Hemophilia aPhase 1NCT06025552
Study of TU7710 in Warfarin Anti-coagulated Healthy Male Subjects
A Phase 1 study testing TU7710 and Normal saline in people with Hemophilia a and Hemophilia B, run by TiumBio Co., Ltd. at 1 site in South Korea.
Trial summary
This is a Phase 1a, double-blind, randomized, placebo- controlled, SAD study to assess safety, tolerability, PK, and PD of TU7710 in warfarin treated healthy male participants. Read more about this study
- Who is it for?
- Ages 19 to 45 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Hemophilia a, Hemophilia B, Blood Coagulation Disorders
- What is being tested?
- TU7710 and Normal saline
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteSouth Korea
- Study dates
- 2 Aug 2023to 21 Aug 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 45 years
- Men only
- Healthy volunteers accepted
- Adult
Who may be able to join
- Age ≥19 and ≤45
- BMI of ≥18.0 kg/m2 and ≤30.0 kg/m2
- Body weight of ≥55.0 kg and ≤90.0 kg
- Provide informed consent and willing to comply with study requirements.
Who cannot join
- History or at risk of developing diseases related to venous thromboembolic events or has family history of such disease
- History of major bleeding/traumatic event or major surgery within 6 month
- History of any other clinically relevant coagulation disorder (such as gastrointestinal bleeding, hemorrhoid hemorrhage)
- Abnormal coagulation related laboratory abnormal test results, including protein C, protein S, PT, aPTT
- history or current symptoms of gastrointestinal, liver, or renal disease that may affect the pharmacokinetics of the IP
- History of or are currently with hepatitis B or C (active or carrier state) or human immunodeficiency virus (HIV) or syphilis infection.
Show all 14Show fewer
- Currently smoking or have smoked within 1 month before IP or positive cotinine results
- History of alcohol abuse or positive alcohol breath test
- Excessive caffeine intake within 7 days before IP
- INR results not between 2.0~3.0 range after warfarin treatment
- History of hypersensitivity to medicinal product similar to TU7710 active ingredient or excipient
- Laboratory abnormal test results, such as QTcF <340msec or >450msec (or family history of long QT syndrome), LDL >190mg/dl , Total cholesterol >300mg/dl, triglycerides > 350mg/dl, ALT >1.5*ULN, AST >1.5*ULN, bilirubin >1.5*ULN
- Abnormal vital sign SBP >140mmHG, DBP <90mmHg, heart rate <40bpm or >85bpm
- Any medical history that may increase the risk or affect the evaluation of study objectives by participating in this study at the discretion of the investigator. (e.g., neurology or psychiatric history)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A First-in-Human (FIH), Randomized, Double-Blind, Placebo-controlled, Phase 1a Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Activity of TU7710 Following Single, Ascending, Intravenous, Dose Administration in Warfarin Anti-coagulated Healthy Male Subjects
This is a Phase 1a, double-blind, randomized, placebo- controlled, SAD study to assess safety, tolerability, PK, and PD of TU7710 in warfarin treated healthy male participants.
Read the detailed description
The 40 subjects will be divided into 5 cohorts, and the subjects assigned to each cohort will be randomly assigned with 6 persons receiving TU7710 and 2 persons receiving a placebo for TU7710. Each cohort will proceed in sequence and the next cohort study will be decided by the Safety Monitoring Committee (SMC) .
Subjects will be participated in the study after warfarin anti-coagulation to maintain the INR between 2.00 and 3.00 as a preventive measure for potential thrombosis prior to the IP administration.
- Conditions
- Hemophilia a, Hemophilia B, Blood Coagulation Disorders
- Being tested
- TU7710, Normal saline
- Medical subject headings
- Hemophilia A, Hemophilia B, Blood Coagulation Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TU7710Drug
In each dose level, 6 subjects will be assigned to TU7710. Anticipated escalating dose levels are 100mcg/kg, 200mcg/kg, 400mcg/kg, 800mcg/kg and the last dose will be decided after assessing cohort 1~4 PK, PD, safety, and exploratory efficacy data.
Normal salineDrug
Placebo of TU7710 at corresponding TU7710 dose level. In each dose level, 2 subjects will be assigned to placebo group.
| Study group | Role | What they receive |
|---|---|---|
| TU7710 | Experimental | TU7710 of escalating 5 doses Receives: Drug: TU7710 |
| Normal Saline (placebo of TU7710) | Placebo comparator | Placebo of TU7710 at corresponding TU7710 dose level Receives: Drug: Normal saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and proportion of participants with adverse events
Number and proportion of participants with adverse events/ adverse reaction /SAE overall and by treatment group
Measured: 30 days post-dose
Number of subjects with significant abnormal laboratory values
Mean with standard deviation, median, maximum, minimum results of laboratory values in each treatment group. The laboratory parameters that will be assessed are clinical chemistry, hematology and urinalysis.
Measured: 30 days post-dose
ADA and Neutralizing antibody results
Incidence of subjects with ADA and Nab positive results
Measured: 30 days post-dose
Number of subjects with significant abnormal Electrocardiography (ECG) findings
Mean with standard deviation, median, maximum, minimum results of ECG results in each treatment group. The ECG parameters that will be assessed are heart rate, PR interval, QRS interval, QT interval, and QTcF interval.
Measured: 30 days post-dose
Number of subjects With Significant Abnormal vital sign findings
Mean with standard deviation, median, maximum, minimum results of vital sign values in each treatment group. The vital signs that will be assessed are body temperature, pulse rate, respiratory rate, and systolic and diastolic blood pressure.
Measured: 30 days post-dose
Secondary outcomes (11)
Pharmacokinetics assessment_Maximum concentration
Maximum plasma VIIa activity level in each dose level
Measured: 4 days post-dose
Pharmacokinetics assessment_AUC last
Area under plasma activity-time curve after TU7710 single administration from time zero to last quantifiable concentration
Measured: 4 days post-dose
Pharmacokinetics assessment_AUC inf
Area Under the Plasma activity-time curve after TU7710 single administration From Time Zero Extrapolated to Infinity
Measured: 4 days post-dose
Pharmacokinetics assessment_Clearance
Clearance after TU7710 single administration
Measured: 4 days post-dose
Pharmacokinetics assessment_Volume of distribution
Volume of distribution after TU7710 single administration
Measured: 4 days post-dose
Pharmacokinetics assessment_Dose proportionality
Regression analysis using the power model between the log-converted Cmax, AUClast, and the log-converted dose can be performed, and each parameter adjusted by dose can be calculated and compared between the dose groups
Measured: 4 days post-dose
Pharmacokinetics assessment_Tmax
Time from administration to maximum plasma VIIa level in each dose level
Measured: 4 days post-dose
Pharmacodynamic assessment_INR change from baseline
INR measurement change from baseline to day 5 in each treatment group and dose level
Measured: 5 days post-dose
Pharmacodynamic assessment_PT change from baseline
PT measurement change from baseline to day 5 in each treatment group and dose level
Measured: 5 days post-dose
Pharmacodynamic assessment_aPTT change from baseline
aPTT measurement change from baseline to day 5 in each treatment group and dose level
Measured: 5 days post-dose
Pharmacokinetics assessment_incremental recovery
Incremental recovery after TU7710 single administration expressed as the ratio of measured peak level against dose per bodyweight
Measured: 4 days post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Sep 2023
- Study start2 Aug 2023
- Primary completion21 Aug 2024
- Study completion21 Aug 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia aStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Randomised
- Intervention model
- Sequential
- Model description
- 5 cohorts with 5 dose levels will sequentially be escalated after safety review. 8 subjects in each cohort who will be randomly assigned to placebo or TU7710 group in 2:6 ratio.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- TiumBio Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06025552
- Sponsor's protocol ID
- TUB4PI-01
- Status last verified
- Sep 2024
- First submitted
- 31 Jul 2023
- Last updated
- 27 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A First-in-Human (FIH), Randomized, Double-Blind, Placebo-controlled, Phase 1a Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Activity of TU7710 Following Single, Ascending, Intravenous, Dose Administration in Warfarin Anti-coagulated Healthy Male Subjects
- ClinicalTrials.gov ID
- NCT06025552
- Acronym / sponsor ID
- TUB4PI-01
- Conditions
- Hemophilia a, Hemophilia B, Blood Coagulation Disorders
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- TiumBio Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 2 Aug 2023 (actual)
- Primary completion
- 21 Aug 2024 (actual)
- Study completion
- 21 Aug 2024 (actual)
- First posted
- 6 Sep 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 1a, double-blind, randomized, placebo- controlled, SAD study to assess safety, tolerability, PK, and PD of TU7710 in warfarin treated healthy male participants.
Detailed description
The 40 subjects will be divided into 5 cohorts, and the subjects assigned to each cohort will be randomly assigned with 6 persons receiving TU7710 and 2 persons receiving a placebo for TU7710. Each cohort will proceed in sequence and the next cohort study will be decided by the Safety Monitoring Committee (SMC) .
Subjects will be participated in the study after warfarin anti-coagulation to maintain the INR between 2.00 and 3.00 as a preventive measure for potential thrombosis prior to the IP administration.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- 5 cohorts with 5 dose levels will sequentially be escalated after safety review. 8 subjects in each cohort who will be randomly assigned to placebo or TU7710 group in 2:6 ratio.
| Arm | Type | Receives |
|---|---|---|
| TU7710 | Experimental | Drug: TU7710TU7710 of escalating 5 doses |
| Normal Saline (placebo of TU7710) | Placebo comparator | Drug: Normal salinePlacebo of TU7710 at corresponding TU7710 dose level |
Who may qualify
- Ages
- 19 to 45 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Age ≥19 and ≤45
- BMI of ≥18.0 kg/m2 and ≤30.0 kg/m2
- Body weight of ≥55.0 kg and ≤90.0 kg
- Provide informed consent and willing to comply with study requirements.
Key exclusion criteria (14 listed)
- History or at risk of developing diseases related to venous thromboembolic events or has family history of such disease
- History of major bleeding/traumatic event or major surgery within 6 month
- History of any other clinically relevant coagulation disorder (such as gastrointestinal bleeding, hemorrhoid hemorrhage)
- Abnormal coagulation related laboratory abnormal test results, including protein C, protein S, PT, aPTT
- history or current symptoms of gastrointestinal, liver, or renal disease that may affect the pharmacokinetics of the IP
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TU7710In each dose level, 6 subjects will be assigned to TU7710. Anticipated escalating dose levels are 100mcg/kg, 200mcg/kg, 400mcg/kg, 800mcg/kg and the last dose will be decided after assessing cohort 1~4 PK, PD, safety, and exploratory efficacy data. | Drug | TU7710 |
| Normal salinePlacebo of TU7710 at corresponding TU7710 dose level. In each dose level, 2 subjects will be assigned to placebo group. | Drug | Normal Saline (placebo of TU7710) |
Primary outcome
Number and proportion of participants with adverse events
Time frame: 30 days post-dose
Number and proportion of participants with adverse events/ adverse reaction /SAE overall and by treatment group
Number of subjects with significant abnormal laboratory values
Time frame: 30 days post-dose
Mean with standard deviation, median, maximum, minimum results of laboratory values in each treatment group. The laboratory parameters that will be assessed are clinical chemistry, hematology and urinalysis.
ADA and Neutralizing antibody results
Time frame: 30 days post-dose
Incidence of subjects with ADA and Nab positive results
Number of subjects with significant abnormal Electrocardiography (ECG) findings
Time frame: 30 days post-dose
Mean with standard deviation, median, maximum, minimum results of ECG results in each treatment group. The ECG parameters that will be assessed are heart rate, PR interval, QRS interval, QT interval, and QTcF interval.
Number of subjects With Significant Abnormal vital sign findings
Time frame: 30 days post-dose
Mean with standard deviation, median, maximum, minimum results of vital sign values in each treatment group. The vital signs that will be assessed are body temperature, pulse rate, respiratory rate, and systolic and diastolic blood pressure.
11 secondary outcomes
Pharmacokinetics assessment_Maximum concentration
Time frame: 4 days post-dose
Pharmacokinetics assessment_AUC last
Time frame: 4 days post-dose
Pharmacokinetics assessment_AUC inf
Time frame: 4 days post-dose
Pharmacokinetics assessment_Clearance
Time frame: 4 days post-dose
Pharmacokinetics assessment_Volume of distribution
Time frame: 4 days post-dose
Pharmacokinetics assessment_Dose proportionality
Time frame: 4 days post-dose
Pharmacokinetics assessment_Tmax
Time frame: 4 days post-dose
Pharmacodynamic assessment_INR change from baseline
Time frame: 5 days post-dose
Pharmacodynamic assessment_PT change from baseline
Time frame: 5 days post-dose
Pharmacodynamic assessment_aPTT change from baseline
Time frame: 5 days post-dose
Pharmacokinetics assessment_incremental recovery
Time frame: 4 days post-dose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Sep 2024.
Related trials for Hemophilia a
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TU7710 and Normal saline in people with Hemophilia a and Hemophilia B, run by TiumBio Co., Ltd. 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 4 inclusion and 14 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 27 Sep 2024. 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. Some participants in this study may receive a placebo. 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 and proportion of participants with adverse events (time frame: 30 days post-dose). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Sep 2024.