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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05789537




Registration number
NCT05789537
Ethics application status
Date submitted
16/03/2023
Date registered
29/03/2023

Titles & IDs
Public title
A Study of SerpinPC in Participants with Hemophilia B (HemB) with Inhibitors
Scientific title
A Global, Open-label Study to Investigate the Efficacy and Safety of SerpinPC in Subjects with Hemophilia B with Inhibitors
Secondary ID [1] 0 0
2022-502881-25-00
Secondary ID [2] 0 0
AP-0103
Universal Trial Number (UTN)
Trial acronym
PRESent-3
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia B With Inhibitor 0 0
Hemophilia B 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - SerpinPC

Experimental: SerpinPC - 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.


Treatment: Drugs: SerpinPC
Administered as SC injection.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized Bleeding Rate (ABR) for Treated Bleeds up to Week 24
Timepoint [1] 0 0
Up to Week 24
Secondary outcome [1] 0 0
Annualized Bleeding Rate (ABR) for Treated Bleeds Up to Week 48
Timepoint [1] 0 0
Up to Week 48
Secondary outcome [2] 0 0
Annualized Bleeding Rate (ABR) for Treated Spontaneous Bleeds
Timepoint [2] 0 0
Up to Week 48
Secondary outcome [3] 0 0
Annualized Bleeding Rate (ABR) for Treated Spontaneous Joint Bleeds
Timepoint [3] 0 0
Up to Week 48
Secondary outcome [4] 0 0
Total Coagulation Factor and/or Bypass Product Consumption During SerpinPC Treatment
Timepoint [4] 0 0
Up to Week 48
Secondary outcome [5] 0 0
Pharmacokinetic Concentrations of SerpinPC
Timepoint [5] 0 0
From Day 1(Pre-dose) up to Week 48(Post-dose)
Secondary outcome [6] 0 0
Haemophilia Quality-of-Life Questionnaire for Adults (Haem-A-QoL) Physical Health Scale
Timepoint [6] 0 0
From Baseline up to Week 48
Secondary outcome [7] 0 0
Number of participants with Adverse events (AEs)
Timepoint [7] 0 0
From Baseline up to Week 52
Secondary outcome [8] 0 0
Number of Participants with Persistent High-titer Antidrug Antibodies (ADAs)
Timepoint [8] 0 0
From Baseline up to Week 48
Secondary outcome [9] 0 0
Number of Participants with Severity of Injection-site Reactions
Timepoint [9] 0 0
Baseline up to Week 44

Eligibility
Key inclusion criteria
1. Male participants greater than or equal to (>=) 12 and less than or equal to (<=) 65 years of age at the time of informed consent.
2. 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.
3. Historically documented HemB (defined as factor IX <=0.05 international unit/Milliliter (IU/mL) [<=5 percent (%)]).
4. 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.
5. Historical or ongoing Factor IX inhibitor with bypass agents based on medical records or laboratory reports.
6. 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
7. 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.
8. 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).
9. 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.
10. 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.
11. 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.
12. Adequate renal function, defined as a serum creatinine level of <=2.0*ULN at Screening 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 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.
Minimum age
12 Years
Maximum age
65 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
1. Known severe thrombophilia (defined as antithrombin deficiency and/or protein S deficiency and/or protein C deficiency).
2. Participant with previous factor IX inhibitor who responded to immune tolerance 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 (mm Hg); diastolic blood pressure >100 mm Hg).
6. Weight >150 kilograms (kg) OR body mass index >40 kg/meter square (m^2).
7. Has active cancer and/or requires therapy for cancer, except for basal cell carcinoma.
8. Participation in another clinical trial (except for AP-0105 [NCT05605678]) during the 30 days before screening.
9. Prior, ongoing, or planned treatment with gene therapy for HemB
10. 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.
11. History of or other evidence of recent alcohol or drug abuse as determined by the Investigator (in the 12 months before screening).
12. 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.
13. Current or planned treatment with anticoagulant or antiplatelet drugs
14. Is planning to donate/bank sperm during SerpinPC treatment AND within 30 days of last dose of SerpinPC.
15. 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/07/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
28/10/2025
Actual
Sample size
Target
12
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
The Alfred Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
North Carolina
Country [4] 0 0
Armenia
State/province [4] 0 0
Yerevan
Country [5] 0 0
France
State/province [5] 0 0
Le Kremlin-Bicêtre
Country [6] 0 0
France
State/province [6] 0 0
Lyon
Country [7] 0 0
France
State/province [7] 0 0
Paris
Country [8] 0 0
Germany
State/province [8] 0 0
Frankfurt
Country [9] 0 0
India
State/province [9] 0 0
Maharashtra
Country [10] 0 0
Italy
State/province [10] 0 0
Milan
Country [11] 0 0
Spain
State/province [11] 0 0
Madrid
Country [12] 0 0
Taiwan
State/province [12] 0 0
Taichung city
Country [13] 0 0
Taiwan
State/province [13] 0 0
Taichung
Country [14] 0 0
Turkey
State/province [14] 0 0
Istanbul
Country [15] 0 0
Turkey
State/province [15] 0 0
Izmir
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Nottingham

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
ApcinteX Ltd
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Centessa Pharmaceuticals plc
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Centessa Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
617-468-5770
Fax 0 0
Email 0 0
presentprogram@centessa.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05789537