Clinical study ANB-002-1/SAFRAN: An Open-Label, Non-Controlled, Single-Dose Escalation Study of the Safety, Pharmacodynamics, and Efficacy of ANB-002 in Patients with Hemophilia B.
The ANB-002-1/SAFRAN clinical trilal will evaluate the safety, pharmacodynamics, and efficacy of single escalating doses of ANB-002 in male patients who:
Are 18 years of age or older.
Have a confirmed diagnosis of hemophilia B: FIX1 activity ≤ 1% or > 1% to ≤ 2% with at least 3 episodes of spontaneous bleeding in the past 52 weeks at screening2.
Are negative for FIX inhibitors.
Have received FIX concentrates for at least 150 days before the screening.
This trial has exclusion criteria that make a patient ineligible, and investigators communicate the full list of which the researcher will inform the potential participant. The exclusion criteria include:
A history of prior use of any gene therapy.
Other blood disorders, except for hemophilia B.
Known allergy or intolerance to FIX concentrates and glucocorticoids.
A diagnosis of HIV infection (a patient with an established diagnosis of HIV infection receiving antiretroviral therapy may participate in the study in case of an undetectable viral load <50 copies/mL and a CD4+ cell count >200 cells×mm3).
A diagnosis of hepatitis B.
Acute or chronic hepatitis C (a patient without HCV RNA may participate in the study; a patient receiving antiviral therapy may participate in the study 12 weeks after completion of this therapy).
*All uncertain cases of HIV infection, hepatitis B and C will be discussed with the infectious disease specialist of the study to determine the possibility of participation of the patient.
A positive history of arterial or venous thrombosis.
Diseases accompanied by severe immunosuppression.
Alcohol or drug addiction.
If necessary, with approval of the research doctor and with consent of the patient, they can be provided with transport services for carrying out appropriate trips within the framework of participation in the Clinical trial, as well as with accommodation services. Compensation for transportation/ accommodation costs associated with participation in the Clinical trial of the investigational drug ANB-002 is provided by JSC BIOCAD.
The ANB-002-1/SAFRAN study is being conducted in study centers in the cities of the Russian Federation, including Moscow, St. Petersburg, Ufa, Nizhny Novgorod, Samara, Chelyabinsk, Kemerovo, Novosibirsk, Syktyvkar, and Kirov.
You can ask questions about participation in the clinical study and clarify contact information of the study centers via a request form a«Do you have any questions?».
Investigators will explain to potential participants the other eligibility criteria and contraindications for participation in this clinical study. The potential participant should make him/herself familiar with the study details and consult the investigator and/or treating physician.