A Phase 1/2 interventional study of Copanlisib (BAY806946) in Relapsed or Refractory Solid Tumors or Lymphoma in Children, Neuroblastoma and Osteosarcoma, sponsored by Bayer. Terminated at 14 sites in United States. Open to participants aged 6 Months to 21 Years. Per ClinicalTrials.gov, last updated 2023-12-04.
Sponsored by Bayer · Phase 1/2, Interventional, and Treatment
This study is designed to investigate whether the use of copanlisib is safe, feasible and beneficial to pediatric patients with solid solid tumors or lymphoma that are recurrent or refractory to standard therapy.
675 studies on the registry are indexed under Neuroblastoma; 127 are open to participants now.
This study's enrollment of 31 is close to the median of 32 across 507 interventional studies indexed under Neuroblastoma.
Browse Neuroblastoma studies →Bayer is the lead sponsor of 1,674 studies on the registry; 51 are open to participants now.
Of its 214 completed or terminated interventional studies of FDA-regulated products, 132 (62%) have results posted.
Compared within Neuroblastoma, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Recurrence. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Confirmation of diagnosis:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Line of therapy
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Measurable disease
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CTCAE grade
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
It is estimated that 2 or 3 dose cohorts may be evaluated in phase 1 of the study. Safety and MTD/RP2D dose will be evaluated in 2 age groups (\< 1 year old and ≥ 1 year old).
Drug: Copanlisib (BAY806946)
Recommended Phase 2 dose (RP2D) for copanlisib in pediatric patients, as defined in the Phase I part of the study, will be used.
Drug: Copanlisib (BAY806946)
RP2D for copanlisib in pediatric patients, as defined in the Phase I part of the study, will be used.
Drug: Copanlisib (BAY806946)
RP2D for copanlisib in pediatric patients, as defined in the Phase I part of the study, will be used.
Drug: Copanlisib (BAY806946)
RP2D for copanlisib in pediatric patients, as defined in the Phase I part of the study, will be used.
Drug: Copanlisib (BAY806946)
Copanlisib will be dosed on Day 1, Day 8, and Day 15 of every 28-day cycle. Phase 1: 2 or 3 dose cohorts may be evaluated in phase 1 of the study. Phase 2: RP2D for copanlisib in pediatric patients, as defined in the Phase I part of the study, will be used.
Phase 1: The Maximum Tolerated Dose (MTD): the Highest Dose Level of Copanlisib That Can be Given so That Not More Than 1 Out of 6 Patients Experience a DLT During the DLT Evaluation Period.
Maximum tolerated dose (MTD) for copanlisib was defined as the highest dose level where 6 patients have been treated and ≤ 1 participant experienced a DLT. This endpoint was performed on SAF.
Time frame: Cycle 1 (28 days)
Phase 1: Number of Subjects With Dose Limiting Toxicity (DLT)
DLT was observed during first cycle of treatment, and assessed as possibly, probably or definitely related to treatment with copanlisib. The DLT observation period for the purposes of dose-escalation was the first cycle of therapy.
Time frame: Cycle 1 (28 days)
Phase 1: Number of Subjects With Treatment-emergent Adverse Events (TEAEs)
TEAE was defined as any event arising or worsening after start of study drug administration until 30 days after the last dose of the study drug intake (end of safety follow-up). This endpoint was performed on SAF.
Time frame: After the first study intervention up to 30 days after the last dose of the study drug intake (end of safety follow up), with a maximum of 145 days.
Phase 1: Number of Subjects With Serious Adverse Events (SAEs)
This endpoint was performed on SAF.
Time frame: Up to 150 days.
Phase 1: Number of Participants With Treatment-related Adverse Events (AEs).
This endpoint was performed on SAF.
Time frame: Up to 145 days.
Phase 2: Objective Response Rate (ORR)
ORR was defined separately in each indication, as the number of responders divided by the number of subjects in FAS in the indication.
Time frame: Data was not collected for this endpoint due to study was terminated before the initiation of phase 2.
Phase 2: Disease Control Rate (DCR)
The DCR was defined as the number of subjects with disease control divided by the number of subjects in FAS or per protocol set (PPS) in the indication.
Time frame: Data was not collected for this endpoint due to study was terminated before the initiation of phase 2.
Phase 2: Progression-free Survival (PFS)
Time frame: Data was not collected for this endpoint due to study was terminated before the initiation of phase 2.
Phase 1: Copanlisib Maximum Drug Concentration (Cmax)
Cmax: maximum concentration after the 3rd dose in a sequence of 3 nominal doses of copanlisib. PK analysis set: All participants with at least one intake of study drug and with at least one valid measurement for copanlisib were included in the copanlisib PK analysis.
Time frame: Age ≥ 6 years: Pre-dose, Post-dose on Cycle 1 Day 1 and Day 15 (1-1.25 hour (h), 1.5- 3h, 22-24h). Age < 6 years: Pre-dose, Post-dose on Cycle 1 Day 1 and Day 15 (1-1.25h, 22-24h). Cycle length is 28 days.
Phase 1: Area Under the Curve (AUC(0-168))
AUC(0-168): Area under the concentration-time curve \[AUC\] from 0 to 168 hours after the 3rd dose in a sequence of 3 nominal doses of copanlisib. PK analysis set: All participants with at least one intake of study drug and with at least one valid measurement for copanlisib were included in the copanlisib PK analysis.
Time frame: Age ≥ 6 years: Pre-dose, Post-dose on Cycle 1 Day 1 and Day 15 (1-1.25 hour (h), 1.5- 3h, 22-24h). Age < 6 years: Pre-dose, Post-dose on Cycle 1 Day 1 and Day 15 (1-1.25h, 22-24h). Cycle length is 28 days.
Phase 1: Objective Response Rate (ORR)
ORR by dose cohort is defined as the number of responders divided by the number of subjects in FAS in the indication. The analysis of ORR was performed on FAS.
Time frame: Up to 150 days
Phase 2: Duration of Response (DOR)
Time frame: Data was not collected for this endpoint due to study was terminated before the initiation of phase 2.
Phase 2: PFS in Each Indication Except for Osteosarcoma
Time frame: Data was not collected for this endpoint due to study was terminated before the initiation of phase 2.
Phase 2: Overall Survival (OS)
Time frame: Data was not collected for this endpoint due to study was terminated before the initiation of phase 2.
Phase 2: Number of Participants With Treatment-emergent AEs
Time frame: Data was not collected for this endpoint due to study was terminated before the initiation of phase 2.
Phase 2: Number of Subjects With Treatment Emergent SAEs
Time frame: Data was not collected for this endpoint due to study was terminated before the initiation of phase 2.
Phase 2: Number of Subjects With Treatment-emergent Clinically Significant Change in Laboratory Parameters, ECGs and Vital Signs
Time frame: Data was not collected for this endpoint due to study was terminated before the initiation of phase 2.
The study was conducted at 14 centers in United States between 30 APR 2018 (First participant first visit) and 1-Feb-2023 (Last participant last visit).
| Milestone | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 |
|---|---|---|
| Started | 24 | 7 |
| Started treatment | 24 | 7 |
| Terminated treatment | 24 | 7 |
| Completed | 0 | 0 |
| Not completed | 24 | 7 |
| Withdrew: Adverse event | 1 | 0 |
| Withdrew: Physician decision | 1 | 0 |
| Withdrew: Progressive disease - clinical assessment | 1 | 0 |
| Withdrew: Progressive disease - radiological progression | 21 | 6 |
| Withdrew: Withdrawal by subject | 0 | 1 |
Maximum tolerated dose (MTD) for copanlisib was defined as the highest dose level where 6 patients have been treated and ≤ 1 participant experienced a DLT. This endpoint was performed on SAF.
| mg/m*2/dose | Copanlisib_Phase 1 |
|---|---|
| Phase 1: The Maximum Tolerated Dose (MTD): the Highest Dose Level of Copanlisib That Can be Given so That Not More Than 1 Out of 6 Patients Experience a DLT During the DLT Evaluation Period. | 28 |
DLT was observed during first cycle of treatment, and assessed as possibly, probably or definitely related to treatment with copanlisib. The DLT observation period for the purposes of dose-escalation was the first cycle of therapy.
| Participants | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 |
|---|---|---|
| Phase 1: Number of Subjects With Dose Limiting Toxicity (DLT) | 3 | 2 |
TEAE was defined as any event arising or worsening after start of study drug administration until 30 days after the last dose of the study drug intake (end of safety follow-up). This endpoint was performed on SAF.
| Participants | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 |
|---|---|---|
| Phase 1: Number of Subjects With Treatment-emergent Adverse Events (TEAEs) | 24 | 7 |
This endpoint was performed on SAF.
| Participants | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 |
|---|---|---|
| Phase 1: Number of Subjects With Serious Adverse Events (SAEs) | 10 | 0 |
This endpoint was performed on SAF.
| Participants | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 |
|---|---|---|
| Phase 1: Number of Participants With Treatment-related Adverse Events (AEs). | 5 | 0 |
ORR was defined separately in each indication, as the number of responders divided by the number of subjects in FAS in the indication.
No measurements were reported for this outcome.
The DCR was defined as the number of subjects with disease control divided by the number of subjects in FAS or per protocol set (PPS) in the indication.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
Cmax: maximum concentration after the 3rd dose in a sequence of 3 nominal doses of copanlisib. PK analysis set: All participants with at least one intake of study drug and with at least one valid measurement for copanlisib were included in the copanlisib PK analysis.
| μg/L | Copanlisib_Phase 1 |
|---|---|
| Phase 1: Copanlisib Maximum Drug Concentration (Cmax) | 359 ± 22.6 |
AUC(0-168): Area under the concentration-time curve \[AUC\] from 0 to 168 hours after the 3rd dose in a sequence of 3 nominal doses of copanlisib. PK analysis set: All participants with at least one intake of study drug and with at least one valid measurement for copanlisib were included in the copanlisib PK analysis.
| μg∙h/L | Copanlisib_Phase 1 |
|---|---|
| Phase 1: Area Under the Curve (AUC(0-168)) | 2900 ± 35.4 |
ORR by dose cohort is defined as the number of responders divided by the number of subjects in FAS in the indication. The analysis of ORR was performed on FAS.
| Participants | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 |
|---|---|---|
| Phase 1: Objective Response Rate (ORR) | 0 | 0 |
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
Collected over After the first study intervention up to 30 days after the last dose of the study drug intake (end of safety follow up), with a maximum of 150 days. Adverse event reporting for the all-cause mortality considers all deaths that occurred at any time during the study before the last contact, with a maximum of 150 days.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Copanlisib 28mg/m*2, Total | 20/24 (83.3%) | 10/24 (41.7%) | 23/24 (95.8%) |
| Copanlisib 35mg/m*2 | 5/7 (71.4%) | 0/7 (0%) | 7/7 (100%) |
| Event | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 |
|---|---|---|
| PyrexiaGeneral disorders | 5/24 | 0/7 |
| HyperglycaemiaMetabolism and nutrition disorders | 3/24 | 0/7 |
| Pain in extremityMusculoskeletal and connective tissue disorders | 2/24 | 0/7 |
| AnaemiaBlood and lymphatic system disorders | 1/24 | 0/7 |
| Pericardial effusionCardiac disorders | 1/24 | 0/7 |
| Sinus bradycardiaCardiac disorders | 1/24 | 0/7 |
| Abdominal painGastrointestinal disorders | 1/24 | 0/7 |
| Non-cardiac chest painGeneral disorders | 1/24 | 0/7 |
| Blood creatinine increasedInvestigations | 1/24 | 0/7 |
| Neutrophil count decreasedInvestigations | 1/24 | 0/7 |
| Event | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 |
|---|---|---|
| White blood cell count decreasedInvestigations | 12/24 | 5/7 |
| HyperglycaemiaMetabolism and nutrition disorders | 17/24 | 4/7 |
| NauseaGastrointestinal disorders | 15/24 | 4/7 |
| ConstipationGastrointestinal disorders | 4/24 | 4/7 |
| VomitingGastrointestinal disorders | 7/24 | 4/7 |
| PyrexiaGeneral disorders | 8/24 | 4/7 |
| AnaemiaBlood and lymphatic system disorders | 13/24 | 3/7 |
| Lymphocyte count decreasedInvestigations | 13/24 | 3/7 |
| HyponatraemiaMetabolism and nutrition disorders | 11/24 | 2/7 |
| ThrombocytopeniaBlood and lymphatic system disorders | 7/24 | 3/7 |
| Age, Continuous(years) | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 | Total |
|---|---|---|---|
| Mean | 12.5 ± 4.6 | 12.3 ± 3.8 | 12.5 ± 4.3 |
| Age, Customized(Participants) | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 | Total |
|---|---|---|---|
| In utero | 0 | 0 | 0 |
| Preterm newborn infants (gestational age < 37 wks) | 0 | 0 | 0 |
| Newborns (0-27 days) | 0 | 0 | 0 |
| Infants and toddlers (28 days-23 months) | 0 | 0 | 0 |
| Children (2-11 years) | 11 | 3 | 14 |
| Adolescents (12-17 years) | 10 | 4 | 14 |
| Adults (18-64 years) | 3 | 0 | 3 |
| From 65-84 years | 0 | 0 | 0 |
| 85 years and over | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 | Total |
|---|---|---|---|
| Female | 10 | 3 | 13 |
| Male | 14 | 4 | 18 |
| Ethnicity (NIH/OMB)(Participants) | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 | Total |
|---|---|---|---|
| Hispanic or Latino | 5 | 1 | 6 |
| Not Hispanic or Latino | 18 | 6 | 24 |
| Unknown or Not Reported | 1 | 0 | 1 |
| Race (NIH/OMB)(Participants) | Copanlisib 28mg/m*2, Total | Copanlisib 35mg/m*2 | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 1 | 2 | 3 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 3 | 1 | 4 |
| White | 17 | 3 | 20 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 3 | 1 | 4 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No — Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.
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Studies of the drug tested here: Copanlisib