NeuroblastomaPhase 1/2NCT03373097
Anti-GD2 CAR T Cells in Pediatric Patients Affected by High Risk and/or Relapsed/Refractory Neuroblastoma or Other GD2-positive Solid Tumors
A Phase 1/2 study testing GD2-CART01 in people with Neuroblastoma and Neuroblastoma Recurrent, run by Bambino Gesù Hospital and Research Institute at 1 site in Italy.
Trial summary
The purpose of this study is to test the safety and efficacy of GD2-CART01, a CAR T cell treatment targeting GD2 in paediatric or young adult patients with High Risk and/or relapsed/refractory Neuroblastoma. A small exploratory cohort of patients with GD2-positive tumors other than Neuroblastoma has also been included. Read more about this study
- Who is it for?
- Ages 12 months to 25 years
- Conditions the study is for
- Neuroblastoma, Neuroblastoma Recurrent, GD2-positive Solid Tumors
- What is being tested?
- GD2-CART01
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 1 siteItaly
- Study dates
- 5 Jan 2018to Dec 2027
- 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 12 months to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Phase I The patient must meet the following eligibility inclusion criteria to be enrolled to receive treatment in the Phase I study.
- Diagnosis of NBL that have been treated with frontline therapy and is judged to be incurable, based upon the following criteria:
- Relapse after first-line treatment, proved by a positive 123-I-mMIBG-scan
- Persistence/progression of disease after the initiation of the upfront treatment
- Patients must have measurable or evaluable disease at the time of treatment enrollment, as shown by bone marrow biopsy/aspirate, US or CT/MRI scan or by 123-I-mMIBG scan.
- Recover from the toxic effect of previous chemotherapies: grade 4 and or 3 non-hematologic toxicities must have resolved to grade ≤2; if some effects of the therapies have become chronic (i.e. treatment associated thrombocytopenia), the patient must be clinically stable, according to the opinion of the treating physicians, and meet all other eligibility criteria.
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- Age: 12 months -18 years.
- Voluntary informed consent is given. For subjects < 18 years old their legal guardian must give informed consent. Pediatric subjects will be included in age appropriate discussion and verbal assent will be obtained for those greater than or equal to 12 years of age, when appropriate.
- Clinical performance status: Patients > 16 years of age: Karnofsky greater than or equal to 60%; Patients less than or equal to 16 years of age: Lansky scale greater than or equal to 60%.
- Patients of child-bearing or child-fathering potential must be willing to practice birth control from the time of enrollment on this study and for four months after receiving the preparative regimen.
- Females of child-bearing potential must have a negative pregnancy test because of the potentially dangerous effects on the fetus.
- Phase II
- The patient must meet the following eligibility inclusion criteria to be enrolled to receive treatment in the Phase II study.
- Diagnosis of NBL that have been treated with frontline therapy and is judged to be incurable, based upon the following criteria:
- Relapse after first-line treatment, proved by a positive MIBG-scan
- Persistence/progression of disease after the initiation of the upfront treatment
- OR
- Diagnosis of extremely High Risk NBL at high risk of relapse, defined by stage III/IV and Myc-N amplification, at the end of the first-line treatment according to the Standard of Care, even if NED.
- OR
- Diagnosis of GD2+ tumors other than Neuroblastoma, considered incurable with conventional treatments by the treating physician.
- Patients with relapsed/refractory disease must have measurable or evaluable disease at the time of treatment enrollment, as shown by bone marrow biopsy/aspirate, US or CT/MRI scan or by MIBG-scan.
- Recover from the toxic effect of previous chemotherapies: grade 4 and or 3 non-hematologic toxicities must have resolved to grade ≤2; if some effects of the therapies have become chronic (i.e. treatment associated thrombocytopenia), the patient must be clinically stable, according to the opinion of the treating physicians, and meet all other eligibility criteria.
- Age: 12 months - 18 years.
- Voluntary informed consent is given. For subjects < 18 years old their legal guardian must give informed consent. Pediatric subjects will be included in age appropriate discussion and verbal assent will be obtained for those greater than or equal to 12 years of age, when appropriate.
- Clinical performance status: Patients > 16 years of age: Karnofsky greater than or equal to 60%; Patients less than or equal to 16 years of age: Lansky scale greater than or equal to 60%.
- Patients of child-bearing or child-fathering potential must be willing to practice birth control from the time of enrollment on this study and for four months after receiving the preparative regimen.
- Females of child-bearing potential must have a negative pregnancy test because of the potentially dangerous effects on the fetus
Who cannot join
- Pregnant or lactating women
- Severe, uncontrolled active intercurrent infections
- Active hepatitis B or hepatitis C infection
- HIV infection
- Rapidly progressive disease with life-expectancy < 6 weeks
- History of grade 3 or 4 hypersensitivity to murine protein-containing products
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- Hepatic function: Inadequate liver function defined as total bilirubin > 4x upper limit of normal (ULN) or transaminase (ALT and AST) > 6 x ULN based on age and laboratory specific normal ranges
- Renal function: serum creatinine > 3x ULN for age.
- Blood oxygen saturation < 90%.
- Cardiac function: Left ventricular ejection fraction lower than 45% by ECHO.
- Marrow function: ANC lower than 500/mm3 and/or platelets lower than 20.000 (not reached by transfusion).
- Congestive heart failure, cardiac arrhythmia, psychiatric illness, or social situations that would limit compliance with study requirements or in the opinion of the PI would pose an unacceptable risk to the subject.
- Untreated CNS metastasis; patients with previous CNS tumor involvement that has been treated and is stable for at least 6 weeks following completion of therapy are eligible.
- Concurrent or recent prior therapies, before infusion:
- Systemic steroids (at a dose equivalent to or greater 2 mg/kg prednisone) in the 2 weeks before infusion. Recent or current use of inhaled/topical/non-absorbable steroids is not exclusionary.
- Systemic chemotherapy in the 2 weeks preceding infusion.
- Immunosuppressive agents less than or equal to 30 days.
- Radiation therapy must have been completed at least 3 weeks prior to enrollment.
- I131-MIBG therapy must have been completes at least 6 weeks prior to enrollment
- Anti-GD2 murine monoclonal antibody (ch14.18 antibody) in the 2 weeks preceding infusion
- Other anti-neoplastic investigational agents currently or within 30 days prior to start of protocol therapy;
- Exceptions:
- Subjects receiving steroid therapy at physiologic replacement doses only are allowed provided there has been no increase in dose for at least 2 weeks prior to starting apheresis
- Patient-derived GD2-CART01 production failure.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I/II Study of Anti-GD2 Chimeric Antigen Receptor-Expressing T Cells in Pediatric Patients Affected by High Risk and/or Relapsed/Refractory Neuroblastoma or Other GD2-positive Solid Tumors
The purpose of this study is to test the safety and efficacy of GD2-CART01, a CAR T cell treatment targeting GD2 in paediatric or young adult patients with High Risk and/or relapsed/refractory Neuroblastoma.
A small exploratory cohort of patients with GD2-positive tumors other than Neuroblastoma has also been included.
Read the detailed description
The study will consist of 2 phases, a Phase I or dose escalation phase and a Phase II or expansion phase. Paediatric or young adult patients with relapsed High Risk and/or relapsed/refractory Neuroblastoma will be enrolled in the study.
After completion of the phase I portion of the study, a small cohort of patients with GD2-positive tumors other than Neuroblastoma has also been included.
Eligible patients will undergo leukapheresis in order to harvest T cells, which will be manufactured to obtain the autologous CAR T product GD2-CART01, a GD2-targeting CAR T product. Briefly, the patients will be treated with a lymphodepleting regimen containing conventional chemotherapic agents and subsequently will receive a single infusion of GD2-CART01. Moreover, the product contains a suicide gene safety switch (namely inducible Caspase 9): in case of relevant toxicities, the patient will receive the dimerizing agent in order to activate the apoptotic pathway in the infused T cells.
After infusion of CAR T cells, the patients will enter a 5-year active follow-up period.
- Conditions
- Neuroblastoma, Neuroblastoma Recurrent, GD2-positive Solid Tumors, Osteosarcoma, Ewing Sarcoma, Sarcoma
- Being tested
- GD2-CART01
- Keywords
- Neuroblastoma, GD2 CAR T cell, CAR T, GD2-positive solid tumors, Osteosarcoma, Ewing Sarcoma, GD2-positive sarcoma
- Medical subject headings
- Neuroblastoma, Osteosarcoma, Sarcoma, Ewing, Sarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GD2-CART01Biological
Following a lymphodepleting treatment with conventional chemotherapy, patients will be treated with 1.0 to 10.0 x 10⁶/kg GD2 Chimeric Antigen Receptor (CAR) positive T cells as a single dose.
| Study group | Role | What they receive |
|---|---|---|
| GD2-CART01 | Experimental | After a lymphodepleting regimen the patients will receive 1.0 to 10.0 x 10⁶/kg GD2 Chimeric Antigen Receptor (CAR) positive T cells. Receives: Biological: GD2-CART01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase I - Identification of the dose limiting toxicity (DLT)
Toxicity will be assessed according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) scale, version 4 and the number of patients experiencing DLT will be evaluated
Measured: 4 weeks after T cell infusion
Phase II - Antitumor effect
Assessment of Best Overall Response (BOR)
Measured: Up to 6 months after T cell infusion
Secondary outcomes (7)
In vivo persistence/expansion of infused CAR T cell
Detection of infused CAR T cell in the peripheral and bone marrow blood
Measured: UP to 5 years
Serum cytokine profiling
Assessment of the seric cytokines profile
Measured: First 2 weeks after T cell infusion
Time To Progression (TTP)
Measured: Up 5 years after T cell infusion
Event-Free Survival (EFS)
Measured: Up 5 years after T cell infusion
Overall Survival (OS)
Measured: Up 5 years after T cell infusion
Disease outcome according to INRC vs irRC
Tumor response assessment through the two different criteria
Measured: Up to 5 years
Elimination of CAR T cell through iC9 in case of toxicity
Assessment of the kinetic of CAR T cell elimination after infusion of the dimerizer
Measured: Up to 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Dec 2017
- Study start5 Jan 2018
- Primary completionDec 2026 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for NeuroblastomaStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bambino Gesù Hospital and Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Franco Locatelli, Bambino Gesù Hospital and Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03373097
- Sponsor's protocol ID
- GD2CAR01
- Status last verified
- Sep 2026
- First submitted
- 6 Dec 2017
- Last updated
- 30 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Locatelli F, Pagliara D, De Ioris MA, Becilli M, Del Baldo G, Serra A, Mastronuzzi A, Cefalo MG, Li Pira G, Leone G, Bertaina V, Fabozzi F, Di Nardo M, Rosignoli C, D'Andrea ML, Crocoli A, Vennarini S, Sinibaldi M, Di Cecca S, Guercio M, Iaffaldano L, Lucarelli B, Algeri M, Merli P, Colafati GS, De Angelis B, Quintarelli C, Del Bufalo F. GD2-targeting CAR T cells in high-risk neuroblastoma: a phase 1/2 trial. Nat Med. 2025 Nov;31(11):3689-3699. doi: 10.1038/s41591-025-03874-6. Epub 2025 Aug 21.
PubMed 40841488Yeku OO, Longo DL. CAR T Cells for Neuroblastoma. N Engl J Med. 2023 Apr 6;388(14):1328-1331. doi: 10.1056/NEJMe2300317. No abstract available.
PubMed 37018497Del Bufalo F, De Angelis B, Caruana I, Del Baldo G, De Ioris MA, Serra A, Mastronuzzi A, Cefalo MG, Pagliara D, Amicucci M, Li Pira G, Leone G, Bertaina V, Sinibaldi M, Di Cecca S, Guercio M, Abbaszadeh Z, Iaffaldano L, Gunetti M, Iacovelli S, Bugianesi R, Macchia S, Algeri M, Merli P, Galaverna F, Abbas R, Garganese MC, Villani MF, Colafati GS, Bonetti F, Rabusin M, Perruccio K, Folsi V, Quintarelli C, Locatelli F; Precision Medicine Team-IRCCS Ospedale Pediatrico Bambino Gesu. GD2-CART01 for Relapsed or Refractory High-Risk Neuroblastoma. N Engl J Med. 2023 Apr 6;388(14):1284-1295. doi: 10.1056/NEJMoa2210859.
PubMed 37018492Tumino N, Weber G, Besi F, Del Bufalo F, Bertaina V, Paci P, Quatrini L, Antonucci L, Sinibaldi M, Quintarelli C, Maggi E, De Angelis B, Locatelli F, Moretta L, Vacca P, Caruana I. Polymorphonuclear myeloid-derived suppressor cells impair the anti-tumor efficacy of GD2.CAR T-cells in patients with neuroblastoma. J Hematol Oncol. 2021 Nov 12;14(1):191. doi: 10.1186/s13045-021-01193-0.
PubMed 34772439
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Study of Anti-GD2 Chimeric Antigen Receptor-Expressing T Cells in Pediatric Patients Affected by High Risk and/or Relapsed/Refractory Neuroblastoma or Other GD2-positive Solid Tumors
- ClinicalTrials.gov ID
- NCT03373097
- Acronym / sponsor ID
- GD2CAR01
- Conditions
- Neuroblastoma, Neuroblastoma Recurrent, GD2-positive Solid Tumors, Osteosarcoma, Ewing Sarcoma, Sarcoma
- Keywords
- Neuroblastoma, GD2 CAR T cell, CAR T, GD2-positive solid tumors, Osteosarcoma, Ewing Sarcoma, GD2-positive sarcoma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Bambino Gesù Hospital and Research InstituteUniversity, hospital or other
- Study start
- 5 Jan 2018 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 14 Dec 2017 (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 purpose of this study is to test the safety and efficacy of GD2-CART01, a CAR T cell treatment targeting GD2 in paediatric or young adult patients with High Risk and/or relapsed/refractory Neuroblastoma.
A small exploratory cohort of patients with GD2-positive tumors other than Neuroblastoma has also been included.
Detailed description
The study will consist of 2 phases, a Phase I or dose escalation phase and a Phase II or expansion phase. Paediatric or young adult patients with relapsed High Risk and/or relapsed/refractory Neuroblastoma will be enrolled in the study.
After completion of the phase I portion of the study, a small cohort of patients with GD2-positive tumors other than Neuroblastoma has also been included.
Eligible patients will undergo leukapheresis in order to harvest T cells, which will be manufactured to obtain the autologous CAR T product GD2-CART01, a GD2-targeting CAR T product. Briefly, the patients will be treated with a lymphodepleting regimen containing conventional chemotherapic agents and subsequently will receive a single infusion of GD2-CART01. Moreover, the product contains a suicide gene safety switch (namely inducible Caspase 9): in case of relevant toxicities, the patient will receive the dimerizing agent in order to activate the apoptotic pathway in the infused T cells.
After infusion of CAR T cells, the patients will enter a 5-year active follow-up period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GD2-CART01 | Experimental | Biological: GD2-CART01After a lymphodepleting regimen the patients will receive 1.0 to 10.0 x 10⁶/kg GD2 Chimeric Antigen Receptor (CAR) positive T cells. |
Who may qualify
- Ages
- 12 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (27 listed)
- Phase I The patient must meet the following eligibility inclusion criteria to be enrolled to receive treatment in the Phase I study.
- Diagnosis of NBL that have been treated with frontline therapy and is judged to be incurable, based upon the following criteria:
- Patients must have measurable or evaluable disease at the time of treatment enrollment, as shown by bone marrow biopsy/aspirate, US or CT/MRI scan or by 123-I-mMIBG scan.
- Recover from the toxic effect of previous chemotherapies: grade 4 and or 3 non-hematologic toxicities must have resolved to grade ≤2; if some effects of the therapies have become chronic (i.e. treatment associated thrombocytopenia), the patient must be clinically stable, according to the opinion of the treating physicians, and meet all other eligibility criteria.
- Age: 12 months -18 years.
Key exclusion criteria (24 listed)
- Pregnant or lactating women
- Severe, uncontrolled active intercurrent infections
- Active hepatitis B or hepatitis C infection
- HIV infection
- Rapidly progressive disease with life-expectancy < 6 weeks
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 |
|---|---|---|
| GD2-CART01Following a lymphodepleting treatment with conventional chemotherapy, patients will be treated with 1.0 to 10.0 x 10⁶/kg GD2 Chimeric Antigen Receptor (CAR) positive T cells as a single dose. | Biological | GD2-CART01 |
Primary outcome
Phase I - Identification of the dose limiting toxicity (DLT)
Time frame: 4 weeks after T cell infusion
Toxicity will be assessed according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) scale, version 4 and the number of patients experiencing DLT will be evaluated
Phase II - Antitumor effect
Time frame: Up to 6 months after T cell infusion
Assessment of Best Overall Response (BOR)
7 secondary outcomes
In vivo persistence/expansion of infused CAR T cell
Time frame: UP to 5 years
Serum cytokine profiling
Time frame: First 2 weeks after T cell infusion
Time To Progression (TTP)
Time frame: Up 5 years after T cell infusion
Event-Free Survival (EFS)
Time frame: Up 5 years after T cell infusion
Overall Survival (OS)
Time frame: Up 5 years after T cell infusion
Disease outcome according to INRC vs irRC
Time frame: Up to 5 years
Elimination of CAR T cell through iC9 in case of toxicity
Time frame: Up to 15 years
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 Sep 2026.
- Derived from this study: PubMed 40841488Locatelli F, Pagliara D, De Ioris MA, Becilli M, Del Baldo G, Serra A, Mastronuzzi A, Cefalo MG, Li Pira G, Leone G, Bertaina V, Fabozzi F, Di Nardo M, Rosignoli C, D'Andrea ML, Crocoli A, Vennarini S, Sinibaldi M, Di Cecca S, Guercio M, Iaffaldano L, Lucarelli B, Algeri M, Merli P, Colafati GS, De Angelis B, Quintarelli C, Del Bufalo F. GD2-targeting CAR T cells in high-risk neuroblastoma: a phase 1/2 trial. Nat Med. 2025 Nov;31(11):3689-3699. doi: 10.1038/s41591-025-03874-6. Epub 2025 Aug 21.
- Derived from this study: PubMed 37018497Yeku OO, Longo DL. CAR T Cells for Neuroblastoma. N Engl J Med. 2023 Apr 6;388(14):1328-1331. doi: 10.1056/NEJMe2300317. No abstract available.
- Derived from this study: PubMed 37018492Del Bufalo F, De Angelis B, Caruana I, Del Baldo G, De Ioris MA, Serra A, Mastronuzzi A, Cefalo MG, Pagliara D, Amicucci M, Li Pira G, Leone G, Bertaina V, Sinibaldi M, Di Cecca S, Guercio M, Abbaszadeh Z, Iaffaldano L, Gunetti M, Iacovelli S, Bugianesi R, Macchia S, Algeri M, Merli P, Galaverna F, Abbas R, Garganese MC, Villani MF, Colafati GS, Bonetti F, Rabusin M, Perruccio K, Folsi V, Quintarelli C, Locatelli F; Precision Medicine Team-IRCCS Ospedale Pediatrico Bambino Gesu. GD2-CART01 for Relapsed or Refractory High-Risk Neuroblastoma. N Engl J Med. 2023 Apr 6;388(14):1284-1295. doi: 10.1056/NEJMoa2210859.
- Derived from this study: PubMed 34772439Tumino N, Weber G, Besi F, Del Bufalo F, Bertaina V, Paci P, Quatrini L, Antonucci L, Sinibaldi M, Quintarelli C, Maggi E, De Angelis B, Locatelli F, Moretta L, Vacca P, Caruana I. Polymorphonuclear myeloid-derived suppressor cells impair the anti-tumor efficacy of GD2.CAR T-cells in patients with neuroblastoma. J Hematol Oncol. 2021 Nov 12;14(1):191. doi: 10.1186/s13045-021-01193-0.
- Individual participant dataSharing planned: Undecided
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing GD2-CART01 in people with Neuroblastoma and Neuroblastoma Recurrent, run by Bambino Gesù Hospital and Research Institute at 1 site in Italy.
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 27 inclusion and 24 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 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. 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?
Phase I - Identification of the dose limiting toxicity (DLT) (time frame: 4 weeks after T cell infusion). 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 NCT03373097, which also lists 4 linked publications.
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 Sep 2026.