NeuroblastomaPhase 2NB2013-HRNCT02641782
NB2013-HR German (GPOH) / Dutch (DCOG) Trial
A Phase 2 study testing antibody ch14.18, GM-CSF and IL-2 i.v. in people with Neuroblastoma, run by University of Cologne at 1 site in Germany.
Trial summary
Although the five year survival rate of children with high risk neuroblastoma have increased over the last three decades from 4 to 44 % (1), neuroblastoma is the second most frequent cause for cancer related death in childhood (11 %). Read more about this study
- Who is it for?
- Ages 18 months to 25 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- antibody ch14.18, GM-CSF and IL-2 i.v.
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteGermany
- Study dates
- Nov 2015to 30 Jan 2017
- Phase
- Phase 2
- 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 months to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Established diagnosis of neuroblastoma according to the international criteria (INSS)
- High risk (HR): stage 4 over 18 months of age and oncogen MYCN (MYCN) amplified neuroblastoma of any stage and any age until 25 years (recurrent disease (Germany and The Netherlands) after re-induction chemotherapy (+/- other modalities) or newly diagnosed disease (only The Netherlands):
- Complete front-line treatment including induction chemotherapy, radioisotope (mIBG) treatment, appropriate local therapy such as surgical removal and/ or local irradiation of the primary tumor and myeloablative chemotherapy with autologous stem cell reinfusion according to the actual guidelines of the GPOH/DCOG
- achieved response status: stable disease or better (CR, VGPR, PR, SD).
- Written informed consent of parents or guardian and - if appropriate - of the patient.
- For at least two weeks prior to start of trial medication off any standard or experimental treatment no tumour surgery no immediate requirements for palliative chemotherapy, radiotherapy or surgery
Show all 10Show fewer
- The patient may have had prior central nervous system (CNS) metastases provided the following criteria are all met:
- The patient's CNS disease has been previously treated The patient's CNS disease has been clinically stable for four weeks prior to starting this study (assessed clinically and by MRI or CT) The patient is off steroids for four weeks prior to starting the study and will not require them during the course of the study A patient with seizure disorders may be enrolled if well controlled on anticonvulsants and if no seizures have occurred within a 6 week period prior to starting trial treatment
- HIV sero-negative and neither active nor chronic-replicative hepatitis B infection
- Laboratory testing: The patients should have adequate functions of the cor, lung, bone marrow, liver, kidney
Who cannot join
- -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Randomized Phase 2 Trial Comparing Experimental Immunotherapy in Recurrent High Risk Neuroblastoma Patients With Standard Immunotherapy in Patients With Recurrent and Newly Diagnosed High Risk Neuroblastoma
Although the five year survival rate of children with high risk neuroblastoma have increased over the last three decades from 4 to 44 % (1), neuroblastoma is the second most frequent cause for cancer related death in childhood (11 %). Most patients show good initial response rates (complete (CR) and partial remission (PR) rate 95 %), but 55 % experience a largely treatment-resistant tumor progression.
Recently, a breakthrough with immunotherapy was reported by US investigators from the Children's Oncology Group (2) using the anti-ganglioside D2 (GD2) monoclonal antibody ch14.18 for tumor cell destruction and granulocyte macrophage-colony stimulating factor (GM-CSF) plus interleukin 2 (IL-2) for immunostimulation. This immune therapy resulted in an increase of 20 % Event free survival (EFS) at 2 year from randomization. However, this was associated with a high toxicity rate (pain, capillary leak syndrome).
Read more
The proposed trial compares the Childrens' Oncology Group (COG) "standard of care" arm (anti-GD2 + GM-CSF + IL-2 i.v. + retinoic acid oral) with an experimental arm (anti-GD2 + GM-CSF + IL-2 s.c. + retinoic acid oral) designed to reduce toxicity.
The potential benefit from this trial consists of the confirmation that the American trial design is feasible in an independent set of patients with different preceding therapy, at a different time point regarding to immune reconstitution after autologous stem cell transplantation (ASCT), the feasibility of a newly designed immunotherapy (which is hopefully less toxic) and the investigation of immune response parameters. This pilot study is the prerequisite for a consecutive randomized clinical trial comparing two immunotherapeutic approaches in a larger set of patients.
Read the detailed description
Neuroblastoma is the second most frequent solid tumour (7.6%) and the second cause of cancer related death (11%) in childhood. In particular the large high risk (HR) group has remained a challenge to Paediatric Oncologists. Although the 5 year survival rates of children with HR disease have increased over the last 3 decades from 4 to 44.4%, the vast majority of those children will finally succumb to disease [1]. Most patients show good initial responses to chemotherapy (CR + PR-rate 95 %), but a majority experiences a highly treatment resistant tumour progression (55%). Therefore new therapeutic modalities are urgently needed.
Recently, a randomized trial demonstrated that an immunotherapeutic concept using the anti-GD2 antibody ch14.18 together with interleukin 2, GM-CSF and retinoic acid improved the outcome of neuroblastoma patients which achieved CR or very good partial remission (VGPR) response to the preceding therapy. This treatment was associated with a high rate of toxic effects (neuropathic pain 52% of patients, capillary leak syndrome 23%). An earlier study using the antibody ch14.18 alone made comparable observations: pain despite of analgesia was seen in 33% of patients and severe capillary leak syndromes in 3 of 151 children. Differences of the reported frequencies are due to the different definitions of side effects.
The investigators therefore propose a randomized clinical trial comparing the COG immunotherapy concept with newly designed hopefully equally effective but less toxic concept. This modifies the application route of IL-2 from i.v. to s.c. and increases the IL-2 dose to 6.0 mio/m²xd (from 3.0 first week and 4.5 mio second week i.v.). Oral retinoic acid is used in both arms. The proposed randomized trial will answer the following questions:
(i) Confirmation of the feasibility to apply the COG immunotherapy as consolidation treatment after a different remission induction therapy in patients with recurrent and de novo high risk neuroblastoma (ii) Investigation of the feasibility to apply the new immunotherapy concept in patients with recurrent high risk neuroblastoma (iii) Comparison of the toxicity of both immunotherapy regimens with the aim to reduce grade 2 - 4 toxicities in the experimental arm.
(iv) Comparison of the immune response (antiidiotype antibodies, immune cell phenotypes, immune mediators, functional assays as antibody dependent cellular cytotoxicity (ADCC) and complement dependent cytotoxicity (CDC) between treatment cycles (intraindividual), treatment arms (interindividual) and between recurrent and newly diagnosed patients (v) Comparison of pharmacokinetics of antibody ch14.18 in both arms (12.10.) (vi) Comparison of therapeutic efficacy by response evaluation at the end of the 25 week treatment (descriptive).
(vii) Comparison of patients´ QoL experienced in both immunotherapy regimens as indicated by parents rating in appropriate questionnaires.
- Conditions
- Neuroblastoma
- Being tested
- antibody ch14.18, GM-CSF, IL-2 i.v., IL-2 s.c., Retinoic acid
- Keywords
- High Risk Neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
antibody ch14.18Drug
Also called Unituxin
17.5mg/m²d 10-20h i.v, d4-7 in cycles 1,3,5 and d8-11 in cycles 2,4.
GM-CSFDrug
Also called Sargramostim, Leukine
250µg/m²xd, d1-14 s.c. or i.v (2h) in cycles 1,3,5
IL-2 i.v.Drug
Also called Proleukin
3.0 mio U/m²xd d1-4 continuous infusion i.v. and 4.5 mio U/m²xd d8-11 continuous infusion i.v. in cycles 2,4
IL-2 s.c.Drug
Also called Proleukin
0.06 mio U/m² i.v. test dosis for 30min. i.v. at least 2h before first subcutaneous (s.c.) application. 6 mio U/m²xd d1-5 and 8-12 s.c. in cycles 2,4
Retinoic acidDrug
Also called isotretinoin
160mg/m²xd b.i.d.oral d 11-24 in cycles 1,3,5,6 and d15-28 in cycles 2,4
| Study group | Role | What they receive |
|---|---|---|
| Standard Arm | Active comparator | Standard IL-2 i.v. together with antibody ch14.18, GM-CSF and retinoic acid Receives: Drug: antibody ch14.18; Drug: GM-CSF; Drug: IL-2 i.v.; Drug: Retinoic acid |
| Experimental Arm | Experimental | IL-2 s.c. together with antibody ch14.18, GM-CSF and retinoic acid Receives: Drug: antibody ch14.18; Drug: GM-CSF; Drug: IL-2 s.c.; Drug: Retinoic acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
toxic death or relevant grade 4 toxicity
Relevant grade 4 toxicities are defined as ascites, adult respiratory distress syndrome, capillary leak syndrome, cytokine release syndrome, dyspnea, hypotension, motor neuropathy, sensory neuropathy.
Measured: up to 7 months
Secondary outcomes (3)
Reduction of key side effects
Key side effects are defined as capillary leak syndrome and cytokine release syndrome. Reduction is defined by at least 1 grade or score point or day less.
Measured: up to 7 months
Maximum of antibody-related pain
Neuralgia (with assessment of pain duration by days requiring morphine and maximum grade of pain scores during first 2 antibody cycles) will compared between both arms.
Measured: up to 5 months
Comparative pharmacokinetics
Maximum plasma concentration of the antibody assessed during the first 2 months of treatment.
Measured: up to 2 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2015 (estimated)
- Study startNov 2015
- Primary completion23 Dec 2016
- Study completion30 Jan 2017
Why it stopped: Antibody supply was discontinued by United Therapeutics Corporation.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Cologne
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Frank Berthold, University of Cologne
- Investigators
- Frank Berthold, Prof. Dr., Study chair, University of Cologne
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02641782
- Sponsor's protocol ID
- Uni-Koeln-1694
- Acronym
- NB2013-HR
- Status last verified
- Nov 2022
- First submitted
- 12 Dec 2015
- Last updated
- 4 Nov 2022
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Yu AL, Gilman AL, Ozkaynak MF, London WB, Kreissman SG, Chen HX, Smith M, Anderson B, Villablanca JG, Matthay KK, Shimada H, Grupp SA, Seeger R, Reynolds CP, Buxton A, Reisfeld RA, Gillies SD, Cohn SL, Maris JM, Sondel PM; Children's Oncology Group. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med. 2010 Sep 30;363(14):1324-34. doi: 10.1056/NEJMoa0911123.
PubMed 20879881Simon T, Hero B, Faldum A, Handgretinger R, Schrappe M, Klingebiel T, Berthold F. Long term outcome of high-risk neuroblastoma patients after immunotherapy with antibody ch14.18 or oral metronomic chemotherapy. BMC Cancer. 2011 Jan 18;11:21. doi: 10.1186/1471-2407-11-21.
PubMed 21244693Simon T, Hero B, Faldum A, Handgretinger R, Schrappe M, Niethammer D, Berthold F. Consolidation treatment with chimeric anti-GD2-antibody ch14.18 in children older than 1 year with metastatic neuroblastoma. J Clin Oncol. 2004 Sep 1;22(17):3549-57. doi: 10.1200/JCO.2004.08.143.
PubMed 15337804
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Phase 2 Trial Comparing Experimental Immunotherapy in Recurrent High Risk Neuroblastoma Patients With Standard Immunotherapy in Patients With Recurrent and Newly Diagnosed High Risk Neuroblastoma
- ClinicalTrials.gov ID
- NCT02641782
- Acronym / sponsor ID
- NB2013-HR / Uni-Koeln-1694
- Conditions
- Neuroblastoma
- Keywords
- High Risk Neuroblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Antibody supply was discontinued by United Therapeutics Corporation.
- Enrolment
- 3 participants (actual)
- Lead sponsor
- University of CologneUniversity, hospital or other
- Study start
- Nov 2015 (actual)
- Primary completion
- 23 Dec 2016 (actual)
- Study completion
- 30 Jan 2017 (actual)
- First posted
- 29 Dec 2015 (estimated)
- 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.
Although the five year survival rate of children with high risk neuroblastoma have increased over the last three decades from 4 to 44 % (1), neuroblastoma is the second most frequent cause for cancer related death in childhood (11 %). Most patients show good initial response rates (complete (CR) and partial remission (PR) rate 95 %), but 55 % experience a largely treatment-resistant tumor progression.
Recently, a breakthrough with immunotherapy was reported by US investigators from the Children's Oncology Group (2) using the anti-ganglioside D2 (GD2) monoclonal antibody ch14.18 for tumor cell destruction and granulocyte macrophage-colony stimulating factor (GM-CSF) plus interleukin 2 (IL-2) for immunostimulation. This immune therapy resulted in an increase of 20 % Event free survival (EFS) at 2 year from randomization. However, this was associated with a high toxicity rate (pain, capillary leak syndrome).
The proposed trial compares the Childrens' Oncology Group (COG) "standard of care" arm (anti-GD2 + GM-CSF + IL-2 i.v. + retinoic acid oral) with an experimental arm (anti-GD2 + GM-CSF + IL-2 s.c. + retinoic acid oral) designed to reduce toxicity.
The potential benefit from this trial consists of the confirmation that the American trial design is feasible in an independent set of patients with different preceding therapy, at a different time point regarding to immune reconstitution after autologous stem cell transplantation (ASCT), the feasibility of a newly designed immunotherapy (which is hopefully less toxic) and the investigation of immune response parameters. This pilot study is the prerequisite for a consecutive randomized clinical trial comparing two immunotherapeutic approaches in a larger set of patients.
Detailed description
Neuroblastoma is the second most frequent solid tumour (7.6%) and the second cause of cancer related death (11%) in childhood. In particular the large high risk (HR) group has remained a challenge to Paediatric Oncologists. Although the 5 year survival rates of children with HR disease have increased over the last 3 decades from 4 to 44.4%, the vast majority of those children will finally succumb to disease [1]. Most patients show good initial responses to chemotherapy (CR + PR-rate 95 %), but a majority experiences a highly treatment resistant tumour progression (55%). Therefore new therapeutic modalities are urgently needed.
Recently, a randomized trial demonstrated that an immunotherapeutic concept using the anti-GD2 antibody ch14.18 together with interleukin 2, GM-CSF and retinoic acid improved the outcome of neuroblastoma patients which achieved CR or very good partial remission (VGPR) response to the preceding therapy. This treatment was associated with a high rate of toxic effects (neuropathic pain 52% of patients, capillary leak syndrome 23%). An earlier study using the antibody ch14.18 alone made comparable observations: pain despite of analgesia was seen in 33% of patients and severe capillary leak syndromes in 3 of 151 children. Differences of the reported frequencies are due to the different definitions of side effects.
The investigators therefore propose a randomized clinical trial comparing the COG immunotherapy concept with newly designed hopefully equally effective but less toxic concept. This modifies the application route of IL-2 from i.v. to s.c. and increases the IL-2 dose to 6.0 mio/m²xd (from 3.0 first week and 4.5 mio second week i.v.). Oral retinoic acid is used in both arms. The proposed randomized trial will answer the following questions:
(i) Confirmation of the feasibility to apply the COG immunotherapy as consolidation treatment after a different remission induction therapy in patients with recurrent and de novo high risk neuroblastoma (ii) Investigation of the feasibility to apply the new immunotherapy concept in patients with recurrent high risk neuroblastoma (iii) Comparison of the toxicity of both immunotherapy regimens with the aim to reduce grade 2 - 4 toxicities in the experimental arm.
(iv) Comparison of the immune response (antiidiotype antibodies, immune cell phenotypes, immune mediators, functional assays as antibody dependent cellular cytotoxicity (ADCC) and complement dependent cytotoxicity (CDC) between treatment cycles (intraindividual), treatment arms (interindividual) and between recurrent and newly diagnosed patients (v) Comparison of pharmacokinetics of antibody ch14.18 in both arms (12.10.) (vi) Comparison of therapeutic efficacy by response evaluation at the end of the 25 week treatment (descriptive).
(vii) Comparison of patients´ QoL experienced in both immunotherapy regimens as indicated by parents rating in appropriate questionnaires.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Standard Arm | Active comparator | Drug: antibody ch14.18, Drug: GM-CSF, Drug: IL-2 i.v., Drug: Retinoic acidStandard IL-2 i.v. together with antibody ch14.18, GM-CSF and retinoic acid |
| Experimental Arm | Experimental | Drug: antibody ch14.18, Drug: GM-CSF, Drug: IL-2 s.c., Drug: Retinoic acidIL-2 s.c. together with antibody ch14.18, GM-CSF and retinoic acid |
Who may qualify
- Ages
- 18 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Established diagnosis of neuroblastoma according to the international criteria (INSS)
- High risk (HR): stage 4 over 18 months of age and oncogen MYCN (MYCN) amplified neuroblastoma of any stage and any age until 25 years (recurrent disease (Germany and The Netherlands) after re-induction chemotherapy (+/- other modalities) or newly diagnosed disease (only The Netherlands):
- Complete front-line treatment including induction chemotherapy, radioisotope (mIBG) treatment, appropriate local therapy such as surgical removal and/ or local irradiation of the primary tumor and myeloablative chemotherapy with autologous stem cell reinfusion according to the actual guidelines of the GPOH/DCOG
- achieved response status: stable disease or better (CR, VGPR, PR, SD).
- Written informed consent of parents or guardian and - if appropriate - of the patient.
Key exclusion criteria (1 listed)
- -
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 |
|---|---|---|
| antibody ch14.18Also: Unituxin17.5mg/m²d 10-20h i.v, d4-7 in cycles 1,3,5 and d8-11 in cycles 2,4. | Drug | Experimental Arm, Standard Arm |
| GM-CSFAlso: Sargramostim, Leukine250µg/m²xd, d1-14 s.c. or i.v (2h) in cycles 1,3,5 | Drug | Experimental Arm, Standard Arm |
| IL-2 i.v.Also: Proleukin3.0 mio U/m²xd d1-4 continuous infusion i.v. and 4.5 mio U/m²xd d8-11 continuous infusion i.v. in cycles 2,4 | Drug | Standard Arm |
| IL-2 s.c.Also: Proleukin0.06 mio U/m² i.v. test dosis for 30min. i.v. at least 2h before first subcutaneous (s.c.) application. 6 mio U/m²xd d1-5 and 8-12 s.c. in cycles 2,4 | Drug | Experimental Arm |
| Retinoic acidAlso: isotretinoin160mg/m²xd b.i.d.oral d 11-24 in cycles 1,3,5,6 and d15-28 in cycles 2,4 | Drug | Experimental Arm, Standard Arm |
Primary outcome
toxic death or relevant grade 4 toxicity
Time frame: up to 7 months
Relevant grade 4 toxicities are defined as ascites, adult respiratory distress syndrome, capillary leak syndrome, cytokine release syndrome, dyspnea, hypotension, motor neuropathy, sensory neuropathy.
3 secondary outcomes
Reduction of key side effects
Time frame: up to 7 months
Maximum of antibody-related pain
Time frame: up to 5 months
Comparative pharmacokinetics
Time frame: up to 2 months
Results and safety
The study was stopped early (reason given: Antibody supply was discontinued by United Therapeutics Corporation.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2022.
- Background: PubMed 20879881Yu AL, Gilman AL, Ozkaynak MF, London WB, Kreissman SG, Chen HX, Smith M, Anderson B, Villablanca JG, Matthay KK, Shimada H, Grupp SA, Seeger R, Reynolds CP, Buxton A, Reisfeld RA, Gillies SD, Cohn SL, Maris JM, Sondel PM; Children's Oncology Group. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med. 2010 Sep 30;363(14):1324-34. doi: 10.1056/NEJMoa0911123.
- Background: PubMed 21244693Simon T, Hero B, Faldum A, Handgretinger R, Schrappe M, Klingebiel T, Berthold F. Long term outcome of high-risk neuroblastoma patients after immunotherapy with antibody ch14.18 or oral metronomic chemotherapy. BMC Cancer. 2011 Jan 18;11:21. doi: 10.1186/1471-2407-11-21.
- Results publication: PubMed 15337804Simon T, Hero B, Faldum A, Handgretinger R, Schrappe M, Niethammer D, Berthold F. Consolidation treatment with chimeric anti-GD2-antibody ch14.18 in children older than 1 year with metastatic neuroblastoma. J Clin Oncol. 2004 Sep 1;22(17):3549-57. doi: 10.1200/JCO.2004.08.143.
- Individual participant dataSharing planned: No
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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.
Frank Berthold, Prof. Dr. study chair
University of Cologne
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing antibody ch14.18, GM-CSF and IL-2 i.v. in people with Neuroblastoma, run by University of Cologne at 1 site in Germany.
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 10 inclusion and 1 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: stopped early, as of the sponsor's last update on 4 Nov 2022. 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 Germany. 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 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?
The study was stopped early (reason given: Antibody supply was discontinued by United Therapeutics Corporation.). Results are not available in the source record.
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?
toxic death or relevant grade 4 toxicity (time frame: up to 7 months). 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 NCT02641782, which also lists 3 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 4 Nov 2022.