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Completed

NCT Number: NCT02448680

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

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).

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Key information

Conditions

Hemophilia A With Inhibitors Hemophilia B With Inhibitors

Age range

Up to 11 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

University Multiprofile Hospital for Active Treatment "Sveti Georgi", Plovdiv, Bulgaria

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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

Primary outcomes

  1. 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

  2. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

Other outcomes

  1. 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.

Sponsors and collaborators

Lead sponsor

Laboratoire français de Fractionnement et de Biotechnologies

Industry

Collaborators

  • LFB USA, Inc.

Registry information

Official study 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

Acronym: PERSEPT2

Important dates

Study start
2015
Primary completion
2017
Study completion
2017
First posted
May 19, 2015
Registry last updated
Feb 25, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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