15 Participants Needed

SerpinPC for Hemophilia

(PRESent-6 Trial)

Recruiting at 36 trial locations
CP
Overseen ByCentessa Pharmaceuticals
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Do I have to stop taking my current medications?

The trial requires that you stop taking anticoagulant or antiplatelet drugs.

What data supports the idea that SerpinPC for Hemophilia is an effective treatment?

The available research shows that targeting natural serpins, which are proteins that help control bleeding, can reduce bleeding in people with hemophilia. This suggests that SerpinPC, which works by blocking these proteins, could be an effective treatment. While the research doesn't directly compare SerpinPC to other treatments, it highlights the potential of using serpins to balance blood clotting in hemophilia, offering a promising alternative to traditional therapies that require frequent injections.12345

What safety data is available for SerpinPC in treating hemophilia?

The provided research does not contain specific safety data for SerpinPC in treating hemophilia. The titles and abstracts focus on other treatments and general safety monitoring practices, but do not mention SerpinPC or its safety data.678910

Is the drug SerpinPC a promising treatment for hemophilia?

Yes, SerpinPC is a promising drug for hemophilia because it targets natural proteins in the body to help reduce bleeding. It works by blocking certain proteins that prevent blood from clotting, which can help restore balance and improve blood clotting in people with hemophilia.35111213

What is the purpose of this trial?

The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with hemophilia

Eligibility Criteria

This trial is for males aged 12-65 with Hemophilia who have already completed a SerpinPC clinical trial and were compliant. They must be able to give informed consent, or if underage, obtain parental/guardian consent.

Inclusion Criteria

I am a male between 12 and 65 years old.
Completed participation in a sponsored SerpinPC hemophilia clinical trial and demonstrated compliance with the study requirements, including diary entries
Capable of providing written informed consent, with adolescent assent and parent/guardian consent when appropriate

Exclusion Criteria

I have had a blood clot, heart attack, or stroke.
Participation in any interventional clinical trial other than SerpinPC trials
Presence of significant conditions or comorbidities that, in the investigator's opinion, would make the subject unsuitable for enrolment or could interfere with study participation
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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive SerpinPC 1.2 mg/kg subcutaneous injection every 2 weeks

Long-term
Bi-weekly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive SerpinPC to observe long-term safety and efficacy

Long-term

Treatment Details

Interventions

  • SerpinPC
Trial Overview The study aims to assess the long-term effects of SerpinPC on people with Hemophilia. It looks at how well it works (efficacy), its safety, how the body tolerates it (tolerability), and how the body processes it (pharmacokinetics).
Participant Groups
1Treatment groups
Experimental Treatment
Group I: SerpinPCExperimental Treatment1 Intervention
Participants will receive SerpinPC 1.2 milligrams/kilograms (mg/kg) subcutaneous injection every 2 weeks (Q2W) once they have completed treatment in a SerpinPC parent study.

Find a Clinic Near You

Who Is Running the Clinical Trial?

ApcinteX Ltd

Lead Sponsor

Trials
5
Recruited
230+

Centessa Pharmaceuticals plc

Industry Sponsor

Trials
10
Recruited
370+

Findings from Research

After two years of prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc), participants with severe hemophilia B reported a significant reduction in chronic pain and the number of activities affected by pain, indicating improved quality of life.
The study showed a 25% reduction in the number of affected activities of daily living (ADLs) after switching to rFIXFc, highlighting its positive impact on daily functioning despite the median health-related quality of life score remaining stable.
Recombinant factor IX-Fc fusion protein in severe hemophilia B: Patient-reported outcomes and health-related quality of life.O'Donovan, M., Quinn, E., Johnston, K., et al.[2023]
Switching from standard half-life factor IX (SHL FIX) treatment to recombinant factor IX Fc fusion protein (rFIXFc) prophylaxis significantly reduced annualized bleeding rates (ABRs) from an average of 4.7 to 1.7, indicating improved clinical outcomes for patients with hemophilia B.
Patients who switched to rFIXFc experienced a reduction in injection frequency and factor consumption, with a mean decrease of 1.0 injections per week and a 27.7 IU/kg/week reduction in factor usage, suggesting a lower treatment burden and potential improvement in quality of life.
Real-World Effectiveness of rFIXFc Prophylaxis in Patients with Haemophilia B Switched from Standard Half-Life Therapy in Three European Countries.Funding, E., Lowe, G., Poulsen, LH., et al.[2023]
The recombinant factor IX Fc fusion protein (rFIXFc) significantly reduces the frequency of injections needed for hemophilia B treatment, with a prolonged half-life of 82.1 hours, allowing for administration every 1 to 2 weeks.
In a study of 123 male patients, rFIXFc resulted in low annualized bleeding rates (3.0 for weekly prophylaxis and 1.4 for interval-adjusted prophylaxis), with 90.4% of bleeding episodes resolving after just one injection, and no inhibitors detected in participants.
Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B.Powell, JS., Pasi, KJ., Ragni, MV., et al.[2022]

References

Recombinant factor IX-Fc fusion protein in severe hemophilia B: Patient-reported outcomes and health-related quality of life. [2023]
Real-World Effectiveness of rFIXFc Prophylaxis in Patients with Haemophilia B Switched from Standard Half-Life Therapy in Three European Countries. [2023]
Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. [2022]
Low-dose Immune Tolerance Induction in Hemophilia: A Single-Center Experience. [2020]
Serpins, New Therapeutic Targets for Hemophilia. [2021]
Monitoring product safety in the postmarketing environment. [2021]
Canadian Adverse Events Following Immunization Surveillance System (CAEFISS): Annual report for vaccines administered in 2012. [2023]
Safety and efficacy of nonacog alfa for the treatment of haemophilia B in children younger than 6 years of age in a routine clinical care setting: the EUREKIX registry study. [2021]
Vaccinovigilance in Europe--need for timeliness, standardization and resources. [2015]
[Evaluation of safety of haemophilus influenza type b(Hib) conjugate vaccine in postmarketing based on the immunization information management system]. [2018]
11.United Statespubmed.ncbi.nlm.nih.gov
Design and characterization of an APC-specific serpin for the treatment of hemophilia. [2021]
Treatment of inherited protein C deficiency by replacement therapy with the French purified plasma-derived protein C concentrate (PROTEXEL). [2007]
Hemostatic assessment of combined anticoagulant therapy using warfarin and prothrombin complex concentrates in a case of severe protein C deficiency. [2020]
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