Neoplasms

All Conditions

Blood and Lymph Conditions

Immune System Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Rare Diseases

Sarcoma

Hodgkin Disease

Wilms Tumor

Sarcoma, Ewing

Hepatoblastoma

Neoplasms, Germ Cell and Embryonal

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Osteosarcoma

Neoplasms, Bone Tissue

Neoplasms, Connective Tissue

Lymphoma

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Soft Tissue Sarcoma

Hodgkin Lymphoma

Germ Cells Tumors

Wilms' Tumor

Ewing Sarcoma

Lymphosarcoma

Antineoplastic Agents

All Drugs and Chemicals

Antirheumatic Agents

Etoposide

Cyclophosphamide

Carboplatin

Melphalan

Etoposide phosphate

Mesna

Immunosuppressive Agents

Immunologic Factors

Antineoplastic Agents, Alkylating

Alkylating Agents

Patients on this study will undergo a tandem Peripheral Blood Stem Cell Rescue following high-dose chemotherapy. The first Peripheral Blood Stem Cell Rescue will consist of Etoposide, Carboplatin, Cyclophosphamide, and Mesna. Once the patient recovers, the patient will be evaluated again and will then undergo a second stem cell transplant consisting of the chemotherapy drugs; Melphalan, Cyclophosphamide, and Mesna.

High-Dose Chemotherapy with Tandem PBSC Rescue.

Morris Kletzel, M.D.

Meredith Marshall

This study uses a double autologous peripheral blood stem cell rescue (PBSC) following dose-intensive chemotherapy for the treatment of high-risk pediatric solid tumors.

Significant advances have been made in recent years in the treatment of solid tumors of childhood. However, much of the improvement in survival has been made in low stage and localized disease. Of significance is the fact that the improvements have come in up-front remission rates without translation into significantly high event-free survival(EFS) or overall survival (OS). This is despite the fact that these tumors as a whole are largely chemotherapy responsive.

Recent advances in the understanding of the biology of hematopoeitic stem cells have driven the design of treatment regimens that allow for dose intensification without unacceptable hematologic toxicity. Protocol development has focused on active agents that have a broad range between hematologic and non-hematologic toxicities. This study uses a double autologous peripheral blood stem cell rescue (PBSC) following dose-intensive chemotherapy for the treatment of high-risk pediatric solid tumors. This study utilizes PBSC to limit the risk of tumor cell contamination while retaining prompt hematologic recovery from these highly intensified treatments.

Tandem Peripheral Blood Stem Cell (PBSC) Rescue for High Risk Solid Tumors

High-Dose Chemotherapy With Tandem Peripheral Blood Stem Cell (PBSC) Rescue for the Treatment of High-Risk Pediatric Solid Tumors.

Ann & Robert H Lurie Children's Hospital of Chicago

Morris Kletzel

Symptoms and General Pathology

Recurrence

Neuroblastoma

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Kidney Neoplasms

Urologic Neoplasms

Urogenital Neoplasms

Syndrome

Neoplastic Syndromes, Hereditary

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Kidney Diseases

Urologic Diseases

Male Urogenital Diseases

Genetic Diseases, Inborn

Neuroepithelioma

Vaccines

Tumor lysate-pulsed dendritic cell (DC) vaccine following HSCT

Tumor lysate-pulsed dendritic cell vaccine

Tumor lysate-pulsed dendritic cell (DC) vaccine

Hematopoietic stem cell transplantation (HSCT)

James D Geiger, M.D.

Localized solid tumors such as, sarcoma, neuroblastoma, and Wilms' tumor, can generally be effectively treated with a combination of surgery, radiation and chemotherapy. However, patients with metastatic or relapsed disease have a very poor prognosis.

New approaches to the management of these difficult groups of patients are needed. There is evidence to suggest that solid tumors may be good candidates for immunotherapy approaches. In fact, recent experimental evidence indicates that the period of lymphopenia that occurs after stem cell transplant may be an opportune time to use an immunotherapy treatment approach. In light of the very poor prognosis of young patients with advanced solid tumors, this treatment approach warrants further investigation.

Localized solid tumors such as, sarcoma, neuroblastoma, and Wilms' tumor, can generally be effectively treated with a combination of surgery, radiation and chemotherapy. However, patients with metastatic or relapsed disease have a very poor prognosis.

For the past decade, efforts to increase overall survival and progression-free survival for patients with high-risk pediatric and young adult tumors, have evaluated the use of high-dose chemotherapy and hematopoietic stem cell transplantation (HSCT). The proportion of patients who enter a complete remission with HSCT is high, ranging from 81 to 90%. While autologous HSCT renders a large proportion of patients temporarily disease-free, relapse develops in the majority of patients.

Survival appears to have been most improved with this strategy for neuroblastoma, but relapses occur in the majority of patients. Similar strategies have also been tried for patients with advanced stage sarcoma and Wilms' tumor, but relapses are even more problematic.

New approaches to the management of these difficult groups of patients are needed. There is evidence to suggest that solid tumors may be good candidates for immunotherapy approaches. In fact, recent experimental evidence indicates that the period of lymphopenia that occurs after HSCT may be an opportune time to use this treatment approach. In light of the very poor prognosis of young patients with advanced solid tumors, this treatment approach warrants further investigation.

A Pilot Study of Tumor Cell Vaccine for High-risk Solid Tumor Patients Following Stem Cell Transplantation

A Pilot Study of Tumor Lysate-pulsed Dendritic Cell Vaccine for Immune Augmentation for High-risk Solid Tumor Patients Following Autologous Stem Cell Transplantation

University of Michigan Rogel Cancer Center

Professor of Surgery, Pediatric Surgery Section

Nervous System Diseases

Digestive System Diseases

Brain Neoplasms

Liver Neoplasms

Rhabdomyosarcoma

Trabectedin

Trabectedin (ecteinascidin-743, ET-743, YONDELIS [R])

Howard A Fine, M.D.

Background:

* Trabectedin is an experimental drug that kills some cancer cells in the laboratory and in mice by interfering with genetic material (DNA) in cancer cells.
* In some adult patients with cancer who received trabectedin, tumors grew slower or shrank.

Objectives:

* To determine a dose of trabectedin that can be given safely to children and adolescents as a 24-hour continuous infusion through a vein.
* To determine the side effects of trabectedin in children and adolescents.
* To study how the body handles trabectedin by measuring the amount of the drug in the bloodstream over time after a dose is given.
* To measure the effect of trabectedin on DNA in white blood cells.
* To determine if an individual's tumor cells have a specific proteins involved in DNA repair and if a pattern of genes can be identified in tumor samples that might help explain why trabectedin reduces tumors in some individuals and not others.
* To study genetic factors that may influence the way the body handles trabectedin.
* To see if trabectedin is beneficial in certain types of cancer.

Eligibility:

-Children between 4 year and 17 years of age with tumors that recur or no longer respond to standard treatment.

Design:

* Patients receive trabectedin as a 24-hour continuous infusion repeated every 21 days. The first three children entering the study receive a dose of 1.1 mg/m2. Subsequent groups of up to six patients receive higher doses (1.5 mg/m2 and 1.7 mg/m2) as long as the preceding dose is well tolerated. Patients enrolled at the lowest dose level may have their dose increased to the next level if they tolerated the lower dose well. Treatment may continue as long as the cancer does not worsen and the treatment is tolerated.
* Patients have blood drawn on days 1, 2, 3, 4, 5 and 7 of the first treatment cycle to study how the body handles trabectedin.
* A tumor sample obtained from a prior surgery or biopsy is examined for proteins involved in DNA repair.
* A blood sample is drawn to look for genetic factors that may influence how the body handles trabectedin.
* Patients have periodic physical examinations and blood tests. MRI or CT scans are done before starting therapy and after every two treatment cycles to evaluate the tumor.

Background:

Trabectedin (ET-743) is a natural product derived from the marine tunicate Ecteinascidia turbinata. It binds to the minor groove of DNA and interacts with various transcription factors resulting in cell cycle arrest. It also inhibits transcription coupled nucleotide excision repair system inducing lethal DNA strand breaks.

In preclinical and clinical studies, trabectedin has been found to be active in many soft tissue sarcomas including leiomyosarcoma, synovial cell sarcoma, neuroblastoma, rhabdomyosarcoma, melanoma, breast, ovarian, non-small cell lung, renal and prostate carcinoma.

In adult phase I and phase II studies, trabectedin has been well-tolerated up to dose levels of 1.9mg/m(2)/dose with the most common toxicities being fatigue, neutropenia, and reversible transaminase elevations. Objective responses were seen at doses equal to or greater than 1.5mg/m(2)/dose. Trabectedin 1.5mg/m(2) administered as a 24-hour continuous intravenous infusion is the recommended dose and schedule in adults.

A pediatric phase I study of trabectedin administered as a 3-hour infusion has been completed in the Children's Oncology Group. The maximum tolerated dose was 1.1 mg/m(2). Dose limiting toxicity was reversible elevation of hepatic transaminases.

Objectives:

Determine the maximum tolerated dose (MTD) of trabectedin administered as a 24-hour continuous infusion in children and adolescents with relapsed or refractory solid tumors.

Define the toxicity profile of trabectedin administered as a 24-hr infusion in children and adolescents.

Describe the pharmacokinetics of trabectedin administered as a 24-hr infusion in children and adolescents.

Quantify double strand DNA breaks in peripheral blood mononuclear cells of patients receiving trabectedin.

Evaluate archival formalin fixed paraffin embedded tumor tissue, when available from prior biopsies or surgical procedures, for the expression of the nucleotide excision repair proteins and genomic instability markers (histone gamma-H2AX, phospho-ATM, phospho-Chk2) by immunohistochemistry

Molecular characterization of DNA repair genes and correlation to outcome using mRNA isolated from microdissected archival formalin fixed, paraffin embedded tumor sections.

Study the pharmacogenetics of host DNA repair proteins and drug metabolizing enzymes to explore the impact of host factors on trabectedin toxicity.

Eligibility:

Children greater than or equal to 4 years and less than 17 years of age with relapsed or refractory solid tumors.

Design:

Patients will receive trabectedin as a 24-hour continuous infusion repeated every 21 days. The starting dose level is 1.1mg/m(2)/dose with escalations to 1.5mg/m(2)/dose and 1.7mg/m(2)/dose.

All patients will receive dexamethasone pretreatment and growth factor support with filgrastim or pegfilgrastim.

The definition of dose-limiting hepatic toxicity will be identical to the definition used in phase I trials in adults on the same dosing schedule.

Pharmacokinetic analysis will be performed on days 1, 2, 3, 4, 5 and 7 of the first treatment cycle.

Three to six patients will be enrolled at the first dose level and up to 6 patients will be enrolled on subsequent dose levels. Dose escalation will be based on tolerability of trabectedin at the prior dose level. For patients enrolled at dose level 1 (1.1mg/m(2)), intrapatient dose escalation to dose level 2 (1.5mg/m(2)) will be allowed with subsequent cycles if no dose limiting toxicities occurred at the lower dose level. Total accrual will be up to 24 patients.

Tabectedin to Treat Children and Adolescents With Cancer

A Phase I Trial and Pharmacokinetic Study of Trabectedin (YONDELIS [R], ET-743) in Children and Adolescents With Relapsed or Refractory Solid Tumors

National Cancer Institute (NCI)

Brigitte C. Widemann, M.D./National Cancer Institute

Infections

Virus Diseases

Vaccinia

Nerve Sheath Neoplasms

Neurofibrosarcoma

Myosarcoma

Communicable Diseases

Poxviridae Infections

DNA Virus Infections

Malignant Peripheral Nerve Sheath Tumor

Molgramostim

Sargramostim

Intratumoral Injection Dosage from 1 x 10\^6 pfu/kg to 3 x 10\^7 pfu/kg is administered once to 1-3 injectable tumors in pediatric patients.

Recombinant Vaccinia GM-CSF; RAC VAC GM-CSF (JX-594)

Timothy Cripe, MD, PhD

This is a Phase I, open-label, dose-escalation trial of JX-594 (Pexa-Vec) in pediatric patients with advanced/metastatic, unresectable solid tumors refractory to standard therapy and/or the patient does not tolerate standard therapies. Tumors are likely to include neuroblastoma, lymphoma, Wilms' tumor, rhabdomyosarcoma, Ewing's sarcoma, osteosarcoma, non-rhabdomyosarcoma soft tissue sarcomas, and malignant peripheral nerve sheath tumors. Benign tumors are excluded. These tumor types were selected because evidence of biological activity was observed in cancer cells lines and ex vivo infected primary human tissue samples, specifically pediatric cancer types such as sarcomas and neuroblastomas.

Safety Study of Recombinant Vaccinia Virus to Treat Refractory Solid Tumors in Pediatric Patients

A Phase I, Open-Label, Dose Escalation Study of JX-594 (Vaccinia GM-CSF/Thymidine Kinase-Deactivated Virus) Administered by Intratumoral Injection in Pediatric Patients With Unresectable Refractory Solid Tumors.

Determine the maximally-tolerated dose (MTD) and/or maximum-feasible dose (MFD) of JX-594 administered by intratumoral (IT) injection in pediatric patients with advanced/metastatic, unresectable refractory solid tumors

Solving Kids' Cancer

Jennerex Biotherapeutics

Irinotecan

Vincristine

Topoisomerase I Inhibitors

Enzyme Inhibitors

Tubulin Modulators

Antimitotic Agents

Expansion phase: Schedule B - Sequential dosing schedule: Of a 21-day cycle, regorafenib will be dosed sequentially, following administration of VI: Vincristine：intravenous bolus, 1.5 mg/m2 (0.05 mg/kg for subjects ≤ 10 kg), Day 1 and Day 8. Irinotecan: intravenously over 1 hour, 50mg/m2/day, Day 1 to Day 5. Regorafenib: orally, at a starting dose level of 72 mg/m2 (subjects 2 to less than 18 years old) or 60 mg/m2 (subjects 6 to less than 24 months old) once daily, Day 8 to Day 21.

Expansion phase: Schedule A - Concomitant dosing schedule: Of a 21-day cycle, regorafenib will be concomitantly administered with vincristine and irinotecan (VI): Vincristine: intravenous bolus, 1.5 mg/m2 (0.05 mg/kg for subjects ≤ 10 kg), Day 1 and Day 8. Irinotecan: intravenously over 1 hour, 50 mg/m2/day, Day 1 to Day 5. Regorafenib: orally, at a starting dose level of 72 mg/m2 (subjects 2 to less than 18 years old) or 60 mg/m2 (subjects 6 to less than 24 months old) once daily, Day 1 to Day 14.

Dose escalation phase: This phase of the study has been completed

Regorafenib will be given orally once a day, across cycles of 21 days each. During each cycle regorafenib is taken for 2 weeks followed by one week off the drug. Doses of the study drug used in this study are age-dependent and the children's dose will been adjusted based on the age and the body surface area and given either as tablets or granulate.

Regorafenib (BAY73-4506)

Vincristine will be given at a dose of 1.5 mg/m2 (0.05 mg/kg for subjects ≤ 10 kg, maximum 2.0 mg) on Day 1 and Day 8 in 21-day cycles.

Vincristine (Cellcristin®)

Irinotecan will be administered at a starting dose of 50 mg/m2/day from Day 1 to Day 5, in 21 day cycles.

Irinotecan (Irinotecan Cell pharm®)

Bayer Study Director

Dose escalation phase of the study :

To define the safety profile, maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of regorafenib administered orally as a single agent in a 3-weeks-on/1- week-off schedule in repeating cycles of 28 days in pediatric subjects with solid malignant tumors recurrent or refractory to standard therapy. To characterize the pharmacokinetics (PK) of regorafenib The dose escalation phase of the study has been completed.

Expansion phase:

To define the safety profile, MTD and the RP2D of regorafenib administered orally in combination with backbone chemotherapy (vincristine and irinotecan) at relapse in pediatric subjects with rhabdomyosarcoma (RMS) and other solid malignant tumors recurrent or refractory to standard therapy.

Expansion Phase of the study:

Subjects must have relapsed/refractory RMS or a solid malignant tumor (Ewing sarcoma, hepatoblastoma, neuroblastoma and Wilms tumor).

A Phase I Dose Finding Study in Children With Solid Tumors Recurrent or Refractory to Standard Therapy

A Multi-center, Open-label, Non-randomized, Phase I Dose Escalation Study of Regorafenib (BAY 73-4506) in Pediatric Subjects With Solid Malignant Tumors That Are Recurrent or Refractory to Standard Therapy

Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

MTD is defined as the dose level at which none or 1 of 6 participants experiences dose-limiting toxicity (DLT), when at least 2 of 3-6 participants experience a DLT at the next highest dose

In order to establish a RP2D, the MTD cohort will be expanded to have at least 12 evaluable subjects to confirm the RP2D. It is expected that at least 15 subjects evaluable for DLTs will be necessary to establish the RP2D of the combination"

Individual listings of adverse events will be provided. The incidence of treatment-emergent adverse events and drug-related adverse events, respectively, will be summarized by worst NCI-CTCAE v 4.0 grade and by dose level

Bayer

Eye Diseases

Glioma

Retinoblastoma

Retinal Neoplasms

Eye Neoplasms

Eye Diseases, Hereditary

Retinal Diseases

Antibodies

Immunoglobulins

Antibodies, Anti-Idiotypic

Immunoglobulin Idiotypes

Dosage form: intradermal injection. Dosage: 0.15 mg; 0.25 mg; 0.4 mg. Frequency: 3 biweekly injections or 6 biweekly injections. Duration: 4 weeks or 10 weeks.

racotumomab

Walter Cacciavillano, MD

Guillermo Chantada, MD

This study will be carried out in children with diagnosis of cancer with tumors known to express N-glycolylated gangliosides. The disease must be resistant to conventional therapy. The acute toxicity and immune response will be evaluated.

The expression of N-glycolylated gangliosides in tumors has previously been investigated in the tumor sample bank at this Hospital. The expression of N-glycolyl GM3 was shown in neuroblastoma, Ewing's sarcoma, Wilm's tumor and retinoblastoma.

Gliomas and the aforementioned tumor types have a very bad prognosis when conventional treatment is ineffective.

New therapeutic strategies have thus been examined, and several immunotherapeutic approaches, including dendritic cell vaccines, peptide vaccines and anti-idiotype vaccines are currently being assessed.

Racotumomab is an anti-idiotype antibody capable of inducing anti-N-glycolyl GM3 antibodies in patients with melanoma, breast cancer and lung cancer.

Dose escalation studies have shown the safety of racotumomab in the 0.5 to 2 mg dose range. The 1 mg dose level was selected for the ensuing clinical studies.

This clinical trial in children involves three dose levels: 0.15 mg, 0.25 mg and 0.4 mg, owing to the difference in body surface between an adult (1.73 sq. m in average) and the candidate population for this study (0.55 to 0.7 sq. m).

Use of Racotumomab in Patients With Pediatric Tumors Expressing N-glycolylated Gangliosides

Phase 1 Study on the Use of Racotumomab Anti-idiotype Antibody in Patients With Pediatric Malignancies That Express N-glycolylated Gangliosides and Are Resistant to Conventional Treatment.

One of the three dose levels assessed in this study will be selected for further clinical testing in children: 0.15 mg, 0,25 mg or 0.4 mg.

Active specific immunotherapy with racotumomab has shown to elicit antigen-specific immune responses in adult patients. The elicitation of anti-immunogen and anti-ganglioside antibodies will be assessed in serum samples prior and after racotumomab treatment.

Ministerio de Ciencia e Innovación, Spain

Laboratorio Elea Phoenix S.A.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Tandem Peripheral Blood Stem Cell (PBSC) Rescue for High Risk Solid Tumors	NCT00179816	Ewing's Sarcoma Soft Tissue Sar... Hepatoblastoma Hodgkin's Disea... Germ Cell Tumor	High-Dose Chemo...	- 21 Years	Ann & Robert H Lurie Children's Hospital of Chicago
A Pilot Study of Tumor Cell Vaccine for High-risk Solid Tumor Patients Following Stem Cell Transplantation	NCT00405327	Sarcoma Neuroblastoma Wilm's Tumor	Tumor lysate-pu... Hematopoietic s...	- 30 Years	University of Michigan Rogel Cancer Center
Tabectedin to Treat Children and Adolescents With Cancer	NCT01453283	Solid Tumors	Trabectedin (ec...	4 Years - 16 Years	National Institutes of Health Clinical Center (CC)
Tandem Peripheral Blood Stem Cell (PBSC) Rescue for High Risk Solid Tumors	NCT00179816	Ewing's Sarcoma Soft Tissue Sar... Hepatoblastoma Hodgkin's Disea... Germ Cell Tumor	High-Dose Chemo...	- 21 Years	Ann & Robert H Lurie Children's Hospital of Chicago
Safety Study of Recombinant Vaccinia Virus to Treat Refractory Solid Tumors in Pediatric Patients	NCT01169584	Neuroblastoma Rhabdomyosarcom... Lymphoma Wilm's Tumor Ewing's Sarcoma	Recombinant Vac...	2 Years - 21 Years	SillaJen, Inc.
Safety Study of Recombinant Vaccinia Virus to Treat Refractory Solid Tumors in Pediatric Patients	NCT01169584	Neuroblastoma Rhabdomyosarcom... Lymphoma Wilm's Tumor Ewing's Sarcoma	Recombinant Vac...	2 Years - 21 Years	SillaJen, Inc.
A Phase I Dose Finding Study in Children With Solid Tumors Recurrent or Refractory to Standard Therapy	NCT02085148	Pediatric Oncol...	Regorafenib (BA... Vincristine (Ce... Irinotecan (Iri...	6 Months - 18 Years	Bayer
Use of Racotumomab in Patients With Pediatric Tumors Expressing N-glycolylated Gangliosides	NCT01598454	Neuroblastoma Ewing's Sarcoma Wilm's Tumor Retinoblastoma Glioma	racotumomab	1 Year - 10 Years	Laboratorio Elea Phoenix S.A.

Spots Global Cancer Trial Database for wilm's tumor

Every month we try and update this database with for wilm's tumor cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.