Hemophilia AEarly Phase 1NCT06238908
Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
A Early Phase 1 study testing NGGT003 in people with Hemophilia A, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- NGGT003
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 17 Jan 2024to 31 Jan 2030
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
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 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Voluntarily sign the informed consent form;
- Male, age ≥18 years old;
- Diagnosed with hemophilia A according to the "Guidelines for Diagnosis and Treatment of Hemophilia A (2022 Edition)", and the endogenous FVIII activity level was <1 IU/dL (<1%);
- The exposure days (EDs) of treatment with any recombinant or plasma-derived FVIII product were ≥150 days;
- Anti-AAV neutralizing antibody titer ≤1:5, binding antibody titer ≤1:100;
- Bleeding events and/or FVIII product injections have occurred within 12 weeks before screening;
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- No history of allergy to FVIII products;
- FVIII inhibitor titer﹤0.6BU/mL;
- Commitment to use other drugs during the study requires the consent of the investigator;
- Willing and able to comply with study procedures and requirements;
- Willing to use effective contraceptive methods within 52 weeks after administration.
Who cannot join
- Positive for hepatitis B surface antigen, hepatitis C, human immunodeficiency virus (HIV)，syphilis test;
- Clinically significant abnormalities in liver function test: alanine aminotransferase (ALT) >1.5 × upper limit of normal (ULN) and/or aspartate aminotransferase (AST) >1.5× ULN;TBil）>1.5×ULN；Serum creatinine (Scr) >1.5×ULN; hemoglobin <110g/L, platelets <10e9/L;
- History of being positive for FVIII inhibitors;
- Have other bleeding factors except hemophilia;
- Plan major surgery within 52 weeks;
- Have contraindications to glucocorticoid, including but not limited to allergy to glucocorticoids, epilepsy, new unhealed fractures, in trauma repair period, uncontrolled infection, severe osteoporosis, etc, which assessed and determined by the investigators;
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- History of allergy to human albumin;
- Have serious diseases or active infections in cardiovascular, respiratory, digestive tract, endocrine, renal, blood, nervous, mental and other systems before screening;
- With hepatitis, cirrhosis, liver cancer or other major liver diseases;
- History of malignant tumors;
- Abnormal and clinical significant vital signs, physical examination, laboratory examination or other related examination results during the screen, which are not suitable for trial according to the investigator;
- Previous gene therapy treatment;
- Participation in any other clinical trial before the screening and have taken medication within four weeks or five half-lives of the study drug;
- Any other condition that may not be appropriate for the study in the opinion of the investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
Lei Zhang, MD, +862223909240, zhanglei1@ihcams.ac.cn
About this study
Official title: Clinical Study on the Safety and Efficacy of an Intravenous Infusion of NGGT003 in the Treatment of Hemophilia A
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients. NGGT003 uses adeno-associated virus (AAV) as a vector, carrying a liver specific promoter and codon optimized human FVIII gene B domain deletion mutant (hFVIII BDD), and expresses human FVIII protein in the liver through intravenous injection.
Read the detailed description
Hemophilia A (HA) is an X-linked recessive genetic disease caused by mutations in the FVIII gene on the X chromosome, leading to abnormal coagulation function. In the male population, the incidence rate of hemophilia A was about 1/5000, and female patients with hemophilia A were extremely rare. Type A hemophilia patients mainly exhibit a tendency for bleeding, with a wide range of bleeding sites and frequent recurrence, which can form hematoma and joint deformation. This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of a single intravenous infusion of NGGT003 in hemophilia A patients. 4-6 subjects will be enrolled and divided into 3 groups according to the principle of dose escalation, respectively administered intravenous infusion of NGGT003 at low dose (4e11vg/kg), medium dose (1e12vg/kg) and high dose (2.5e12vg/kg). All subjects will undergo 52 weeks of treatment observation and further 260 weeks of long-term follow-up.
- Conditions
- Hemophilia A
- Being tested
- NGGT003
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NGGT003Drug
Single intravenous infusion of NGGT003 at low dose (4e11vg/kg), medium dose (1e12vg/kg) and high dose (2.5e12vg/kg)
| Study group | Role | What they receive |
|---|---|---|
| Experimental | Experimental | 3 doses of NGGT003 will be administered according to the principle of dose escalation Receives: Drug: NGGT003 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events (AEs) and serious adverse events (SAEs)
Incidence of AE and SAE, as assessed by physical examinations, clinical laboratory parameters and adverse event reporting
Measured: 52 weeks
Changes in annualized bleeding rate (ABR)
Changes in annualized bleeding rate (ABR) from baseline to 52 weeks.
Measured: 52 weeks
Secondary outcomes (4)
FVIII activity levels
Change in FVIII activity levels from baseline to week 52.
Measured: 52 weeks
FVIII protein product infusions
Calculate the number and volume of FVIII protein product infusions from baseline to week 52.
Measured: 52 weeks
Target joints
Changes the numbers of target joints from baseline to week 52.
Measured: 52 weeks
HA-QOL scores
Change in HA-QOL scores from baseline to 52 weeks.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2024
- Study start17 Jan 2024
- Primary completion31 Jan 2026 (estimated)
- Study completion31 Jan 2030 (estimated)
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.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, MD, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06238908
- Sponsor's protocol ID
- IIT2023043
- Status last verified
- Jan 2025
- First submitted
- 26 Jan 2024
- Last updated
- 21 Feb 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study on the Safety and Efficacy of an Intravenous Infusion of NGGT003 in the Treatment of Hemophilia A
- ClinicalTrials.gov ID
- NCT06238908
- Acronym / sponsor ID
- IIT2023043
- Conditions
- Hemophilia A
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 17 Jan 2024 (actual)
- Primary completion
- 31 Jan 2026 (estimated)
- Study completion
- 31 Jan 2030 (estimated)
- First posted
- 2 Feb 2024 (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 an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients. NGGT003 uses adeno-associated virus (AAV) as a vector, carrying a liver specific promoter and codon optimized human FVIII gene B domain deletion mutant (hFVIII BDD), and expresses human FVIII protein in the liver through intravenous injection.
Detailed description
Hemophilia A (HA) is an X-linked recessive genetic disease caused by mutations in the FVIII gene on the X chromosome, leading to abnormal coagulation function. In the male population, the incidence rate of hemophilia A was about 1/5000, and female patients with hemophilia A were extremely rare. Type A hemophilia patients mainly exhibit a tendency for bleeding, with a wide range of bleeding sites and frequent recurrence, which can form hematoma and joint deformation. This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of a single intravenous infusion of NGGT003 in hemophilia A patients. 4-6 subjects will be enrolled and divided into 3 groups according to the principle of dose escalation, respectively administered intravenous infusion of NGGT003 at low dose (4e11vg/kg), medium dose (1e12vg/kg) and high dose (2.5e12vg/kg). All subjects will undergo 52 weeks of treatment observation and further 260 weeks of long-term follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental | Experimental | Drug: NGGT0033 doses of NGGT003 will be administered according to the principle of dose escalation |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Voluntarily sign the informed consent form;
- Male, age ≥18 years old;
- Diagnosed with hemophilia A according to the "Guidelines for Diagnosis and Treatment of Hemophilia A (2022 Edition)", and the endogenous FVIII activity level was <1 IU/dL (<1%);
- The exposure days (EDs) of treatment with any recombinant or plasma-derived FVIII product were ≥150 days;
- Anti-AAV neutralizing antibody titer ≤1:5, binding antibody titer ≤1:100;
Key exclusion criteria (14 listed)
- Positive for hepatitis B surface antigen, hepatitis C, human immunodeficiency virus (HIV)，syphilis test;
- Clinically significant abnormalities in liver function test: alanine aminotransferase (ALT) >1.5 × upper limit of normal (ULN) and/or aspartate aminotransferase (AST) >1.5× ULN;TBil）>1.5×ULN；Serum creatinine (Scr) >1.5×ULN; hemoglobin <110g/L, platelets <10e9/L;
- History of being positive for FVIII inhibitors;
- Have other bleeding factors except hemophilia;
- Plan major surgery within 52 weeks;
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 |
|---|---|---|
| NGGT003Single intravenous infusion of NGGT003 at low dose (4e11vg/kg), medium dose (1e12vg/kg) and high dose (2.5e12vg/kg) | Drug | Experimental |
Primary outcome
Adverse events (AEs) and serious adverse events (SAEs)
Time frame: 52 weeks
Incidence of AE and SAE, as assessed by physical examinations, clinical laboratory parameters and adverse event reporting
Changes in annualized bleeding rate (ABR)
Time frame: 52 weeks
Changes in annualized bleeding rate (ABR) from baseline to 52 weeks.
4 secondary outcomes
FVIII activity levels
Time frame: 52 weeks
FVIII protein product infusions
Time frame: 52 weeks
Target joints
Time frame: 52 weeks
HA-QOL scores
Time frame: 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2025.
- Individual participant dataSharing planned: No
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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.
Lei Zhang, MD principal investigator
Institute of Hematology & Blood Diseases Hospital, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing NGGT003 in people with Hemophilia A, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
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 11 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: looking for participants now, as of the sponsor's last update on 21 Feb 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 China. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Wei Liu, MD (+862223909240, liuwei1@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (AEs) and serious adverse events (SAEs) (time frame: 52 weeks). The study lists 2 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 21 Feb 2025.