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An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B

ConditionHemophilia B
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexMale
Age18+
SponsorBiocad

About this trial

The aim of the study is to demonstrate non-inferiority of ANB-002 compared with preventive use of coagulation factor IX (FIX) in adult subjects with hemophilia B with FIX activity ≤2% and without FIX inhibitor. The study will have an open-label single-arm design.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Men diagnosed with hemophilia B aged 18 or older

FIX activity ≤2%

Absense of FIX inhibitor

≥150 previous exposure days of treatment with FIX concentrates

Disqualifiers

Any diseases of blood and hematopoietic organs other than hemophilia B

A history of any gene therapy, including ANB-002

Diagnosed HIV-infection, not controlled with anti-viral therapy

Active HBV or HCV infection

Trial design

Design model

Single group

Treatments tested in this trial

  • ANB-002

    Genetic

    Adeno-associated viral vector carrying the FIX gene single infusion

Treatment groups

24 Participants
are divided into 2 treatment groups
Group A: Lead-inNo intervention 0 interventions
Group B: ANB-002 (arvenacogene sanparvovec)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Annualized bleeding rate (ABR) before and after a single dose of ANB-002

A comparison will be made between the mean ABR values in the lead-in period and within 12 months after stabilization of endogenous FIX activity, but not later than 18 months after the ANB-002 dose

Time frame
Lead-in period: at least 6 months prior to ANB-002 infusion (Lead-in arm); Month 12 to Month 18 post infusion (ANB-002 arm)

Secondary outcomes

1

FIX activity

Time frame
18 months after drug administration; final assessment - 5 years
2

Annualized FIX consumption

Time frame
18 months after drug administration; final assessment - 5 years
3

Proportion of subjects not receiving FIX prevention

Time frame
18 months after drug administration
4

Annualized bleeding rate (ABR)

Time frame
18 months after drug administration; final assessment - 5 years

Other outcomes

1

Proportion of subjects with adverse reactions

Time frame
18 months after drug administration; final assessment - 5 years
2

Haemo-A-QoL (Hemophilia-Specific Quality of Life) score

Haemo-A-QoL is a quality of life (QoL) assessment instrument for patients with haemophilia, including the domains of consequences of bleeding, emotional impact, physical functioning, role functioning, treatment concern, and worry. Using scale overall score ranges from 0 to 100.The high score represent low quality of life.

Time frame
18 months after drug administration; final assessment - 5 years
3

EuroQol-5D-3L (European Quality of Life Questionnaire) score

The EQ-5D-5L descriptive system of health-related QoL states consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression). The EQ-5D-5L VAS overall score ranges from 0 to 100. A higher score is considered to be more favorable.

Time frame
18 months after drug administration; final assessment - 5 years
4

SF-36 (Short Form-36) score

SF-36 is a questionnaire for evaluating health-related QoL (Quality of Life). The 36 questions are meant to reflect 8 domains of health, including physical functioning, physical role, pain, general health, vitality, social function, emotional role, and mental health. SF-3 overall score ranges from 0 to 100. The lower the score the more disability

Time frame
18 months after drug administration; final assessment - 5 years

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Biocad

Lead sponsor

Source ClinicalTrials.gov
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