| NCT ID | NCT03217032 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | Shenzhen Geno-Immune Medical Institute |
| Condition | Hemophilia A |
| Study Type | INTERVENTIONAL |
| Enrollment | 10 participants |
| Start Date | 2025-06-01 |
| Primary Completion | 2027-05-31 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 10 participants in total. It began in 2025-06-01 with a primary completion date of 2027-05-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.
Eligibility Criteria
Inclusion Criteria: 1. Able to provide informed consent and comply with requirements of the study. 2. Males ≥2 years with confirmed diagnosis of hemophilia A (endogenous factor VIII ≤2 IU/dL or ≤2% of normal). 3. A minimum average of 4 bleeding events per year requiring episodic treatment of factor VIII infusions or prophylactic factor VIII infusions. 4. No measurable factor VIII inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor VIII protein. 5. Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences. Exclusion Criteria: 1. Significant liver dysfunction as defined by abnormal alanine transaminase, bilirubin and alkaline phosphatase. 2. History of inhibitor against factor VIII. 3. Evidence of active hepatitis B or C and currently on antiviral therapy. 4. Have serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 (subjects who are HIV+ and stable with CD4 count \>200/mm3 and undetectable viral load are eligible to enroll). 5. Any evidence of active infection or any immunosuppressive disorder. 6. Participated in a gene transfer trial within the last 6 months or in a clinical trial with an investigational drug within the last 12 weeks. 7. Unable or unwilling to comply with study assessments.
Contact & Investigator
Lung-Ji Chang
PRINCIPAL INVESTIGATOR
Shenzhen Geno-Immune Medical Institute
Frequently Asked Questions
Who can join the NCT03217032 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, up to 65 Years, studying Hemophilia A. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT03217032 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT03217032 currently recruiting?
Yes, NCT03217032 is actively recruiting participants. Contact the research team at c@szgimi.org for enrollment information.
Where is the NCT03217032 trial being conducted?
This trial is being conducted at Shenzhen, China.
Who is sponsoring the NCT03217032 clinical trial?
NCT03217032 is sponsored by Shenzhen Geno-Immune Medical Institute. The principal investigator is Lung-Ji Chang at Shenzhen Geno-Immune Medical Institute. The trial plans to enroll 10 participants.