SerpinPC for Hemophilia B (PRESent-3 Trial)

Phase 2

Recruiting

Research Sponsored by ApcinteX Ltd

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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.

Male participants greater than or equal to (>=) 12 and less than or equal to (<=) 65 years of age at the time of informed consent.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline up to week 48

Awards & highlights

No Placebo-Only Group

Summary

This trial studies a new medicine to help treat hemophilia B with inhibitors. It looks at safety, effectiveness, and how the body processes it.

Who is the study for?

This trial is for males aged 12-65 with Hemophilia B and inhibitors, who haven't bled in the week before starting. They must have a history of frequent bleeding episodes and be willing to stop current prophylaxis treatments. Participants need normal platelet counts and hemoglobin levels, plus documented bleeding history or agree to an observation period.

What is being tested?

The study tests SerpinPC's effectiveness, safety, tolerability, and how it moves through the body when used as a preventive treatment in those with Hemophilia B with inhibitors. It aims to become part of the standard care for these patients.

What are the potential side effects?

While specific side effects are not listed here, common ones associated with new hemophilia treatments may include reactions at injection sites, headaches, nausea, allergic reactions or development of antibodies that might reduce treatment efficacy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am willing to stop my current prophylaxis before starting SerpinPC.

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I am a male between 12 and 65 years old.

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My blood tests show enough platelets and hemoglobin.

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I have a history of Factor IX inhibitor with treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline up to week 48

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline up to week 48

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline up to week 48 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Haemophilia Quality-of-Life Questionnaire for Adults (Haem-A-QoL) Physical Health Scale

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: SerpinPCExperimental Treatment1 Intervention

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.

0 / 4Eligibility criteria met