Desmoplastic Small Round Cell TumorPhase 1/2NCT04145349
CAMPFIRE: A Study of Ramucirumab (LY3009806) in Children and Young Adults With Desmoplastic Small Round Cell Tumor
A Phase 1/2 study testing Ramucirumab, Cyclophosphamide and Vinorelbine in people with Desmoplastic Small Round Cell Tumor, run by Eli Lilly and Company at 22 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 months to 29 years
- Conditions the study is for
- Desmoplastic Small Round Cell Tumor
- What is being tested?
- Ramucirumab, Cyclophosphamide and Vinorelbine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 22 sitesin 7 countries
- Study dates
- 22 Jan 2020to 23 Jan 2026
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 months to 29 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
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- 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:
- Creatinine clearance or radioscope glomerular filtration rate (GFR) ≥60 milliliters/minute/meters squared OR serum creatinine meeting the following parameters:
- for participants ≥18 years of age serum creatinine ≤1.5×upper limit of normal (ULN);
- for participants <18 years of age, serum creatinine based on age/gender as follows: Age 1 to <2 years maximum serum creatinine 0.6, Age 2 to <6 years maximum serum creatinine 0.8, Age 6 to <10 years maximum serum creatinine 1.0, Age 10 to <13 years maximum serum creatinine 1.2, Age 13 to <16 years maximum serum creatinine 1.5 for males and 1.4 for females, Age 16 to <18 years maximum serum creatinine 1.7 for males and 1.4 for females.
- Urine protein meeting the following parameters:
- for participants ≥18 years of age: <2+ on dipstick or routine urinalysis. If urine dipstick or routine analysis indicates proteinuria ≥2+, then a 24-hour urine must be collected and must demonstrate <2 grams of protein in 24 hours to allow participation in the study.
- for participants <18 years of age: ≤30 milligrams per deciliter urine analysis or <2+ on dipstick. If urine dipstick or routine analysis indicates proteinuria ≥2+, then a 24-hour urine must be collected and must demonstrate <1 g of protein in 24 hours to allow participation in the study.
- Adequate liver function:
- Total bilirubin: ≤1.5×ULN. Except participants with document history of Gilbert Syndrome who must have a total bilirubin level of <3.0×ULN.
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST): ≤2.5×ULN OR ≤5.0×ULN if the liver has tumor involvement.
- The participant has an adequate coagulation function as defined by International Normalized Ratio ≤1.5 or prothrombin time ≤1.5×ULN, and partial thromboplastin time ≤1.5×ULN if not receiving anticoagulation therapy. For participants receiving anticoagulants, exceptions to these coagulation parameters are allowed if they are within the intended or expected range for their therapeutic use. Participants must have no history of clinically significant active bleeding (defined as within 14 days of first dose of study drug) or pathological condition that carries a high risk of bleeding (for example, tumor involving major vessels or known esophageal varices).
- The participant has adequate hematologic and organ function ≤1 week (7 days) prior to first dose of study drug.
- Female participants of childbearing potential must have a negative urine or serum pregnancy test within 7 days prior to randomization. Male and female participants must agree to use highly effective contraception for the duration of the study and up to 3 months following the last dose of ramucirumab and vinorelbine, and 12 months following the last dose of cyclophosphamide in order to prevent pregnancy.
Who cannot join
- 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.
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- 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.
- Cardiac:
- Participants 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 prior to study enrollment are not eligible.
- Participants with myocardial infarction or unstable angina within the prior 6 months.
- Participants with New York Heart Association Grade 2 or greater congestive heart failure (CHF).
- Participants with serious and inadequately controlled cardiac arrhythmia.
- Participants with significant vascular disease (eg, aortic aneurysm, history of aortic dissection).
- Participants with clinically significant peripheral vascular disease.
- Participants who have a history of fistula, gastrointestinal (GI) ulcer or perforation, or intra-abdominal abscess within 3 months of study enrollment are not eligible.
- Participants with a history of hypertensive crisis or hypertensive encephalopathy within 6 months of study enrollment are not eligible.
- Participants who have non-healing wound, unhealed or incompletely healed fracture, or a compound (open) bone fracture at the time of enrollment are not eligible.
- Participants previously treated and progressed on combination cyclophosphamide and vinorelbine regimen. Participants who received combination as maintenance therapy, without progression, would be eligible.
- Participants with a known hypersensitivity to ramucirumab, cyclophosphamide, vinorelbine or any of the excipients of the medicinal products.
- Hepatic impairment:
- Severe liver cirrhosis Child-Pugh Class B (or worse).
- Cirrhosis with a history of hepatic encephalopathy.
- Clinically meaningful ascites resulting from cirrhosis and requiring ongoing treatment with diuretics and/or paracentesis.
- History of hepatorenal syndrome.
- The participant has a bowel obstruction, history or presence of inflammatory enteropathy or extensive intestinal resection (eg, hemicolectomy or extensive small intestine resection with chronic diarrhea), Crohn's disease, ulcerative colitis, or chronic diarrhea.
- The participant has a urinary outflow obstruction.
- The participant has Grade 2 hematuria or non-infectious cystitis at the time of screening.
- Participants with central nervous system (CNS) involvement are ineligible.
Trial locations and contacts
22 sites in 7 countries.
22 sites in 7 countries
United States6 sites
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Washington University
St Louis, Missouri, 63110
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Italy4 sites
Fondazione IRCCS Istituto Nazionale dei Tumori
Milan, Lombardy, 20133
Azienda Ospedaliera di Padova
Padova, Veneto, 35128
Ospedale Pediatrico Bambino Gesù
Rome, Roma, 165
Humanitas
Rozzano, Milano, 20089
United Kingdom4 sites
University College London Hospital
London, London, City of, NW1 2PG
Royal Marsden Hospital (Sutton)
London, Sutton, SM2 5PT
Royal Manchester Children's Hospital
Manchester, M13 9WL
The Christie
Manchester, M20 4BX
Germany3 sites
Universitaetsklinikum Essen
Essen, North Rhine-Westphalia, 45122
Universitaetsklinikum Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, 79106
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20251
Japan2 sites
National Cancer Center Hospital
Chuo-ku, Tokyo, 104-0045
Kyushu University Hospital
Fukuoka, 812-8582
Spain2 sites
Hospital Universitari Vall d'Hebron
Barcelona, Barcelona [Barcelona], 08035
Hospital Universitario Virgen Del Rocio
Seville, 41013
About this study
Official title: A Randomized, Open-Label Phase 1/2 Study Evaluating Ramucirumab in Pediatric Patients and Young Adults With Relapsed, Recurrent, or Refractory Desmoplastic Small Round Cell Tumor
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
- Being tested
- Ramucirumab, Cyclophosphamide, Vinorelbine
- Keywords
- soft tissue sarcoma, adolescents and young adults (AYAs), adolescent
- Medical subject headings
- Desmoplastic Small Round Cell Tumor, Sarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RamucirumabDrug
Also called LY3009806
Administered IV
CyclophosphamideDrug
Administered orally
VinorelbineDrug
Administered IV
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Cyclophosphamide; Drug: Vinorelbine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
- 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.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: Baseline Up to 23 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2019
- Study start22 Jan 2020
- Primary completion14 Jun 2024
- Study completion23 Jan 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Desmoplastic Small Round Cell TumorStudies of the same condition, open and under way.
- More trials run by Eli Lilly and CompanyThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eli Lilly and Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- 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), Study director, Eli Lilly and Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04145349
- Sponsor's protocol ID
- 17305
- Other IDs
- J1S-MC-JV01, 2018-004242-42, 2018
- Status last verified
- Jul 2026
- First submitted
- 29 Oct 2019
- Last updated
- 7 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.