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Hemophilia aPhase 2PRESent-2NCT05789524

The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B

A Phase 2 study testing SerpinPC in people with Hemophilia a and Hemophilia B, run by ApcinteX Ltd at 59 sites in 15 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of prophylactic SerpinPC administered subcutaneously (SC) to participants with severe hemophilia A (HemA) (with or without inhibitors) or moderately severe to severe hemophilia B (HemB) (without inhibitors) as part of the… Read more about this study

Who is it for?
Ages 12 to 65 years, men only
Conditions the study is for
Hemophilia a, 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
60 participants
Where
59 sitesin 15 countries
Study dates
6 Jul 2023to 28 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 ≥12 and ≤65 years of age at the time of informed consent. Enrollment of adolescents (aged ≥12 to <18 years) will be deferred until at least 12 adult participants from each SerpinPC treatment regimen have completed at least 12 weeks of dosing in Part 1 and safety of SerpinPC has been assessed
  • 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 severe HemA (defined as factor VIII less than (<) 0.01 international unit (IU)/milliliter(mL) [<1%]), with or without inhibitors, or moderately severe to severe HemB (defined as factor IX ≤0.02 IU/mL [≤2%]), without inhibitors high titer inhibitor (high titer inhibitor defined as ≥5
  • Participant is currently included in a prophylaxis program. Fulfillment of this criterion will be based on investigator's judgment of adequate prophylaxis regimen OR participant is undergoing an on-demand treatment regimen and must have had greater than or equal to (≥) 6 documented acute bleeding episodes (spontaneous or traumatic) that required treatment during the 6 months before screening. Irrespective of the treatment program that the participant is currently undergoing, they must be willing to remain in the same program for the duration of the prospective observational period
  • 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
  • For Part 1: At least 12 weeks of prospective documentation of bleeding episodes in the AP-0105 non-interventional study before SerpinPC dosing, or willing to complete a 12-week observational period (at minimum) in AP-0102
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  • For Part 2: At least 24 weeks of prospective documentation of bleeding episodes in the AP-0105 non-interventional study before SerpinPC dosing or willing to complete a 24-week observational period (at minimum) in AP-0102
  • 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 less than or equal to (≤) 750 micrograms(μg)/Liter(L). In cases where there is a resolving bleed, the exclusion threshold is ≤1750 milligrams(mg)/L at Screening and Pre-dosing visits
  • Adequate hematologic function, defined as a platelet count of ≥100,000/microliters(μL) (≥100 × 109/L) and hemoglobin level of ≥10 grams(g)/deciliter(dL) (≥100 g/L or ≥6.206 millimols(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 measures 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 VIII or 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 kg OR body mass index >40 Kilograms(kg)/meter square (m2)
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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) during the 30 days before Screening
  • Use of emicizumab in the 24 weeks before Baseline (Day 0)
  • Prior, ongoing, or planned treatment with gene therapy for hemophilia
  • 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/μL within 24 weeks before Screening and Pre-dosing visits. Participants 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

59 sites in 15 countries.

59 sites in 15 countries

United States10 sites

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • University of Colorado School of Medicine

    Aurora, Colorado, 80045-7202

  • East Carolina Univeristy

    Greenville, North Carolina, 27834

  • Penn State Hershey Medical Center

    Hershey, Pennsylvania, 17033

  • University of Texas Health Science Center at Houston-Gulf States HTC

    Houston, Texas, 77030

  • Indiana Hemophilia and Thrombosis Center, Inc.

    Indianapolis, Indiana, 46260

  • University of Iowa Healthcare

    Iowa City, Iowa, 52246

  • University of Minnesota Medical Center

    Minneapolis, Minnesota, 55455

  • Hemophilia Center of Western Pennsylvania

    Pittsburgh, Pennsylvania, 15213

  • University of South Florida

    Tampa, Florida, 33612

Other trials in United States

United Kingdom10 sites

  • University Hospitals Birmingham NHS Foundation Trust

    Birmingham, Birmingham, B15 2GW

  • Kent Canterbury Hospital

    Canterbury, Kent, CT1 3NG

  • University Hospital of Wales

    Cardiff, Cardiff, CF14 4XW

  • Glasgow Royal Infirmary

    Glasgow, Glasgow, G4 0SF

  • Barts and London School of Medicine and Dentistry

    London, London, E1 2AT

  • Royal Free London NHS Foundation Trust

    London, London, NW3 2QG

  • Imperial College Healthcare NHS Trust

    London, London, W2 1NY

  • Royal Victoria Infirmary

    Newcastle upon Tyne, Tyne and Wear, NE1 4LP

  • Oxford University Hospital

    Oxford, Oxfordshire, OX3 9DU

  • Southampton General Hospital

    Southampton, Southampton, S016 6YD

Other trials in United Kingdom

Turkey (Türkiye)6 sites

  • Trakya University Haematology Clinic

    Edirne, 22030

  • Istanbul University Oncology Institute

    Istanbul, 34093

  • Ege University Medical Faculty Pediatric Hospital

    Izmir, İzmir, 35100

  • Kocaeli Universitesi Tip Fakultesi

    Izmir, 1000

  • Ege University Hospital Internal Disease

    Izmir, 35100

  • Ondokuz Mayis University Medical Faculty

    Samsun, 55200

Other trials in Turkey (Türkiye)

Italy5 sites

  • Azienda Ospedaliero-Universitaria Careggi

    Florence, 50134

  • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

    Milan, MI, 20122

  • A.O.U Citt della Salute e della Scienza di Torino

    Torino, Torino, 10126

  • Presidio Ospedaliero Universitario S. Maria della Misericordia - ASUFC

    Udine, UD, 33100

  • Azienda Ospedaliera Universitaria Integrata Verona

    Verona, Verona, 37126

Other trials in Italy

Spain5 sites

  • Hospital Universitari Vall d'Hebron

    Barcelona, Spain, 8035

  • Hospital Universitario La Paz

    Madrid

  • Hospital Regional Universitario de Malaga Hospital Carlos Haya - Hospital Materno-Infantil

    Málaga, 29010

  • Hospital Clínico Universitario Virgen de la Arrixaca

    Murcia, Murcia, 30120

  • Hospital Universitario Miguel Servet

    Zaragoza, Zaragoza, 50009

Other trials in Spain

Taiwan4 sites

  • China Medical University Hospital

    Taichung, 40447

  • Taichung Veterans General Hospital

    Taichung, 40705

  • Taipei Veterans General Hospital

    Taipei, Taipei, 11217

  • National Taiwan University Hospital

    Taipei, 10004

Other trials in Taiwan

Canada3 sites

  • Hamilton Health Sciences Corporation

    Hamilton, Hamilton, L8S 4K1

  • Hamilton Health Sciences Corporation

    Hamilton, Ontario, L8S4K1

  • Unity Health Toronto

    Toronto, M5B 1W8

Other trials in Canada

France3 sites

  • Hôpital Bicêtre

    Le Kremlin-Bicêtre, France, 94270

  • Hospices Civils de Lyon (HCL) - Hopital Femme-Mere-Enfant (HFME)

    Lyon, Rhone, 69500

  • Hopital Necker - Enfants Malades

    Paris, IDF, 75015

Other trials in France

Germany3 sites

  • Klinik fur Angiologie Hamostaseologie Haus 12 A Gerinnungssprechstunde

    Berlin, State of Berlin, 10249

  • Universitätsklinikum Carl Gustav Carus Dresden

    Dresden, Saxony, 1307

  • University Hospital Frankfurt

    Frankfurt am Main, Hesse, 60590

Other trials in Germany

Australia2 sites

  • Royal Prince Alfred Hospital

    Camperdown, New South Wales, 2050

  • The Alfred Hospital

    Melbourne, Victoria, 3004

Other trials in Australia

Belgium2 sites

  • Queen Fabiola Children

    Brussels, Brussels Capital, 1020

  • Cliniques Universitaires Saint-Luc

    Brussels, Brussels Capital, 1200

Other trials in Belgium

India2 sites

  • Christian Medical College & Hospital

    Ludhiana, Punjab, 141008

  • K J Somaiya Super Speciality Hospital & Research Centre

    Mumbai, Maharashtra, 400022

Other trials in India

Poland2 sites

  • Korczowski Bartosz, Gabinet Lekarski

    Rzeszów, Podkarpackie Voivodeship, 35-302

  • Kl Hemat Now Krwi i Trans USK

    Wroclaw, Woj. Dolnośląskie, 50-367

Other trials in Poland

Armenia1 site

  • Yeolyan Hematology and Oncology Center, MoH of Armenia CJSC

    Yerevan, Yerevan, 14

Other trials in Armenia

South Africa1 site

  • Phoenix Pharma Pty Ltd

    Port Elizabeth, Eastern Cape, 6001

Other trials in South Africa

About this study

Official title: A Global, Open-label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC in Subjects with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B

The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of prophylactic SerpinPC administered subcutaneously (SC) to participants with severe hemophilia A (HemA) (with or without inhibitors) or moderately severe to severe hemophilia B (HemB) (without inhibitors) as part of the SerpinPC registrational program.

This study consists of 3 parts: Part 1: dose-justification phase, Part 2: dose-confirmatory phase, Part 3: extension phase for participants who complete either Part 1 or Part 2.

This adaptive design study has a randomized dose-justification component to investigate the efficacy and safety of SerpinPC as a therapeutic option, principally for participants with HemB without inhibitors. SerpinPC has a novel mechanism of action compared with marketed treatments and those that are in development.

Being tested
SerpinPC
Keywords
SerpinPC, Hemophilia
Medical subject headings
Hemophilia A, Hemophilia B

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 groups in NCT05789524
Study groupRoleWhat they receive
Part 1 - Cohort 1: SerpinPCExperimentalParticipants will receive SerpinPC 1.2 mg/kg SC Injection QW for 24 weeks after a minimum of 12 weeks of a prospective observation period.
Receives: Drug: SerpinPC
Part 1 - Cohort 2: SerpinPCExperimentalParticipants will receive SerpinPC 1.2 mg/kg SC Injection Q2W for 24 weeks after a minimum of 12 weeks of a prospective observation period.
Receives: Drug: SerpinPC
Part 1 - Cohort 3: SerpinPCExperimentalParticipants will receive SerpinPC 1.2 mg/kg SC Injection Q4W for 24 weeks after a minimum of 12 weeks of a prospective observation period.
Receives: Drug: SerpinPC
Part 2 - SerpinPC (Dose-confirmatory phase)ExperimentalAfter a minimum of 24 weeks of prospective observation, participants will receive SerpinPC at dose of 1.2 mg/kg Q2W for 24 weeks in Part 2, unless the Interim Analysis (IA) shows a greater benefit-risk profile with either the 1.2 mg/kg QW or Q4W treatment regimens.
Receives: Drug: SerpinPC
Part 3 - SerpinPC (Extension phase)ExperimentalAfter completion of dosing in Part 1 or Part 2, participants will continue treatment with SerpinPC at the dose of SerpinPC selected for Part 2 in a 24-week extension phase (Part 3).
Receives: Drug: SerpinPC

What is being measured

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

  1. Annualized Bleeding Rate (ABR) for Treated Bleeds up to Week 24

    Measured: Up to Week 24

Secondary outcomes (7)

  1. Annualized Bleeding Rate (ABR) for Treated Bleeds Up to Week 48

    Measured: Up to Week 48

  2. Annualized Bleeding Rate (ABR) for Treated Spontaneous Bleeds

    Measured: Up to Week 48

  3. Annualized Bleeding Rate (ABR) for Treated Spontaneous Joint Bleeds

    Measured: Up to Week 48

  4. Total Coagulation Factor and/or Bypass Product Consumption During Parts 2 and 3

    Measured: Up to Week 48

  5. Pharmacokinetic Plasma Concentrations of SerpinPC

    Measured: From Day 1 up to 24 weeks

  6. Haemophilia-specific QoL Instrument for Adults (Haem-A-QoL) Physical Health scale in participants aged 17 to ≤65 years with hemophilia

    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 24 weeks

  7. Number of Participants With Adverse Events (AEs)

    Measured: From Baseline up to Week 48

Timeline

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

  1. First posted29 Mar 2023
  2. Study start6 Jul 2023
  3. Primary completion14 Nov 2024
  4. Study completion28 Feb 2025

Why it stopped: Study terminated due to business and strategic decision

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
60 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05789524
Sponsor's protocol ID
AP-0102
Other IDs
2022-502880-39-00
Acronym
PRESent-2
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

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing SerpinPC in people with Hemophilia a and Hemophilia B, run by ApcinteX Ltd at 59 sites in 15 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 14 inclusion and 16 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 59 sites in United States, United Kingdom, Turkey (Türkiye), Italy, Spain and 10 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?

On the ClinicalTrials.gov record for NCT05789524.

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.

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