New hope for rare bone cancer? drug shows promise in early trial
NCT ID NCT01267955
First seen Jun 27, 2026 · Last updated Aug 14, 2026 · Updated 2 times
Summary
This phase 2 trial tested vismodegib, a pill that targets a specific growth pathway, in 45 people with advanced chondrosarcoma (a rare bone cancer). The goal was to see if the drug could shrink tumors or stop them from growing. Participants had to have measurable disease and no more than three prior chemotherapy treatments. The study used a two-stage design to decide if the drug was worth further study.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Vismodegib (a targeted cancer drug taken by mouth)
- What this could lead to
- If it works, this could provide a new treatment option for people with advanced chondrosarcoma, a rare bone cancer that currently has few effective therapies.
- What could go wrong
- This is a small, early-phase trial with only 45 participants, so results may not apply to everyone. The drug may not shrink tumors or improve survival, and side effects like muscle spasms, hair loss, and fatigue are common.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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45 people
The number who actually took part.
- Started
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Dec 2010
- Expected to finish
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Jul 2027
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Patients must have histologically confirmed diagnosis of chondrosarcoma (conventional, mesenchymal, dedifferentiated or clear cell subtypes) * Patients must have measurable disease (outside any previously irradiated field) defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded) as \>= 10 mm with spiral computed tomography (CT) scan * No more than three prior lines of chemotherapy for advanced disease (including no more than 450 mg/m\^2 doxorubicin); at least three weeks since last chemotherapy (six weeks in case of nitrosoureas and mitomycin C), immunotherapy or any other pharmacological treatment and/or radiotherapy * Life expectancy of greater than 3 months * Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 (Karnofsky \>= 60%) * Leukocytes \>= 3,000/mcL * Absolute neutrophil count \>= 1,500/mcL * Platelets \>= 100,000/mcL * Total bilirubin within normal institutional limits * Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 2.5 X institutional upper limit of normal * Creatinine within normal institutional limits OR creatinine clearance \> 60 mL/min/1.73 m\^2 for patients with creatinine levels above institutional normal * Metastatic or unresectable locally advanced disease * Documented disease progression (as per RECIST) before study entry * Women of child-bearing potential and men must use two forms of contraception (i.e., barrier contraception and one other method of contraception) at least 4 weeks prior to study entry, for the duration of study participation, and for at least 24 months post-treatment for female patients and for 2 months for male patients; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately * Women of childbearing potential are required to have a negative serum pregnancy test (with a sensitivity of at least 25 mIU/mL) within 7 days prior to initiation of GDC-0449 (serum or urine); a pregnancy test (serum or urine) will be administered every 4 weeks while on study within the 24-hour period prior to the administration of GDC-0449; a positive urine test must be confirmed by a serum pregnancy test; prior to dispensing GDC-0449, the investigator must confirm and document the patient's use of two contraceptive methods, dates of negative pregnancy test, and confirm the patient's understanding of the teratogenic potential of GDC-0449 * Women of childbearing potential are defined as follows: * Patients with regular menses * Patients with amenorrhea, irregular cycles, or using a contraceptive method that precludes withdrawal bleeding * Women who have had a tubal ligation * Women are considered not to be of childbearing potential for the following reasons: * The patient has undergone hysterectomy and/or bilateral oophorectomy * The patient is post-menopausal defined by amenorrhea for at least 1 year in a woman \> 50 years old * The patient has permanent premature ovarian failure confirmed by specialist gynecologist * Ability to understand and the willingness to sign a written informed consent document * In accordance with French Regulatory Authorities: Patients with French Social Security in compliance with the French law relating to biomedical research (Huriet Law 88-1138 and related decrees) Exclusion Criteria: * Patients who have had chemotherapy or radiotherapy within 3 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 3 weeks earlier * Patients may not be receiving any other investigational agents * Patients with known brain metastases should be excluded from this clinical trial * History of allergic reactions attributed to compounds of similar chemical or biologic composition to GDC-0449 or other agents used in the study * Patients with malabsorption syndrome or other condition that would interfere with intestinal absorption; patients must be able to swallow capsules * Patients with clinically important history of liver disease, including viral or other hepatitis, or cirrhosis are ineligible * Patients with uncontrolled hypocalcemia, hypomagnesemia, hyponatremia, or hypokalemia defined as less than the lower limit of normal for the institution, despite adequate electrolyte supplementation, are excluded from this study * Tumor tissue sample not available for pathological review and/or correlative studies * Uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements * Pregnant women are excluded from this study; breastfeeding should be discontinued if the mother is treated with GDC-0449; these potential risks may also apply to other agents used in this study * Human immunodeficiency virus (HIV)-positive patients on combination antiretroviral therapy are ineligible
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Centre Leon Berard
Lyon, 69373, France
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Centre Oscar Lambert
Lille, 59020, France
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Gustave Roussy
Villejuif, 94805, France
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Hopital De La Timone
Marseille, 13385, France
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Institut Bergonie Cancer Center
Bordeaux, 33076, France
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Institut Curie Paris
Paris, 75005, France
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Experimental drug tested for rare cancer, trial stopped early
- Blood test could spot cartilage cancer early, without radiation
- Rare bone tumor under the microscope: can we predict its behavior?
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- Could your own tumor cells fight cancer? new trial tests immune cell therapy