Overall Number of Baseline Participants 12 13 25
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 12 participants 13 participants 25 participants
4.9  (3.02) 4.8  (3.63) 4.9  (3.28)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Age, Categorical Number Analyzed 12 participants 13 participants 25 participants
Birth to < 6 years old
6
  50.0%
7
  53.8%
13
  52.0%
>= 6 years to < 12 years old
6
  50.0%
6
  46.2%
12
  48.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants 13 participants 25 participants
Female 0 0 0
Male 12 13 25
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants 13 participants 25 participants
Hispanic or Latino
1
   8.3%
2
  15.4%
3
  12.0%
Not Hispanic or Latino
11
  91.7%
11
  84.6%
22
  88.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants 13 participants 25 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
4
  33.3%
3
  23.1%
7
  28.0%
White
8
  66.7%
10
  76.9%
18
  72.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Weight  
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 12 participants 13 participants 25 participants
19.73  (7.432) 21.94  (13.391) 20.88  (10.782)
Height  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 12 participants 13 participants 25 participants
104.21  (21.210) 108.60  (25.904) 106.49  (23.382)
Body Mass Index (BMI)  
Mean (Standard Deviation)
Unit of measure:  Kg/m^2
Number Analyzed 12 participants 13 participants 25 participants
17.74  (2.505) 17.35  (3.842) 17.53  (3.209)
1.Primary Outcome
Title Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement.
Hide Description

For the primary efficacy endpoint, successful treatment of mild/moderate bleeding episode was defined as meeting all of the following:

  • "Good" or "excellent" response noted by the patient/parent/legal guardian or other caregiver, depending on patient's age and maturity
  • Study drug treatment: No further treatment with LR769 beyond timepoint where a "good" or "excellent" response for this bleeding episode was noted
  • No other hemostatic treatment needed for this bleeding episode
  • No administration of blood products that would indicate continuation of bleeding beyond timepoint where a "good" or "excellent" response for this bleeding episode was noted
  • No increase of pain beyond timepoint where a "good" or "excellent" response for this bleeding episode was noted that could not be explained other than as continuation of bleeding
Time Frame 12 hours after first administration of study drug
Hide Outcome Measure Data
Hide Analysis Population Description
Treated Population
Arm/Group Title FVIIa 75 µg/kg FVIIa 225µg/kg
Hide Arm/Group Description:
75 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode
225 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode
Overall Number of Participants Analyzed 23 23
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding Episodes (BEs)
239 307
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Proportion of successfully treated BEs
0.654
(0.523 to 0.785)
0.603
(0.482 to 0.723)
2.Primary Outcome
Title Proportion of Successfully Treated Bleeding Episodes (Mild/Moderate/Severe) Per EMA Definition
Hide Description
  • "Good" or "excellent" response noted by the patient/caregiver for mild/moderate bleeding episodes;
  • "Good" or "excellent" response noted by the physician for severe bleeding episodes.
Time Frame 12 hours after first administration of study drug
Hide Outcome Measure Data
Hide Analysis Population Description
Treated Population
Arm/Group Title FVIIa 75 µg/kg FVIIa 225 µg/kg
Hide Arm/Group Description:
75 µg/kg Treatment Regimen at Time of Bleeding Episode
225 µg/kg Treatment Regimen at Time of Bleeding Episode
Overall Number of Participants Analyzed 23 24
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding Episodes (BEs)
239 310
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Proportion of successfully treated BEs
0.667
(0.533 to 0.800)
0.625
(0.500 to 0.750)
3.Secondary Outcome
Title Patient-Reported "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes
Hide Description

Based on Patient-Reported "Good" or "Excellent" responses as per the below descriptions:

Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) had largely been reduced by the treatment, but had not completely disappeared. Symptoms had improved enough to not require more infusions of the study drug.

Excellent: Full relief of pain and cessation of objective signs of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage). No additional infusion of study drug was required.

Time Frame 12 hour after first administration of study drug
Hide Outcome Measure Data
Hide Analysis Population Description
Treated Population
Arm/Group Title FVIIa 75 µg/kg FVIIa 225µg/kg
Hide Arm/Group Description:
75 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode
225 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode
Overall Number of Participants Analyzed 23 23
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding Episodes (BEs)
239 307
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Proportion of successfully treated BEs
0.667
(0.533 to 0.800)
0.629
(0.502 to 0.756)
4.Secondary Outcome
Title Time to Patient Assessment of a "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes
Hide Description

Categories of Response to Treatment are Described as Follows:

None: No noticeable effect of the treatment on the bleed or worsening of patient's condition. Continuation of treatment with the study drug was needed.

Moderate: Some effect of the treatment on the bleed was noticed, e.g., pain decreased or bleeding signs improved, but bleed continued and required continued treatment with the study drug.

Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had largely been reduced by the treatment, but had not completely disappeared. Symptoms had improved enough to not require more infusions of the study drug.

Excellent: Full relief of pain and cessation of objective signs of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage). No additional infusion of study drug was required.

Time Frame Within 24 hours of Bleeding Episode
Hide Outcome Measure Data
Hide Analysis Population Description
Treated Population with non-missing measurements
Arm/Group Title FVIIa 75 µg/kg FVIIa 225µg/kg
Hide Arm/Group Description:
75 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode
225 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode
Overall Number of Participants Analyzed 23 23
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding Episodes with Event
233 299
Median (95% Confidence Interval)
Unit of Measure: Hours
9.00
(8.92 to 11.83)
12.00
(11.83 to 12.00)
5.Secondary Outcome
Title Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode
Hide Description The number of study drug administrations with non-missing dose information in order to treat one mild/moderate bleeding episode.
Time Frame Within 24 hours of Bleeding Episode
Hide Outcome Measure Data
Hide Analysis Population Description
Treated Population
Arm/Group Title FVIIa 75 µg/kg FVIIa 225µg/kg
Hide Arm/Group Description:
75 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode
225 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode
Overall Number of Participants Analyzed 23 23
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding Episodes (BEs)
239 307
Mean (Standard Deviation)
Unit of Measure: Administrations of Study Drug
3.6  (2.27) 2.6  (2.48)
6.Secondary Outcome
Title Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode
Hide Description The total amount of study drug administered in order to treat one mild/moderate bleeding episode.
Time Frame Within 24 hours of Bleeding Episode
Hide Outcome Measure Data
Hide Analysis Population Description
Treated Population with non-missing measurements
Arm/Group Title FVIIa 75 µg/kg FVIIa 225µg/kg
Hide Arm/Group Description:
75 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode
225 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode
Overall Number of Participants Analyzed 23 23
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding Episodes (BEs)
239 307
Mean (Standard Deviation)
Unit of Measure: mL per bleeding episode
6.733  (6.3693) 8.287  (5.6531)
7.Other Pre-specified Outcome
Title Mild/Moderate Bleeding Episodes With Successful Pain Relief
Hide Description Successful pain relief was defined as a Visual Analogue Scale (VAS) pain score at 12 hours after initial study drug administration that was less than the pain score at the start of treatment with study drug.
Time Frame 12 hour after first administration of study drug
Hide Outcome Measure Data
Hide Analysis Population Description
Treated Population
Arm/Group Title FVIIa 75 µg/kg FVIIa 225µg/kg
Hide Arm/Group Description:
75 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode
225 µg/kg Treatment Regimen at Time of Mild/Moderate Bleeding Episode
Overall Number of Participants Analyzed 23 23
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding Episodes (BEs)
239 307
Count of Units
Unit of Measure: Bleeding Episodes (BEs)
220
  92.1%
275
  89.6%
Time Frame From signing of the informed consent/assent until resolution or 30 days after the last dose of LR769 or early termination, whichever came first.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Coagulation Factor VIIa (Recombinant): 75 µg/kg Coagulation Factor VIIa (Recombinant): 225 µg/kg
Hide Arm/Group Description

75 µg/kg treatment regimen for 3 months

Coagulation FVIIa (Recombinant): A cross over design to assess the efficacy of 2 separate dose regimens (75 µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX

225 µg/kg treatment regimen for 3 months

Coagulation FVIIa (Recombinant): A cross over design to assess the efficacy of 2 separate dose regimens (75 µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX

All-Cause Mortality
Coagulation Factor VIIa (Recombinant): 75 µg/kg Coagulation Factor VIIa (Recombinant): 225 µg/kg
Affected / at Risk (%) Affected / at Risk (%)
Total   0/23 (0.00%)      0/25 (0.00%)    
Hide Serious Adverse Events
Coagulation Factor VIIa (Recombinant): 75 µg/kg Coagulation Factor VIIa (Recombinant): 225 µg/kg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/23 (0.00%)      2/25 (8.00%)    
Infections and infestations     
Dysentery  [1]  0/23 (0.00%)  0 1/25 (4.00%)  1
Nervous system disorders     
Paresis  [1]  0/23 (0.00%)  0 1/25 (4.00%)  1
Indicates events were collected by systematic assessment
[1]
Term from vocabulary, MedDRA 15.1
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Coagulation Factor VIIa (Recombinant): 75 µg/kg Coagulation Factor VIIa (Recombinant): 225 µg/kg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   12/23 (52.17%)      15/25 (60.00%)    
Blood and lymphatic system disorders     
Anaemia  [1]  2/23 (8.70%)  2 0/25 (0.00%)  0
Gastrointestinal disorders     
Diarrhoea  [1]  1/23 (4.35%)  1 3/25 (12.00%)  3
Vomiting  [1]  1/23 (4.35%)  1 2/25 (8.00%)  2
Infections and infestations     
Bronchitis  [1]  1/23 (4.35%)  1 3/25 (12.00%)  4
Nasopharyngitis  [1]  4/23 (17.39%)  6 3/25 (12.00%)  3
Respiratory tract infection viral  [1]  1/23 (4.35%)  2 2/25 (8.00%)  3
Rhinitis  [1]  3/23 (13.04%)  3 3/25 (12.00%)  3
Respiratory, thoracic and mediastinal disorders     
Cough  [1]  3/23 (13.04%)  4 3/25 (12.00%)  4
Indicates events were collected by systematic assessment
[1]
Term from vocabulary, MedDRA 15.1
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Kerry Biron, Director US Clinical Operations
Organization: LFB USA, Inc.
Phone: 508-370-5166
EMail: kerry.biron@lfb-usa.com
Layout table for additonal information
Responsible Party: LFB USA, Inc.
ClinicalTrials.gov Identifier: NCT02448680    
Other Study ID Numbers: LFB-FVIIa-007-14
First Submitted: May 15, 2015
First Posted: May 19, 2015
Results First Submitted: July 31, 2020
Results First Posted: August 28, 2020
Last Update Posted: August 28, 2020