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

NCT06987864 | Completed Phase 3

• 1 other identifier: BCD-148-EXT
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 11

Brief Summary

To study the long-term safety and efficacy of BCD-148 in patients with paroxysmal nocturnal hemoglobinuria (PNH), previously treated in clinical study (CS) BCD-148-2/NOCTURN

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Keywords

Paroxysmal nocturnal hemoglobinuria; eculizumab; PNH

Outcome Measures

Primary Outcomes (1)

• Change in lactate dehydrogenase (LDH) level from baseline estimated at Visit 1/Week 0 prior to drug administration

  105 weeks

Secondary Outcomes (10)

• Proportion of patients with thrombotic complications

  105 weeks

• Change in the size of the PNH clone (change in the number of circulating red blood cells with the PNH phenotype) from baseline estimated at Visit 1/Week 0 prior to drug administration

  105 weeks

• Change in the mean Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score from baseline as assessed at Visit 1/Week 0 prior to drug administration

  105 weeks

• Proportion of patients with adverse reactions (ARs), including those meeting the seriousness criteria

  105 weeks

• Proportion of patients with grade 3 or higher adverse reactions (ARs) according to the Common Toxicity Criteria for Adverse Events (CTCAE) version 5.0

  105 weeks

Study Arms (1)

To study the long-term safety and efficacy of BCD-148 in patients with paroxysmal nocturnal hemoglob

EXPERIMENTAL

Biological: eculizumab

Interventions

eculizumab BIOLOGICAL

Intravenous infusion of BCD-148 every 2 weeks

To study the long-term safety and efficacy of BCD-148 in patients with paroxysmal nocturnal hemoglob

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Biocad: lead

Study Sites (11)

Territorial State Budgetary Healthcare Institution "Territorial Clinical Hospital"

Barnaul, Russia

Location

State Autonomous Healthcare Institution "S.V. Belyaev Kuzbass Regional Clinical Hospital"

Kemerovo, Russia

Location

Federal State Budgetary Institution of Science "Kirov Research Institute of Hematology and Blood Transfusion of the Federal Medical and Biological Agency"

Kirov, Russia

Location

Moscow State Budgetary Healthcare Institution "S.P. Botkin Municipal Clinical Hospital of the Moscow Healthcare Department"

Moscow, Russia

Location

Federal State Budgetary Educational Institution of Higher Education "Rostov State Medical University" of the Ministry of Health of the Russian Federation

Rostov-on-Don, Russia

Location

Federal State Budgetary Educational Institution of Higher Education "Academician I.P. Pavlov First St. Petersburg State Medical University" of the Ministry of Health of the Russian Federation

Saint Petersburg, Russia

Location

Federal State Budgetary Educational Institution of Higher Education "I.I. Mechnikov North-Western State Medical University" of the Ministry of Health of the Russian Federation

Saint Petersburg, Russia

Location

Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Health of the Russian Federation

Samara, Russia

Location

State Institution "Komi Republican Cancer Clinic"

Syktyvkar, Russia

Location

Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Health of the Russian Federation

Ufa, Russia

Location

State Autonomous Healthcare Institution of the Sverdlovsk Region "Sverdlovsk Regional Clinical Hospital No. 1"

Yekaterinburg, Russia

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 16, 2025
• First Posted: May 23, 2025
• Study Start: April 13, 2020
• Primary Completion: November 2, 2022
• Study Completion: November 2, 2022
• Last Updated: May 29, 2025

Record last verified: 2025-05

Locations

RU (11)