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Coagulation Factor Replacement Therapy

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 day 1 up to 24 weeks

Awards & highlights

PRESent-2 Trial Summary

This trial will investigate the efficacy & safety of SerpinPC, a novel therapy, for people with HemA & HemB without inhibitors.

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.Check my eligibility

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.See study design

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.

PRESent-2 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from day 1 up to 24 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from day 1 up to 24 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and from day 1 up to 24 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Annualized Bleeding Rate (ABR) for Treated Bleeds up to Week 24

Secondary outcome measures

Annualized Bleeding Rate (ABR) for Treated Bleeds Up to Week 48

Annualized Bleeding Rate (ABR) for Treated Spontaneous Bleeds

Annualized Bleeding Rate (ABR) for Treated Spontaneous Joint Bleeds

+4 more

Side effects data

From 2010 Phase 3 trial • 216 Patients • NCT00708435

Constipation

Oedema peripheral

Hypokalaemia

Diarrhoea

Cardiac failure congestive

Pyrexia

Headache

Pulmonary oedema

Insomnia

Hypomagnesaemia

Nausea

Dyspnoea

Chest pain

Hyperkalaemia

Fluid overload

Urinary tract infection

Hypotension

Rash

Anxiety

Anaemia

Vomiting

Myocardial ischaemia

Encephalopathy

Subarachnoid haemorrhage

Device dislocation

Spinal fracture

Myocardial infarction

Hypoglycaemia

Agitation

Gastrointestinal haemorrhage

Tachycardia

Cardiac failure

Cardiac failure chronic

Cerebrovascular accident

Pneumonia

Bacteraemia

Cellulitis

Gastroenteritis

Infection

Septic shock

Small intestinal obstruction

Respiratory failure

Pleural effusion

Lung cancer metastatic

Metastases to liver

Haemorrhagic anaemia

Hepatic failure

Renal failure acute

Arthralgia

100%

80%

60%

40%

20%

Study treatment Arm

Fresh Frozen Plasma

Beriplex® P/N

PRESent-2 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.

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

ApcinteX LtdLead Sponsor

3 Previous Clinical Trials

251 Total Patients Enrolled

3 Trials studying Hemophilia A

251 Patients Enrolled for Hemophilia A

Centessa Pharmaceuticals plcIndustry Sponsor

8 Previous Clinical Trials

396 Total Patients Enrolled

3 Trials studying Hemophilia A

251 Patients Enrolled for Hemophilia A

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there still openings for volunteers in this research endeavor?

"According to the records posted on clinicaltrials.gov, recruitment for this trial is ongoing and began on July 6th 2023 with recent updates made on November 7th of the same year."

Answered by AI

Is eligibility for this trial limited to those above the age of 20?

"According to the inclusion criteria, this trial is open to adults aged 12-65. Additionally, there are 52 trials for children under 18 years of age and 73 clinical studies catered towards seniors above 65."

Answered by AI

What is the uppermost limit of individuals taking part in this medical research?

"Affirmative. Clinicaltrials.gov evidences that this investigation, initially posted on July 6th 2023, is currently seeking participants. The research has an ambition of recruiting 120 patients from 17 distinct locations."

Answered by AI

What are the risks associated with Part 1 - Cohort 1: SerpinPC?

"Our internal assessment of SerpinPC's safety was a 2 -- there is evidence that it can be ingested without major risk, but no empirical proof that the drug serves its intended purpose."

Answered by AI

Where are the various sites conducting this research project?

"This clinical trial is being conducted at multiple locations, including East Carolina University in Greenville, North carolina; Royal Prince Alfred Hospital in Camperdown, New South Wales; and The Alfred Hospital in Melbourne, Victoria. There are 17 other sites which have been approved to participate as well."

Answered by AI

Who may enlist in this trial according to the criteria established?

"This clinical trial is enrolling a total of 120 participants aged 12-65 who suffer from Hemophilia A. In order to be admissible, individuals must have had at least 24 weeks of prospective monitoring in the preceding non-interventional study (AP0105) or agree to participate in an observational period for the same length of time; must provide informed consent and discuss their participation with the investigator; should have historically documented severe HemA (factor VIII <1%) without inhibitors or moderately to severely low levels of factor IX (<2%) without high titer inhibitor (>5 BU); currently participating in either prophylaxis/epis"

Answered by AI

Recent research and studies

clinicaltrials.govStudy

The Efficacy and Safety of SerpinPC in Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

2023. "The Efficacy and Safety of SerpinPC in Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05789524.

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