Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT06185335

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

18 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital", Chelyabinsk, Russia

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Male subjects aged ≥18 years at the time of signing the informed consent form.
  • Established diagnosis of hemophilia A with a documented history of endogenous FVIII activity ≤1% AND ≤2% at screening.
  • Therapy with FVIII concentrates for at least 150 exposure days.

Exclusion criteria

  • History of use of any gene therapy product.
  • Use of emicizumab within less than 6 months before the date of signing the ICF.
  • The presence of other blood or hematopoietic disorders other than hemophilia A.
  • Presence of AAV6 antibodies detected by ELISA.
  • BMI <16 kg/m² or ≥35 kg/m².
  • Diagnosis of HIV infection.
  • HBV infection.
  • HCV infection.
  • Any active systemic infections or recurrent infections requiring systemic therapy at screening.
  • Any other disorders associated with severe immunodeficiency.
  • Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder.
  • Malignancies with remission duration of less than 5 years at the time of signing the ICF, except for cured basal cell carcinoma.

Treatment and study plan

ANB-010, dose 1

Genetic

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

ANB-010, dose 2

Genetic

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

ANB-010, dose 3

Genetic

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

Primary outcomes

  1. Change in FVIII activity from baseline to Week 52

    Time frame: 12 months

  2. Assessment of ANB-010 safety

    Time frame: 12 months

    Proportion and characteristics of adverse events

Secondary outcomes

  1. Change in FVIII activity from baseline to scheduled assessment visits

    Time frame: 12 months

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

  2. Proportion of subjects achieving clinical response

    Time frame: 12 months

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

  3. Proportion of subjects who achieved normalized response

    Time frame: 12 months

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

  4. Annualized consumption of FVIII concentrates by a subject

    Time frame: 12 months

  5. Annualized bleeding rate

    Time frame: 12 months

  6. Annualized rate of bleeding requiring therapy with FVIII concentrates

    Time frame: 12 months

  7. Duration of response based on activity FVIII

    Time frame: 12 months

Other outcomes

  1. Pharmacodynamics of ANB-010

    Time frame: 12 months

    Evaluation of peak and steady-state FVIII concentrations

  2. Proportion of subjects with FVIII inhibitor

    Time frame: 12 months

  3. Proportion of subjects with antibodies to capsid

    Time frame: 12 months

  4. Proportion of subjects with anti-FVIII antibodies

    Time frame: 12 months

  5. Proportion of subjects with T cells specific to AAV6 and FVIII transgene product

    Time frame: 12 months

  6. ANB-010 biodistribution (in blood, saliva, urine, semen and feces)

    Time frame: 12 months

  7. Annualized rate of spontaneous bleeding

    Time frame: 12 months

  8. Annualized rate of intraarticular bleeding

    Time frame: 12 months

  9. Annualized rate of trauma-related bleeding

    Time frame: 12 months

  10. Change from baseline in the quality of life measured with Haemo-A-QoL

    Time frame: 12 months

    The assessment will be provided at scheduled assessment visits (if the scale is available in the CS).

  11. Change from baseline in the quality of life measured with EuroQol-5D-3L

    Time frame: 12 months

    The assessment will be provided at scheduled assessment visits (if the scale is available in the CS).

  12. Change from baseline in the quality of life measured with SF-36

    Time frame: 12 months

    The assessment will be provided at scheduled assessment visits (if the scale is available in the CS).

  13. Change from baseline in the assessment on the Health Needs Questionnaire for Adults with Hemophilia A at scheduled assessment visits

    Time frame: 12 months

    The assessment is be performed if the scales are available in the CS.

  14. Joint assessment based on HJHS v.2.1

    Time frame: 12 months

    The assessment is be performed if the scales are available in the CS.

Sponsors and collaborators

Lead sponsor

Biocad

Industry

Registry information

Official study title

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

Acronym: EDELWEISS

Important dates

Study start
2023
Primary completion
2025
Study completion
2033
First posted
Dec 29, 2023
Registry last updated
Apr 24, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.