A Trial of the Safety and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A

Phase 2Active Not RecruitingNCT06185335

Biocad50 enrolled

Overview

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.

The study will be conducted in 2 stages: Stage 1: pilot efficacy and safety study of different doses to select a potentially therapeutic dose for further study. Stage 2: study of the efficacy and safety of ANB-010 at the selected potentially therapeutic dose. The stage 1 design is typical of phase I clinical trials with a modified "3+3" design and dose escalation. Three subjects are to be sequentially included in each cohort, each of whom will recieved a pre-specified cohort dose of ANB-010 as a single inravenous infusion. Subjects will be monitored for dose-limiting toxicity (DLT) events for 4 weeks after the drug infusion. The decision concerning dose escalation will be made at the Independent Data Monitoring Committee (IDMC) meetings. At the second stage the main study period will include 6 subjects who will receive ANB-010 at the optimal dose selected based on the results of stage 1 data analysis.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hemophilia A

Interventions

ANB-010, dose 1GENETIC

Adeno-associated viral vector carrying the FVIII gene single infusion at dose 1.

ANB-010, dose 2GENETIC

Adeno-associated viral vector carrying the FVIII gene single infusion at dose 2.

ANB-010, dose 3GENETIC

Adeno-associated viral vector carrying the FVIII gene single infusion at dose 3.

Eligibility

Sex: MALEMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (17)

State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital"

Outcomes

Primary Outcomes

Change in FVIII activity from baseline to Week 52

Time frame: 12 months

Assessment of ANB-010 safety

Proportion and characteristics of adverse events

Time frame: 12 months

Secondary Outcomes

Change in FVIII activity from baseline to scheduled assessment visits

FVIII activity will be assessed at every scheduled visits and compared to baseline

Time frame: 12 months

Proportion of subjects achieving clinical response

Clinical response is formulated as FVIII activity of 5-150%

Time frame: 12 months

Proportion of subjects who achieved normalized response

Normalized response is formulated as FVIII activity of 50-150%

Time frame: 12 months

Annualized consumption of FVIII concentrates by a subject

Time frame: 12 months

Annualized bleeding rate

Time frame: 12 months

Annualized rate of bleeding requiring therapy with FVIII concentrates

Time frame: 12 months

Duration of response based on activity FVIII

Time frame: 12 months

Data from ClinicalTrials.gov

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