SerpinPC for Severe Hemophilia (PRESent-2 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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline up to 24 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new injection called SerpinPC to prevent bleeding in people with severe hemophilia A or B. It aims to see if SerpinPC is safe and effective, especially for those who don't respond well to other treatments. SerpinPC works by helping the blood clot better. Serpins are being explored as potential treatments for hemophilia by rebalancing coagulation.

Who is the study for?

This trial is for males aged 12-65 with severe Hemophilia A or moderately severe to severe Hemophilia B, without inhibitors. Participants must have a history of bleeding episodes and be on or willing to commit to a prophylaxis program. Adolescents can join after safety assessments in adults.

What is being tested?

The study tests SerpinPC's effectiveness and safety when given subcutaneously as a preventive treatment for hemophilia. It has three phases: dose justification, confirmation, and an extension phase for ongoing evaluation.

What are the potential side effects?

While specific side effects are not listed here, participants will be monitored for any adverse reactions related to SerpinPC administration which could include typical drug-related responses such as injection site reactions, allergies, or other systemic effects.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline up to 24 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline up to 24 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline up to 24 weeks 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-specific QoL Instrument for Adults (Haem-A-QoL) Physical Health scale in participants aged 17 to ≤65 years with hemophilia

Awards & Highlights

No Placebo-Only Group

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

Trial Design

5Treatment groups

Experimental Treatment

Group I: Part 3 - SerpinPC (Extension phase)Experimental Treatment1 Intervention

After completion of dosing in Part 1 or Part 2, participants will continue treatment with SerpinPC at the dose of SerpinPC selected for Part 2 in a 24-week extension phase (Part 3).

Group II: Part 2 - SerpinPC (Dose-confirmatory phase)Experimental Treatment1 Intervention

After a minimum of 24 weeks of prospective observation, participants will receive SerpinPC at dose of 1.2 mg/kg Q2W for 24 weeks in Part 2, unless the Interim Analysis (IA) shows a greater benefit-risk profile with either the 1.2 mg/kg QW or Q4W treatment regimens.

Group III: Part 1 - Cohort 3: SerpinPCExperimental Treatment1 Intervention

Participants will receive SerpinPC 1.2 mg/kg SC Injection Q4W for 24 weeks after a minimum of 12 weeks of a prospective observation period.

Group IV: Part 1 - Cohort 2: SerpinPCExperimental Treatment1 Intervention

Participants will receive SerpinPC 1.2 mg/kg SC Injection Q2W for 24 weeks after a minimum of 12 weeks of a prospective observation period.

Group V: Part 1 - Cohort 1: SerpinPCExperimental Treatment1 Intervention

Participants will receive SerpinPC 1.2 mg/kg SC Injection QW for 24 weeks after a minimum of 12 weeks of a prospective observation period.

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