This study is being conducted to test the safety and efficacy of ramucirumab in combination with other chemotherapy in the treatment of relapsed, recurrent, or refractory desmoplastic small round cell tumor (DSRCT) in children and young adults. This trial is part of the CAMPFIRE master protocol (NCT05999994) which is a platform to accelerate the development of new treatments for pediatric and young adult participants with cancer. Your participation in this trial could last 12 months or longer, depending on how you and your tumor respond.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Administered IV
Administered orally
Administered IV
Phoenix Children's Hospital
Phoenix, Arizona, United States
Children's Hospital of Los Angeles
Los Angeles, California, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Washington University
St Louis, Missouri, United States
Memorial Sloan Kettering Cancer Center
New York, New York, United States
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
Royal Children's Hospital
Parkville, Victoria, Australia
Universitaetsklinikum Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, Germany
Universitaetsklinikum Essen
Essen, North Rhine-Westphalia, Germany
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, Germany
...and 12 more locations
Progression Free Survival (PFS)
PFS was defined as the time from randomization to the date of investigator-determined objective progression as defined by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1, or death from any cause in the absence of disease progression. Progressive disease (PD) was defined as at least a 20% increase in the sum of the diameters of target lesions, with reference being the smallest sum on study and an absolute increase of at least 5 mm, or unequivocal progression of non-target lesions, or 1 or more new lesions.Participants known to be alive and without disease progression were censored at the date of their last adequate tumor assessment per RECIST 1.1 criteria, or date of randomization (whichever is later). The posterior median and 98% credible interval were estimated using Bayesian analysis. Data are presented as the posterior median with 98% credible interval.
Time frame: Randomization to Objective Progression or Death Due to Any Cause (Up To 23 Months)
Overall Response Rate (ORR): Percentage of Participants Who Achieved a Complete Response (CR) or Partial Response (PR)
* The ORR was defined as the percentage of participants achieving either a CR or PR. Tumor response was assessed based on histology: Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) was used for solid tumors, and Response Assessment in Neuro-Oncology criteria were used for glioblastoma. * CR was defined as the disappearance of all target lesions, with any pathological lymph nodes (whether target or non-target) showing a reduction in the short axis to \<10 mm. Tumor marker results were required to have normalized. PR was defined as a decrease of at least 30% in the sum of the diameters of target lesions, using baseline diameters as the reference.
Time frame: Randomization until measured progressive disease (Up To 23 Months)
Duration of Response (DoR)
DoR is defined as the time from the date that measurement criteria for CR or PR (whichever is first recorded) are first met until the first date that disease is recurrent or objective disease progression is observed, per RECIST 1.1 criteria, or the date of death from any cause in the absence of documented disease progression or recurrence .Participants known to be alive and without disease progression were censored at the date of their last adequate tumor assessment per RECIST 1.1 criteria, or date of randomization (whichever is later).
Time frame: Date of CR or PR to Date of Objective Disease Progression or Death Due to Any Cause (Up To 23 Months)
Complete Response (CR) : Percentage of Participants Who Achieved a CR
CR was defined as the disappearance of all target lesions, with any pathological lymph nodes (whether target or non-target) showing a reduction in the short axis to \<10 mm.Tumor marker results were required to have normalized.
Time frame: Randomization until measured progressive disease (Up To 23 Months)
Pharmacokinetics (PK): Maximum Serum Concentration of Ramucirumab (Cmax)
Cmax was the concentration of study drug in the blood after the dose is administered. It was measured post-dose and was summarized using descriptive statistics.
Time frame: End of ramucirumab infusion on Day 1 of Cycle 1
PK: Minimum Serum Concentration of Ramucirumab (Cmin)
Cmin was the concentration of study drug in the blood immediately before the next dose was administered. It was measured pre-dose at all visits and was summarized using descriptive statistics.
Time frame: Prior to ramucirumab infusion on - Day 15 of Cycle 1 and Day 1 of Cycles 2, 4, 7, and 10
Number of Participants With Treatment-Emergent Anti-Drug Antibodies (TE-ADA)
A TE-ADA evaluable participant is considered TE-ADA positive if they have at least one post-baseline ADA titer that is a 4-fold or greater increase from their baseline titer (treatment-boosted); alternatively, if the baseline ADA result is Not Present, the participant is considered TE-ADA positive if there is at least one post-baseline ADA result that is Present with a titer greater than or equal to 1:20 (treatment-induced).
Time frame: Baseline Up to 23 months
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