ENSAYO CLÍNICO

Reclutando

Fase II

Coagulopatías hemorrágicas, Leucemia mieloide aguda, --> Otros, Terapia celular y T-CARs

Agosto, 2023

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Male subjects 12 and 65 years of age at the time of informed consent
Adequate hematologic function, defined as a platelet count of 100,000/L (100 × 109/L) and hemoglobin level of 10 g/dL (100 g/L or 6.206 mmol/L) at Screening and Pre-dosing visits
Adequate hepatic function, defined as a total bilirubin level of 1.5 × upper level 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 (caregiver assistance allowed for adolescents)
Sexually active subjects 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
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 IU/mL [5%])
Subjects 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 requiring current treatment with bypass agents based on medical records or laboratory reports
Documented ABR of 6 in the 12 months before screening (subjects not on prophylaxis regimen) or documented ABR of 2 for subjects on prophylaxis regimen
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-0103
No bleeding in the 7 days before Baseline (the prospective observation period can be extended if there is an ongoing active bleed)
D-dimer of 750 g/L; in cases where there is a resolving bleed, the exclusion threshold is 1750 mg/L at Screening and Pre-dosing visits

Known severe thrombophilia (defined as antithrombin deficiency and/or protein S deficiency and/or protein C deficiency)
Any major medical, psychological, or psychiatric condition that could cause the subject 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 HIV infection with CD4 count (or T-cell count) of <200 cells/L 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 subject unsuitable for enrollment, or could interfere with participation in, or completion of the study
Patient with previous factor IX inhibitor who responded to immune tolerance induction and remains on prophylactic factor concentrate
Previous deep vein thrombosis (excluding line-associated thrombosis), pulmonary embolism, myocardial infarction, or stroke
History of intolerance to SC injections
Uncontrolled hypertension (systolic blood pressure >160 mm Hg; diastolic blood pressure >100 mm Hg)
Weight >150 kg OR body mass index >40 kg/m2
Has active cancer and/or requires therapy for cancer, except for basal cell carcinoma
Participation in another interventional clinical trial (except for AP-0105) during the 30 days before screening
Ongoing, or planned treatment with gene therapy for HemB

To evaluate the efficacy and safety of prophylactic SerpinPC administered subcutaneously in subjects with hemophilia B (HemB) with inhibitors

check-point inhibitor, complement inhibitor, hemophilia, ph + acute lymphoblastic leukemia