Nilotinib With Radiation for High Risk Chordoma

NCT01407198 | Completed Phase 1 DMC

• 1 other identifier: 11-072
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 1

Brief Summary

The study drug, Nilotinib, is believed to slow down tumor growth by regulating a gene involved in cellular growth of chordoma cells. During this research study, subjects will also receive radiation therapy which is considered a standard treatment for advanced chordomas. It is hoped by adding nilotinib, the benefits of radiation therapy can be enhanced without adding significant toxicities. The purpose of this research study is to determine the safety of nilotinib when used in combination with radiation therapy, and the highest dose of nilotinib that can be given safely with radiation therapy.

Conditions

Chordoma

Keywords

High Risk Chordoma

Outcome Measures

Primary Outcomes (1)

• To determine the dose limiting toxicities (DLTs) for participants when treated above the maximum tolerated dose (MTD).

  A DLT will be defined as any of the following events occurring during days 1-56 of study according to CTCAE version 4.0. MTD is then determined by identifying a dose cohort demonstrating \> or = 30% rate of DLTs, which are defined as: * Any Grade ≥ 3 nonhematologic toxicity except alopecia, nausea, or vomiting not otherwise controlled by maximual supportive care. * Grade 4 neutropenia (ANC \< 500/µL) lasting \> 5 days, Grade 3 thrombocytopenia lasting \> 7 days, or Grade 4 thrombocytopenia * Failure to resume treatment delays within a defined period of time.

  2 years

Secondary Outcomes (3)

• Number of Participants with Adverse Events

  2 years

• Survival

  2 years

• PDGFR signaling

  2 years

Study Arms (1)

Nilotinib/XRT

EXPERIMENTAL

Nilotinib is administered 400mg PO BID for 2 weeks and then administered concurrently with daily radiation therapy until completion of radiation therapy. Participants can then undergo surgery if clinically possible. After either surgery or definitive radiation, participants have the option to continue on nilotinib therapy.

Drug: Nilotinib; Radiation: Radiation therapy

Interventions

Nilotinib DRUG

Orally, daily 200 - 400 mg BID

Also known as: AMN 107, Tasigna

Nilotinib/XRT

Radiation therapy RADIATION

External beam radiation will be delivered at 1.8 Gy per day, 5 days per week (excluding holidays)for a total of 28 fractions over a 6 weeks period.

Also known as: External beam radiation

Nilotinib/XRT

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Histologically confirmed chordoma
• Considered to have high risk disease
• Measurable disease
• Life expectancy \> 3 months
• Adequate organ function
• Able to swallow oral capsules

You may not qualify if:

• Previous treatment with any other tyrosine kinase inhibitor
• Previous treatment with radiotherapy to the primary or recurrent chordomas
• Impaired cardiac function
• Currently receiving treatment with strong CYP3A4 inhibitors
• Requires anticoagulation with coumadin
• Impaired GI function or GI disease that may significantly alter the absorption of study drug
• Acute or chronic pancreatic disease
• Known cytopathologically confirmed CNS infiltration
• Another primary malignant disease which requires systemic treatment
• Acute or chronic liver disease or severe renal disease considered unrelated to the cancer
• History of significant congenital or acquired bleeding disorder unrelated to cancer
• Major surgery within 4 weeks prior to Day 1 of the study or who have not recovered from prior surgery
• Treatment with other investigational agents within 30 days of Day 1
• History of non-compliance to medical regimens
• Pregnant or breast-feeding

Sponsors & Collaborators

• Massachusetts General Hospital: lead

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Chordoma

Interventions

nilotinib; Radiotherapy

Condition Hierarchy (Ancestors)

Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Therapeutics

Study Officials

• Edwin Choy, MD, PhD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Instructor

Study Record Dates

• First Submitted: July 28, 2011
• First Posted: August 2, 2011
• Study Start: August 1, 2011
• Primary Completion: February 1, 2016
• Study Completion: June 1, 2025
• Last Updated: December 19, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)