GM1 GangliosidosisPhase 1/2Imagine-1NCT04713475
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
A Phase 1/2 study testing PBGM01 in people with GM1 Gangliosidosis and GM1 Gangliosidosis, Type I, run by Gemma Biotherapeutics at 7 sites in 4 countries.
Trial summary
PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues. This study will assess in a 2 part design the safety, tolerability and efficacy of PBGM01 in patients with early onset infantile (Type 1) and late onset infantile (Type 2a) GM1… Read more about this study
- Who is it for?
- Ages 1 month to 24 months
- Conditions the study is for
- GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2
- What is being tested?
- PBGM01
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 7 sitesin 4 countries
- Study dates
- 17 Mar 2021to Feb 2029
- 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 1 month to 24 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- All Patients: Documented GM1 gangliosidosis diagnosis based on genotyping confirming 2 mutations in the GLB1 gene and documented deficiency of beta-galactosidase enzyme by laboratory testing.
- Early onset infantile (Type 1) must be ≥1 month and <12 months of age at enrollment and have signs and/or symptoms of GM1 gangliosidosis that started before 6 months of age with specific minimum developmental milestones remaining.
- Late onset infantile (Type 2a) must be ≥6 months and ≤24 months of age at enrollment and have signs and/or symptoms of GM1 gangliosidosis that started between 6 and 18 months of age with specific minimum developmental milestones remaining including the ability to sit independently at screening as defined by the WHO Multicenter Growth Reference Study (WHO-MGRS) criteria of being able to sit up unsupported with head erect for at least 10 seconds.
Who cannot join
- Any clinically significant neurocognitive deficit not attributable to GM1 gangliosidosis or any other condition that may, in the opinion of the investigator, confound interpretation of study results.
- If a subject had an acute illness requiring hospitalization within 30 days of enrollment, the history must be discussed with the sponsor's medical monitor before allowing the subject to be enrolled.
- History of ventilation assisted respiratory support or a need for tracheostomy as a result of their disease. Chronic ventilatory support is defined as use of invasive or noninvasive (BiPAP) mechanical ventilation. Note: This does not exclude participants who use respiratory vests or noninvasive (BiPAP) mechanical ventilation for obstructive sleep apnea regardless of cause for less than 12 hours per day.
- Intractable seizure or uncontrolled epilepsy defined as having had an episode of status epilepticus, or seizures requiring hospitalization within 30 days prior to dosing of PBGM01. Note: This does not exclude participants who have a history of staring spells that have not been associated with EEG findings.
- Any contraindication to the ICM administration procedure, including contraindications to fluoroscopic imaging and anesthesia or any condition that would increase the risk of adverse outcomes from the ICM procedure including, but not limited to, the presence of a space occupying lesion causing mass effects or signs of increased intracranial pressure, space occupying lesion in the posterior fossa or foramen magnum, aberrant vascular anatomy such as a large midline posterior inferior cerebellar artery, aberrant venous anatomy such as a large cerebellar vein or occipital sinus, or congenital anatomical abnormalities such as a Chiari malformation.
- Any contraindication to MRI or lumbar puncture (LP).
Show all 18Show fewer
- Prior gene therapy.
- Use of miglustat within 48 hours prior to dosing of PBGM01. The use of miglustat is prohibited throughout the study.
- Use of enzyme replacement therapy or other investigational therapy within 5 half-lives prior to dosing of PBGM01. The use of enzyme replacement is prohibited throughout the study.
- Receipt of a vaccine within 14 days prior to dosing and/or scheduled vaccine within 30 days after dosing.
- Estimate glomerular filtration rate (eGFR) <30 mL/minute based on creatinine
- Coagulopathy (INR > 1.5) or activated partial thromboplastin time [aPTT] > 40 seconds
- Thrombocytopenia (platelet count < 100,000 per μL.
- AST or ALT > 3 times the upper limit of normal (ULN) or total bilirubin > 1.5x ULN
- Cardiomyopathy (screening troponin level above the ULN).
- Peripheral neuropathy
- Medical conditions or laboratory or vital sign abnormalities that would increase risk of complications from intra-cisterna magna injection, anesthesia, fluoroscopy, LP, and/or MRI including temperature over 38°C, oxygen saturation below 95% on room air or baseline oxygen requirement, heart rate or respiratory rate abnormal for age of the subject, abnormal blood pressure for age, or evidence of infection.
- Any condition (e.g., history of any disease, evidence of any current disease, any finding upon physical examination, or any laboratory abnormality) that, in the opinion of the investigator, would put the subject at undue risk during the administration procedure or would interfere with evaluation of PBGM01 or interpretation of subject safety or study results.
Trial locations and contacts
7 sites in 4 countries.
7 sites in 4 countries
United States4 sites
University of Minnesota
Minneapolis, Minnesota, 55455
Children's Hospital at St. Peter's University Hospital
New Brunswick, New Jersey, 08901
Benioff Children's Hospital
Oakland, California, 94158
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Pediatric Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis
PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues. This study will assess in a 2 part design the safety, tolerability and efficacy of PBGM01 in patients with early onset infantile (Type 1) and late onset infantile (Type 2a) GM1 gangliosidosis
Read the detailed description
GM1 gangliosidosis (GM1) is an autosomal recessive disorder that results from mutations in the galactosidase beta 1 gene (GLB1), which encodes beta-galactosidase (β-gal). β-gal is a lysosomal enzyme that catalyzes the first step in the degradation of GM1 ganglioside and keratan sulfate, and GM1 patients carry GLB1 alleles that produce little or no residual β-gal activity. PBGM01 is an adeno-associated viral vector serotype hu68 carrying GLB1, the gene encoding for human beta-galactosidase, formulated as a solution for injection into the cisterna magna. This is a global interventional, multicenter, single-arm, dose escalation, adaptive design study of PBGM01 delivered as a one-time dose administered into the cisterna magna to patients with early onset infantile (Type 1) and late onset infantile (Type 2a) GM1 gangliosidosis.
In Part 1 of the study, the dose-escalation phase will assess three dose levels of PBGM01 as a one-time dose in six independent cohorts of patients with either Type 1 or Type 2a GM1 gangliosidosis. The cohorts for patients with Type 1 and Type 2a will be assessed independently from each other.
Part 2 of the study will test the safety and efficacy of PBGM01 in confirmatory cohorts for Types 1 and Type 2a GM1 gangliosidosis with a dose chosen based on the data obtained in part 1 of the study. This will be a 2-year study with a 3-year safety extension.
- Conditions
- GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
- Being tested
- PBGM01
- Keywords
- Infantile, Late Infantile, Rare disease, Lysosomal storage disease
- Medical subject headings
- Gangliosidosis, GM1, Rare Diseases, Lysosomal Storage Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PBGM01Biological
AAVhu68 viral vector
| Study group | Role | What they receive |
|---|---|---|
| Part 1: Dose I of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01 | Experimental | Assigned Intervention:
PBGM01 Dose I: 3.3 x 10^10 GC/g estimated brain weight, single dose of PBGM01 via intra cisterna magna in Late Onset Infantile GM1 Gangliosidosis (Type 2a) and Early Onset Infantile GM1 Gangliosidosis (Type 1) Receives: Biological: PBGM01 |
| Part 1: Dose II of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01 | Experimental | Assigned Intervention:
PBGM01 Dose II: 1.1 x 10^11 GC/g estimated brain weight, single dose of PBGM01 via intra cisterna magna in Late Onset Infantile GM1 Gangliosidosis (Type 2a) and Early Onset Infantile GM1 Gangliosidosis (Type 1) Receives: Biological: PBGM01 |
| Part 1: Dose III of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01 | Experimental | Assigned Intervention:
PBGM01 Dose III: 2.2 x 10^11 GC/g estimated brain weight, single dose of PBGM01 via intra cisterna magna in Late Onset Infantile GM1 Gangliosidosis (Type 2a) and Early Onset Infantile GM1 Gangliosidosis (Type 1) Receives: Biological: PBGM01 |
| Part 2: Expansion Cohort designed to confirm the safety and efficacy of PBGM01 | Experimental | Confirmatory Cohorts: Late Onset Infantile GM1 Gangliosidosis (Type 2a) and Early Onset Infantile GM1 Gangliosidosis (Type 1)
Assigned Intervention:
PBGM01 Single dose of PBGM01, via intra cisterna magna Dose to be determined Receives: Biological: PBGM01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Treatment Related AEs and SAEs as Characterized by CTCAEv5.0
Assess the number of treatment-related adverse events (AEs) and serious adverse events (SAEs) as characterized by CTCAEv5.0
Measured: Up to 5 years (multiple visits)
Change from Baseline in Developmental Milestones as Assessed by the Bayley Scale of Infant and Toddler Development, Third Edition
Assess changes in the developmental age and the total number of developmental milestones using the Bayley Scale of Infant and Toddler Development, Third Edition instrument
Measured: From baseline to 2 years (multiple visits)
Secondary outcomes (7)
Change from Baseline in Developmental Milestones as Assessed by the Vineland Adaptive Behavior Scale-II
Assess changes in the developmental age and the total number of developmental milestones using the Vineland Adaptive Behavior Scale-II instrument
Measured: From baseline to 2 years (multiple visits)
Change in Baseline in Biomarkers of Beta-Galactosidase Activity in Blood and CSF
Assess change in biomarkers of beta-galactosidase activity including enzyme activity in blood and CSF
Measured: From baseline to 2 years (multiple visits)
Change in Baseline in Biomarkers of Beta-Galactosidase Substrates in Blood and CSF
Assess change in concentration of beta-galactosidase substrates including GM1 ganglioside in blood and CSF
Measured: From baseline to 2 years (multiple visits)
Change in Concentration of Biomarker of Disease Progression in Plasma and CSF
Assess change in neurofilament light chain (NfL) concentration as a marker for neurodegeneration and disease progression in plasma and CSF
Measured: From baseline to 2 years (multiple visits)
Change in Brain Anatomy as Assessed by MRI
Assess change in disease severity, volumetric MRI measures, brain diffusivity, and white matter lesions by MRI imaging
Measured: From baseline to 2 years (multiple visits)
Change in Quality of Life Using Pediatric Quality of Life Scales
Assess change in quality of life as measured by Pediatric Quality of Life Scale (Peds QL) and the Pediatric Quality of Life-Infant Scale (PedsQL-IS)
Measured: From baseline to 2 years (multiple visits)
Change in Ventilator-Free Survival Compared with Natural History Data
Assess change compared with natural history data in ventilator-free survival and chronic ventilatory support
Measured: From baseline to 2 years (multiple visits)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2021
- Study start17 Mar 2021
- Primary completionFeb 2028 (estimated)
- Study completionFeb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for GM1 GangliosidosisStudies of the same condition, open and under way.
- 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
- Open-label, multi-center, dose escalation
- Masking
- None (open label)
- Enrolment
- 26 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Gemma Biotherapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- May Orfali, MD, Study director, Gemma Biotherapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04713475
- Sponsor's protocol ID
- PBGM01-001
- Other IDs
- 2020-001109-22
- Acronym
- Imagine-1
- Status last verified
- Jun 2026
- First submitted
- 4 Jan 2021
- Last updated
- 30 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Zhang X, Brind'Amour K, King KE, Hartmaier S, Harris K, Weinstein DA, Girman CJ. Strategy for Generating Blinded Evidence for Single-Arm Trials with External Controls Using Expert Review of Home Video. Ther Innov Regul Sci. 2023 Nov;57(6):1304-1313. doi: 10.1007/s43441-023-00568-4. Epub 2023 Aug 17.
PubMed 37592153
Study at a glance
From the registry record, last updated by the sponsor on 30 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Pediatric Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis
- ClinicalTrials.gov ID
- NCT04713475
- Acronym / sponsor ID
- Imagine-1 / PBGM01-001
- Conditions
- GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
- Keywords
- Infantile, Late Infantile, Rare disease, Lysosomal storage disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 26 participants (estimated)
- Lead sponsor
- Gemma BiotherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 17 Mar 2021 (actual)
- Primary completion
- Feb 2028 (estimated)
- Study completion
- Feb 2029 (estimated)
- First posted
- 19 Jan 2021 (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.
PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues. This study will assess in a 2 part design the safety, tolerability and efficacy of PBGM01 in patients with early onset infantile (Type 1) and late onset infantile (Type 2a) GM1 gangliosidosis
Detailed description
GM1 gangliosidosis (GM1) is an autosomal recessive disorder that results from mutations in the galactosidase beta 1 gene (GLB1), which encodes beta-galactosidase (β-gal). β-gal is a lysosomal enzyme that catalyzes the first step in the degradation of GM1 ganglioside and keratan sulfate, and GM1 patients carry GLB1 alleles that produce little or no residual β-gal activity. PBGM01 is an adeno-associated viral vector serotype hu68 carrying GLB1, the gene encoding for human beta-galactosidase, formulated as a solution for injection into the cisterna magna. This is a global interventional, multicenter, single-arm, dose escalation, adaptive design study of PBGM01 delivered as a one-time dose administered into the cisterna magna to patients with early onset infantile (Type 1) and late onset infantile (Type 2a) GM1 gangliosidosis.
In Part 1 of the study, the dose-escalation phase will assess three dose levels of PBGM01 as a one-time dose in six independent cohorts of patients with either Type 1 or Type 2a GM1 gangliosidosis. The cohorts for patients with Type 1 and Type 2a will be assessed independently from each other.
Part 2 of the study will test the safety and efficacy of PBGM01 in confirmatory cohorts for Types 1 and Type 2a GM1 gangliosidosis with a dose chosen based on the data obtained in part 1 of the study. This will be a 2-year study with a 3-year safety extension.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- Open-label, multi-center, dose escalation
| Arm | Type | Receives |
|---|---|---|
| Part 1: Dose I of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01 | Experimental | Biological: PBGM01Assigned Intervention: PBGM01 Dose I: 3.3 x 10^10 GC/g estimated brain weight, single dose of PBGM01 via intra cisterna magna in Late Onset Infantile GM1 Gangliosidosis (Type 2a) and Early Onset Infantile GM1 Gangliosidosis (Type 1) |
| Part 1: Dose II of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01 | Experimental | Biological: PBGM01Assigned Intervention: PBGM01 Dose II: 1.1 x 10^11 GC/g estimated brain weight, single dose of PBGM01 via intra cisterna magna in Late Onset Infantile GM1 Gangliosidosis (Type 2a) and Early Onset Infantile GM1 Gangliosidosis (Type 1) |
| Part 1: Dose III of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01 | Experimental | Biological: PBGM01Assigned Intervention: PBGM01 Dose III: 2.2 x 10^11 GC/g estimated brain weight, single dose of PBGM01 via intra cisterna magna in Late Onset Infantile GM1 Gangliosidosis (Type 2a) and Early Onset Infantile GM1 Gangliosidosis (Type 1) |
| Part 2: Expansion Cohort designed to confirm the safety and efficacy of PBGM01 | Experimental | Biological: PBGM01Confirmatory Cohorts: Late Onset Infantile GM1 Gangliosidosis (Type 2a) and Early Onset Infantile GM1 Gangliosidosis (Type 1) Assigned Intervention: PBGM01 Single dose of PBGM01, via intra cisterna magna Dose to be determined |
Who may qualify
- Ages
- 1 month to 24 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- All Patients: Documented GM1 gangliosidosis diagnosis based on genotyping confirming 2 mutations in the GLB1 gene and documented deficiency of beta-galactosidase enzyme by laboratory testing.
- Early onset infantile (Type 1) must be ≥1 month and <12 months of age at enrollment and have signs and/or symptoms of GM1 gangliosidosis that started before 6 months of age with specific minimum developmental milestones remaining.
- Late onset infantile (Type 2a) must be ≥6 months and ≤24 months of age at enrollment and have signs and/or symptoms of GM1 gangliosidosis that started between 6 and 18 months of age with specific minimum developmental milestones remaining including the ability to sit independently at screening as defined by the WHO Multicenter Growth Reference Study (WHO-MGRS) criteria of being able to sit up unsupported with head erect for at least 10 seconds.
Key exclusion criteria (18 listed)
- Any clinically significant neurocognitive deficit not attributable to GM1 gangliosidosis or any other condition that may, in the opinion of the investigator, confound interpretation of study results.
- If a subject had an acute illness requiring hospitalization within 30 days of enrollment, the history must be discussed with the sponsor's medical monitor before allowing the subject to be enrolled.
- History of ventilation assisted respiratory support or a need for tracheostomy as a result of their disease. Chronic ventilatory support is defined as use of invasive or noninvasive (BiPAP) mechanical ventilation. Note: This does not exclude participants who use respiratory vests or noninvasive (BiPAP) mechanical ventilation for obstructive sleep apnea regardless of cause for less than 12 hours per day.
- Intractable seizure or uncontrolled epilepsy defined as having had an episode of status epilepticus, or seizures requiring hospitalization within 30 days prior to dosing of PBGM01. Note: This does not exclude participants who have a history of staring spells that have not been associated with EEG findings.
- Any contraindication to the ICM administration procedure, including contraindications to fluoroscopic imaging and anesthesia or any condition that would increase the risk of adverse outcomes from the ICM procedure including, but not limited to, the presence of a space occupying lesion causing mass effects or signs of increased intracranial pressure, space occupying lesion in the posterior fossa or foramen magnum, aberrant vascular anatomy such as a large midline posterior inferior cerebellar artery, aberrant venous anatomy such as a large cerebellar vein or occipital sinus, or congenital anatomical abnormalities such as a Chiari malformation.
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 |
|---|---|---|
| PBGM01AAVhu68 viral vector | Biological | Part 1: Dose I of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01, Part 1: Dose II of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01, Part 1: Dose III of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01, Part 2: Expansion Cohort designed to confirm the safety and efficacy of PBGM01 |
Primary outcome
Number of Participants with Treatment Related AEs and SAEs as Characterized by CTCAEv5.0
Time frame: Up to 5 years (multiple visits)
Assess the number of treatment-related adverse events (AEs) and serious adverse events (SAEs) as characterized by CTCAEv5.0
Change from Baseline in Developmental Milestones as Assessed by the Bayley Scale of Infant and Toddler Development, Third Edition
Time frame: From baseline to 2 years (multiple visits)
Assess changes in the developmental age and the total number of developmental milestones using the Bayley Scale of Infant and Toddler Development, Third Edition instrument
7 secondary outcomes
Change from Baseline in Developmental Milestones as Assessed by the Vineland Adaptive Behavior Scale-II
Time frame: From baseline to 2 years (multiple visits)
Change in Baseline in Biomarkers of Beta-Galactosidase Activity in Blood and CSF
Time frame: From baseline to 2 years (multiple visits)
Change in Baseline in Biomarkers of Beta-Galactosidase Substrates in Blood and CSF
Time frame: From baseline to 2 years (multiple visits)
Change in Concentration of Biomarker of Disease Progression in Plasma and CSF
Time frame: From baseline to 2 years (multiple visits)
Change in Brain Anatomy as Assessed by MRI
Time frame: From baseline to 2 years (multiple visits)
Change in Quality of Life Using Pediatric Quality of Life Scales
Time frame: From baseline to 2 years (multiple visits)
Change in Ventilator-Free Survival Compared with Natural History Data
Time frame: From baseline to 2 years (multiple visits)
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 30 Jun 2026.
- Derived from this study: PubMed 37592153Zhang X, Brind'Amour K, King KE, Hartmaier S, Harris K, Weinstein DA, Girman CJ. Strategy for Generating Blinded Evidence for Single-Arm Trials with External Controls Using Expert Review of Home Video. Ther Innov Regul Sci. 2023 Nov;57(6):1304-1313. doi: 10.1007/s43441-023-00568-4. Epub 2023 Aug 17.
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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.
May Orfali, MD study director
Gemma Biotherapeutics
7 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PBGM01 in people with GM1 Gangliosidosis and GM1 Gangliosidosis, Type I, run by Gemma Biotherapeutics at 7 sites in 4 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 3 inclusion and 18 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: under way, not looking for participants, as of the sponsor's last update on 30 Jun 2026. 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 United States, Brazil, Turkey (Türkiye), United Kingdom. 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?
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?
Number of Participants with Treatment Related AEs and SAEs as Characterized by CTCAEv5.0 (time frame: Up to 5 years (multiple visits)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04713475, which also lists 1 linked publication.
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 30 Jun 2026.