Hemophilia AHANCT04728841
Gene Therapy for Chinese Hemophilia A
A study testing Injection of GS001 in people with Hemophilia A and Gene Therapy, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with <1 IU/dl residual FVIII levels. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A, Gene Therapy
- What is being tested?
- Injection of GS001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 4 Mar 2021to 31 Jul 2028
- Phase
- Interventional
- 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
- Be able to understand the purpose and risks of the study and provide informed consent according to national and local privacy laws;
- Male subjects and ≥ 18 years of age;
- Have hemophilia A with ≤1 IU/dL (≤1%) endogenous FVIII activity levels at the time of screening. If the screening result is >1% due to previous treatment with FVIII product, then it may be confirmed by documented historical evidence from a certified clinical laboratory demonstrating ≤1% FVIII activity levels ;
- No history of hypersensitivity or anaphylaxis associated with FVIII product administration;
- Have no measurable FVII inhibitor as assessed by laboratory two times that were at least one week apart; or documented no prior history of FVIII inhibitor after 150 EDs and no clinical signs or symptoms of decreased response to FVIII infusion ;
- Have acceptable laboratory values sampled at screening and repeated prior to Day 0; A. Hemoglobin ≥ 11 g/dL; B. Platelets ≥ 100 x 10^9/L; C. AST, ALT, alkaline phosphatase ≤ 1.25 upper limit of normal (ULN); D. Bilirubin ≤ 1.25 ULN; E. Creatinine ≤ 2 mg/dL.
- Agree to use reliable barrier contraception until the end of the 52 weeks observation period, and three consecutive semen samples are negative for vector sequences after GS001 infusion.
Who cannot join
- Have Hepatitis B, hepatitis C or HBsAg, HCVAb, HBV-DNA, HCV-RNA are positive and have clinical significance. Both natural clearers and those who have cleared HCV on antiviral therapy are deemed eligible;
- Currently Receiving antiviral therapy for hepatitis B and C;
- Have history of chronic infections or other chronic diseases that may pose a risk to the study participation;
- Have participated in a previous gene therapy research trial within the last 52 weeks or in a clinical study with an investigational drug within the past 30 days;
- The subject has any concurrent diseases that cannot tolerate treatments of prednisone or prednisolone as judged by the investigator;
- History of arterial or venous thromboembolic events (e.g., deep vein thrombosis, non-hemorrhagic stroke, pulmonary embolism, myocardial infarction, arterial embolism);
Show all 12Show fewer
- Known inherited or acquired thrombophilia, including conditions associated with increased risk of thromboembolism, such as atrial fibrillation;
- Major surgery planned in 1 year period following the infusion with GS001;
- Hypersensitivity to the study vector;
- Have clinically major diseases or any other unspecified conditions that, in the opinion of the Investigator, makes the subject unsuitable for participating in the study;
- Patients who are unable or unwilling to comply with the schedule of visits and study assessments described in the clinical protocol;
- Evidence of other bleeding disorders not associated with hemophilia A.
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
Chinese Academy of Medical Science and Blood Disease Hospital
Tianjin, Tianjin Municipality, 300020
Shuo Chen, MD, +862223909240, zhanglei1@ihcams.ac.cn
Jun L Liu, MD, +862223909095, gcp@ihcams.ac.cn
About this study
Official title: Clinical Exploration of Clinical Exploration Adeno-associated Virus Vector Expression of Human Coagulation Factor VIII Gene Therapy for Hemophilia A
IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with <1 IU/dl residual FVIII levels.
Read the detailed description
IHBDH-GTHA-2020 is a open- label, non- randomized study to evaluate the safety, tolerability and kinetics of GS001 in hemophilia A subjects with residual FVIII levels<1 IU/dl. The first patient will receive a single intravenous infusion of GS001 at a dose level of 2 x 10^12 vg/kg body weight. After three weeks of follow-up for the first patient post infusion of GS001, the dose of GS001 can be adjusted for the other two patients based on the activity level of FVIII:C and safety assessment. Following completion of the first 3 patients received GS001 infusion at 2 x 10^12 vg/kg dose level , the following 3 patients will continue to be enrolled to this dose group if there isn't any unexpected safety risk based on the assessments of FVIII expression and safety profile. The safety data and the activity level of FVIII:C from the first 6 subjects at 2 x 10^12 vg/kg dose level will undergo review by an independent DMC prior to dosing the first subject in the next dose level, DMC may recommend dose escalation to 6 x 10^12 vg/kg body weight or other recommended dose levels.
If the DMC recommends dose escalation to 6 x 10 ^12 vg/kg body weight or other recommended doses, at least 10 weeks of safety data and the activity level of FVIII:C post GS001 infusion from the first 3 subjects in this given dose level will undergo review by an independent DMC prior to dosing the following subjects. Based on the data from the first 3 subjects, DMC may recommend the dose adjustments for subsequent enrolled subjects, and expand the number of subject enrolled. Based on the safety, preliminary efficacy and vector kinetics profile of single intravenous infusions of GS001 at different dose levels in severe hemophilia A patients with endogenous factor FVIII activity levels ≤ 1% in this study, and based on the benefit-risk assessment of the subjects, the dose level to be administered for future larger clinical trial in patients with severe hemophilia A will be determined.
After all enrolled subjects have been followed for at least 12 weeks after intravenous infusion GS001, periodic analysis will be performed. Primary analysis of safety and efficacy will be performed 52 (± 2) weeks after intravenous infusion GS001, and all subjects will receive 52 (± 2) weeks of comprehensive safety and efficacy assessments. After completing the 52 (± 2) week visit (end-of-study visit), subjects will continue to be followed in this study for up to an additional 4 years for long-term safety and efficacy assessment to evaluate the long-term safety and efficacy of GS001 treatment.
A total of 12 to 15 subjects are expected to be enrolled in this study. Subjects will provide informed consent and then undergo screening assessments up to 4-8 weeks prior administration of GS001. All subjects will undergo 260 weeks (5 years) safety observation.
- Conditions
- Hemophilia A, Gene Therapy
- Being tested
- Injection of GS001
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Injection of GS001Genetic
Patients will be enrolled sequentially every 3 weeks or more between cohorts. Dose escalation may occur after a single patient has been safely dosed if the resulting FVIII activity at Week 3 is < 5 IU/dL.The dose levels are as follows:
- 2×10^12 vg/kg
- 6×10^12vg/kg or other recommended doses
- 2×10^13 vg/kg or other recommended doses
| Study group | Role | What they receive |
|---|---|---|
| Treatment group | Experimental | Arm of GS001 Receives: Genetic: Injection of GS001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment- related adverse events
Number of patients experiencing treatment-related adverse events.
Measured: From screening through up to the end of study (about 5 years).
Percentage of subjects in each dose group with newly occurred clinically significant abnormalities in physical examination compared to the baseline.
The number of subjects in each dose group with clinically significant changes in physical examination compared to the baseline.
Measured: From the start of study treatment (Day 1) through up to the end of study (about 5 years).
Changes of Weighted Mean of vital signs (systolic blood pressure [SBP] and diastolic blood pressure [DBP], pulse rate, temperature, respiratory rate) from baseline at each assessment time point for each dose group
The vital signs (SBP, DBP, pulse rate, temperature, respiratory rate) of the subjects were measured. The maximum, minimum, and mean observed values of vital signs (SBP, DBP, pulse rate, temperature, respiratory rate) from the dosing (Day 1) to the end of the study were calculated for each subject.
Measured: From the start of study treatment (Day 1) through up to the end of study (about 5 years).
Changes of Mean Alkaline Phosphatase (ALP), Alanine Amino Transferase (ALT), Aspartate Amio Transferase (AST) from baseline at each assessment time point for each dose group
Blood samples of subjects were collected for the evaluation of liver function throughout this study. All of these parameters are measured to help assess the condition of the liver. For ALP, AST and ALT, the percentage of subjects with the worst post-treatment results in each dose group will be summarized as follows: > 1.0 x ULN ≤ 1.5x ULN; > 1.5 x ULN ≤ 3.0 x ULN; > 3.0 x ULN ≤ 5.0 x ULN; > 5.0 x ULN
Measured: From the start of study treatment (Day 1) through up to the end of study (about 5 years).
Immune response to AAV capsid proteins
Changes in the expression levels of neutralizing and binding antibodies of AAV.
Measured: From screening period through up to 5 years.
Immune response to FVIII transgene
The changes of FVIII inhibitor and antibody levels
Measured: From screening period through up to 5 years.
Viral vector shedding of GS001
The vector shedding in serum, PMBC, saliva, urine, semen and feces will be monitored
Measured: From date of infusion until the date of 3 consecutive documented negative results, assessed up to 1 year.
Thrombosis risk assessment
For any individual who reaches >150% vector-derived FVIII: C activity levels following the infusion of GS001, laboratory parameters of thrombotic potential will be assessed.
Measured: From the start of study treatment (Day 1) through up to the end of study (about 5 years).
Secondary outcomes (4)
Vector- derived FVIII:C and FVIII antigen levels
Vector- derived FVIII:C and FVIII antigen levels will be measured after dosing.
Measured: From pre-dose phase through up to 1 years post-dose
FVIII usage within 1 year after GS001 infusion
Number of FVIII replacement therapies and total utilization of exogenous FVIII (IU/kg) replacement therapy.
Measured: From week 3 to week 52 post GS001 infusion.
Number of bleeding events requiring exogenous FVIII replacement therapy within 1 year after GS001 infusion
Number of bleeding events (Spontaneous and Traumatic) requiring exogenous FVIII replacement therapy
Measured: Week 3 to Week 52 post GS001 Infusion
Number of bleeding events within 1 year after GS001 infusion
Number of bleeding events (spontaneous or traumatic) including untreated bleeding events
Measured: Week 3 to Week 52 post GS001 infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2021
- Study start4 Mar 2021
- Primary completion31 Jul 2028 (estimated)
- Study completion31 Jul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, MD, Principal investigator, Chinese Academy of Medical Science and Blood Disease Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04728841
- Sponsor's protocol ID
- IIT2020030
- Acronym
- HA
- Status last verified
- Jan 2025
- First submitted
- 25 Jan 2021
- Last updated
- 1 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Liu W, Pei X, Yu T, Xu B, Dai X, Xue F, Chen L, Wang X, Wang Y, Chen Y, Liu X, Fu R, Wang W, Chi Y, Li HY, Yang R, Zhang L. Phase 1 pilot study for hemophilia-A: AAV8 vector with prophylactic tacrolimus-glucocorticoid achieves therapeutic FVIII activity. Signal Transduct Target Ther. 2026 Mar 11;11(1):88. doi: 10.1038/s41392-026-02599-3.
PubMed 41813646
Study at a glance
From the registry record, last updated by the sponsor on 1 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Exploration of Clinical Exploration Adeno-associated Virus Vector Expression of Human Coagulation Factor VIII Gene Therapy for Hemophilia A
- ClinicalTrials.gov ID
- NCT04728841
- Acronym / sponsor ID
- HA / IIT2020030
- Conditions
- Hemophilia A, Gene Therapy
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 4 Mar 2021 (actual)
- Primary completion
- 31 Jul 2028 (estimated)
- Study completion
- 31 Jul 2028 (estimated)
- First posted
- 28 Jan 2021 (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.
IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with <1 IU/dl residual FVIII levels.
Detailed description
IHBDH-GTHA-2020 is a open- label, non- randomized study to evaluate the safety, tolerability and kinetics of GS001 in hemophilia A subjects with residual FVIII levels<1 IU/dl. The first patient will receive a single intravenous infusion of GS001 at a dose level of 2 x 10^12 vg/kg body weight. After three weeks of follow-up for the first patient post infusion of GS001, the dose of GS001 can be adjusted for the other two patients based on the activity level of FVIII:C and safety assessment. Following completion of the first 3 patients received GS001 infusion at 2 x 10^12 vg/kg dose level , the following 3 patients will continue to be enrolled to this dose group if there isn't any unexpected safety risk based on the assessments of FVIII expression and safety profile. The safety data and the activity level of FVIII:C from the first 6 subjects at 2 x 10^12 vg/kg dose level will undergo review by an independent DMC prior to dosing the first subject in the next dose level, DMC may recommend dose escalation to 6 x 10^12 vg/kg body weight or other recommended dose levels.
If the DMC recommends dose escalation to 6 x 10 ^12 vg/kg body weight or other recommended doses, at least 10 weeks of safety data and the activity level of FVIII:C post GS001 infusion from the first 3 subjects in this given dose level will undergo review by an independent DMC prior to dosing the following subjects. Based on the data from the first 3 subjects, DMC may recommend the dose adjustments for subsequent enrolled subjects, and expand the number of subject enrolled. Based on the safety, preliminary efficacy and vector kinetics profile of single intravenous infusions of GS001 at different dose levels in severe hemophilia A patients with endogenous factor FVIII activity levels ≤ 1% in this study, and based on the benefit-risk assessment of the subjects, the dose level to be administered for future larger clinical trial in patients with severe hemophilia A will be determined.
After all enrolled subjects have been followed for at least 12 weeks after intravenous infusion GS001, periodic analysis will be performed. Primary analysis of safety and efficacy will be performed 52 (± 2) weeks after intravenous infusion GS001, and all subjects will receive 52 (± 2) weeks of comprehensive safety and efficacy assessments. After completing the 52 (± 2) week visit (end-of-study visit), subjects will continue to be followed in this study for up to an additional 4 years for long-term safety and efficacy assessment to evaluate the long-term safety and efficacy of GS001 treatment.
A total of 12 to 15 subjects are expected to be enrolled in this study. Subjects will provide informed consent and then undergo screening assessments up to 4-8 weeks prior administration of GS001. All subjects will undergo 260 weeks (5 years) safety observation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment group | Experimental | Genetic: Injection of GS001Arm of GS001 |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Be able to understand the purpose and risks of the study and provide informed consent according to national and local privacy laws;
- Male subjects and ≥ 18 years of age;
- Have hemophilia A with ≤1 IU/dL (≤1%) endogenous FVIII activity levels at the time of screening. If the screening result is >1% due to previous treatment with FVIII product, then it may be confirmed by documented historical evidence from a certified clinical laboratory demonstrating ≤1% FVIII activity levels ;
- No history of hypersensitivity or anaphylaxis associated with FVIII product administration;
- Have no measurable FVII inhibitor as assessed by laboratory two times that were at least one week apart; or documented no prior history of FVIII inhibitor after 150 EDs and no clinical signs or symptoms of decreased response to FVIII infusion ;
Key exclusion criteria (12 listed)
- Have Hepatitis B, hepatitis C or HBsAg, HCVAb, HBV-DNA, HCV-RNA are positive and have clinical significance. Both natural clearers and those who have cleared HCV on antiviral therapy are deemed eligible;
- Currently Receiving antiviral therapy for hepatitis B and C;
- Have history of chronic infections or other chronic diseases that may pose a risk to the study participation;
- Have participated in a previous gene therapy research trial within the last 52 weeks or in a clinical study with an investigational drug within the past 30 days;
- The subject has any concurrent diseases that cannot tolerate treatments of prednisone or prednisolone as judged by the investigator;
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 |
|---|---|---|
| Injection of GS001Patients will be enrolled sequentially every 3 weeks or more between cohorts. Dose escalation may occur after a single patient has been safely dosed if the resulting FVIII activity at Week 3 is < 5 IU/dL.The dose levels are as follows: 1. 2×10^12 vg/kg 2. 6×10^12vg/kg or other recommended doses 3. 2×10^13 vg/kg or other recommended doses | Genetic | Treatment group |
Primary outcome
Incidence of treatment- related adverse events
Time frame: From screening through up to the end of study (about 5 years).
Number of patients experiencing treatment-related adverse events.
Percentage of subjects in each dose group with newly occurred clinically significant abnormalities in physical examination compared to the baseline.
Time frame: From the start of study treatment (Day 1) through up to the end of study (about 5 years).
The number of subjects in each dose group with clinically significant changes in physical examination compared to the baseline.
Changes of Weighted Mean of vital signs (systolic blood pressure [SBP] and diastolic blood pressure [DBP], pulse rate, temperature, respiratory rate) from baseline at each assessment time point for each dose group
Time frame: From the start of study treatment (Day 1) through up to the end of study (about 5 years).
The vital signs (SBP, DBP, pulse rate, temperature, respiratory rate) of the subjects were measured. The maximum, minimum, and mean observed values of vital signs (SBP, DBP, pulse rate, temperature, respiratory rate) from the dosing (Day 1) to the end of the study were calculated for each subject.
Changes of Mean Alkaline Phosphatase (ALP), Alanine Amino Transferase (ALT), Aspartate Amio Transferase (AST) from baseline at each assessment time point for each dose group
Time frame: From the start of study treatment (Day 1) through up to the end of study (about 5 years).
Blood samples of subjects were collected for the evaluation of liver function throughout this study. All of these parameters are measured to help assess the condition of the liver. For ALP, AST and ALT, the percentage of subjects with the worst post-treatment results in each dose group will be summarized as follows: > 1.0 x ULN ≤ 1.5x ULN; > 1.5 x ULN ≤ 3.0 x ULN; > 3.0 x ULN ≤ 5.0 x ULN; > 5.0 x ULN
Immune response to AAV capsid proteins
Time frame: From screening period through up to 5 years.
Changes in the expression levels of neutralizing and binding antibodies of AAV.
Immune response to FVIII transgene
Time frame: From screening period through up to 5 years.
The changes of FVIII inhibitor and antibody levels
Viral vector shedding of GS001
Time frame: From date of infusion until the date of 3 consecutive documented negative results, assessed up to 1 year.
The vector shedding in serum, PMBC, saliva, urine, semen and feces will be monitored
Thrombosis risk assessment
Time frame: From the start of study treatment (Day 1) through up to the end of study (about 5 years).
For any individual who reaches >150% vector-derived FVIII: C activity levels following the infusion of GS001, laboratory parameters of thrombotic potential will be assessed.
4 secondary outcomes
Vector- derived FVIII:C and FVIII antigen levels
Time frame: From pre-dose phase through up to 1 years post-dose
FVIII usage within 1 year after GS001 infusion
Time frame: From week 3 to week 52 post GS001 infusion.
Number of bleeding events requiring exogenous FVIII replacement therapy within 1 year after GS001 infusion
Time frame: Week 3 to Week 52 post GS001 Infusion
Number of bleeding events within 1 year after GS001 infusion
Time frame: Week 3 to Week 52 post GS001 infusion
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 1 Aug 2025.
- Derived from this study: PubMed 41813646Liu W, Pei X, Yu T, Xu B, Dai X, Xue F, Chen L, Wang X, Wang Y, Chen Y, Liu X, Fu R, Wang W, Chi Y, Li HY, Yang R, Zhang L. Phase 1 pilot study for hemophilia-A: AAV8 vector with prophylactic tacrolimus-glucocorticoid achieves therapeutic FVIII activity. Signal Transduct Target Ther. 2026 Mar 11;11(1):88. doi: 10.1038/s41392-026-02599-3.
- 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
Chinese Academy of Medical Science and Blood Disease Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Injection of GS001 in people with Hemophilia A and Gene Therapy, 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 7 inclusion and 12 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 1 Aug 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.
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 (+82223909240, liuwei1@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment- related adverse events (time frame: From screening through up to the end of study (about 5 years).). The study lists 8 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 NCT04728841, 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 1 Aug 2025.