Phase
Condition
Hemophilia
Treatment
SerpinPC
Clinical Study ID
Ages 12-65 Male
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
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
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
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
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
Participants who are currently in a prophylaxis program must be willing to stopprophylaxis (including episodic prophylaxis for sporting events) before the firstdose of SerpinPC
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
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
No bleeding in the 7 days before baseline (the prospective observation period can beextended 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 wherethere is a resolving bleed, the exclusion threshold is ≤1750 milligrams(mg)/L atScreening and Pre-dosing visits
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
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
Adequate renal function, defined as a serum creatinine level of ≤2.0*ULN atScreening 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 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:
Known severe thrombophilia (defined as antithrombin deficiency and/or protein Sdeficiency and or protein C deficiency).
Participant with previous factor VIII or factor IX inhibitor who responded to immunetolerance 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 (mmHg); diastolic blood pressure >100 mm Hg)
Weight >150 kg OR body mass index >40 Kilograms(kg)/meter square (m2)
Has active cancer and/or requires therapy for cancer, except for basal cellcarcinoma
Participation in another clinical trial (except for AP-0105) during the 30 daysbefore 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 theparticipant to be unsuitable for the study or could interfere with theinterpretation of the study results
History of or other evidence of recent alcohol or drug abuse as determined by theinvestigator (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 areeligible
Current or planned treatment with anticoagulant or antiplatelet drugs
Is planning to donate/bank sperm during SerpinPC treatment AND within 30 days oflast dose of SerpinPC
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
Connect with a study center
Centre of Haematology named after prof. R. O. Yeolian
Yerevan, 14
ArmeniaActive - Recruiting
Yeolyan Hematology and Oncology Center, MoH of Armenia CJSC
Yerevan, 14
ArmeniaSite Not Available
Royal Prince Alfred Hospital
Camperdown, New South Wales 2050
AustraliaSite Not Available
The Alfred Hospital
Melbourne, Victoria 3004
AustraliaSite Not Available
Cliniques Universitaires Saint-Luc
Brussels, 1200
BelgiumSite Not Available
Queen Fabiola Children
Brussels, 1020
BelgiumSite Not Available
Hamilton Health Sciences Corporation
Hamilton, L8S 4K1
CanadaSite Not Available
Unity Health Toronto
Toronto, M5B 1W8
CanadaSite Not Available
Hopital Necker - Enfants Malades
Paris, IDF 75015
FranceSite Not Available
Hospices Civils de Lyon (HCL) - Hopital Femme-Mere-Enfant (HFME)
Lyon, Rhone 69500
FranceSite Not Available
Hôpital Bicêtre
Le Kremlin-Bicêtre, 94270
FranceSite Not Available
CHU Hotel Dieu
Nantes, 44000
FranceSite Not Available
University Hospital Frankfurt
Frankfurt, Hesse 60590
GermanySite Not Available
Universitätsklinikum Carl Gustav Carus Dresden
Dresden, Sachsen 1307
GermanySite Not Available
Klinik fur Angiologie Hamostaseologie Haus 12 A Gerinnungssprechstunde
Berlin, 10249
GermanySite Not Available
K J Somaiya Super Speciality Hospital & Research Centre
Mumbai, Maharashtra 400022
IndiaSite Not Available
Christian Medical College & Hospital
Ludhiāna, Punjab 141008
IndiaSite Not Available
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milano, MI 20122
ItalySite Not Available
Presidio Ospedaliero Universitario S. Maria della Misericordia - ASUFC
Udine, UD 33100
ItalySite Not Available
Azienda Ospedaliero-Universitaria Careggi
Florence, 50134
ItalySite Not Available
A.O.U Citt della Salute e della Scienza di Torino
Torino, 10126
ItalySite Not Available
Azienda Ospedaliera Universitaria Integrata Verona
Verona, 37126
ItalySite Not Available
Korczowski Bartosz, Gabinet Lekarski
Rzeszów, Podkarpackie 35-302
PolandSite Not Available
Kl Hemat Now Krwi i Trans USK
Wrocław, Woj. Dolnośląskie 50-367
PolandSite Not Available
Phoenix Pharma Pty Ltd
Port Elizabeth, Eastern Cape 6001
South AfricaSite Not Available
Hospital Universitari Vall d'Hebron
Barcelona, 8035
SpainSite Not Available
Hospital Universitario La Paz
Madrid,
SpainSite Not Available
Hospital Clínico Universitario Virgen de la Arrixaca
Murcia, 30120
SpainSite Not Available
Hospital Regional Universitario de Malaga Hospital Carlos Haya - Hospital Materno-Infantil
Málaga, 29010
SpainSite Not Available
Hospital Universitario Miguel Servet
Zaragoza, 50009
SpainSite Not Available
China Medical University Hospital
Taichung, 40447
TaiwanSite Not Available
Taichung Veterans General Hospital
Taichung, 40705
TaiwanSite Not Available
Taipei Veterans General Hospital
Taipei, 11217
TaiwanSite Not Available
National Taiwan University Hospital
Taipei City, 10004
TaiwanSite Not Available
Trakya University Haematology Clinic
Edirne, 22030
TurkeySite Not Available
Istanbul University Oncology Institute
Istanbul, 34093
TurkeySite Not Available
Ondokuz Mayis University Medical Faculty
Samsun, 55200
TurkeySite Not Available
Ege University Hospital Internal Disease
İzmir, 35100
TurkeySite Not Available
Ege University Medical Faculty Pediatric Hospital
İzmir, 35100
TurkeySite Not Available
Kocaeli Universitesi Tip Fakultesi
İzmir, 1000
TurkeySite Not Available
Kent Canterbury Hospital
Canterbury, Kent CT1 3NG
United KingdomSite Not Available
Oxford University Hospital
Oxford, Oxfordshire OX3 9DU
United KingdomSite Not Available
Royal Victoria Infirmary
Newcastle Upon Tyne, Tyne and Wear NE1 4LP
United KingdomSite Not Available
University Hospitals Birmingham NHS Foundation Trust
Birmingham, B15 2GW
United KingdomSite Not Available
University Hospital of Wales
Cardiff, CF14 4XW
United KingdomSite Not Available
Glasgow Royal Infirmary
Glasgow, G4 0SF
United KingdomSite Not Available
Barts and London School of Medicine and Dentistry
London, E1 2AT
United KingdomSite Not Available
Imperial College Healthcare NHS Trust
London, W2 1NY
United KingdomSite Not Available
Royal Free London NHS Foundation Trust
London, NW3 2QG
United KingdomSite Not Available
Southampton General Hospital
Southampton, S016 6YD
United KingdomSite Not Available
University of Colorado School of Medicine
Aurora, Colorado 80045-7202
United StatesSite Not Available
University of South Florida
Tampa, Florida 33612
United StatesSite Not Available
Indiana Hemophilia and Thrombosis Center, Inc.
Indianapolis, Indiana 46260
United StatesSite Not Available
University of Iowa Healthcare
Iowa City, Iowa 52246
United StatesSite Not Available
University of Michigan
Ann Arbor, Michigan 48109
United StatesSite Not Available
University of Minnesota Medical Center
Minneapolis, Minnesota 55455
United StatesSite Not Available
East Carolina Univeristy
Greenville, North Carolina 27834
United StatesSite Not Available
Penn State Hershey Medical Center
Hershey, Pennsylvania 17033
United StatesSite Not Available
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania 15213
United StatesSite Not Available
University of Texas Health Science Center at Houston-Gulf States HTC
Houston, Texas 77030
United StatesSite Not Available
Not the study for you?
Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.