Clinical trial
An Open-Label Two-Stage Trial of the Safety, Pharmacodynamics, Biodistribution, Immunogenicity and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A
Name
ANB-010-1
Description
The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-010 in subjects with hemophilia A. The study will have a dose-escalation design with elements of phase I/II seamless adaptive design.
Trial arms
Trial start
2023-07-26
Estimated PCD
2025-12-01
Trial end
2033-06-01
Status
Recruiting
Phase
Early phase I
Treatment
ANB-010, dose 1
Adeno-associated viral vector carrying the FVIII gene single infusion at dose 1.
Arms:
Cohort 1
ANB-010, dose 2
Adeno-associated viral vector carrying the FVIII gene single infusion at dose 2.
Arms:
Cohort 2
ANB-010, dose 3
Adeno-associated viral vector carrying the FVIII gene single infusion at dose 3.
Arms:
Cohort 3
Size
50
Primary endpoint
Change in FVIII activity from baseline to Week 52
12 months
Assessment of ANB-010 safety
12 months
Eligibility criteria
Inclusion Criteria:
1. Male subjects aged ≥18 years at the time of signing the informed consent form.
3. Established diagnosis of hemophilia A with a documented history of endogenous FVIII activity ≤1% AND ≤2% at screening.
4. Therapy with FVIII concentrates for at least 150 exposure days.
Exclusion Criteria:
1. History of use of any gene therapy product.
2. Use of emicizumab within less than 6 months before the date of signing the ICF.
3. The presence of other blood or hematopoietic disorders other than hemophilia A.
4. Presence of AAV6 antibodies detected by ELISA.
5. BMI \<16 kg/m² or ≥35 kg/m².
6. Diagnosis of HIV infection.
7. HBV infection.
8. HCV infection.
9. Any active systemic infections or recurrent infections requiring systemic therapy at screening.
10. Any other disorders associated with severe immunodeficiency.
11. Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder.
12. Malignancies with remission duration of less than 5 years at the time of signing the ICF, except for cured basal cell carcinoma.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 50, 'type': 'ESTIMATED'}}
Updated at
2023-12-29
1 organization
1 product
1 indication
Product
ANB-010Indication
Hemophilia AOrganization
Biocad