Neoplasms

All Conditions

Blood and Lymph Conditions

Immune System Diseases

Skin and Connective Tissue Diseases

Rare Diseases

Osteosarcoma

Neuroblastoma

Sarcoma

Lymphoma

Sarcoma, Ewing

Rhabdomyosarcoma

Neoplasms by Histologic Type

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Neoplasms, Connective and Soft Tissue

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Neoplasms, Bone Tissue

Neoplasms, Connective Tissue

Myosarcoma

Soft Tissue Sarcoma

Lymphosarcoma

Ewing Sarcoma

Neuroepithelioma

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).

Recommended Phase 2 dose (RP2D) for copanlisib in pediatric patients, as defined in the Phase I part of the study, will be used.

RP2D for copanlisib in pediatric patients, as defined in the Phase I part of the study, will be used.

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.

Copanlisib (BAY806946)

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.

Safety, Tolerability, Efficacy and Pharmacokinetics of Copanlisib in Pediatric Patients

A Non-randomized, Open-label, Multi-center, Phase I/II Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor Copanlisib in Pediatric Patients With Relapsed/Refractory Solid Tumors or Lymphoma

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.

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.

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.

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.

ORR was defined separately in each indication, as the number of responders divided by the number of subjects in FAS in the indication.

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.

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.

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.

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.

Bayer

Copanlisib was administered Intravenous (IV) on Days 1, 8 and 15 of each 28-day treatment cycle. Treatment was continued until radiological progressive disease, unacceptable toxicity, withdrawal of consent, death or other event specified by the protocol. Copanlisib 28mg/m\*2 (Total) included AMD0 (5 participants who were under the original DLT criteria) and AMD1+ (19 participants who were under the amended DLT criteria).

Copanlisib 28mg/m*2, Total

Copanlisib was administered Intravenous (IV) on Days 1, 8 and 15 of each 28-day treatment cycle. Treatment was continued until radiological progressive disease,unacceptable toxicity, withdrawal of consent, death or other event specified by the protocol.

Copanlisib 35mg/m*2

Copanlisib was administered Intravenous (IV) on Days 1, 8 and 15 of each 28-day treatment cycle. Treatment was continued until radiological progressive disease, unacceptable toxicity, withdrawal of consent, death or other event specified by the protocol.

Total

Age, Continuous

In utero

Preterm newborn infants (gestational age < 37 wks)

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

From 65-84 years

85 years and over

Age, Customized

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Therapeutic Area Head

Copanlisib was administered on Day 1, Day 8, and Day 15 of every 28-day cycle. Subjects received copanlisib IV infusion with intermittent (3 weeks on / 1 week off) dosing schedule at the assigned dose level.

Copanlisib_Phase 1

Safety analysis set (SAF): The SAF population was defined as all participants with at least one intake of study drug.

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.

Phase 1: Number of Subjects With Dose Limiting Toxicity (DLT)

Phase 1: Number of Subjects With Treatment-emergent Adverse Events (TEAEs)

Phase 1: Number of Subjects With Serious Adverse Events (SAEs)

Phase 1: Number of Participants With Treatment-related Adverse Events (AEs).

As the study was terminated before the initiation of phase 2, data was not collected in phase 2.

Copanlisib_Phase 2

Data was not collected for this endpoint due to study was terminated before the initiation of phase 2.

Phase 2: Objective Response Rate (ORR)

Phase 2: Disease Control Rate (DCR)

Phase 2: Progression-free Survival (PFS)

For all participants included in the PK analysis set, copanlisib PK was analyzed using an established comprehensive population PK model with incorporation of allometric scaling by Body surface area (BSA) on copanlisib disposition parameters (clearance and volume parameters). The model with allometric scaling enables combining of treatment groups and was used to estimate copanlisib PK parameters (AUC0-168h and Cmax).

Phase 1: Copanlisib Maximum Drug Concentration (Cmax)

Phase 1: Area Under the Curve (AUC(0-168))

Full analysis set (FAS): All subjects with at least one intake of study drug.

Spots Global Cancer Trial Database for Safety, Tolerability, Efficacy and Pharmacokinetics of Copanlisib in Pediatric Patients

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Study Description

Keywords

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