Hemophilia B with InhibitorPhase 2PRESent-3NCT05789537
A Study of SerpinPC in Participants with Hemophilia B (HemB) with Inhibitors
A Phase 2 study testing SerpinPC in people with Hemophilia B with Inhibitor and Hemophilia B, run by ApcinteX Ltd at 20 sites in 11 countries.
Trial summary
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with Hemophilia B with inhibitors, as part of the SerpinPC registrational program. Read more about this study
- Who is it for?
- Ages 12 to 65 years, men only
- Conditions the study is for
- Hemophilia B with Inhibitor, Hemophilia B
- What is being tested?
- SerpinPC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 20 sitesin 11 countries
- Study dates
- 28 Jul 2023to 24 Feb 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 12 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male participants greater than or equal to (>=) 12 and less than or equal to (<=) 65 years of age at the time of informed consent.
- Capable of providing written informed consent (adolescent assent and parental/guardian/legal representative consent when appropriate) for participation and having the opportunity to discuss the study with the Investigator or delegate.
- Historically documented HemB (defined as factor IX <=0.05 international unit/Milliliter (IU/mL) [<=5 percent (%)]).
- Participants who are currently in a prophylaxis program must be willing to stop prophylaxis (including episodic prophylaxis for sporting events) before the first dose of SerpinPC.
- Historical or ongoing Factor IX inhibitor with bypass agents based on medical records or laboratory reports.
- Documented ABR of 6 in the 12 months before screening (participants not on prophylaxis regimen) or documented ABR of ≥2 for participants on prophylaxis regimen
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- At least 12 weeks of prospective documentation of bleeding episodes in the AP-0105 (NCT05605678) non-interventional study before SerpinPC dosing, or willing to complete a 12-week observational period (at minimum) in AP-0103.
- No bleeding in the 7 days before Baseline (the prospective observation period can be extended by 10 days if there is an ongoing active bleed).
- D-dimer of <=750 micrograms/Liter (mc/L); in cases where there is a resolving bleed, the exclusion threshold is <=1750 mg/L at Screening and Pre-dosing visits.
- Adequate hematologic function, defined as a platelet count of >=100,000/microliters (mcL) (>=100*10^9/L) and hemoglobin level of >=10 grams/deciliter(g/dL) (>=100 g/L or >= 6.206 millimoles per liter (mmol/L) at Screening and Pre-dosing visits.
- Adequate hepatic function, defined as a total bilirubin level of <=1.5*upper limit of normal (ULN) (excluding Gilbert syndrome) and aspartate aminotransferase and/or alanine aminotransferase of <=3*ULN at Screening and Pre-dosing visits; no clinical signs or known laboratory or radiographic evidence consistent with cirrhosis of the liver.
- Adequate renal function, defined as a serum creatinine level of <=2.0*ULN at Screening and Pre-dosing visits.
- Able to use a diary to document bleeding events and medication usage.
- Sexually active participants with a partner who could become pregnant should agree to use effective contraception for the duration of the study.
- Effective contraceptive measure include condom with or without spermicide, a combination of male condom with either cap, diaphragm, or sponge with spermicide (double barrier methods), vasectomy, partner using stable contraceptive measures (combined [ estrogen and progestogen-containing] hormonal contraception or progestogen-only hormonal contraception initiated 2 or more menstrual cycles prior to screening, intrauterine device [IUD]. Intrauterine hormone-releasing system [IUS], bilateral tubal ligation), and/or sexual abstinence.
Who cannot join
- Known severe thrombophilia (defined as antithrombin deficiency and/or protein S deficiency and/or protein C deficiency).
- Participant with previous factor IX inhibitor who responded to immune tolerance induction and remains on prophylactic factor concentrate.
- Previous deep vein thrombosis, pulmonary embolism, myocardial infarction, or stroke.
- History of intolerance to SC injections.
- Uncontrolled hypertension (systolic blood pressure >160 millimeter of mercury (mm Hg); diastolic blood pressure >100 mm Hg).
- Weight >150 kilograms (kg) OR body mass index >40 kg/meter square (m^2).
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- Has active cancer and/or requires therapy for cancer, except for basal cell carcinoma.
- Participation in another clinical trial (except for AP-0105 [NCT05605678]) during the 30 days before screening.
- Prior, ongoing, or planned treatment with gene therapy for HemB
- Any major medical, psychological, or psychiatric condition that could cause the participant to be unsuitable for the study or could interfere with the interpretation of the study results.
- History of or other evidence of recent alcohol or drug abuse as determined by the Investigator (in the 12 months before screening).
- Known human immunodeficiency virus (HIV) infection with CD4 count (or T-cell count) of <200 cells/mcL within 24 weeks before Screening and Pre-dosing visits. Patients with HIV infection who have CD4 > 200 and meet all other criteria are eligible.
- Current or planned treatment with anticoagulant or antiplatelet drugs
- Is planning to donate/bank sperm during SerpinPC treatment AND within 30 days of last dose of SerpinPC.
- Any other significant conditions or comorbidities that, in the opinion of the Investigator, would make the participant unsuitable for enrollment, or could interfere with participation in, or completion of the study.
Trial locations and contacts
20 sites in 11 countries.
20 sites in 11 countries
France4 sites
Hopital Bicetre
Le Kremlin-Bicêtre, 94270
CHRU de Lille Centre de biologie et pathologie
Lille, 59000
Hospices Civils de Lyon (HCL) - Hopital Femme-Mere-Enfant (HFME)
Lyon, 69500
Hopital Necker - Enfants Malades
Paris, 75015
United States3 sites
University of Colorado School of Medicine
Aurora, Colorado, 80045-7202
East Carolina University
Greenville, North Carolina, 27834
University of South Florida
Tampa, Florida, 33612
Taiwan3 sites
Chung Shan Medical University
Taichung, 40201
China Medical University Hospital
Taichung, 40447
Taichung Veterans General Hospital
Taichung, 407
Australia2 sites
Royal Prince Alfred Hospital
Camperdown, New South Wales, 2050
The Alfred Hospital
Melbourne, Victoria, 3004
Turkey (Türkiye)2 sites
Istanbul University Oncology Institute
Istanbul, 34093
Ege University Medical Faculty Hospital
Izmir, 35100
Armenia1 site
Centre of Haematology named after prof. R. O. Yeolian/ Hemophilia center
Yerevan, 0014
India1 site
K J Somaiya Super Speciality Hospital & Research Centre
Mumbai, Maharashtra, 400022
Italy1 site
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, 20122
United Kingdom1 site
Nottingham University Hospitals NHS Trust
Nottingham, NG7 2UH
About this study
Official title: A Global, Open-label Study to Investigate the Efficacy and Safety of SerpinPC in Subjects with Hemophilia B with Inhibitors
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with Hemophilia B with inhibitors, as part of the SerpinPC registrational program.
- Conditions
- Hemophilia B with Inhibitor, Hemophilia B
- Being tested
- SerpinPC
- Keywords
- SerpinPC, Hemophilia
- Medical subject headings
- Hemophilia B, Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SerpinPCDrug
Also called Activated Protein C (APC) inhibitor
Administered as SC injection.
| Study group | Role | What they receive |
|---|---|---|
| SerpinPC | Experimental | Participants will receive SerpinPC 1.2 milligrams/kilogram (mg/kg) subcutaneous (SC) injection every 2 weeks (Q2W) for 48 weeks after a prospective observation of 12 weeks for all participants, either in a prior non-interventional study (AP-0105[NCT05605678]) or as part of the ongoing study observational period. Receives: Drug: SerpinPC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Bleeding Rate (ABR) for Treated Bleeds up to Week 24
Measured: Up to Week 24
Secondary outcomes (9)
Annualized Bleeding Rate (ABR) for Treated Bleeds Up to Week 48
Measured: Up to Week 48
Annualized Bleeding Rate (ABR) for Treated Spontaneous Bleeds
Measured: Up to Week 48
Annualized Bleeding Rate (ABR) for Treated Spontaneous Joint Bleeds
Measured: Up to Week 48
Total Coagulation Factor and/or Bypass Product Consumption During SerpinPC Treatment
Measured: Up to Week 48
Pharmacokinetic Concentrations of SerpinPC
Measured: From Day 1(Pre-dose) up to Week 48(Post-dose)
Haemophilia Quality-of-Life Questionnaire for Adults (Haem-A-QoL) Physical Health Scale
The Haem-A-QoL instrument contains 44 items across 10 domains relevant to HRQoL in adults (physical health, feelings, view of participant's self, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, partnership, and sexuality). Each item is rated on a 5-point scale (1=never, 2=rarely, 3=sometimes, 4=often, 5=all the time). Higher scores are indicative of greater impairment in HRQoL.
Measured: From Baseline up to Week 48
Number of participants with Adverse events (AEs)
Measured: From Baseline up to Week 52
Number of Participants with Persistent High-titer Antidrug Antibodies (ADAs)
Measured: From Baseline up to Week 48
Number of Participants with Severity of Injection-site Reactions
Measured: Baseline up to Week 44
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2023
- Study start28 Jul 2023
- Primary completion14 Nov 2024
- Study completion24 Feb 2025
Why it stopped: Study terminated due to business and strategic decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia B with InhibitorStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- ApcinteX Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Centessa Pharmaceuticals plc
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05789537
- Sponsor's protocol ID
- AP-0103
- Other IDs
- 2022-502881-25-00
- Acronym
- PRESent-3
- Status last verified
- Mar 2025
- First submitted
- 16 Mar 2023
- Last updated
- 11 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Global, Open-label Study to Investigate the Efficacy and Safety of SerpinPC in Subjects with Hemophilia B with Inhibitors
- ClinicalTrials.gov ID
- NCT05789537
- Acronym / sponsor ID
- PRESent-3 / AP-0103
- Conditions
- Hemophilia B with Inhibitor, Hemophilia B
- Keywords
- SerpinPC, Hemophilia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study terminated due to business and strategic decision
- Enrolment
- 3 participants (actual)
- Lead sponsor
- ApcinteX LtdIndustry (pharma, biotech, devices)
- Collaborators
- Centessa Pharmaceuticals plc
- Study start
- 28 Jul 2023 (actual)
- Primary completion
- 14 Nov 2024 (actual)
- Study completion
- 24 Feb 2025 (actual)
- First posted
- 29 Mar 2023 (actual)
- Results
- Not posted in the source record
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 evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with Hemophilia B with inhibitors, as part of the SerpinPC registrational program.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SerpinPC | Experimental | Drug: SerpinPCParticipants will receive SerpinPC 1.2 milligrams/kilogram (mg/kg) subcutaneous (SC) injection every 2 weeks (Q2W) for 48 weeks after a prospective observation of 12 weeks for all participants, either in a prior non-interventional study (AP-0105[NCT05605678]) or as part of the ongoing study observational period. |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (15 listed)
- Male participants greater than or equal to (>=) 12 and less than or equal to (<=) 65 years of age at the time of informed consent.
- Capable of providing written informed consent (adolescent assent and parental/guardian/legal representative consent when appropriate) for participation and having the opportunity to discuss the study with the Investigator or delegate.
- Historically documented HemB (defined as factor IX <=0.05 international unit/Milliliter (IU/mL) [<=5 percent (%)]).
- Participants who are currently in a prophylaxis program must be willing to stop prophylaxis (including episodic prophylaxis for sporting events) before the first dose of SerpinPC.
- Historical or ongoing Factor IX inhibitor with bypass agents based on medical records or laboratory reports.
Key exclusion criteria (15 listed)
- Known severe thrombophilia (defined as antithrombin deficiency and/or protein S deficiency and/or protein C deficiency).
- Participant with previous factor IX inhibitor who responded to immune tolerance induction and remains on prophylactic factor concentrate.
- Previous deep vein thrombosis, pulmonary embolism, myocardial infarction, or stroke.
- History of intolerance to SC injections.
- Uncontrolled hypertension (systolic blood pressure >160 millimeter of mercury (mm Hg); diastolic blood pressure >100 mm Hg).
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 |
|---|---|---|
| SerpinPCAlso: Activated Protein C (APC) inhibitorAdministered as SC injection. | Drug | SerpinPC |
Primary outcome
Annualized Bleeding Rate (ABR) for Treated Bleeds up to Week 24
Time frame: Up to Week 24
9 secondary outcomes
Annualized Bleeding Rate (ABR) for Treated Bleeds Up to Week 48
Time frame: Up to Week 48
Annualized Bleeding Rate (ABR) for Treated Spontaneous Bleeds
Time frame: Up to Week 48
Annualized Bleeding Rate (ABR) for Treated Spontaneous Joint Bleeds
Time frame: Up to Week 48
Total Coagulation Factor and/or Bypass Product Consumption During SerpinPC Treatment
Time frame: Up to Week 48
Pharmacokinetic Concentrations of SerpinPC
Time frame: From Day 1(Pre-dose) up to Week 48(Post-dose)
Haemophilia Quality-of-Life Questionnaire for Adults (Haem-A-QoL) Physical Health Scale
Time frame: From Baseline up to Week 48
Number of participants with Adverse events (AEs)
Time frame: From Baseline up to Week 52
Number of Participants with Persistent High-titer Antidrug Antibodies (ADAs)
Time frame: From Baseline up to Week 48
Number of Participants with Severity of Injection-site Reactions
Time frame: Baseline up to Week 44
Results and safety
The study was stopped early (reason given: Study terminated due to business and strategic decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Mar 2025.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
20 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SerpinPC in people with Hemophilia B with Inhibitor and Hemophilia B, run by ApcinteX Ltd at 20 sites in 11 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 15 inclusion and 15 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: stopped early, as of the sponsor's last update on 11 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in France, United States, Taiwan, Australia, Turkey (Türkiye) and 6 more countries. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Study terminated due to business and strategic decision). Results are not available in the source record.
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) for Treated Bleeds up to Week 24 (time frame: Up to Week 24). 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 11 Mar 2025.