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The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B | Clinical Trials | Clareo Health

TERMINATEDPHASE2

The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B

A Global, Open-label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC in Subjects with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B

NCT05789524•ApcinteX Ltd

View on ClinicalTrials.gov

Summary

The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of prophylactic SerpinPC administered subcutaneously (SC) to participants with severe hemophilia A (HemA) (with or without inhibitors) or moderately severe to severe hemophilia B (HemB) (without inhibitors) as part of the SerpinPC registrational program. This study consists of 3 parts: Part 1: dose-justification phase, Part 2: dose-confirmatory phase, Part 3: extension phase for participants who complete either Part 1 or Part 2. This adaptive design study has a randomized dose-justification component to investigate the efficacy and safety of SerpinPC as a therapeutic option, principally for participants with HemB without inhibitors. SerpinPC has a novel mechanism of action compared with marketed treatments and those that are in development.

Eligibility

Age

12 - 65 years

Sex

MALE

Healthy Volunteers

Inclusion Criteria

•1. Male participants ≥12 and ≤65 years of age at the time of informed consent. Enrollment of adolescents (aged ≥12 to \<18 years) will be deferred until at least 12 adult participants from each SerpinPC treatment regimen have completed at least 12 weeks of dosing in Part 1 and safety of SerpinPC has been assessed
•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 severe HemA (defined as factor VIII less than (\<) 0.01 international unit (IU)/milliliter(mL) \[\<1%\]), with or without inhibitors, or moderately severe to severe HemB (defined as factor IX ≤0.02 IU/mL \[≤2%\]), without inhibitors high titer inhibitor (high titer inhibitor defined as ≥5
•4. Participant is currently included in a prophylaxis program. Fulfillment of this criterion will be based on investigator's judgment of adequate prophylaxis regimen OR participant is undergoing an on-demand treatment regimen and must have had greater than or equal to (≥) 6 documented acute bleeding episodes (spontaneous or traumatic) that required treatment during the 6 months before screening. Irrespective of the treatment program that the participant is currently undergoing, they must be willing to remain in the same program for the duration of the prospective observational period
•5. 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
•6. For Part 1: 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-0102
•7. For Part 2: At least 24 weeks of prospective documentation of bleeding episodes in the AP-0105 non-interventional study before SerpinPC dosing or willing to complete a 24-week observational period (at minimum) in AP-0102
•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 less than or equal to (≤) 750 micrograms(μg)/Liter(L). In cases where there is a resolving bleed, the exclusion threshold is ≤1750 milligrams(mg)/L at Screening and Pre-dosing visits
•10. Adequate hematologic function, defined as a platelet count of ≥100,000/microliters(μL) (≥100 × 109/L) and hemoglobin level of ≥10 grams(g)/deciliter(dL) (≥100 g/L or ≥6.206 millimols(mmol)/L) at Screening and Pre-dosing visits
+4 more criteria

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 VIII or 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 kg OR body mass index \>40 Kilograms(kg)/meter square (m2)
•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) during the 30 days before Screening
•9. Use of emicizumab in the 24 weeks before Baseline (Day 0)
•10. Prior, ongoing, or planned treatment with gene therapy for hemophilia
+6 more criteria

Locations (59)

University of Colorado School of Medicine

Aurora, Colorado, United States

University of South Florida

Tampa, Florida, United States

Indiana Hemophilia and Thrombosis Center, Inc.

Indianapolis, Indiana, United States

University of Iowa Healthcare

Iowa City, Iowa, United States

University of Michigan

Ann Arbor, Michigan, United States

University of Minnesota Medical Center

Minneapolis, Minnesota, United States

East Carolina Univeristy

Greenville, North Carolina, United States

Penn State Hershey Medical Center

Hershey, Pennsylvania, United States

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, United States

University of Texas Health Science Center at Houston-Gulf States HTC

Houston, Texas, United States

Key Dates

Start Date

July 6, 2023

Primary Completion Date

November 14, 2024

Completion Date

February 28, 2025

Last Updated

March 11, 2025

Enrollment

ACTUAL participants

Conditions

Hemophilia aHemophilia B

Interventions

SerpinPC

DRUG

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

NCT06379789

Hemophilia B

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RECRUITINGPHASE1/PHASE2

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

NCT07226206

Hemophilia A

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RECRUITINGPHASE1/PHASE2

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 11, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B

Inclusion Criteria

Exclusion Criteria

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

National Longitudinal Cohort of Hematological Diseases

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B

Inclusion Criteria

Exclusion Criteria

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

National Longitudinal Cohort of Hematological Diseases

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to &lt; 18 Years of Age) With Severe or Moderately Severe Hemophilia B

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B