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Hemophilia BPhase 3NCT03855280

Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B

A Phase 3 study testing APVO101 in people with Hemophilia B, run by Medexus Pharma, Inc. at 11 sites in 6 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Phase 3/4, single arm, open-label study to evaluate PK, safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age. Read more about this study

Who is it for?
Ages Up to 11 years
Conditions the study is for
Hemophilia B
What is being tested?
APVO101
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
11 sitesin 6 countries
Study dates
16 Jan 2020to 4 Jul 2022
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
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Age: < 11.5 years of age at the time of the first dose and < 12 years throughout the Treatment Phase of the study (for at least 50 ED).
  • Informed consent: subject's parent or legal guardian written Institutional Review Board (IRB)/Ethics Committee (EC)-approved informed consent. An assent form (IRB/EC-approved) will be obtained, when required by local regulations/guidelines.
  • Willingness and ability to make the required study visits, and follow instructions while enrolled in the study (for at least 50 ED; approximately 6 months).
  • Documented severe or moderately severe hemophilia B diagnosis (factor IX activity ≤ 2 IU/dL); in addition, severity may be indicated by the occurrence of one or more joint bleeding episode(s) at any point in the child's medical history requiring infusion(s) to replace factor IX.
  • Subjects must be on prophylaxis or switch to a prophylaxis regimen for the duration of the study.
  • Previously treated patients with a minimum of 50 ED (as documented and determined by the investigator) to a preparation/blood components containing factor IX.
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  • Willingness to adhere to the 4-day washout period of any factor IX replacement therapy prior to PK evaluation. In case of previous exposure to a factor IX product with a prolonged half-life, a washout period of 3 half-lives is required in order to achieve steady state factor IX level prior to exposure to APVO101.
  • Immunocompetent (CD4 count > 400/mm3) and not receiving immune modulating or chemotherapeutic agents.
  • Platelet count at least 150,000/mm3.
  • Liver function: alanine transaminase (ALT) and aspartate transaminase (AST) ≤ 2 times the upper limit of the normal range.
  • Total bilirubin ≤ 1.5 times the upper limit of the normal range.
  • Renal function: serum creatinine ≤ 1.25 times the upper limit of the normal range.
  • Hemoglobin ≥ 7 g/dL.

Who cannot join

  • History of factor IX inhibitor ≥ 0.6 Bethesda Units (BU); confirmed by the screening result.
  • Existence of another coagulation disorder.
  • Evidence of thrombotic disease, fibrinolysis, or disseminated intravascular coagulation (DIC).
  • Use of an investigational drug within 30 days prior to study entry.
  • Previous use of APVO101.
  • Use of medications that could impact hemostasis, such as aspirin.
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  • Known hypersensitivity to the active substance or to any of the excipients in the investigational products.
  • Known allergic reaction to hamster proteins.
  • History of poor compliance, geographic isolation, unreliable transportation, a serious medical or social condition, or any other circumstance that, in the opinion of the investigator, would interfere with participation or compliance with the study protocol.
  • History of adverse reaction to either plasma-derived factor IX or recombinant factor IX that interfered with the subject's ability to treat bleeding episodes with a factor IX product.
  • History of any medical condition that would impact the efficacy evaluation and/or safety evaluation of the study product.

Trial locations and contacts

11 sites in 6 countries.

11 sites in 6 countries

Brazil3 sites
  • Universidade Estadual de Campinas - Centro de Hematologia e Hemoterapia

    Campinas, 13083-878

  • Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo

    Ribeirão Preto, 14051-140

  • Centro Estadual de Hemopterapia e Hematologia do Espirito Santo

    Vitória, Espírito Santo, 29040-090

Other trials in Brazil

South Africa2 sites
  • Worthwhile Clinical Trials, Lakeview Hospital

    Benoni, Gauteng, 1500

  • Haemophilia Comprehensive Care Centre

    Johannesburg, 2193

Other trials in South Africa

Turkey (Türkiye)2 sites
  • Cukurova University School of Medicine

    Adana, 01330

  • Ege University School ofMedicine

    Izmir, 35100

Other trials in Turkey (Türkiye)

Ukraine2 sites
  • National Specialized Children's Hospital OKHMATDYT

    Kyiv, 01135

  • State Institute: Institute of Blood Pathology and Transfusion Medicine of the National Academy of Medical Sciences of Ukraine

    Lviv, 79044

Other trials in Ukraine

Georgia1 site
  • JSC K Eristavi National Center for Experimental and Clinical Surgery

    Tbilisi, 0159

Other trials in Georgia

Moldova1 site
  • PMSI Institute of Mother and Child

    Chisinau, MD-2062

Other trials in Moldova

About this study

Phase 3/4, single arm, open-label study to evaluate PK, safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age.

Read the detailed description

Study APVO101-903 is a Phase 3/4, single arm, open-label clinical trial. The purpose of the study is to evaluate pharmacokinetics (PK), safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age. The study is designed to gather information in two age groups of previously treated (with a minimum of 50 previous ED to factor IX replacement therapy) pediatric patients, specifically those < 6 years of age and 6 to <12 years of age.

Study APVO101-903 consists of three distinct phases:

  • PK Phase - PK evaluation will consist of administration of a single 75 ± 5 IU/kg dose, followed by factor IX activity and safety assessments up to 50 hours post-infusion.
  • Treatment Phase - subjects will receive APVO101 prophylaxis (starting prophylaxis dose to be determined based on APVO101 recovery; ideally within the recommended dose range: 35 - 75 IU/kg; twice weekly) for 50 ED (approximately 6 months).
  • Continuation Phase - subjects may continue to receive APVO101 prophylaxis (recommended dose range: 35 - 75 IU/kg; twice weekly) for an additional ≥ 50 ED.
Conditions
Hemophilia B
Being tested
APVO101
Medical subject headings
Hemophilia B

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • APVO101Drug

    Also called IB1001, Recombinant factor IX, IXINITY

    Subjects will receive a single IV dose of APVO101 twice weekly or at a frequency of infusions as determined appropriate by the investigator for the particular study subject for a total of 50 ED. The starting prophylaxis dose will be based on APVO101 recovery from PK Phase assessments (only pre-infusion and 15-30 minute post-infusion samples).

Study groups in NCT03855280
Study groupRoleWhat they receive
APVO101ExperimentalAPVO101: 35 - 75 IU/kg; twice weekly
Receives: Drug: APVO101

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Annualized Bleeding Rate (ABR)

    The primary efficacy variable was the ABR while on prophylaxis to prevent bleeding episodes. The ABR was defined as the number of bleeding episodes per year.

    Measured: Exposure Day 1 up to 50 exposure days (approximately 6 months)

  2. Annualized Bleeding Rate (ABR) Overall

    The primary efficacy variable was the ABR while on prophylaxis to prevent bleeding episodes. The ABR was defined as the number of bleeding episodes per year.

    Measured: Exposure Day 1 through study completion (up to 2.5 years)

Secondary outcomes (13)
  1. Concentration (Cmax)

    Maximum post-infusion plasma concentration of FIX activity was measured at the following time points post infusion: 15-30 minutes, 4-6 hours, 24-26 hours and 46-50 hours.

    Measured: Pre-infusion to 50 hours post-infusion

  2. Area Under the Curve (0-inf)

    Area under plasma concentration curve, FIX activity-time profile from time zero extrapolated to infinity. FIX activity was measured at the following time points post infusion: 15-30 minutes, 4-6 hours, 24-26 hours and 46-50 hours.

    Measured: Pre-infusion to 50 hours post-infusion

  3. Mean Residence Time (MRT)

    MRT is the average time the molecules of drug reside in the body before elimination.

    Measured: Pre-infusion to 50 hours post-infusion

  4. Terminal Half-Life (t 1/2)

    Terminal half-life is the length of time required for the concentration of drug to decrease by one half of its starting dose in the body. FIX activity was measured at the following time points post infusion: 15-30 minutes, 4-6 hours, 24-26 hours and 46-50 hours.

    Measured: Pre-infusion to 50 hours post-infusion

  5. Clearance (CL)

    Clearance is a measure of the volume of plasma from which FIX activity is removed per unit time. Weight normalized clearance calculated as CL=Dose/AUC 0-inf. FIX activity was measured at the following time points post infusion: 15-30 minutes, 4-6 hours, 24-26 hours and 46-50 hours.

    Measured: Pre-infusion to 50 hours post-infusion

  6. Volume of Distribution at Steady-State (Vdss)

    Volume of distribution is defined as the theoretical volume in which the total amount of FIX would need to be uniformly distributed to produce the observed plasma concentration of FIX. Steady state volume of distribution (Vdss) is the apparent volume of distribution at steady-state. FIX activity was measured at the following time points post infusion: 15-30 minutes, 4-6 hours, 24-26 hours and 46-50 hours.

    Measured: Pre-infusion to 50 hours post-infusion

  7. Incremental Recovery (IR)

    Incremental recovery was the increase in circulating FIX activity for every international unit (IU) of APVO101 administered per kilogram of body weight of participant. FIX activity was measured at the following time points post infusion: 15-30 minutes, 4-6 hours, 24-26 hours and 46-50 hours.

    Measured: Pre-infusion to 50 hours post-infusion

  8. Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Treatment Phase)

    Subjects rated APVO101 efficacy for each bleeding episode based on a four-point scale:

    1. Excellent: a dramatic response with abrupt pain relief and clear reduction in joint or hemorrhage site size
    2. Good: pain relief or reduction in hemorrhage site size that may have required an additional infusion for resolution;
    3. Fair: probable or slight beneficial response usually requiring one of more additional infusions for resolution;
    4. Poor: no improvement or condition worsens.

    Measured: Exposure Day 1 up to 50 exposure days (approximately 6 months)

  9. Subject Rating of APVO101 Efficacy - Evaluated at the Bleeding Episode Level (Overall)

    Subjects rated APVO101 efficacy for each bleeding episode based on a four-point scale:

    1. Excellent: a dramatic response with abrupt pain relief and clear reduction in joint or hemorrhage site size
    2. Good: pain relief or reduction in hemorrhage site size that may have required an additional infusion for resolution;
    3. Fair: probable or slight beneficial response usually requiring one of more additional infusions for resolution;
    4. Poor: no improvement or condition worsens.

    Measured: Exposure Day 1 through study completion (up to 2.5 years)

  10. Investigator Rating of APVO101 Prophylaxis Efficacy (Treatment Phase)

    The investigator will indicate the overall assessment of APVO101 prophylaxis efficacy, considering the absence of bleeding episodes, site, severity and types of bleeding episodes treated, and other factors that might influence the therapeutic response.

    The investigator's efficacy assessment categories for prophylaxis will include: 'effective', 'partially effective' and 'not effective'.

    Measured: Exposure Day 1 up to 50 exposure days (approximately 6 months)

  11. Investigator Rating of APVO101 Prophylaxis Efficacy (Overall)

    The investigator will indicate the overall assessment of APVO101 prophylaxis efficacy, considering the absence of bleeding episodes, site, severity and types of bleeding episodes treated, and other factors that might influence the therapeutic response.

    The investigator's efficacy assessment categories for prophylaxis will include: 'effective', 'partially effective' and 'not effective'.

    Measured: Exposure Day 1 through study completion (up to 2.5 years)

  12. Investigator Rating of APVO101 Efficacy for Control and Management of Bleeding Episodes (Treatment Phase)

    Of the bleeding episodes requiring treatment, the investigator considered the site, severity and type of the bleeding episode while evaluating efficacy for control and management of the bleeding episode.

    The investigator's efficacy assessment categories control of bleeding episodes included: 'effective', 'partially effective' and 'not effective'.

    Measured: Exposure Day 1 up to 50 exposure days (approximately 6 months)

  13. Investigator Rating of APVO101 Efficacy for Control and Management of Bleeding Episodes (Overall)

    Of the bleeding episodes requiring treatment, the investigator considered the site, severity and type of the bleeding episode while evaluating efficacy for control and management of the bleeding episode.

    The investigator's efficacy assessment categories control of bleeding episodes included: 'effective', 'partially effective' and 'not effective'.

    Measured: Exposure Day 1 through study completion (up to 2.5 years)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Feb 2019
  2. Study start16 Jan 2020
  3. Primary completion4 Jul 2022
  4. Study completion4 Jul 2022

Resources

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
Not applicable
Intervention model
Single group
Model description
Phase 3/4, single arm, open-label study with three defined phases: * PK Phase: Initial PK evaluation - single dose of APVO101 * Treatment Phase: APVO101 prophylaxis treatment for 50 ED * Continuation Phase: After completion of the Treatment Phase, subjects may continue APVO101 prophylaxis treatment (for an additional ≥ 50 ED)
Masking
None (open label)
Enrolment
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03855280
Sponsor's protocol ID
APVO101-903
Status last verified
Apr 2024
First submitted
25 Feb 2019
Last updated
16 May 2024
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing APVO101 in people with Hemophilia B, run by Medexus Pharma, Inc. at 11 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 13 inclusion and 11 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 16 May 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites in Brazil, South Africa, Turkey (Türkiye), Ukraine, 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 16 May 2024). 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?

Annualized Bleeding Rate (ABR) (time frame: Exposure Day 1 up to 50 exposure days (approximately 6 months)). 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?
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 16 May 2024.

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