Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 3NCT06987864
An Extension Clinical Study of BCD-148 for the Treatment of Patients With PNH Previously Treated in Clinical Study No. BCD-148-2/NOCTURN
A Phase 3 study testing eculizumab in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Biocad at 11 sites in Russia.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- What is being tested?
- eculizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 11 sitesRussia
- Study dates
- 13 Apr 2020to 2 Nov 2022
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
Russia11 sites
Territorial State Budgetary Healthcare Institution "Territorial Clinical Hospital"
Barnaul
State Autonomous Healthcare Institution "S.V. Belyaev Kuzbass Regional Clinical Hospital"
Kemerovo
Federal State Budgetary Institution of Science "Kirov Research Institute of Hematology and Blood Transfusion of the Federal Medical and Biological Agency"
Kirov
Moscow State Budgetary Healthcare Institution "S.P. Botkin Municipal Clinical Hospital of the Moscow Healthcare Department"
Moscow
Federal State Budgetary Educational Institution of Higher Education "Rostov State Medical University" of the Ministry of Health of the Russian Federation
Rostov-on-Don
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
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
Federal State Budgetary Educational Institution of Higher Education "Samara State Medical University" of the Ministry of Health of the Russian Federation
Samara
State Institution "Komi Republican Cancer Clinic"
Syktyvkar
Federal State Budgetary Educational Institution of Higher Education "Bashkir State Medical University" of the Ministry of Health of the Russian Federation
Ufa
State Autonomous Healthcare Institution of the Sverdlovsk Region "Sverdlovsk Regional Clinical Hospital No. 1"
Yekaterinburg
About this study
Official title: An Extension Clinical Study of BCD-148 (JSC BIOCAD) for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria Previously Treated in Clinical Study No. BCD-148-2/NOCTURN
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
Read the detailed description
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.
- Being tested
- eculizumab
- Keywords
- Paroxysmal nocturnal hemoglobinuria, eculizumab, PNH
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
eculizumabBiological
Intravenous infusion of BCD-148 every 2 weeks
| Study group | Role | What they receive |
|---|---|---|
| To study the long-term safety and efficacy of BCD-148 in patients with paroxysmal nocturnal hemoglob | Experimental | Not described Receives: Biological: eculizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in lactate dehydrogenase (LDH) level from baseline estimated at Visit 1/Week 0 prior to drug administration
Measured: 105 weeks
Secondary outcomes (10)
Proportion of patients with thrombotic complications
Measured: 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
Measured: 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
The assessment of the quality of life of patients was carried out on the basis of the analysis of change in the mean score on the FACIT-Fatigue scale at Visits 1-14 (Weeks 0-104) compared to the baseline values. An increase in the score indicates a decrease in the patient's fatigue severity.
Measured: 105 weeks
Proportion of patients with adverse reactions (ARs), including those meeting the seriousness criteria
Measured: 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
Proportion of patients with grade 3, 4 or 5 ARs according to the CTCAE v.5.0
Measured: 105 weeks
Proportion of patients who prematurely withdrew from the study due to ARs
Measured: 105 weeks
Proportion of BAb (binding antibodies) and NAb (neutralizing antibodies)-positive patients
Measured: 105 weeks
Eculizumab Ctrough (steady-state eculizumab concentration at the end of the dosing interval, before the next scheduled dose, after multiple intravenous infusions of BCD-148)
Measured: 105 weeks
Emin of serum complement hemolytic activity and C5 level (minimum serum complement hemolytic activity and C5 levels after administration of BCD-148)
Measured: 105 weeks
Etrough of serum complement hemolytic activity and C5 level (the effect magnitude measured at the end of the dosing interval, before the next scheduled dose, at steady state after multiple intravenous infusions of BCD-148)
Measured: 105 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2025
- Study start13 Apr 2020
- Primary completion2 Nov 2022
- Study completion2 Nov 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)Studies of the same condition, open and under way.
- More trials run by BiocadThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biocad
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06987864
- Sponsor's protocol ID
- BCD-148-EXT
- Status last verified
- May 2025
- First submitted
- 16 May 2025
- Last updated
- 29 May 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Extension Clinical Study of BCD-148 (JSC BIOCAD) for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria Previously Treated in Clinical Study No. BCD-148-2/NOCTURN
- ClinicalTrials.gov ID
- NCT06987864
- Acronym / sponsor ID
- BCD-148-EXT
- Keywords
- Paroxysmal nocturnal hemoglobinuria, eculizumab, PNH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- BiocadIndustry (pharma, biotech, devices)
- Study start
- 13 Apr 2020 (actual)
- Primary completion
- 2 Nov 2022 (actual)
- Study completion
- 2 Nov 2022 (actual)
- First posted
- 23 May 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| To study the long-term safety and efficacy of BCD-148 in patients with paroxysmal nocturnal hemoglob | Experimental | Biological: eculizumab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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) .
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| eculizumabIntravenous infusion of BCD-148 every 2 weeks | Biological | To study the long-term safety and efficacy of BCD-148 in patients with paroxysmal nocturnal hemoglob |
Primary outcome
Change in lactate dehydrogenase (LDH) level from baseline estimated at Visit 1/Week 0 prior to drug administration
Time frame: 105 weeks
10 secondary outcomes
Proportion of patients with thrombotic complications
Time frame: 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
Time frame: 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
Time frame: 105 weeks
Proportion of patients with adverse reactions (ARs), including those meeting the seriousness criteria
Time frame: 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
Time frame: 105 weeks
Proportion of patients who prematurely withdrew from the study due to ARs
Time frame: 105 weeks
Proportion of BAb (binding antibodies) and NAb (neutralizing antibodies)-positive patients
Time frame: 105 weeks
Eculizumab Ctrough (steady-state eculizumab concentration at the end of the dosing interval, before the next scheduled dose, after multiple intravenous infusions of BCD-148)
Time frame: 105 weeks
Emin of serum complement hemolytic activity and C5 level (minimum serum complement hemolytic activity and C5 levels after administration of BCD-148)
Time frame: 105 weeks
Etrough of serum complement hemolytic activity and C5 level (the effect magnitude measured at the end of the dosing interval, before the next scheduled dose, at steady state after multiple intravenous infusions of BCD-148)
Time frame: 105 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing eculizumab in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Biocad at 11 sites in Russia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in lactate dehydrogenase (LDH) level from baseline estimated at Visit 1/Week 0 prior to drug administration (time frame: 105 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 May 2025.