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

Primary Purpose

Hemophilia A With Inhibitors, Hemophilia B With Inhibitors

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Coagulation FVIIa (Recombinant)
Sponsored by
Laboratoire français de Fractionnement et de Biotechnologies
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A With Inhibitors

Eligibility Criteria

undefined - 11 Years (Child)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • be male with a diagnosis of congenital hemophilia A or B of any severity
  • have one of the following:
  • a positive inhibitor test BU ≥5, OR
  • a Bethesda Unit (BU) <5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of factor VIII or IX products to treat bleeding episodes, OR
  • a BU <5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of factor VIII or IX products to treat bleeding episodes
  • be aged from birth to <12 years old
  • have experienced at least 3 bleeding episodes of any severity in the past 6 months
  • parents or legal guardians must be capable of understanding and be willing to comply with the conditions of the protocol
  • parents or legal guardians must have read, understood, and provided written informed consent

Exclusion Criteria:

  • have any coagulation disorder other than hemophilia A or B
  • be immunosuppressed (i.e., the patient may not be receiving systemic immunosuppressive medication; cluster of differentiation 4 (CD4) counts at screening must be >200/µL)
  • have a known allergy or hypersensitivity to rabbits
  • have platelet count <100,000/mL
  • have had a major surgical procedure (e.g. orthopedic, abdominal) within 1 month prior to first administration of study drug
  • have received an investigational drug within 30 days of first study drug administration, or be expected to receive such drug during participation in this study

Sites / Locations

  • University of Colorado Denver Hemophilia & Thrombosis Center
  • Jimmy Everest Center for Cancer and Bleeding Disorders
  • UT Southwestern Medical Center at Dallas / Children's Medical Center
  • University Multiprofile Hospital for Active Treatment "Sveti Georgi"
  • University Hospital Motol
  • Hematology of Department Hemophilia and Thromboses center
  • Worthwhile Clinical Trials
  • National Specialized Children's Hospital OKHMATDYT, Centre for Hemostatic Pathology (Ukraine)
  • Institute of Blood Pathology and Transfusion Medicine

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Active Comparator

Arm Label

Coagulation Factor VIIa (Recombinant): 75 µg/kg

Coagulation Factor VIIa (Recombinant): 225 µg/kg

Arm Description

75 µg/kg treatment regimen for 3 months

225 µg/kg treatment regimen for 3 months

Outcomes

Primary Outcome Measures

Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement.
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
Proportion of Successfully Treated Bleeding Episodes (Mild/Moderate/Severe) Per EMA Definition
"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.

Secondary Outcome Measures

Patient-Reported "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes
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 to Patient Assessment of a "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes
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.
Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode
The number of study drug administrations with non-missing dose information in order to treat one mild/moderate bleeding episode.
Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode
The total amount of study drug administered in order to treat one mild/moderate bleeding episode.

Full Information

First Posted
May 15, 2015
Last Updated
February 24, 2022
Sponsor
Laboratoire français de Fractionnement et de Biotechnologies
Collaborators
LFB USA, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT02448680
Brief Title
A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa
Acronym
PERSEPT2
Official Title
A Phase III Study on the Safety, Pharmacokinetics, and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Pediatric Patients From Birth to <12 Years Old With Inhibitors to Factor VIII or IX: PerSept 2
Study Type
Interventional

2. Study Status

Record Verification Date
February 2022
Overall Recruitment Status
Completed
Study Start Date
December 7, 2015 (Actual)
Primary Completion Date
June 30, 2017 (Actual)
Study Completion Date
August 30, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Laboratoire français de Fractionnement et de Biotechnologies
Collaborators
LFB USA, Inc.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/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 IX in 12 patients ( birth to <6 years old), and 12 patients (≥6 years old to <12 years old).
Detailed Description
A Phase III Study on the Safety, Pharmacokinetics, and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Pediatric Patients from birth to <12 years old with Inhibitors to Factor VIII or IX: PerSept 2

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A With Inhibitors, Hemophilia B With Inhibitors

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
25 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Coagulation Factor VIIa (Recombinant): 75 µg/kg
Arm Type
Active Comparator
Arm Description
75 µg/kg treatment regimen for 3 months
Arm Title
Coagulation Factor VIIa (Recombinant): 225 µg/kg
Arm Type
Active Comparator
Arm Description
225 µg/kg treatment regimen for 3 months
Intervention Type
Biological
Intervention Name(s)
Coagulation FVIIa (Recombinant)
Intervention Description
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
Primary Outcome Measure Information:
Title
Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement.
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
Title
Proportion of Successfully Treated Bleeding Episodes (Mild/Moderate/Severe) Per EMA Definition
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
Secondary Outcome Measure Information:
Title
Patient-Reported "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes
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
Title
Time to Patient Assessment of a "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes
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
Title
Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode
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
Title
Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode
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
Other Pre-specified Outcome Measures:
Title
Mild/Moderate Bleeding Episodes With Successful Pain Relief
Description
Successful pain relief was defined as a Visual Analogue Scale (VAS: 0-100; 0: no pain at all; 100: the worst pain ever possible) 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

10. Eligibility

Sex
Male
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: be male with a diagnosis of congenital hemophilia A or B of any severity have one of the following: a positive inhibitor test BU ≥5, OR a Bethesda Unit (BU) <5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of factor VIII or IX products to treat bleeding episodes, OR a BU <5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of factor VIII or IX products to treat bleeding episodes be aged from birth to <12 years old have experienced at least 3 bleeding episodes of any severity in the past 6 months parents or legal guardians must be capable of understanding and be willing to comply with the conditions of the protocol parents or legal guardians must have read, understood, and provided written informed consent Exclusion Criteria: have any coagulation disorder other than hemophilia A or B be immunosuppressed (i.e., the patient may not be receiving systemic immunosuppressive medication; cluster of differentiation 4 (CD4) counts at screening must be >200/µL) have a known allergy or hypersensitivity to rabbits have platelet count <100,000/mL have had a major surgical procedure (e.g. orthopedic, abdominal) within 1 month prior to first administration of study drug have received an investigational drug within 30 days of first study drug administration, or be expected to receive such drug during participation in this study
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michael Wang, MD
Organizational Affiliation
University of Colorado, Denver
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Colorado Denver Hemophilia & Thrombosis Center
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Jimmy Everest Center for Cancer and Bleeding Disorders
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73117
Country
United States
Facility Name
UT Southwestern Medical Center at Dallas / Children's Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
University Multiprofile Hospital for Active Treatment "Sveti Georgi"
City
Plovdiv
Country
Bulgaria
Facility Name
University Hospital Motol
City
Prague
Country
Czechia
Facility Name
Hematology of Department Hemophilia and Thromboses center
City
Tbilisi
Country
Georgia
Facility Name
Worthwhile Clinical Trials
City
Benoni
Country
South Africa
Facility Name
National Specialized Children's Hospital OKHMATDYT, Centre for Hemostatic Pathology (Ukraine)
City
Kyiv
Country
Ukraine
Facility Name
Institute of Blood Pathology and Transfusion Medicine
City
Lviv
Country
Ukraine

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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