A Phase 1b Study to Assess the Safety, Tolerability, PK and PD of MG1113 in Hemophilia Patient

NCT05493631 | Completed Phase 1 DMC

• 1 other identifier: MG1113_HP_P0101
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to assess the safety and tolerability, pharmacokinetics and pharmacodynamics of subcutaneous MG1113 in the multiple ascending dose study in patients with severe hemophilia.

Conditions

Hemophilia

Keywords

MG1113; Hemophilia

Outcome Measures

Primary Outcomes (9)

• Number of subject with Adverse events, Adverse Drug Reactions, Serious adverse events and Adverse event of special interest (AESI)

  Through study completion (Study Day 1 to Day 78 visit)

• Incidence of Adverse events, Adverse Drug Reactions, Serious adverse events and Adverse event of special interest (AESI)

  Through study completion (Study Day 1 to Day 78 visit)

• Severity of Adverse events, Adverse Drug Reactions, Serious adverse events and Adverse event of special interest (AESI)

  Through study completion (Study Day 1 to Day 78 visit)

• Incidence of injection site reaction

  Study Day 1 to Day 57 visit

• Severity of injection site reaction

  Study Day 1 to Day 57 visit

• Number of subjects with abnormal Physical examination

  Through study completion (Study Day 1 to Day 78 visit)

• Number of subjects with abnormal 12-lead ECG (Ventricular rate in beat/min, Interval for PR in msec, QRS in msec, QTc in msec)

  Through study completion (Study Day 1 to Day 78 visit)

• Number of subjects with abnormal Vital signs (Blood pressure in mmHg, Pulse rate in beats/min, Respiration rate in breaths/min, Body temperature in ℃)

  Through study completion (Study Day 1 to Day 78 visit)

• Incidence of clinically significant laboratory value abnormalities

  Through study completion (Study Day 1 to Day 78 visit)

Secondary Outcomes (19)

• Pharmacokinetic assessment - Cmax (Peak plasma concentration)

  Through study completion (Study Day 1 to Day 78 visit)

• Pharmacokinetic assessment - Cmin (Minimum plasma concentration)

  Through study completion (Study Day 1 to Day 78 visit)

• Pharmacokinetic assessment - AUC (Area under the plasma concentration versus time curve)

  Through study completion (Study Day 1 to Day 78 visit)

• Pharmacokinetic assessment - Tmax (Time to maximum plasma concentration after administration)

  Through study completion (Study Day 1 to Day 78 visit)

• Pharmacokinetic assessment - half-life (T1/2; the time required to reduce the plasma concentration by half)

  Through study completion (Study Day 1 to Day 78 visit)

Study Arms (3)

Cohort 1 (2.0 mg/kg, once weekly)

ACTIVE COMPARATOR

* Anti-tissue factor pathway inhibitor (TFPI) recombinant antibody * Each vial contains 1mL of study drug * The subjects will be treated with 2.0 mg/kg once weekly in cohort 1.

Biological: MG1113

Cohort 2 (A mg/kg, once weekly)

ACTIVE COMPARATOR

* Anti-tissue factor pathway inhibitor (TFPI) recombinant antibody * Each vial contains 1mL of study drug * The subjects will be treated with A mg/kg once weekly in cohort 2. * The Dose A mg/kg will be determined based on the safety, PK, and PD data obtained from previous dose level (cohort 1).

Biological: MG1113

Cohort 3 (B mg/kg, once weekly)

ACTIVE COMPARATOR

* Anti-tissue factor pathway inhibitor (TFPI) recombinant antibody * Each vial contains 1mL of study drug * The subjects will be treated with B mg/kg once weekly in cohort 3. * The Dose B mg/kg will be determined based on the safety, PK, and PD data obtained from previous dose level (cohort 2).

Biological: MG1113

Interventions

MG1113 BIOLOGICAL

MG1113 subcutaneous (SC) injection

Cohort 1 (2.0 mg/kg, once weekly); Cohort 2 (A mg/kg, once weekly); Cohort 3 (B mg/kg, once weekly)

Eligibility Criteria

• Age: 19 Years - 60 Years
• Gender Eligibility Details: Only Male severe hemophilia A or B patients will be enrolled.
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Male severe hemophilia A or B patients (FVIII or FIX activity \<1%) aged 19-60 years (both inclusive) at screening
• Patients without inhibitors against FVIII or FIX (having difficulty in their Self-injection of current standard treatment regimen) OR
• Patients with inhibitors who has a positive inhibitor result of confirmed human factor VIII or IX with an inhibitor titer(≥ 0.6 BU) and failed after ITI treatment or not undergoing ITI
• ≥50 kg in weight with calculated BMI between 18.5 and 29.9 kg/m\^2 (BMI = (Weight \[kg\])/(height \[m\])\^2)
• Documentation of ≥4 bleeding episodes (any type or location of bleeds, treated or not) within 6 months prior to screening
• Agree to use medically acceptable adequate dual contraceptive methods (condom, vasectomy, spermicide, oral contraceptives, intrauterine device, and complete sexual abstinence, etc.) and not to donate sperm until 60 days after administration of the investigational product
• Voluntarily decided to participate in the study and provided written consent to follow precautions after receiving a detailed explanation on this study and fully understanding the information

You may not qualify if:

• Congenital or acquired anticoagulant disorders other than hemophilia A or B, or conditions of other diseases that increase the risk of bleeding or thrombus (e.g., autoimmune disease)
• Be at risk of venous thromboembolism or thrombotic microangiopathy per investigator's judgment or have related medical history or family history
• Be at risk of cardia and/or coronary disease per investigator's judgment or have related medical history or family history
• Risk factors for venous or arterial disease (e.g., uncontrolled hypertension, uncontrolled diabetes)
• Any of the following results from laboratory tests:
• AST(sGOT) or AST(sGPT) \> 3 x UNL
• Total bilirubin \> 2 mg/mL
• Hb \< 9.0 g/dL
• Absolute Neutrophil Count \< 1500 /μL
• Platelet count \< 10\^5 /μL
• Have hepatitis B (HBs Ag positive) or C (anti-HCV positive), or have HIV positive test result If the anti-HCV antibody test is positive, the positive hepatitis virus result must be confirmed by a quantitative HCV RNA test
• Serum Creatinine \> 1.5 x Upper limit of normal (ULN)
• Known or suspected hypersensitivity to the IP or its components
• Treatment history due to symptoms of fever within 28 days of IP administration or any surgery planned during the study period
• Clinically significant active chronic disease

Sponsors & Collaborators

• GC Biopharma Corp: lead

Study Sites (1)

GC Biopharma Corp.

Yongin-si, Gyeonggi-do, 16924, South Korea

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Eun-Jin Choi

  Daegu Catholic University Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 21, 2022
• First Posted: August 9, 2022
• Study Start: August 24, 2022
• Primary Completion: December 11, 2024
• Study Completion: December 11, 2024
• Last Updated: November 24, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)