Childhood Soft Tissue SarcomaPhase 1NCT00019630
Liposomal Doxorubicin in Treating Children With Refractory Solid Tumors
A Phase 1 study testing doxorubicin HCl liposome in people with Childhood Soft Tissue Sarcoma and Childhood Liver Cancer, run by National Cancer Institute (NCI) at 2 sites in United States.
Trial summary
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of liposomal doxorubicin in treating children who have refractory solid tumors. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Childhood Soft Tissue Sarcoma, Childhood Liver Cancer, Bone Cancer
- What is being tested?
- doxorubicin HCl liposome
- How many people
- Not given
- Where
- 2 sitesUnited States
- Study dates
- Jul 1999to not given
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- Child, Adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics-- Histologically confirmed solid tumor, including but not limited to: Rhabdomyosarcoma and other soft tissue sarcomas Ewing's family tumors Osteosarcoma Neuroblastoma Wilms' tumor Hepatic tumors Germ cell tumors Primary brain tumors Histological confirmation for brain stem gliomas may be waived Refractory to standard treatment and no curative therapy available Measurable or evaluable disease Evidence of progressive disease on prior chemotherapy or radiotherapy or persistent disease after prior surgery --Prior/Concurrent Therapy-- Biologic therapy: At least 1 week since prior colony-stimulating factors (e.g., filgrastim (G-CSF), sargramostim (GM-CSF), or epoetin alfa) At least 4 months since prior bone marrow transplantation No concurrent anticancer immunotherapy Chemotherapy: See Disease Characteristics At least 3 weeks since prior chemotherapy (4 weeks for nitrosoureas) and recovered Prior anthracyclines as adjuvant front-line therapy allowed provided: No relapse during therapy At least 6 months since last dose Cumulative dose is no greater than 400 mg/m2 for patients who received bolus administration without a concurrent cardioprotectant (e.g., dexrazoxane) or received cardiac irradiation and no greater than 450 mg/m2 for patients who received either continuous infusion or administration with a concurrent cardioprotectant and have not received cardiac irradiation No other concurrent anticancer chemotherapy Endocrine therapy: Concurrent corticosteroids for brain tumor-associated edema allowed (must be on stable or decreasing dose for at least 1 week prior to study) Radiotherapy: See Disease Characteristics At least 4 weeks since prior radiotherapy and recovered No more than 1,500 cGy of prior cardiac radiotherapy No prior extensive radiotherapy (e.g., craniospinal radiation, total body radiation, or radiation to more than half of the pelvis) No concurrent anticancer radiotherapy Surgery: See Disease Characteristics Other: No other concurrent anticancer investigational agents No other concurrent liposomal formulations of any drug (e.g., liposomal amphotericin B) --Patient Characteristics-- Age: 21 and under Performance status: ECOG 0-2 Life expectancy: At least 2 months Hematopoietic: Absolute granulocyte count greater than 1,500/mm3 Hemoglobin greater than 8 g/dL Platelet count greater than 100,000/mm3 Hepatic: Bilirubin normal SGPT no greater than 2 times upper limit of normal No significant hepatic dysfunction Renal: Creatinine normal OR Creatinine clearance at least 60 mL/min Cardiovascular: Cardiac ejection fraction at least 45% on MUGA (National Cancer Institute patients) OR Shortening fraction at least 28% on echocardiogram (Children's Hospital of Philadelphia patients) No significant or preexisting cardiac dysfunction (e.g., recurrent or persistent cardiac dysrhythmia or an ejection fraction below the lower limit of normal on MUGA or echocardiogram) Pulmonary: No significant pulmonary dysfunction Other: Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception No clinically significant unrelated systemic illness (e.g., serious infections or organ dysfunction) No allergy to doxorubicin or other anthracyclines, eggs, egg products, or other liposomal drug formulations
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Pediatric Oncology Branch
Bethesda, Maryland, 20892
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: Phase I Study of Doxorubicin HCl Liposome in Pediatric Patients With Refractory Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.
PURPOSE: Phase I trial to study the effectiveness of liposomal doxorubicin in treating children who have refractory solid tumors.
Read the detailed description
OBJECTIVES: I. Determine the tolerance to and toxicity profile of doxorubicin HCl liposome (Lipodox) at standard doxorubicin doses and doses of Lipodox that were tolerable in adults administered every 3 weeks in pediatric patients with refractory solid tumors.
II. Determine the maximum tolerated dose of this drug in these patients if dose-limiting toxicity is observed at doses of 105 mg/m2 or less.
III. Determine the pharmacokinetics of this drug in these patients. IV. Assess the cardiotoxicity of this drug in children who have previously been treated with free doxorubicin and in children who have not previously received doxorubicin.
- Evaluate the feasibility of using cardiac MRI functional imaging as a screening tool for the quantitative assessment of doxorubicin-induced cardiotoxicity.
VI. Determine if serum troponin t levels are a useful biomarker for doxorubicin-induced myocardial damage.
PROTOCOL OUTLINE: This is a dose-escalation, multicenter study. Patients receive doxorubicin HCl liposome IV over 60 minutes. Treatment repeats every 4 weeks for a maximum of 10 courses in the absence of disease progression or unacceptable toxicity.
Cohorts of 4-6 patients receive escalating doses of doxorubicin HCl liposome until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which at least 2 of 4 or 2 of 6 patients experience dose-limiting toxicity.
Patients are followed every 3 months for 1 year, every 6 months for 2 years, and then annually thereafter.
PROJECTED ACCRUAL:
A total of 21-36 patients will be accrued for this study within 1-2 years.
- Conditions
- Childhood Soft Tissue Sarcoma, Childhood Liver Cancer, Bone Cancer, Brain Tumor, Kidney Tumor
- Being tested
- doxorubicin HCl liposome
- Keywords
- Ewing's family of tumors, Wilms' tumor, adult solid tumor, body system/site cancer, bone cancer, brain tumor, cancer, central nervous system cancer, childhood brain stem glioma, childhood brain tumor, childhood cancer, childhood central nervous system germ cell tumor, childhood cerebellar astrocytoma, childhood cerebral astrocytoma, childhood choroid plexus tumor, childhood craniopharyngioma, childhood ependymoma, childhood extracranial germ cell tumor, childhood extragonadal malignant germ cell tumor, childhood liver cancer
- Medical subject headings
- Bone Neoplasms, Brain Neoplasms, Carcinoma, Renal Cell, Neuroectodermal Tumors, Primitive, Peripheral, Wilms Tumor, Neoplasms, Central Nervous System Neoplasms, Astrocytoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
doxorubicin HCl liposomeDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2007 (estimated)
- Study startJul 1999
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Childhood Soft Tissue SarcomaStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Investigators
- Elizabeth Lowe, Study chair, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00019630
- Sponsor's protocol ID
- CDR0000066924
- Other IDs
- NCI-99-C-0039F, LIPO-NCI-99-C-0039, NCI-99-C-0039
- Status last verified
- Mar 2024
- First submitted
- 1 Mar 2007
- Last updated
- 4 Mar 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Doxorubicin HCl Liposome in Pediatric Patients With Refractory Solid Tumors
- ClinicalTrials.gov ID
- NCT00019630
- Acronym / sponsor ID
- CDR0000066924
- Conditions
- Childhood Soft Tissue Sarcoma, Childhood Liver Cancer, Bone Cancer, Brain Tumor, Kidney Tumor
- Keywords
- Ewing's family of tumors, Wilms' tumor, adult solid tumor, body system/site cancer, bone cancer, brain tumor, cancer, central nervous system cancer
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- Jul 1999
- First posted
- 5 Mar 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.
PURPOSE: Phase I trial to study the effectiveness of liposomal doxorubicin in treating children who have refractory solid tumors.
Detailed description
OBJECTIVES: I. Determine the tolerance to and toxicity profile of doxorubicin HCl liposome (Lipodox) at standard doxorubicin doses and doses of Lipodox that were tolerable in adults administered every 3 weeks in pediatric patients with refractory solid tumors.
II. Determine the maximum tolerated dose of this drug in these patients if dose-limiting toxicity is observed at doses of 105 mg/m2 or less.
III. Determine the pharmacokinetics of this drug in these patients. IV. Assess the cardiotoxicity of this drug in children who have previously been treated with free doxorubicin and in children who have not previously received doxorubicin.
- Evaluate the feasibility of using cardiac MRI functional imaging as a screening tool for the quantitative assessment of doxorubicin-induced cardiotoxicity.
VI. Determine if serum troponin t levels are a useful biomarker for doxorubicin-induced myocardial damage.
PROTOCOL OUTLINE: This is a dose-escalation, multicenter study. Patients receive doxorubicin HCl liposome IV over 60 minutes. Treatment repeats every 4 weeks for a maximum of 10 courses in the absence of disease progression or unacceptable toxicity.
Cohorts of 4-6 patients receive escalating doses of doxorubicin HCl liposome until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which at least 2 of 4 or 2 of 6 patients experience dose-limiting toxicity.
Patients are followed every 3 months for 1 year, every 6 months for 2 years, and then annually thereafter.
PROJECTED ACCRUAL:
A total of 21-36 patients will be accrued for this study within 1-2 years.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Age groups
- Child, Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| doxorubicin HCl liposome | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Elizabeth Lowe study chair
National Cancer Institute (NCI)
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing doxorubicin HCl liposome in people with Childhood Soft Tissue Sarcoma and Childhood Liver Cancer, run by National Cancer Institute (NCI) at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2024.