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

Last updated: March 7, 2025
Sponsor: ApcinteX Ltd
Overall Status: Terminated

Phase

2

Condition

Hemophilia

Treatment

SerpinPC

Clinical Study ID

NCT05789524
AP-0102
2022-502880-39-00
  • Ages 12-65
  • Male

Study 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 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. 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 12adult participants from each SerpinPC treatment regimen have completed at least 12weeks of dosing in Part 1 and safety of SerpinPC has been assessed

  2. Capable of providing written informed consent (adolescent assent andparental/guardian/legal representative consent when appropriate) for participationand having the opportunity to discuss the study with the investigator or delegate

  3. Historically documented severe HemA (defined as factor VIII less than (<) 0.01international unit (IU)/milliliter(mL) [<1%]), with or without inhibitors, ormoderately severe to severe HemB (defined as factor IX ≤0.02 IU/mL [≤2%]), withoutinhibitors high titer inhibitor (high titer inhibitor defined as ≥5

  4. Participant is currently included in a prophylaxis program. Fulfillment of thiscriterion will be based on investigator's judgment of adequate prophylaxis regimenOR participant is undergoing an on-demand treatment regimen and must have hadgreater than or equal to (≥) 6 documented acute bleeding episodes (spontaneous ortraumatic) 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 theprospective observational period

  5. Participants who are currently in a prophylaxis program must be willing to stopprophylaxis (including episodic prophylaxis for sporting events) before the firstdose of SerpinPC

  6. For Part 1: At least 12 weeks of prospective documentation of bleeding episodes inthe AP-0105 non-interventional study before SerpinPC dosing, or willing to completea 12-week observational period (at minimum) in AP-0102

  7. For Part 2: At least 24 weeks of prospective documentation of bleeding episodes inthe AP-0105 non-interventional study before SerpinPC dosing or willing to complete a 24-week observational period (at minimum) in AP-0102

  8. No bleeding in the 7 days before baseline (the prospective observation period can beextended by 10 days if there is an ongoing active bleed)

  9. D-dimer of less than or equal to (≤) 750 micrograms(μg)/Liter(L). In cases wherethere is a resolving bleed, the exclusion threshold is ≤1750 milligrams(mg)/L atScreening and Pre-dosing visits

  10. Adequate hematologic function, defined as a platelet count of ≥100,000/microliters(μL) (≥100 × 109/L) and hemoglobin level of ≥10grams(g)/deciliter(dL) (≥100 g/L or ≥6.206 millimols(mmol)/L) at Screening andPre-dosing visits

  11. Adequate hepatic function, defined as a total bilirubin level of ≤1.5*upper limit ofnormal (ULN) (excluding Gilbert syndrome) and aspartate aminotransferase and/oralanine aminotransferase of ≤3 × ULN at Screening and Pre-dosing visits; no clinicalsigns or known laboratory or radiographic evidence consistent with cirrhosis of theliver

  12. Adequate renal function, defined as a serum creatinine level of ≤2.0*ULN atScreening and Pre-dosing visits

  13. Able to use a diary to document bleeding events and medication usage

  14. Sexually active participants with a partner who could become pregnant should agreeto use effective contraception for the duration of the study effective contraceptivemeasures include condom with or without spermicide, a combination of male condomwith either cap, diaphragm, or sponge with spermicide (double barrier methods),vasectomy, partner using stable contraceptive measures (combined [estrogen andprogestogen-containing] hormonal contraception or progestogen-only hormonalcontraception initiated 2 or more menstrual cycles prior to screening, intrauterinedevice [IUD], intrauterine hormone-releasing system [IUS], bilateral tuballigation), and/or sexual abstinence.

Exclusion

Exclusion Criteria:

  1. Known severe thrombophilia (defined as antithrombin deficiency and/or protein Sdeficiency and or protein C deficiency).

  2. Participant with previous factor VIII or factor IX inhibitor who responded to immunetolerance induction and remains on prophylactic factor concentrate

  3. Previous deep vein thrombosis, pulmonary embolism, myocardial infarction, or stroke

  4. History of intolerance to SC injections

  5. Uncontrolled hypertension (systolic blood pressure >160 millimeter of mercury (mmHg); diastolic blood pressure >100 mm Hg)

  6. Weight >150 kg OR body mass index >40 Kilograms(kg)/meter square (m2)

  7. Has active cancer and/or requires therapy for cancer, except for basal cellcarcinoma

  8. Participation in another clinical trial (except for AP-0105) during the 30 daysbefore Screening

  9. Use of emicizumab in the 24 weeks before Baseline (Day 0)

  10. Prior, ongoing, or planned treatment with gene therapy for hemophilia

  11. Any major medical, psychological, or psychiatric condition that could cause theparticipant to be unsuitable for the study or could interfere with theinterpretation of the study results

  12. History of or other evidence of recent alcohol or drug abuse as determined by theinvestigator (in the 12 months before Screening)

  13. 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 areeligible

  14. Current or planned treatment with anticoagulant or antiplatelet drugs

  15. Is planning to donate/bank sperm during SerpinPC treatment AND within 30 days oflast dose of SerpinPC

  16. Any other significant conditions or comorbidities that, in the opinion of theinvestigator, would make the participant unsuitable for enrollment or couldinterfere with participation in or completion of the study

Study Design

Total Participants: 60
Treatment Group(s): 1
Primary Treatment: SerpinPC
Phase: 2
Study Start date:
July 06, 2023
Estimated Completion Date:
February 28, 2025

Connect with a study center

  • Centre of Haematology named after prof. R. O. Yeolian

    Yerevan, 14
    Armenia

    Active - Recruiting

  • Yeolyan Hematology and Oncology Center, MoH of Armenia CJSC

    Yerevan, 14
    Armenia

    Site Not Available

  • Royal Prince Alfred Hospital

    Camperdown, New South Wales 2050
    Australia

    Site Not Available

  • The Alfred Hospital

    Melbourne, Victoria 3004
    Australia

    Site Not Available

  • Cliniques Universitaires Saint-Luc

    Brussels, 1200
    Belgium

    Site Not Available

  • Queen Fabiola Children

    Brussels, 1020
    Belgium

    Site Not Available

  • Hamilton Health Sciences Corporation

    Hamilton, L8S 4K1
    Canada

    Site Not Available

  • Unity Health Toronto

    Toronto, M5B 1W8
    Canada

    Site Not Available

  • Hopital Necker - Enfants Malades

    Paris, IDF 75015
    France

    Site Not Available

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

    Lyon, Rhone 69500
    France

    Site Not Available

  • Hôpital Bicêtre

    Le Kremlin-Bicêtre, 94270
    France

    Site Not Available

  • CHU Hotel Dieu

    Nantes, 44000
    France

    Site Not Available

  • University Hospital Frankfurt

    Frankfurt, Hesse 60590
    Germany

    Site Not Available

  • Universitätsklinikum Carl Gustav Carus Dresden

    Dresden, Sachsen 1307
    Germany

    Site Not Available

  • Klinik fur Angiologie Hamostaseologie Haus 12 A Gerinnungssprechstunde

    Berlin, 10249
    Germany

    Site Not Available

  • K J Somaiya Super Speciality Hospital & Research Centre

    Mumbai, Maharashtra 400022
    India

    Site Not Available

  • Christian Medical College & Hospital

    Ludhiāna, Punjab 141008
    India

    Site Not Available

  • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

    Milano, MI 20122
    Italy

    Site Not Available

  • Presidio Ospedaliero Universitario S. Maria della Misericordia - ASUFC

    Udine, UD 33100
    Italy

    Site Not Available

  • Azienda Ospedaliero-Universitaria Careggi

    Florence, 50134
    Italy

    Site Not Available

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

    Torino, 10126
    Italy

    Site Not Available

  • Azienda Ospedaliera Universitaria Integrata Verona

    Verona, 37126
    Italy

    Site Not Available

  • Korczowski Bartosz, Gabinet Lekarski

    Rzeszów, Podkarpackie 35-302
    Poland

    Site Not Available

  • Kl Hemat Now Krwi i Trans USK

    Wrocław, Woj. Dolnośląskie 50-367
    Poland

    Site Not Available

  • Phoenix Pharma Pty Ltd

    Port Elizabeth, Eastern Cape 6001
    South Africa

    Site Not Available

  • Hospital Universitari Vall d'Hebron

    Barcelona, 8035
    Spain

    Site Not Available

  • Hospital Universitario La Paz

    Madrid,
    Spain

    Site Not Available

  • Hospital Clínico Universitario Virgen de la Arrixaca

    Murcia, 30120
    Spain

    Site Not Available

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

    Málaga, 29010
    Spain

    Site Not Available

  • Hospital Universitario Miguel Servet

    Zaragoza, 50009
    Spain

    Site Not Available

  • China Medical University Hospital

    Taichung, 40447
    Taiwan

    Site Not Available

  • Taichung Veterans General Hospital

    Taichung, 40705
    Taiwan

    Site Not Available

  • Taipei Veterans General Hospital

    Taipei, 11217
    Taiwan

    Site Not Available

  • National Taiwan University Hospital

    Taipei City, 10004
    Taiwan

    Site Not Available

  • Trakya University Haematology Clinic

    Edirne, 22030
    Turkey

    Site Not Available

  • Istanbul University Oncology Institute

    Istanbul, 34093
    Turkey

    Site Not Available

  • Ondokuz Mayis University Medical Faculty

    Samsun, 55200
    Turkey

    Site Not Available

  • Ege University Hospital Internal Disease

    İzmir, 35100
    Turkey

    Site Not Available

  • Ege University Medical Faculty Pediatric Hospital

    İzmir, 35100
    Turkey

    Site Not Available

  • Kocaeli Universitesi Tip Fakultesi

    İzmir, 1000
    Turkey

    Site Not Available

  • Kent Canterbury Hospital

    Canterbury, Kent CT1 3NG
    United Kingdom

    Site Not Available

  • Oxford University Hospital

    Oxford, Oxfordshire OX3 9DU
    United Kingdom

    Site Not Available

  • Royal Victoria Infirmary

    Newcastle Upon Tyne, Tyne and Wear NE1 4LP
    United Kingdom

    Site Not Available

  • University Hospitals Birmingham NHS Foundation Trust

    Birmingham, B15 2GW
    United Kingdom

    Site Not Available

  • University Hospital of Wales

    Cardiff, CF14 4XW
    United Kingdom

    Site Not Available

  • Glasgow Royal Infirmary

    Glasgow, G4 0SF
    United Kingdom

    Site Not Available

  • Barts and London School of Medicine and Dentistry

    London, E1 2AT
    United Kingdom

    Site Not Available

  • Imperial College Healthcare NHS Trust

    London, W2 1NY
    United Kingdom

    Site Not Available

  • Royal Free London NHS Foundation Trust

    London, NW3 2QG
    United Kingdom

    Site Not Available

  • Southampton General Hospital

    Southampton, S016 6YD
    United Kingdom

    Site Not Available

  • University of Colorado School of Medicine

    Aurora, Colorado 80045-7202
    United States

    Site Not Available

  • University of South Florida

    Tampa, Florida 33612
    United States

    Site Not Available

  • Indiana Hemophilia and Thrombosis Center, Inc.

    Indianapolis, Indiana 46260
    United States

    Site Not Available

  • University of Iowa Healthcare

    Iowa City, Iowa 52246
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • University of Minnesota Medical Center

    Minneapolis, Minnesota 55455
    United States

    Site Not Available

  • East Carolina Univeristy

    Greenville, North Carolina 27834
    United States

    Site Not Available

  • Penn State Hershey Medical Center

    Hershey, Pennsylvania 17033
    United States

    Site Not Available

  • Hemophilia Center of Western Pennsylvania

    Pittsburgh, Pennsylvania 15213
    United States

    Site Not Available

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

    Houston, Texas 77030
    United States

    Site Not Available

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