NCT01518413

Phase 1 Dose Escalation Study of Sorafenib and Irinotecan Combination Therapy in Pediatric Patients With Relapsed or Refractory Solid Tumors

Completed Phase 1 Last updated 26 February 2015

What this trial tests

Phase 1 trial testing sorafenib in Rhabdomyosarcoma and Other Soft Tissue Sarcomas in 17 participants. Completed in 1 January 2015.

Timeline

1 December 2011

Primary endpoint
1 January 2015

1 January 2015

Quick facts

Lead sponsor	HMeany
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	17
Start date	1 December 2011
Primary completion	1 January 2015
Estimated completion	1 January 2015
Sites	4 locations across United States

Drugs / interventions tested

• sorafenib (SORAFENIB) — full drug profile →
• irinotecan (irinotecan) — full drug profile →

Conditions studied

• Rhabdomyosarcoma and Other Soft Tissue Sarcomas — all drugs for Rhabdomyosarcoma and Other Soft Tissue Sarcomas →
• Ewing's Sarcoma Family of Tumors — all drugs for Ewing's Sarcoma Family of Tumors →
• Osteosarcoma — all drugs for Osteosarcoma →
• Neuroblastoma — all drugs for Neuroblastoma →

Sponsor

HMeany

Who can join

Adults 2 to 22, any sex, with Rhabdomyosarcoma and Other Soft Tissue Sarcomas or Ewing's Sarcoma Family of Tumors. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Toxicity Profile
  Time frame: 24 months
  Determine the toxicity profile, dose-limiting toxicity (DLT) and maximum tolerated dose (MTD) of sorafenib, when administered in combination with oral irinotecan in children with relapsed or refractory solid tumors.
• Patient Related Outcomes
  Time frame: 24 months
  Demonstrate the feasibility of incorporating measurement of patient-related outcome into a four site phase 1 trial of sorafenib and irinotecan for children and adolescents. Demonstrate feasibility: defined as 70% of participants will complete the 5 patient-reported outcomes (PROs) (pain, fatigue, worry, sadness and physical functioning) at both baseline (pre-treatment) and at the end of the first

Sponsor's own description

The purpose of this study is to determine the safest and most effective oral dose combinations of sorafenib and irinotecan in pediatric patients with solid tumors, i.e. relapsed or refractory.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Osteosarcoma: Cells-of-Origin, Cancer Stem Cells, and Targeted Therapies.
   Abarrategi A, Tornin J, Martinez-Cruzado L, Hamilton A, et al · · 2016 · cited 155× · PMID 27366153 · DOI 10.1155/2016/3631764
2. Present Advances and Future Perspectives of Molecular Targeted Therapy for Osteosarcoma.
   Shaikh AB, Li F, Li M, He B, et al · · 2016 · cited 89× · PMID 27058531 · DOI 10.3390/ijms17040506
3. Phase 2 trial of sorafenib in children and young adults with refractory solid tumors: A report from the Children's Oncology Group.
   Kim A, Widemann BC, Krailo M, Jayaprakash N, et al · · 2015 · cited 66× · PMID 26207356 · DOI 10.1002/pbc.25548
4. Advances in paediatric cancer treatment.
   Saletta F, Seng MS, Lau LM. · · 2014 · cited 50× · PMID 26835334 · DOI 10.3978/j.issn.2224-4336.2014.02.01
5. A review of targeted therapies evaluated by the pediatric preclinical testing program for osteosarcoma.
   Sampson VB, Gorlick R, Kamara D, Anders Kolb E. · · 2013 · cited 43× · PMID 23755370 · DOI 10.3389/fonc.2013.00132
6. BRAF mutation and its inhibitors in sarcoma treatment.
   Liu H, Nazmun N, Hassan S, Liu X, et al · · 2020 · cited 40× · PMID 32476297 · DOI 10.1002/cam4.3103
7. A review of the biological and clinical implications of RAS-MAPK pathway alterations in neuroblastoma.
   Mlakar V, Morel E, Mlakar SJ, Ansari M, et al · · 2021 · cited 37× · PMID 34103089 · DOI 10.1186/s13046-021-01967-x
8. Bone Sarcomas in Pediatrics: Progress in Our Understanding of Tumor Biology and Implications for Therapy.
   Rivera-Valentin RK, Zhu L, Hughes DP. · · 2015 · cited 31× · PMID 26002157 · DOI 10.1007/s40272-015-0134-4