A Randomized, Open-Label Phase 1b/2 Study Evaluating Ramucirumab in Pediatric Patients and Young Adults with Relapsed, Recurrent, or Refractory Desmoplastic Small Round Cell Tumor.

Phase 2

Withdrawn

• Conditions: Desmoplastic round cell tumour; abdominal/pelvic tumors

• Registration Number: NL-OMON52509
• Lead Sponsor: Eli Lilly

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Withdrawn
• Sex: Not specified
• Target Recruitment: 1

Inclusion Criteria

- Patients with relapsed, recurrent, or refractory DSRCT.- Patients must be 35
months to <=29 years of age at the time of study enrollment.- Patients must have
received at least one prior line of systemic treatment, have measurable disease
by RECIST 1.1, and must 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.able for thi

Exclusion Criteria

- 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 known hypersensitivity to cyclophosphamide or
vinorelbine.- Patients who have previously received any exposure to ramucirumab
are not eligible.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>To evaluate the efficacy of ramucirumab in combination with cyclophosphamide<br /><br>and vinorelbine compared with cyclophosphamide and vinorelbine in pediatric and<br /><br>young adult patients with DSRCT. With it's primary endpoint, Progression Free<br /><br>Survival (PFS)</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>The secondary endpoints for the study are:<br /><br>- To evaluate the safety and tolerability of ramucirumab in combination with<br /><br>cyclophosphamide and vinorelbine compared with cyclophosphamide and vinorelbine<br /><br>in pediatric and young adult patients with DSRCT.<br /><br>- To evaluate the efficacy of ramucirumab in combinationwith cyclophosphamide<br /><br>and vinorelbine compared with cyclophosphamide and vinorelbine in pediatric and<br /><br>young adult patients with DSRCT. With endpoints Overall Response Rate (ORR),<br /><br>Duration of Response (DoR) & Complete Response (CR)<br /><br>- To characterize the PK of ramucirumab when co-administered with<br /><br>cyclophosphamide and vinorelbine in pediatric and young adult patients with<br /><br>DSRCT.<br /><br>- To assess the immunogenicity of ramucirumab when co-administered with<br /><br>cyclophosphamide and vinorelbine in pediatric and young adult patients with<br /><br>DSRCT</p><br>