Spinal Muscular Atrophy (SMA)Phase 1/2BLUEBELLNCT05747261
Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)
A Phase 1/2 study testing ANB-004, dose 1, ANB-004, dose 2 and ANB-004, dose 3 in people with Spinal Muscular Atrophy (SMA), run by Biocad at 7 sites in 2 countries.
Trial summary
The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular atrophy. The study will have a standard 3+3 dose-escalation design. Read more about this study
- Who is it for?
- Ages Up to 240 days
- Conditions the study is for
- Spinal Muscular Atrophy (SMA)
- What is being tested?
- ANB-004, dose 1, ANB-004, dose 2 and ANB-004, dose 3
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 2 Feb 2023to Aug 2030
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 Up to 240 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Informed consent form for participation in the study signed by the subject's legal representative;
- Subjects of either sex under the age of 240 days at the time of signing the Information Sheet for the Legal Representative of the Clinical Study Subject with Informed Consent Form;
- A diagnosis of 5q-SMA (homozygous deletion of exon 7 of the SMN1 gene or heterozygous deletion of exon 7 + confirmed point mutation of the SMN1 gene) and 2 or 3 copies of the SMN2 gene established based on molecular genetic testing;
- Subjects with 2 copies of the SMN2 gene can be included in the study both at the presymptomatic stage of the disease and in the presence of SMA symptoms. If symptoms are present, the age of onset of the disease should be up to 180 days from birth.
- Subjects with 3 copies of the SMN2 gene can be included in the study if they have symptoms of SMA type 1 and the disease began before the age of 180 days.
- The ability of the subject's legal representative, in the Investigator's opinion, to perceive information and follow the Protocol procedures
Who cannot join
- A diagnosis of HIV infection, hepatitis B, hepatitis C, congenital syphilis in the study subject, as well as a documented diagnosis of HIV infection in the study subject's mother. Note: documented hepatitis B and/or hepatitis C and/or syphilis in the mother of a study subject is not an exclusion criterion in this clinical study, provided that standard breastfeeding rules are followed or the subject is not breastfed due to the low risk of transmission of hepatitis B and C viruses and Treponema pallidum from mother to child with breast milk;
- Unwillingness of the legal representative to use alternative feeding methods (nasogastric tube, gastrostomy) in case of swallowing disorders and a risk of aspiration;
- Anti-AAV9 antibody titer >1:50 determined by ELISA. Note: if a subject's screening anti-AAV9 antibody titer is >1:50, the anti-AAV9 antibody titer may be determined again. Subjects with anti-AAV9 antibody titers ≤1:50 in the second test may be included in the study;
- Need for respiratory support for ≥16 hours per day or tracheostomy ;
- Treatment with nusinersen, risdiplam, branaplam, onasemnogene abeparvovec or other antisense oligonucleotides/selective SMN2 splicing modifiers or gene therapy drugs for SMN1 transduction or other AAV-based gene therapy drugs regardless of serotype used previously (from birth) or planned for the main study period, i.e., within 12 months after the administration of the investigational product.
- A need to use any medications for the treatment of myopathy or neuropathy, drugs for the treatment of diabetes, ongoing immunosuppressive therapy, or the need for immunosuppressive therapy after the start of the study (for example, glucocorticoids (except for premedication and post-medication), cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab, etc.);
Show all 17Show fewer
- Subjects with the following laboratory test results at screening:
- increased activity of transaminases (ALT, AST) or GGT >2×ULN;
- total bilirubin level ≥34 µmol/L;
- creatinine level ≥160 µmol/L;
- hemoglobin <80 g/L and >180 g/L;
- WBC count >20x109/L;
- Troponin I level > ULN.
- Any concomitant diseases that, in the Investigator's opinion, may affect the safety of ANB-004 in the subject or have a significant impact on the assessment of the outcomes of SMA therapy;
- A diagnosis of acute or chronic hepatic failure at screening;
- A known allergy or intolerance to any components of the investigational product or pre- and post-medication drug (glucocorticoids);
- Simultaneous participation of the subject in other clinical studies or previous participation in another clinical study using an experimental therapy.
Trial locations and contacts
7 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 2 countries
Russia6 sites, 6 recruiting
- Recruiting
Federal State Autonomous Educational Institution of Higher Education "N.I. Pirogov Russian National Research Medical University", Ministry of Health of the Russian Federation
Moscow, 117997
Dmitry Vlodavets, mityaus@gmail.com
- Recruiting
Federal State Autonomous Educational Institution of Higher Education "N.I. Pirogov Russian National Research Medical University", Ministry of Health of the Russian Federation
Moscow, 117997
Svetlana Mikhailova, svetychvital@mail.ru
- Recruiting
Federal State Autonomous Institution "National Medical Research Center for Children's Health", Ministry of Health of the Russian Federation
Moscow, 119991
Ludmila Kuzenkova, l.kuzenkova@list.ru
- Recruiting
Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State Pediatric Medical University", Ministry of Health of the Russian Federation
Saint Petersburg, 194100
Valentina Guzeva, viktoryka@mail.ru
- Recruiting
Federal State Budgetary Institution "V. A. Almazov National Medical Research Center" of the Ministry of Health of the Russian Federation
Saint Petersburg, 197341
Natalia Petrova, natalja5@yandex.ru
- Recruiting
State Autonomous Healthcare Institution of the Sverdlovsk Region "Regional Children's Clinical Hospital"
Yekaterinburg, 620149
Olga Lvova, olvova@bk.ru
Belarus1 site, 1 recruiting
- Recruiting
1. State Institution Republican Scientific and Practical Center "Mother and Child"
Minsk
Irina V Zhevneronok, irynazhauniaronak@yandex.ru
About this study
Official title: An Open-Label, Non-Comparative Clinical Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene (ANB-004 (JSC BIOCAD, Russia)) After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy
The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular atrophy. The study will have a standard 3+3 dose-escalation design.
Read the detailed description
The study will be conducted in 2 stages:
Stage 1: pilot efficacy and safety study of different doses to select a potentially therapeutic dose for further study.
Stage 2: study of the efficacy and safety of ANB-004 at the selected potentially therapeutic dose.
At the first stage, the study will have a "3+3" design and involve dose escalation in two cohorts, with the possibility of including a third cohort. Three subjects are to be included in each cohort, each of whom will receive a pre-specified cohort dose of ANB-004 as a single intravenous infusion.
Subjects will be monitored for dose-limiting toxicity (DLT) events for 3 weeks after the drug infusion. In this study, DLT will include any of the CTCAE 5.0 grade 3 or higher adverse events (AEs) at least possibly related to the administration of the investigational product, except for an increase in body temperature at least possibly related to the administration of the investigational product, which will be classified as DLT starting from CTCAE 5.0 grade 4.
At the first stage, in the absence of DLT events in three subjects in the same cohort, an ID will be assigned, and an infusion will be administered to the first subject in subsequent cohort. If DLT events occur in 1 of 3 subjects in the same cohort, this cohort will additionally include 3 subjects who will receive the same ANB-004 dose with which the DLT event was observed. If DLT events occur in 2 or more of 3 subjects within the same cohort, the assignment of ID/infusion in subsequent subjects/study is suspended.
AT the second stage, if the IDMC judges that the potentially therapeutic dose was previously used in one of the cohorts, an additional 6 subjects will be included in this cohort.
- Conditions
- Spinal Muscular Atrophy (SMA)
- Being tested
- ANB-004, dose 1, ANB-004, dose 2, ANB-004, dose 3
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ANB-004, dose 1Genetic
Adeno-associated viral vector carrying the SMN gene single infusion at dose 1. The duration of the infusion is about 60 minutes.
ANB-004, dose 2Genetic
Adeno-associated viral vector carrying the SMN gene single infusion at dose 2. The duration of the infusion is about 60 minutes.
ANB-004, dose 3Genetic
Adeno-associated viral vector carrying the SMN gene single infusion at dose 3. The duration of the infusion is about 60 minutes.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Subjects in Cohort 1 will receive ANB-004 at a dose 1. Depending on the DLT, the cohort may include 1 to 6 subjects in the first stage and 9 to 12 in the second stage. Receives: Genetic: ANB-004, dose 1 |
| Cohort 2 | Experimental | Subjects in Cohort 2 will receive ANB-004 at a dose 2. The dose for Cohort 2 will be determined at the IDMC meeting. Depending on the DLT, the cohort may include 1 to 6 subjects in the first stage and 9 to 12 in the second stage. Receives: Genetic: ANB-004, dose 2 |
| Cohort 3 | Experimental | Subjects of Cohort 3, if included, will receive the drug at a dose 3. The dose for Cohort 3 will be determined at the IDMC meeting. Depending on the DLT, the cohort may include 1 to 6 subjects in the first stage and 9 to 12 in the second stage. Receives: Genetic: ANB-004, dose 3 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects with adverse reactions
Measured: 12 months
Proportion of subjects with serious adverse reactions
Measured: 12 months
Proportion of subjects with CTCAE 5.0 or DAIDS grade 3 or higher adverse reactions
Since some of the severity criteria used in the CTCAE are not applicable to the pediatric population, it is proposed to use the 2017 Division of AIDS (DAIDS) Table for Grading the Severity of Adult and Pediatric Adverse Events of the National Institute of Allergy and Infectious Diseases.
Measured: 12 months
Time from date of birth to event
Time from date of birth to onset of a fatal event or the need for invasive respiratory support using tracheostomy or the need for non-invasive respiratory support for at least 16 hours a day for ≥14 consecutive days (in the absence of acute reversible disease and excluding surgery).
Measured: 12 months
Motor development score
The assessment will be made using the motor development scale of a healthy child, which reflects the motor development milestones from birth to a certain age.
Measured: 12 months
Change in the The Hammersmith Infant Neurological Examination (HINE) score
To assess the motor skills of study subjects, Section 2 of this scale will be used; the assessment will be made in subjects aged 3 to 24 months.
Measured: 12 months
Secondary outcomes (2)
Event occurrence
No fatal event, no need for invasive respiratory support using tracheostomy or non-invasive respiratory support for at least 16 hours a day for ≥14 consecutive days (in the absence of acute reversible disease and excluding surgery).
Measured: 12 months
Documented evidence of efficacy
Changes in functional and motor abilities: percentage and age of children who can turn over and sit without support for more than 5, 10, 20 and 30 seconds on video records obtained during a visit to the study center and/or by a legal representative of the study participant outside the study center.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2023
- Study start2 Feb 2023
- Primary completionSep 2025 (estimated)
- Study completionAug 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular Atrophy (SMA)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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- "3+3" design
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Biocad
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Arina V Zinkina-Orikhan, PhD, Study director, Director of Clinical Development Department, BIOCAD
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05747261
- Sponsor's protocol ID
- ANB-004-1
- Acronym
- BLUEBELL
- Status last verified
- Feb 2024
- First submitted
- 14 Feb 2023
- Last updated
- 22 Feb 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Non-Comparative Clinical Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene (ANB-004 (JSC BIOCAD, Russia)) After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT05747261
- Acronym / sponsor ID
- BLUEBELL / ANB-004-1
- Conditions
- Spinal Muscular Atrophy (SMA)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- BiocadIndustry (pharma, biotech, devices)
- Study start
- 2 Feb 2023 (actual)
- Primary completion
- Sep 2025 (estimated)
- Study completion
- Aug 2030 (estimated)
- First posted
- 28 Feb 2023 (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.
The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular atrophy. The study will have a standard 3+3 dose-escalation design.
Detailed description
The study will be conducted in 2 stages:
Stage 1: pilot efficacy and safety study of different doses to select a potentially therapeutic dose for further study.
Stage 2: study of the efficacy and safety of ANB-004 at the selected potentially therapeutic dose.
At the first stage, the study will have a "3+3" design and involve dose escalation in two cohorts, with the possibility of including a third cohort. Three subjects are to be included in each cohort, each of whom will receive a pre-specified cohort dose of ANB-004 as a single intravenous infusion.
Subjects will be monitored for dose-limiting toxicity (DLT) events for 3 weeks after the drug infusion. In this study, DLT will include any of the CTCAE 5.0 grade 3 or higher adverse events (AEs) at least possibly related to the administration of the investigational product, except for an increase in body temperature at least possibly related to the administration of the investigational product, which will be classified as DLT starting from CTCAE 5.0 grade 4.
At the first stage, in the absence of DLT events in three subjects in the same cohort, an ID will be assigned, and an infusion will be administered to the first subject in subsequent cohort. If DLT events occur in 1 of 3 subjects in the same cohort, this cohort will additionally include 3 subjects who will receive the same ANB-004 dose with which the DLT event was observed. If DLT events occur in 2 or more of 3 subjects within the same cohort, the assignment of ID/infusion in subsequent subjects/study is suspended.
AT the second stage, if the IDMC judges that the potentially therapeutic dose was previously used in one of the cohorts, an additional 6 subjects will be included in this cohort.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- "3+3" design
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Genetic: ANB-004, dose 1Subjects in Cohort 1 will receive ANB-004 at a dose 1. Depending on the DLT, the cohort may include 1 to 6 subjects in the first stage and 9 to 12 in the second stage. |
| Cohort 2 | Experimental | Genetic: ANB-004, dose 2Subjects in Cohort 2 will receive ANB-004 at a dose 2. The dose for Cohort 2 will be determined at the IDMC meeting. Depending on the DLT, the cohort may include 1 to 6 subjects in the first stage and 9 to 12 in the second stage. |
| Cohort 3 | Experimental | Genetic: ANB-004, dose 3Subjects of Cohort 3, if included, will receive the drug at a dose 3. The dose for Cohort 3 will be determined at the IDMC meeting. Depending on the DLT, the cohort may include 1 to 6 subjects in the first stage and 9 to 12 in the second stage. |
Who may qualify
- Ages
- Up to 240 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Informed consent form for participation in the study signed by the subject's legal representative;
- Subjects of either sex under the age of 240 days at the time of signing the Information Sheet for the Legal Representative of the Clinical Study Subject with Informed Consent Form;
- A diagnosis of 5q-SMA (homozygous deletion of exon 7 of the SMN1 gene or heterozygous deletion of exon 7 + confirmed point mutation of the SMN1 gene) and 2 or 3 copies of the SMN2 gene established based on molecular genetic testing;
- Subjects with 2 copies of the SMN2 gene can be included in the study both at the presymptomatic stage of the disease and in the presence of SMA symptoms. If symptoms are present, the age of onset of the disease should be up to 180 days from birth.
- Subjects with 3 copies of the SMN2 gene can be included in the study if they have symptoms of SMA type 1 and the disease began before the age of 180 days.
Key exclusion criteria (17 listed)
- A diagnosis of HIV infection, hepatitis B, hepatitis C, congenital syphilis in the study subject, as well as a documented diagnosis of HIV infection in the study subject's mother. Note: documented hepatitis B and/or hepatitis C and/or syphilis in the mother of a study subject is not an exclusion criterion in this clinical study, provided that standard breastfeeding rules are followed or the subject is not breastfed due to the low risk of transmission of hepatitis B and C viruses and Treponema pallidum from mother to child with breast milk;
- Unwillingness of the legal representative to use alternative feeding methods (nasogastric tube, gastrostomy) in case of swallowing disorders and a risk of aspiration;
- Anti-AAV9 antibody titer >1:50 determined by ELISA. Note: if a subject's screening anti-AAV9 antibody titer is >1:50, the anti-AAV9 antibody titer may be determined again. Subjects with anti-AAV9 antibody titers ≤1:50 in the second test may be included in the study;
- Need for respiratory support for ≥16 hours per day or tracheostomy ;
- Treatment with nusinersen, risdiplam, branaplam, onasemnogene abeparvovec or other antisense oligonucleotides/selective SMN2 splicing modifiers or gene therapy drugs for SMN1 transduction or other AAV-based gene therapy drugs regardless of serotype used previously (from birth) or planned for the main study period, i.e., within 12 months after the administration of the investigational product.
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 |
|---|---|---|
| ANB-004, dose 1Adeno-associated viral vector carrying the SMN gene single infusion at dose 1. The duration of the infusion is about 60 minutes. | Genetic | Cohort 1 |
| ANB-004, dose 2Adeno-associated viral vector carrying the SMN gene single infusion at dose 2. The duration of the infusion is about 60 minutes. | Genetic | Cohort 2 |
| ANB-004, dose 3Adeno-associated viral vector carrying the SMN gene single infusion at dose 3. The duration of the infusion is about 60 minutes. | Genetic | Cohort 3 |
Primary outcome
Proportion of subjects with adverse reactions
Time frame: 12 months
Proportion of subjects with serious adverse reactions
Time frame: 12 months
Proportion of subjects with CTCAE 5.0 or DAIDS grade 3 or higher adverse reactions
Time frame: 12 months
Since some of the severity criteria used in the CTCAE are not applicable to the pediatric population, it is proposed to use the 2017 Division of AIDS (DAIDS) Table for Grading the Severity of Adult and Pediatric Adverse Events of the National Institute of Allergy and Infectious Diseases.
Time from date of birth to event
Time frame: 12 months
Time from date of birth to onset of a fatal event or the need for invasive respiratory support using tracheostomy or the need for non-invasive respiratory support for at least 16 hours a day for ≥14 consecutive days (in the absence of acute reversible disease and excluding surgery).
Motor development score
Time frame: 12 months
The assessment will be made using the motor development scale of a healthy child, which reflects the motor development milestones from birth to a certain age.
Change in the The Hammersmith Infant Neurological Examination (HINE) score
Time frame: 12 months
To assess the motor skills of study subjects, Section 2 of this scale will be used; the assessment will be made in subjects aged 3 to 24 months.
2 secondary outcomes
Event occurrence
Time frame: 12 months
Documented evidence of efficacy
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Feb 2024.
- Individual participant dataSharing planned: No
Related trials for Spinal Muscular Atrophy (SMA)
Active, not recruitingPhase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Muscular Atrophy, Spinal
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingObservational study
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
Spinal Muscle Atrophy
RecruitingPhase 3
A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)
Metastatic Castration Resistant Prostate Cancer
Recruiting
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth
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.
Arina V Zinkina-Orikhan, PhD study director
Director of Clinical Development Department, BIOCAD
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ANB-004, dose 1, ANB-004, dose 2 and ANB-004, dose 3 in people with Spinal Muscular Atrophy (SMA), run by Biocad at 7 sites in 2 countries.
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 17 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: looking for participants now, as of the sponsor's last update on 22 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Russia, Belarus. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Maria Morozova, MD, PhD (+7 (985) 910 28 13, morozovama@biocad.ru), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of subjects with adverse reactions (time frame: 12 months). The study lists 6 primary outcomes in all. 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 22 Feb 2024.