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HemophiliaPhase 1NCT05493631

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

A Phase 1 study testing MG1113 in people with Hemophilia, run by GC Biopharma Corp at 1 site in South Korea.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 19 to 60 years, men only
Conditions the study is for
Hemophilia
What is being tested?
MG1113
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
1 siteSouth Korea
Study dates
24 Aug 2022to 11 Dec 2024
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 19 to 60 years
  • Men only
  • No healthy volunteers
  • Adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 22Show fewer
  • 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
  • Subjects who refuse the following wash-out times of Factor VIII, Factor IX, and bypassing agent from the time of first IP administration (Factor VIII: 72 hrs, Factor IX: 96 hrs, Bypassing agent e.g., rFVIIa or aPCC: 96 hrs)
  • Received immune tolerance induction within 30 days prior to administration of the investigational product
  • Received emicizumab within 30 days prior to administration of the investigational product
  • Currently using systemic immunomodulator treatment (e.g., Corticosteroid*, IVIG, interferon or rituximab)
  • High-dose corticosteroids (it is allowed to administer corticosteroid equivalent to up to 20mg/kg daily based on Prednisolone, but if a dose of more is continuously administered in excess of 14 days, it is considered as high dose, such case is excluded from this study. However, inhaled, intranasal, and topical administration of corticosteroids is allowed irrespective of the dose.)
  • Participated in another clinical trial within 30 days of investigational product administration
  • Determined to be ineligible to participate in the study per investigator's judgment due to other reasons including the laboratory test results

Trial locations and contacts

1 site in 1 country.

South Korea1 site

  • GC Biopharma Corp.

    Yongin-si, Gyeonggi-do, 16924

Other trials in South Korea

About this study

Official title: A Phase 1b, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Subcutaneous MG1113 in Patients With Severe Hemophilia

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.

Read the detailed description

This is a repeat-dose study that assign 5 subjects in each cohort to explore the safety, tolerability, PK, and PD of the study drug by sequentially increasing the study drug.

The route of administration is subcutaneous (SC) injection. Dose escalation will be decided after checking the safety and tolerability at the previous dose to the extent not exceeding the criteria for discontinuation of dose escalation. The dose escalation will be decided by the Steering Committee and Data and Safety Monitoring Boards (DSMB) in the evaluation of the safety and tolerability data obtained from each cohort after repeated administration of MG1113.

The subjects will be treated with 2.0 mg/kg once weekly for 8 weeks in cohort 1. Visit window of ±1 day (calculated from Day1) are allowed for the dosing schedule after first IP administration (Day 1). But next scheduled IP administration must be kept in mind to ensure subjects will not have more than 8 days in between IP dosing interval.

The next dose level (Dose A and B) will be determined based on the safety, PK, and PD data obtained from previous dose level. If a criterion of discontinuation of dose escalation is fulfilled, discussion about dose escalation is available for next cohort. The dose selection and escalation will be finally determined from the Steering Committee and DSMB. The safety, tolerability, PK, and PD data obtained from all subjects up to Cohort 3 will be evaluated by the Steering Committee and Data and Safety Monitoring Boards (DSMB).

Conditions
Hemophilia
Being tested
MG1113
Keywords
MG1113, Hemophilia
Medical subject headings
Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MG1113Biological

    MG1113 subcutaneous (SC) injection

Study groups in NCT05493631
Study groupRoleWhat they receive
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.
Receives: 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).
Receives: 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).
Receives: Biological: MG1113

What is being measured

The primary outcome is the main question the study is designed to answer.

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

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

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

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

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

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

  4. Incidence of injection site reaction

    Measured: Study Day 1 to Day 57 visit

  5. Severity of injection site reaction

    Measured: Study Day 1 to Day 57 visit

  6. Number of subjects with abnormal Physical examination

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

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

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

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

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

  9. Incidence of clinically significant laboratory value abnormalities

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

Secondary outcomes (19)

  1. Pharmacokinetic assessment - Cmax (Peak plasma concentration)

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

  2. Pharmacokinetic assessment - Cmin (Minimum plasma concentration)

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

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

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

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

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

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

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

  6. Pharmacokinetic assessment - CL/F (apparent clearance)

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

  7. Pharmacokinetic assessment - Vd/F (apparent volume of distribution)

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

  8. Pharmacokinetic assessment - Accumulation Index (AUCtau_multiple dose/AUCtau_single dose)

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

  9. Pharmacodynamic assessment - Free TFPI in plasma in ng/mL

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

  10. Pharmacodynamic assessment - Residual TFPI activity in unit/mL

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

  11. Pharmacodynamic assessment - Diluted PT in sec

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

  12. Pharmacodynamic assessment - Thrombin generation (Lag time in min)

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

  13. Pharmacodynamic assessment - Thrombin generation (Peak generation in nM)

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

  14. Pharmacodynamic assessment - Thrombin generation (Endogenous thrombin generation potential [ETP] in nM*min)

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

  15. Pharmacodynamic assessment - Pro-coagulant effect (D-dimer in ug/mL)

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

  16. Pharmacodynamic assessment - Pro-coagulant effect (Fibrinogen in mg/dL)

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

  17. Pharmacodynamic assessment - Pro-coagulant effect (Prothrombin fragments 1+2 in pmol/L)

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

  18. Immunogenicity assessment - Any formation of anti-drug antibody (ADA) to MG1113

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

  19. Efficacy Evaluation - Incidence of new bleeding episode

    Measured: Study Day 1 to Day 57 visit

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Aug 2022
  2. Study start24 Aug 2022
  3. Primary completion11 Dec 2024
  4. Study completion11 Dec 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
15 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05493631
Sponsor's protocol ID
MG1113_HP_P0101
Status last verified
Nov 2025
First submitted
21 Jul 2022
Last updated
24 Nov 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • You CW, Baek HJ, Park YS, Kim BR, Lee H, Kim SH, Hwang SH, Choi EJ. Subcutaneous MG1113 in severe hemophilia A and B: phase 1b study for safety, pharmacokinetics, and pharmacodynamics. Res Pract Thromb Haemost. 2026 Aug 15;10(6):106910. doi: 10.1016/j.rpth.2026.106910. eCollection 2026 Aug.

    PubMed 42757060

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing MG1113 in people with Hemophilia, run by GC Biopharma Corp at 1 site in South Korea.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 24 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of subject with Adverse events, Adverse Drug Reactions, Serious adverse events and Adverse event of special interest (AESI) (time frame: Through study completion (Study Day 1 to Day 78 visit)). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05493631, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Nov 2025.

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