A Study Evaluating Ramucirumab in Children and Young Adults with Desmoplastic Small Round Cell Tumor.
- Conditions
- Desmoplastic small round cell tumour (DSRCT)MedDRA version: 21.1Level: PTClassification code 10064581Term: Desmoplastic small round cell tumourSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)Therapeutic area: Diseases [C] - Cancer [C04]
- Registration Number
- EUCTR2018-004242-42-BE
- Lead Sponsor
- Eli Lilly and Company
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 34
- The washout-period after previous antibody therapy is defined as 4 half-lives after the last dose of the antibody
- Pediatric and young adult patients will be enrolled, where the specific age requirement for each indication will be specified in the protocol addenda.
- Patients must be >11 kg at the time of study enrollment.
- Patients with relapsed, recurrent, or refractory DSRCT.
- Patients must be 36 months to =29 years of age at the time of study enrollment.
- Patients must:
•have measurable disease by RECIST 1.1
•have received at least one prior line of systemic treatment (including neoadjuvant and adjuvant chemotherapy). This prior treatment must include approved therapies for which they are eligible, unless the patient is not a suitable candidate for the approved therapy.
•not be eligible for surgical resection at time of enrollment.
- Patients who have a Lansky (<16 years of age; Lansky et al. 1987) or Karnofsky (=16 years of age; Karnofsky et al. 1948) performance score of at least 50.
- Patient with adequate hematologic, coagulation, liver, cardiac, renal and bladder function, and adequate blood pressure (BP) control as per protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Patients who have had allogeneic bone marrow or solid organ transplant are excluded.
- Patients who have active infections requiring therapy.
- Patients who have a history of fistula, gastrointestinal (GI) ulcer or perforation, or intra-abdominal abscess within 3 months of study enrolment are not eligible.
- Patients with a bowel obstruction, extensive intestinal resection or history or presence of inflammatory enteropathy or other GI pathology as per protocol.
- Patients with a history of hepatorenal syndrome.
- Patients with evidence of active bleeding or a history of significant (=Grade 3) bleeding event, deep vein thrombosis requiring medical intervention (including pulmonary embolism), hemoptysis or other signs of pulmonary haemorrhage, or esophageal varices within 3 months of enrollment are not eligible.
- Patients with a bleeding diathesis or vasculitis are not eligible.
- Patients with a history of central nervous system (CNS) arterial/venous thromboembolic events (VTEs) including transient ischemic attack (TIA) or cerebrovascular accident (CVA) within 6 months of study enrollment are not eligible.
- Patients with myocardial infarction or unstable angina within the prior 6 months.
- Patients with significant vascular disease or peripheral vascular disease.
- Patients with a history of hypertensive crisis or hypertensive encephalopathy within 6 months of study enrollment are not eligible.
- Patients who have non-healing wound, unhealed or incompletely healed fracture, or a compound (open) bone fracture at the time of enrolment are not eligible.
- Patients previously treated and progressed on combination cyclophosphamide and vinorelbine regimen. (Patients who received combination as maintenance therapy, without progression, would be eligible.)
- Patients with a known hypersensitivity to ramucirumab, cyclophosphamide, vinorelbine or any of the excipients of the medicinal products.
- Patients who have previously received any exposure to ramucirumab are not eligible.
- Patients with CNS involvement are ineligible.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the efficacy of ramucirumab in combination with cyclophosphamide and vinorelbine compared with cyclophosphamide and vinorelbine in pediatric and young adult patients with DSRCT (DSRCT= desmoplastic small round cell tumor).;Secondary Objective: -To evaluate the safety and tolerability of ramucirumab in combination with cyclophosphamide and vinorelbine compared with cyclophosphamide and vinorelbine in pediatric and young adult patients with DSRCT<br>- To characterize the PK of ramucirumab when co-administered with cyclophosphamide and vinorelbine in pediatric and young adult patients with DSRCT<br>- To assess the immunogenicity of ramucirumab when co-administered with cyclophosphamide and vinorelbine in pediatric and young adult patients with DSRCT;Primary end point(s): progression-free survival (PFS);Timepoint(s) of evaluation of this end point: The primary analysis will be triggered when PFS events have occurred for approximately 80% of the enrolled patients
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Serious adverse event, Adverse event, Safety laboratory assessments, Vital signs, Overall response rate, Duration of response, Complete response, Incidence of immunogenicity, Cmax (maximum concentration) and Cmin (minimum concentration);Timepoint(s) of evaluation of this end point: Throughout study