34 Participants Needed

Ramucirumab for Desmoplastic Small Round Cell Tumor

Recruiting at 89 trial locations
Tm
Overseen ByThere may be multiple sites in this clinical trial. 1-877-CTLILLY (1-877-285-4559) or
Age: < 65
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Eli Lilly and Company
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires participants to stop all previous cancer treatments or investigational agents at least 7 days before starting the study treatment. However, it does not specify if you need to stop other non-cancer medications, so it's best to discuss your current medications with the trial team.

What evidence supports the effectiveness of the drug combination including Ramucirumab for treating desmoplastic small round cell tumor?

Research shows that a combination of vinorelbine and cyclophosphamide has been effective in some patients with desmoplastic small round cell tumor, suggesting potential benefits of similar drug combinations. Additionally, a regimen including vinorelbine, cyclophosphamide, and temsirolimus showed partial responses in patients, indicating that these drugs can be effective in treating this type of tumor.12345

Is vinorelbine generally safe for humans?

Vinorelbine (Navelbine) is generally considered safe for humans, with a favorable safety profile. However, it can cause some side effects, such as respiratory issues in less than 5% of patients and severe granulocytopenia (a drop in white blood cells) as a major toxicity.26789

What makes the drug combination of Cyclophosphamide, Ramucirumab, and Vinorelbine unique for treating Desmoplastic Small Round Cell Tumor?

This drug combination is unique because it includes Ramucirumab, which is a targeted therapy that works by blocking blood vessel growth in tumors, potentially enhancing the effectiveness of traditional chemotherapy drugs like Cyclophosphamide and Vinorelbine. This approach may offer a novel treatment option for Desmoplastic Small Round Cell Tumor, a condition with limited standard therapies.26101112

Research Team

C1

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)

Principal Investigator

Eli Lilly and Company

Eligibility Criteria

This trial is for children and young adults with relapsed, recurrent, or refractory Desmoplastic Small Round Cell Tumor (DSRCT). Participants must have tried at least one systemic treatment before, not be eligible for surgery, and have adequate organ function. They should not have severe infections, recent significant bleeding events or thrombosis, uncontrolled hypertension, certain heart conditions or a history of severe liver disease.

Inclusion Criteria

I cannot have surgery for my condition right now.
My DSRCT cancer has come back or hasn't responded to treatment.
I have had at least one treatment for my condition before, including chemotherapy.
See 9 more

Exclusion Criteria

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.
I have a history of serious heart, blood vessel problems, or severe reactions to medications.
I have not had significant bleeding or bleeding disorders in the last 3 months.
See 6 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ramucirumab in combination with other chemotherapy drugs, including cyclophosphamide and vinorelbine, for up to 10 cycles of 28 days each

10 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 months

Open-label extension (optional)

Participants may opt into continuation of treatment long-term if beneficial

Long-term

Treatment Details

Interventions

  • Cyclophosphamide
  • Ramucirumab
  • Vinorelbine
Trial Overview The study tests the safety and effectiveness of Ramucirumab combined with chemotherapy drugs Cyclophosphamide and Vinorelbine in treating DSRCT. It's part of a larger protocol aiming to speed up new cancer treatments for young patients. The commitment could exceed 12 months based on individual response to treatment.
Participant Groups
2Treatment groups
Experimental Treatment
Active Control
Group I: Ramucirumab + Cyclophosphamide + VinorelbineExperimental Treatment3 Interventions
Ramucirumab given intravenously (IV), Cyclophosphamide given orally and vinorelbine given IV.
Group II: Cyclophosphamide + VinorelbineActive Control2 Interventions
Cyclophosphamide given orally and vinorelbine given IV.

Cyclophosphamide is already approved in United States, European Union, Canada, Japan for the following indications:

🇺🇸
Approved in United States as Cytoxan for:
  • Breast cancer
  • Ovarian cancer
  • Multiple myeloma
  • Leukemia
  • Lymphoma
  • Rheumatoid arthritis
🇪🇺
Approved in European Union as Endoxan for:
  • Breast cancer
  • Ovarian cancer
  • Multiple myeloma
  • Leukemia
  • Lymphoma
  • Rheumatoid arthritis
🇨🇦
Approved in Canada as Neosar for:
  • Breast cancer
  • Ovarian cancer
  • Multiple myeloma
  • Leukemia
  • Lymphoma
  • Rheumatoid arthritis
🇯🇵
Approved in Japan as Endoxan for:
  • Breast cancer
  • Ovarian cancer
  • Multiple myeloma
  • Leukemia
  • Lymphoma

Find a Clinic Near You

Who Is Running the Clinical Trial?

Eli Lilly and Company

Lead Sponsor

Trials
2,708
Recruited
3,720,000+
Dr. Daniel Skovronsky profile image

Dr. Daniel Skovronsky

Eli Lilly and Company

Chief Medical Officer since 2018

MD from Harvard Medical School

David A. Ricks profile image

David A. Ricks

Eli Lilly and Company

Chief Executive Officer since 2017

BSc from Purdue University, MBA from Indiana University

Findings from Research

Vinorelbine tartrate is an effective treatment for non-small cell lung cancer (NSCLC) and has a low reported prevalence of respiratory adverse effects, typically under 5%.
However, there is a potential risk of increased pulmonary toxicity in patients with diffuse pulmonary NSCLC, as demonstrated by a case of acute respiratory failure following vinorelbine infusion in a patient with a hypercoagulable state.
Respiratory failure following vinorelbine tartrate infusion in a patient with non-small cell lung cancer.Kouroukis, C., Hings, I.[2019]
Vinorelbine, an oral and intravenous chemotherapy drug, was found to have a maximum tolerated dose of 30 mg/m² in children without bone marrow involvement, with myelosuppression (reduced blood cell production) being the main side effect.
The study revealed that children metabolize vinorelbine differently than adults, showing higher plasma clearance and lower overall drug exposure, which may influence dosing strategies for pediatric patients.
Phase I evaluation of oral and intravenous vinorelbine in pediatric cancer patients: a report from the Children's Oncology Group.Johansen, M., Kuttesch, J., Bleyer, WA., et al.[2018]
Vinorelbine tartrate is an FDA-approved treatment for advanced, unresectable non-small cell lung cancer, known for its favorable safety profile and the ability to be administered weekly in an outpatient setting.
While approximately 40% of patients experience severe granulocytopenia, this side effect is quickly reversible, and only about 8% of patients need hospitalization for related complications, indicating that it can be managed effectively.
Vinorelbine tartrate: a promising new chemotherapeutic agent.Baldwin, PD.[2018]

References

Response to vinorelbine and low-dose cyclophosphamide chemotherapy in two patients with desmoplastic small round cell tumor. [2018]
Respiratory failure following vinorelbine tartrate infusion in a patient with non-small cell lung cancer. [2019]
Recurrent desmoplastic small round cell tumor responding to an mTOR inhibitor containing regimen. [2018]
The use of interval-compressed chemotherapy with the addition of vincristine, irinotecan, and temozolomide for pediatric patients with newly diagnosed desmoplastic small round cell tumor. [2021]
[Desmoplastic small round cell tumors in adults: a new entity]. [2017]
Phase I evaluation of oral and intravenous vinorelbine in pediatric cancer patients: a report from the Children's Oncology Group. [2018]
Phase II trial of vinorelbine in recurrent and progressive epithelial ovarian cancer. [2018]
Vinorelbine tartrate: a promising new chemotherapeutic agent. [2018]
Experimental antitumor activity of 5'-nor-anhydrovinblastine navelbine. [2019]
10.United Statespubmed.ncbi.nlm.nih.gov
Response to vinorelbine with or without estramustine as second-line chemotherapy in patients with hormone-refractory prostate cancer. [2018]
11.United Statespubmed.ncbi.nlm.nih.gov
In vivo study of vinorelbine associations with cisplatin, 5-fluorouracil or actinomycin D relative to a non-small-cell lung carcinoma (NSCLCN6-L16). [2018]
Cisplatin and vinorelbine in advanced and/or metastatic adenocarcinoma of the endometrium: a new highly active chemotherapeutic regimen. [2020]