CAMPFIRE: A Study of Ramucirumab (LY3009806) in Children and Young Adults With Desmoplastic Small Round Cell Tumor

NCT04145349 | Active Not Recruiting Phase 1 Results DMC FDA Drug

• 3 other identifiers: 17305J1S-MC-JV012018-004242-42
• Study Type: interventional
• Target: 30
• Locations: 7 countries
• Sites: 22

Brief Summary

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.

Conditions

Desmoplastic Small Round Cell Tumor

Keywords

soft tissue sarcoma; adolescents and young adults (AYAs); adolescent

Outcome Measures

Primary Outcomes (1)

• 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.

  Randomization to Objective Progression or Death Due to Any Cause (Up To 23 Months)

Secondary Outcomes (6)

• Overall Response Rate (ORR): Percentage of Participants Who Achieved a Complete Response (CR) or Partial Response (PR)

  Randomization until measured progressive disease (Up To 23 Months)

• Duration of Response (DoR)

  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

  Randomization until measured progressive disease (Up To 23 Months)

• Pharmacokinetics (PK): Maximum Serum Concentration of Ramucirumab (Cmax)

  End of ramucirumab infusion on Day 1 of Cycle 1

• PK: Minimum Serum Concentration of Ramucirumab (Cmin)

  Prior to ramucirumab infusion on - Day 15 of Cycle 1 and Day 1 of Cycles 2, 4, 7, and 10

Study Arms (2)

Ramucirumab + Cyclophosphamide + Vinorelbine

EXPERIMENTAL

Participants received the following treatments in a 28-day cycle, continuing until disease progression or a criterion for discontinuation was met. * Ramucirumab administered intravenously at a dose of 12 milligrams per kilogram (mg/kg) as a one-hour infusion on days 1 and 15. * Cyclophosphamide administered orally at 25 milligrams per meter square (mg/m2) daily from days 1 to 28. * Vinorelbine administered intravenously at 25 mg/m² on days 1, 8, and 15.

Drug: Ramucirumab; Drug: Cyclophosphamide; Drug: Vinorelbine

Cyclophosphamide + Vinorelbine

ACTIVE COMPARATOR

Participants received the following treatments in a 28-day cycle, continuing until disease progression or a criterion for discontinuation was met. * Cyclophosphamide administered orally at 25 mg/m² daily from days 1 to 28. * Vinorelbine administered intravenously at 25 mg/m² on days 1, 8, and 15.

Drug: Cyclophosphamide; Drug: Vinorelbine

Interventions

Ramucirumab DRUG

Administered IV

Also known as: LY3009806

Ramucirumab + Cyclophosphamide + Vinorelbine

Cyclophosphamide DRUG

Administered orally

Cyclophosphamide + Vinorelbine; Ramucirumab + Cyclophosphamide + Vinorelbine

Vinorelbine DRUG

Administered IV

Cyclophosphamide + Vinorelbine; Ramucirumab + Cyclophosphamide + Vinorelbine

Eligibility Criteria

• Age: 12 Months - 29 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Participants must have discontinued all previous treatments for cancer or investigational agents ≥7 days after the last dose or per the type of previous treatment as stated in the protocol and must have recovered from the acute effects to ≤Grade 2 for alopecia and decreased tendon reflex and to ≤Grade 1 for all other effects at the time of enrollment, unless otherwise noted. Consult with the Lilly clinical research physician or scientist for the appropriate length of time prior to the first dose of study treatment.
• Participants with relapsed, recurrent, or refractory DSRCT.
• Participants must:
• Have measurable disease by Response Evaluation Criteria in Solid Tumors, Version (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 participant is not a suitable candidate for the approved therapy.
• Not be eligible for surgical resection at time of enrollment.
• Adequate cardiac function, defined as: Shortening fraction of ≥27% by echocardiogram, or ejection fraction of ≥50% by gated radionuclide study.
• Adequate blood pressure (BP) control, defined as:
• Participants ≥18 years: Controlled hypertension defined as systolic BP ≤150 millimeters of mercury (mmHg) or diastolic BP ≤90 mmHg where standard medical management is permitted. Please note that ≥2 serial BP readings should be obtained and averaged to determine baseline BP.
• Participants \<18 years: A BP ≤95th percentile for age, height, and gender measured as described in National High Blood Pressure Education Program Working Group (NHBPEPWG) on High Blood Pressure in Children and Adolescents (2004), where standard medical management is permitted. Please note that ≥2 serial BP readings should be obtained and averaged to determine baseline BP.
• Adequate hematologic function, as defined as:
• Absolute neutrophil count (ANC): ≥750/microliters (µL) granulocyte-colony stimulating factor (G-CSF) permitted up to 48 hours prior. Participants with documented history of benign ethnic neutropenia or other conditions could be considered with a lower ANC after discussion with and approval from the Lilly clinical research physician or scientist.
• Platelets: ≥75,000/cubic millimeters. Platelet transfusion permitted up to 72 hours prior.
• Hemoglobin: ≥8 grams per deciliter (g/dL) (≥80 g/liter). Transfusions to increase the participant's hemoglobin level to at least 8 g/dL are permitted; however, study treatment must not begin until 7 days after the transfusion, and complete blood count criteria for eligibility are confirmed within 24 hr of first study dose.
• Adequate renal function, as defined as:

You may not qualify if:

• Participants with severe and/or uncontrolled concurrent medical disease or psychiatric illness/social situation that in the opinion of the investigator could cause unacceptable safety risks or compromise compliance with the protocol.
• Participants who have active infections requiring therapy.
• Participants with an active fungal, bacterial, and/or known severe viral infection including, but not limited to, human immunodeficiency virus (HIV) or viral (A, B, or C) hepatitis (screening is not required).
• Participants who have had allogeneic bone marrow or solid organ transplant are excluded.
• Surgery: Participants who have had, or are planning to have, the following invasive procedures are not eligible:
• Major surgical procedure, laparoscopic procedure, or significant traumatic injury within 28 days prior to enrollment.
• Central line placement or subcutaneous port placement is not considered major surgery.
• Core biopsy, fine needle aspirate, and bone marrow biopsy/aspirate are not considered major surgeries.
• Surgical or other wounds must be adequately healed prior to enrollment.
• Bleeding and thrombosis:
• Participants with evidence of active bleeding or a history of significant (≥Grade 3) bleeding event within 3 months prior to enrollment are not eligible.
• Participants with a bleeding diathesis or vasculitis are not eligible.
• Participants with known or prior history in the prior 3 months of esophageal varices are not eligible.
• Participants with a history of deep vein thrombosis requiring medical intervention (including pulmonary embolism) within 3 months prior to study enrollment are not eligible.
• Participants with a history of hemoptysis or other signs of pulmonary hemorrhage within 3 months prior to study enrollment are not eligible.

Sponsors & Collaborators

• Eli Lilly and Company: lead

Study Sites (22)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, 19104, United States

Location

Royal Children's Hospital

Parkville, Victoria, 3052, Australia

Location

Universitaetsklinikum Freiburg

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

Location

Universitaetsklinikum Essen

Essen, North Rhine-Westphalia, 45122, Germany

Location

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, 20251, Germany

Location

Fondazione IRCCS Istituto Nazionale dei Tumori

Milan, Lombardy, 20133, Italy

Location

Humanitas

Rozzano, Milano, 20089, Italy

Location

Ospedale Pediatrico Bambino Gesù

Rome, Roma, 165, Italy

Location

Azienda Ospedaliera di Padova

Padua, Veneto, 35128, Italy

Location

National Cancer Center Hospital

Chuo-ku, Tokyo, 104-0045, Japan

Location

Kyushu University Hospital

Fukuoka, 812-8582, Japan

Location

Hospital Universitari Vall d'Hebron

Barcelona, Barcelona [Barcelona], 08035, Spain

Location

Hospital Universitario Virgen Del Rocio

Seville, 41013, Spain

Location

University College London Hospital

London, London, City of, NW1 2PG, United Kingdom

Location

Royal Marsden Hospital (Sutton)

London, Sutton, SM2 5PT, United Kingdom

Location

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

Location

The Christie

Manchester, M20 4BX, United Kingdom

Location

Related Links

• CAMFIRE: A Study of Ramucirumab (LY3009806) in Children and Young Adults With Desmoplastic Small Round Cell Tumor

MeSH Terms

Conditions

Desmoplastic Small Round Cell Tumor; Sarcoma

Interventions

Ramucirumab; Cyclophosphamide; Vinorelbine

Condition Hierarchy (Ancestors)

Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Vinca Alkaloids; Secologanin Tryptamine Alkaloids; Indole Alkaloids; Alkaloids; Heterocyclic Compounds; Indoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Indolizidines; Indolizines

Results Point of Contact

• Title: Chief Medical Officer
• Organization: Eli Lilly and Company

ClinicalTrials.gov@lilly.com

800-545-5979

Study Officials

• Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST)

  Eli Lilly and Company

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 29, 2019
• First Posted: October 30, 2019
• Study Start: January 22, 2020
• Primary Completion: June 14, 2024
• Study Completion (Estimated): October 1, 2026
• Last Updated: December 8, 2025
• Results First Posted: July 20, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: Data are available 6 months after the primary publication and approval of the indication studied in the US and EU, whichever is later. Data will be indefinitely available for requesting.
• Access Criteria: A research proposal must be approved by an independent review panel and researchers must sign a data sharing agreement.

Locations

US (6); DE (3); JP (2); AU (1); IT (4); ES (2); GB (4)