A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (PERSEPT2)
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ClinicalTrials.gov Identifier: NCT02448680 |
Recruitment Status :
Completed
First Posted : May 19, 2015
Results First Posted : August 28, 2020
Last Update Posted : February 25, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Hemophilia A With Inhibitors Hemophilia B With Inhibitors | Biological: Coagulation FVIIa (Recombinant) | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 25 participants |
Allocation: | Randomized |
Intervention Model: | Crossover Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | December 7, 2015 |
Actual Primary Completion Date : | June 30, 2017 |
Actual Study Completion Date : | August 30, 2017 |

Arm | Intervention/treatment |
---|---|
Active Comparator: Coagulation Factor VIIa (Recombinant): 75 µg/kg
75 µg/kg treatment regimen for 3 months
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Biological: 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 |
Active Comparator: Coagulation Factor VIIa (Recombinant): 225 µg/kg
225 µg/kg treatment regimen for 3 months
|
Biological: 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 |
- Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement. [ Time Frame: 12 hours after first administration of study drug ]
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 [ Time Frame: 12 hours after first administration of study drug ]
- "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.
- Patient-Reported "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes [ Time Frame: 12 hour after first administration of study drug ]
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 [ Time Frame: Within 24 hours of Bleeding Episode ]
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 [ Time Frame: Within 24 hours of 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 [ Time Frame: Within 24 hours of Bleeding Episode ]The total amount of study drug administered in order to treat one mild/moderate bleeding episode.
- Mild/Moderate Bleeding Episodes With Successful Pain Relief [ Time Frame: 12 hour after first administration of study drug ]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.

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Ages Eligible for Study: | up to 11 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02448680
United States, Colorado | |
University of Colorado Denver Hemophilia & Thrombosis Center | |
Aurora, Colorado, United States, 80045 | |
United States, Oklahoma | |
Jimmy Everest Center for Cancer and Bleeding Disorders | |
Oklahoma City, Oklahoma, United States, 73117 | |
United States, Texas | |
UT Southwestern Medical Center at Dallas / Children's Medical Center | |
Dallas, Texas, United States, 75390 | |
Bulgaria | |
University Multiprofile Hospital for Active Treatment "Sveti Georgi" | |
Plovdiv, Bulgaria | |
Czechia | |
University Hospital Motol | |
Prague, Czechia | |
Georgia | |
Hematology of Department Hemophilia and Thromboses center | |
Tbilisi, Georgia | |
South Africa | |
Worthwhile Clinical Trials | |
Benoni, South Africa | |
Ukraine | |
National Specialized Children's Hospital OKHMATDYT, Centre for Hemostatic Pathology (Ukraine) | |
Kyiv, Ukraine | |
Institute of Blood Pathology and Transfusion Medicine | |
Lviv, Ukraine |
Principal Investigator: | Michael Wang, MD | University of Colorado, Denver |
Documents provided by Laboratoire français de Fractionnement et de Biotechnologies:
Responsible Party: | Laboratoire français de Fractionnement et de Biotechnologies |
ClinicalTrials.gov Identifier: | NCT02448680 |
Other Study ID Numbers: |
LFB-FVIIa-007-14 |
First Posted: | May 19, 2015 Key Record Dates |
Results First Posted: | August 28, 2020 |
Last Update Posted: | February 25, 2022 |
Last Verified: | February 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Hemophilia A Hemophilia B Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn Genetic Diseases, X-Linked |