NCT01962103

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

A Phase 1/2, Multicenter, Open-label, Dose-finding Study to Assess the Safety, Tolerability, and Preliminary Efficacy of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent or Refractory Solid Tumors.

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 107 (actual)
• Sponsor: Celgene · Industry
• Sex: All
• Age: 6 Months – 24 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

ABI-007-PST-001 is a Phase 1/2, multicenter, open-label, dose-finding study to assess the safety , tolerability, and preliminary efficacy of weekly nab-paclitaxel in pediatric patients with recurrent or refractory solid tumors (excluding brain tumors). The Phase 1 portion of the study, with a dose escalation design, ended and the recommended Phase 2 dose (RP2D) was determined as 240 mg/m\^2 intravenously (IV) in patients weighing \> 10 kg and 11.5 mg/kg in patients weighing ≤ 10 kg, on Days 1, 8 and 15 of a 28-day cycle. The Phase 2 portion of the study will enroll additional patients at the RP2D into 1 of 3 solid tumor groups \[neuroblastomas, rhabdomyosarcomas, Ewing's sarcomas\]. Both phases of the study are open-label and conducted at multiple centers. The Phase 2 is using a Simon 2-stage design to monitor patient enrollment for each group separately. The rhabdomyosarcoma group, neuroblastoma or Ewing's sarcoma groups did not reach the expected number of 2 responders out of 14 efficacy eligible patients. Consequently, the groups were stopped.

Conditions

• 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

Interventions

Type	Name	Description
DRUG	nab-paclitaxel	nab-paclitaxel 120-270 mg/m2 IV on Days 1, 8 and 15 of a 28-day cycle
DRUG	nab-paclitaxel	IV infusion

Timeline

• Start date: 2013-12-04
• Primary completion: 2017-12-05
• Completion: 2018-11-06
• First posted: 2013-10-14
• Last updated: 2019-12-27
• Results posted: 2018-12-27

Locations

20 sites across 7 countries: United States, Canada, France, Italy, Spain, Switzerland, United Kingdom