Study to Find a Safe Dose and Show Early Clinical Activity of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent/ Refractory Solid Tumors

Completed · Phase 1/Phase 2

Conditions studied: Neuroblastoma, Rhabdomyosarcoma, Ewing's Sarcoma, Ewing's Tumor, Sarcoma, Ewing's, Sarcomas, Epitheliod, Sarcoma, Soft Tissue, Sarcoma, Spindle Cell, Melanoma, Malignant Melanoma, Clinical Oncology, Oncology, Medical, Pediatrics, Osteosarcoma, Osteogenic Sarcoma, Osteosarcoma Tumor, Sarcoma, Osteogenic, Tumors, Cancer, Neoplasia, Neoplasm, Histiocytoma, Fibrosarcoma, Dermatofibrosarcoma

In brief

The purpose of this study is to find the safe dose of nab-paclitaxel in children with solid tumors, and to see if it works to treat these solid tumors in children and young adults (in Phase 1 ≤ 18 years old and in Phase 2 ≤ 24 years old). After the final dose has been chosen, patients will be enrolled according to the specific solid tumor type, (neuroblastoma, rhabdomyosarcoma, or Ewing's sarcoma), to see how nab-paclitaxel works in treating these tumors.

Key facts

Study ID

NCT01962103

Run by

Celgene

People needed

107

Starts

2013-12-04

Expected to finish

2018-11-06

Last updated by the study team

2019-12-27

Who can join

Age: 1 and older, up to 24. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patients must meet all of the following criteria to be enrolled in the study:
• Patient has a confirmed solid tumor diagnosis according to the
• following:
• Phase 1: patient has a recurrent or refractory solid tumor that has
• progressed or did not respond to standard therapy, or for which no
• standard anticancer therapy exists
• Phase 2: patient has radiologically documented measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 (for neuroblastoma, evaluable disease by 123\^I-metaiodobenzylguanidine [MIBG]/Curie score is also acceptable) in 1 of the following tumor types and has failed up to 3 lines of treatment: Group 1: neuroblastoma, Group 2: rhabdomyosarcoma; Group 3: Ewing's sarcoma.
• The patient has a Lansky/Karnofsky performance status score of ≥ 70%.
• The patient has adequate serum chemistry levels, evidenced by the
• following laboratory values
• aspartate aminotransferase (AST)/serum glutamic-oxaloacetric
• transaminase (SGOT), alanine aminotransferase (ALT)/serum glutamic
• pyruvate transaminase (SGPT) ≤ 2.5 × upper limit of normal range (ULN)
• Total bilirubin ≤ 1.5 × ULN
• Creatinine ≤ 1.5 × ULN
• The patient has adequate bone marrow function, evidenced by the
• following:
• Absolute neutrophil count ≥ 1.0 × 10\^9 cells/L
• Platelets ≥ 80 × 10\^9 cells/L (transfusion independent, defined as not
• receiving platelet transfusions within 7 days prior to laboratory sample). In the phase 2 portion, for patients with known bone marrow involvement, platelets ≥ 50 × 10\^9 cells/L
• Hemoglobin ≥ 8 g/dL (transfusion is permitted to fulfill this criterion).
• The patient (when applicable) or patient's parent(s) or legal guardian(s)
• understand(s) and voluntarily signed an informed consent document prior
• to any study-related assessments/procedures being conducted. Where
• locally applicable, the patient also understands and voluntarily provides

You may not qualify if…

• The presence of any of the following will exclude a patient from enrollment:
• <!-- -->
• The patient has a primary brain tumor(s) or brain metastasis (unless metastasis is treated and stable for > 28 days). In patients who are symptomatic, a brain scan is required to exclude metastasis.
• The patient has received therapeutic dose chemotherapy or radiotherapy ≤ 21 days prior to start of investigational product.
• The patient has received maintenance dose chemotherapy (e.g., low dose cyclophosphamide) ≤ 7 days from the first dose of investigational product.
• The patient has received any investigational therapy ≤ 28 days prior to start of investigational product. Investigational therapy is defined as any medicinal product that is not approved in the country of treatment for any indication, adult or pediatric.
• The patient has received any biological therapy ≤ 7 days prior to the start of investigational product, or monoclonal antibody ≤ 3 half-lives or 28 days, whichever is shorter, prior to the first dose of investigational product.
• The patient has received any hematopoietic stem cell transplantation (HSCT) ≤ 3 months prior to start of investigational product.
• The patient has received allogeneic hematopoietic stem cell transplantation (HSCT) ≤ 3 months or autologous HSCT ≤ 21 days prior to start of investigational product.
• The patient has not recovered from the acute toxic effects of prior chemotherapy, radiation, or major surgery/significant trauma.
• The patient has had minor surgery ≤ 7 days from the start of study treatment (excluding the placement of central/peripheral lines, skin biopsy).
• The patient has a known history of stroke, myocardial infarction, peripheral vascular disease, or recent (within 3 months) uncontrolled deep venous thrombosis.
• The patient has a known history or current diagnosis of human immunodeficiency virus (HIV) infection, regardless of treatment status.
• The patient has an uncontrolled intercurrent illness including but not limited to ongoing or active infection requiring antibiotic, antifungal, or antiviral therapy, symptomatic heart failure, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
• The patient has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the patient from participating in the study.
• The patient has any condition, including the presence of laboratory abnormalities, that places the patient at unacceptable risk if he/she were to participate in the study.
• The patient has any condition that confounds the ability to interpret data from the study.
• The patient or parent(s)/guardian(s) is/are unable to comply with the study visit schedule and other protocol requirements, in the opinion of the investigator.
• The patient has ≥ Grade 2 peripheral neuropathy by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) at screening.

Where it is running

• Phoenix Childrens Hospital — Phoenix, Arizona, United States
• Columbia University Medical Center — New York, New York, United States
• The Hospital for Sick Children — Toronto, Ontario, Canada
• Institute for Pediatric Hematology - Oncology, Leon Berard Cancer Center — Lyon, France
• Hopital d'Enfants, CHU Nancy — Nancy, France
• Institut Curie — Paris, France
• Institut Gustave Roussy — Villejuif, France
• Azienda Ospedaliera Universitaria Meyer — Florence, Italy
• Children's Hospital Largo — Genova, Italy
• Istituto Nazionale Tumori — Milan, Italy
• Clinica di Oncoematologia — Padova, Italy
• Policlinico Agostino Gemelli — Rome, Italy
• l'Azienda Ospedaliera Regina Margherita - Sant Anna — Torino, Italy
• Hospital Universitario Vall D Hebron — Barcelona, Spain
• Hospital Sant Joan de Deu — Barcelona, Spain
• Spanish National Cancer Research Centre — Madrid, Spain
• Hospital Universitario Virgen Del Rocio — Seville, Spain
• Unidad de Oncologia Pediatrica, Hospital Universitario la Fe — Valencia, Spain
• Universitäts-Kinderklinik — Zurich, Switzerland
• Royal Marsden Hospital — Sutton, United Kingdom

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.