An Extension Clinical Study of BCD-148 for the Treatment of Patients With PNH Previously Treated in Clinical Study No. BCD-148-2/NOCTURN

The clinical study BCD-148-EXT was an extension of a multicenter, randomized, open-label, comparative study of the efficacy and safety of BCD-148 in PNH patients versus SOLIRIS® (BCD-148-2/NOCTURN).

Clinical study BCD-148-EXT is a Phase III study extension conducted after completion of BCD-148 900 mg therapy or SOLIRIS® 900 mg by subjects of clinical study BCD-148-2/NOCTURN.

In the BCD-148-EXT clinical study patients continued BCD-148 maintenance therapy for 105 weeks.

The BCD-148-EXT study consisted of two periods: screening period (not more than 7 days inclusive, i.e. 1 week from signing the patient information sheet and IC form) and the treatment period (Weeks 0 to 105 of the study, inclusive). As part of the treatment period, patients received intravenous infusions of the investigational drug BCD-148 in the maintenance therapy regimen.

Inclusion criteria

• Signed IC form for participation in the study.
• Indications for pathogenesis-based therapy of PNH with an anti-C5 monoclonal antibody (eculizumab).
• Consent for revaccination against Neisseria meningitidis .
• Negative pregnancy test for urine human chorionic gonadotropin (HCG) in women at enrollment in study BCD-148-EXT (the test was not performed in women who have been postmenopausal for at least 2 years, or in surgically sterile subjects) .
• Willingness of patients and their sexual partners of childbearing potential to use reliable contraceptive measures from signing the IC form throughout the study and for 4 weeks after the last drug dosing in the clinical study. This requirement did not apply to patients who underwent surgical sterilization and women who have been postmenopausal for over 2 years. Reliable methods of contraception included the use by partners of one barrier method in combination with one of the following: spermicides, intrauterine device, oral contraceptives (for female partners of male subjects only, for female study participants - upon agreement with the Sponsor and with justification by the investigator) .
• The ability of the patient to comply with the Protocol requirements, in the Investigator's opinion.

Exclusion criteria

• Patient withdrawal from clinical study BCD-148-2/NOCTURN until its completion for any reason.
• Pregnancy or breastfeeding, or planning of pregnancy by a female participant/planning conceiving a child by a male participant during the clinical study.
• Any conditions that, in the investigator's opinion, could increase the patient's risk associated with participation in the study or could affect the assessment of the study results.
• Hypersensitivity to BCD-148 components, murine proteins, and other components of the products according to the patient's history; hypersensitivity to any component of the meningococcal vaccine.
• Known alcohol or drug addiction, or signs of alcohol/drug addiction, which, according to the investigator, were a contraindication for the treatment with the study drug or limited the treatment compliance.