Hemophilia A With InhibitorsPhase 3PERSEPT2NCT02448680
A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa
A Phase 3 study testing Coagulation FVIIa (Recombinant) in people with Hemophilia A With Inhibitors and Hemophilia B With Inhibitors, run by Laboratoire français de Fractionnement et de Biotechnologies at 9 sites in 6 countries.
Trial 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)… Read more about this study
- Who is it for?
- Ages Up to 11 years, men only
- Conditions the study is for
- Hemophilia A With Inhibitors, Hemophilia B With Inhibitors
- What is being tested?
- Coagulation FVIIa (Recombinant)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 9 sitesin 6 countries
- Study dates
- 7 Dec 2015to 30 Aug 2017
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 11 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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
Show all 9Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
9 sites in 6 countries.
9 sites in 6 countries
United States3 sites
University of Colorado Denver Hemophilia & Thrombosis Center
Aurora, Colorado, 80045
UT Southwestern Medical Center at Dallas / Children's Medical Center
Dallas, Texas, 75390
Jimmy Everest Center for Cancer and Bleeding Disorders
Oklahoma City, Oklahoma, 73117
Ukraine2 sites
National Specialized Children's Hospital OKHMATDYT, Centre for Hemostatic Pathology (Ukraine)
Kyiv
Institute of Blood Pathology and Transfusion Medicine
Lviv
Bulgaria1 site
University Multiprofile Hospital for Active Treatment "Sveti Georgi"
Plovdiv
Georgia1 site
Hematology of Department Hemophilia and Thromboses center
Tbilisi
About this study
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
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).
Read the 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
- Being tested
- Coagulation FVIIa (Recombinant)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Coagulation FVIIa (Recombinant)Biological
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
| Study group | Role | What they receive |
|---|---|---|
| Coagulation Factor VIIa (Recombinant): 75 µg/kg | Active comparator | 75 µg/kg treatment regimen for 3 months Receives: Biological: Coagulation FVIIa (Recombinant) |
| Coagulation Factor VIIa (Recombinant): 225 µg/kg | Active comparator | 225 µg/kg treatment regimen for 3 months Receives: Biological: Coagulation FVIIa (Recombinant) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 12 hours after first administration of study drug
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.
Measured: 12 hours after first administration of study drug
Secondary outcomes (4)
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.
Measured: 12 hour after first administration of study drug
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.
Measured: Within 24 hours of Bleeding Episode
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.
Measured: Within 24 hours of 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.
Measured: Within 24 hours of Bleeding Episode
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2015 (estimated)
- Study start7 Dec 2015
- Primary completion30 Jun 2017
- Study completion30 Aug 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hemophilia A With InhibitorsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- LFB USA, Inc.
- Responsible party
- Sponsor
- Investigators
- Michael Wang, MD, Principal investigator, University of Colorado, Denver
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02448680
- Sponsor's protocol ID
- LFB-FVIIa-007-14
- Acronym
- PERSEPT2
- Status last verified
- Feb 2022
- First submitted
- 15 May 2015
- Last updated
- 25 Feb 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Young G, Mahlangu J, Boggio LN, Carcao M, Dargaud Y, Escobar M, Giermasz A, Hermans C, Kuriakose P, Miesbach W, Nance D, Rafique A, Sidonio RF Jr, Vilchevska KV, Wang M, Pipe SW. Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of the PERSEPT 1 and 2 trials. Blood Vessel Thromb Hemost. 2025 Mar 27;2(3):100069. doi: 10.1016/j.bvth.2025.100069. eCollection 2025 Aug.
PubMed 40765904Carcao M, Hermans C, Giermasz A, Kessler C, Miesbach W, Quon D, Windyga J, Mahlangu J. Safety and Use of Eptacog Beta 225 microg/kg in Patients With Haemophilia A or B With Inhibitors. Haemophilia. 2025 Sep;31(5):957-965. doi: 10.1111/hae.70083. Epub 2025 Jul 17.
PubMed 40674256
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02448680
- Acronym / sponsor ID
- PERSEPT2 / LFB-FVIIa-007-14
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Laboratoire français de Fractionnement et de BiotechnologiesIndustry (pharma, biotech, devices)
- Collaborators
- LFB USA, Inc.
- Study start
- 7 Dec 2015 (actual)
- Primary completion
- 30 Jun 2017 (actual)
- Study completion
- 30 Aug 2017 (actual)
- First posted
- 19 May 2015 (estimated)
- Results
- Posted 28 Aug 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Coagulation Factor VIIa (Recombinant): 75 µg/kg | Active comparator | Biological: Coagulation FVIIa (Recombinant)75 µg/kg treatment regimen for 3 months |
| Coagulation Factor VIIa (Recombinant): 225 µg/kg | Active comparator | Biological: Coagulation FVIIa (Recombinant)225 µg/kg treatment regimen for 3 months |
Who may qualify
- Ages
- Up to 11 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 | Biological | Coagulation Factor VIIa (Recombinant): 225 µg/kg, Coagulation Factor VIIa (Recombinant): 75 µg/kg |
Primary outcome
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.
4 secondary outcomes
Patient-Reported "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes
Time frame: 12 hour after first administration of study drug
Time to Patient Assessment of a "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes
Time frame: Within 24 hours of Bleeding Episode
Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode
Time frame: Within 24 hours of Bleeding Episode
Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode
Time frame: Within 24 hours of Bleeding Episode
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40765904Young G, Mahlangu J, Boggio LN, Carcao M, Dargaud Y, Escobar M, Giermasz A, Hermans C, Kuriakose P, Miesbach W, Nance D, Rafique A, Sidonio RF Jr, Vilchevska KV, Wang M, Pipe SW. Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of the PERSEPT 1 and 2 trials. Blood Vessel Thromb Hemost. 2025 Mar 27;2(3):100069. doi: 10.1016/j.bvth.2025.100069. eCollection 2025 Aug.
- Derived from this study: PubMed 40674256Carcao M, Hermans C, Giermasz A, Kessler C, Miesbach W, Quon D, Windyga J, Mahlangu J. Safety and Use of Eptacog Beta 225 microg/kg in Patients With Haemophilia A or B With Inhibitors. Haemophilia. 2025 Sep;31(5):957-965. doi: 10.1111/hae.70083. Epub 2025 Jul 17.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael Wang, MD principal investigator
University of Colorado, Denver
9 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Coagulation FVIIa (Recombinant) in people with Hemophilia A With Inhibitors and Hemophilia B With Inhibitors, run by Laboratoire français de Fractionnement et de Biotechnologies at 9 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Ukraine, Bulgaria, Czechia, Georgia and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Aug 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement. (time frame: 12 hours after first administration of study drug). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02448680, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Feb 2022.