stop_circleTerminated/Withdrawn
Relapsed or refractory solid tumors or lymphoma in children, Neuroblastoma, Osteosarcoma, Rhabdomyosarcoma, Ewing sarcoma
Bayer Identifier:
19176
ClinicalTrials.gov Identifier:
EudraCT Number:
EU CT Number:
Not Available
Safety, tolerability, efficacy and pharmacokinetics of copanlisib in pediatric patients
Trial purpose
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.
beneficial to pediatric patients with solid solid tumors or lymphoma that are recurrent or refractory to standard therapy.
Key Participants Requirements
Sex
AllAge
6 - 21 YearsTrial summary
Enrollment Goal
31Trial Dates
April 2018 - February 2023Phase
Phase 1/Phase 2Could I Receive a placebo
NoProducts
Aliqopa (Copanlisib, BAY80-6946)Accepts Healthy Volunteer
NoWhere to participate
Status | Institution | Location |
---|---|---|
Completed | The Children's Hospital | Aurora, 80045, United States |
Completed | Children's Hospital of Philadelphia | Philadelphia, 19104, United States |
Completed | St. Jude Children's Research Hospital | Memphis, 38105, United States |
Completed | Cincinnati Children's Hospital and Medical Center | Cincinnati, 45229, United States |
Completed | Dana-Farber Cancer Institute | Boston, 02215, United States |
Completed | Columbia University Medical Center | New York, 10032, United States |
Completed | Children's Hospital of Alabama | Birmingham, 35233, United States |
Completed | Children's Healthcare of Atlanta | Atlanta, 30322, United States |
Completed | Riley Hospital For Children | Indianapolis, 46202, United States |
Completed | Seattle Children's Hospital | Seattle, 98105, United States |
Completed | Children's Hospital of Orange County | Orange, 92868-3974, United States |
Completed | Children's National Medical Center | Washington, 20010-2970, United States |
Completed | Children's Hospital of Pittsburgh of UPMC | Pittsburgh, 15224, United States |
Withdrawn | Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago, 60611, United States |
Completed | Texas Children's Hospital | Houston, 77030, United States |
Withdrawn | University of Minnesota Medical Center | Minneapolis, 55455, United States |
Withdrawn | C.S. Mott Children's Hospital | Ann Arbor, 48109, United States |
Withdrawn | Children's Hospital of Los Angeles | Los Angeles, 90027-6089, United States |
Withdrawn | Memorial Sloan-Kettering Cancer Center | New York, 10065, United States |
Withdrawn | University of Texas Southwestern Medical Center | Dallas, 75390, United States |
Withdrawn | Cook Children's Medical Center | Fort Worth, 76104-2796, United States |
Primary Outcome
- The maximum tolerated dose (MTD)Phase 1: 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.date_rangeTime Frame:Cycle 1 (28 days)
- Dose-limiting Toxicities(DLTs)Phase 1date_rangeTime Frame:Cycle 1 (28 days)
- Number of participants with Treatment-emergent Adverse Events(TEAEs)Phase 1date_rangeTime Frame:Approximately 13 months
- Number of participants with Serious Adverse Events (SAEs)Phase 1date_rangeTime Frame:Approximately 13 months
- Number of participants with Treatment-related Adverse Events (AEs).Phase 1date_rangeTime Frame:Approximately 13 months
- Objective response rate (ORR)Phase 2:ORR is the primary efficacy variable in neuroblastoma, Ewing sarcoma and rhabdomyosarcoma.date_rangeTime Frame:Up to 31 months
- Disease control rate (DCR)Phase 2:DCR is the primary efficacy variable in osteosarcoma.date_rangeTime Frame:Up to 31 months
- Progression-free survival (PFS)Phase 2: PFS is considered as co-primary (descriptively evaluated) variable in patients with osteosarcoma.date_rangeTime Frame:Up to 31 months
Secondary Outcome
- Copanlisib maximum drug concentration (Cmax)Phase 1.date_rangeTime 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.
- Area under the curve (AUC(0-168))Phase 1date_rangeTime 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.
- Objective response rate (ORR)Phase 1: ORR by dose cohort is defined as the number of responders divided by the number of patients in full analysis set (FAS) in the indicationdate_rangeTime Frame:Approximately 12 months
- Duration of response (DOR)Phase 2: DOR is defined as the time from the date of first observed tumor response (Complete response (CR) or Partial response (PR)) until first subsequent disease progression or until death (if death occurs before progression is documented) due to any causedate_rangeTime Frame:Up to 31 months
- PFS in each indication except for osteosarcomaPhase 2: PFS is defined as the time from first dose of study drug to disease progression according to Response Evaluation Criteria in Solid Tumors 1.1 (RECIST1.1) for solid tumor patients (except osteosarcoma) and SIOPEN or Curie score for neuroblastoma patients with Iodine-123 metaiodobenzylguanidine (MIBG)-avid disease, or death (if death occurs before progression is documented).date_rangeTime Frame:Up to 31 months
- Overall survival (OS)Phase 2: OS is defined as the time from first dose of study drug until death from any cause or until the last date the patient is known to be alive.date_rangeTime Frame:Up to 31 months
- Number of participants with Treatment-emergent AEsPhase 2: A treatment emergent AE is defined as any event arising or worsening after start of test drug administration until 30 days after the last dose of the study drug intake (end of safety followup).date_rangeTime Frame:Up to 32 months
- Number of participants with treatment emergent SAEsPhase 2: The severity of AEs will be graded using the NCI CTCAE v 4.03 dictionarydate_rangeTime Frame:Up to 32 months
- Number of participants with treatment-emergent clinically significant change in laboratory parameters, ECGs and vital signsPhase 2:The severity of AEs will be graded using the NCI CTCAE v 4.03 dictionarydate_rangeTime Frame:Up to 32 months
Trial design
Trial Type
InterventionalIntervention Type
DrugTrial Purpose
TreatmentAllocation
Non-randomizedBlinding
N/AAssignment
Parallel AssignmentTrial Arms
5