NeuroblastomaPhase 1NCT00003191
Fenretinide in Treating Children With Solid Tumors
A Phase 1 study testing fenretinide in people with Neuroblastoma and Unspecified Childhood Solid Tumor, Protocol Specific, run by National Cancer Institute (NCI) at 26 sites in 2 countries.
Trial summary
Phase I trial to study the effectiveness of fenretinide in treating children who have solid tumors that have not responded to standard therapy. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Neuroblastoma, Unspecified Childhood Solid Tumor, Protocol Specific
- What is being tested?
- fenretinide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 26 sitesin 2 countries
- Study dates
- Mar 1998to Mar 2005
- 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
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS:
- Histologically confirmed malignant solid tumor that is refractory to conventional therapy or recurrent neuroblastoma treated with myeloablative therapy and autologous stem cell transplant in second complete or partial response
- Bone marrow metastases with granulocytopenia, anemia, and/or thrombocytopenia are eligible
PATIENT CHARACTERISTICS:
- Age: Under 21 at diagnosis
- Performance status: CCG 0-2
- Life expectancy: At least 2 months
- Absolute neutrophil count at least 750/mm3
- Platelet count at least 50,000/mm3
- Hemoglobin at least 7.0 g/dL
- Bilirubin no greater than 1.5 mg/dL
- SGOT and SGPT less than 2.5 times normal
- Creatinine no greater than 1.5 g/dL OR creatinine clearance at least 50 mL/min OR radioisotope GFR at least 50 mL/min
- Seizure disorders controlled with anticonvulsants allowed
- No CNS toxicity greater than grade 2
- Not pregnant
- Fertile patients must use effective contraception
PRIOR CONCURRENT THERAPY:
- At least 1 month since prior autologous stem cell transplantation
- No prior allogeneic transplantation
- At least 2 weeks since prior chemotherapy (4 weeks for nitrosourea) and recovered
- No other concurrent chemotherapy
- No concurrent immunomodulating agents (including steroids)
- Concurrent corticosteroid therapy for increased intracranial pressure allowed
- Concurrent dexamethasone for CNS tumor allowed
- At least 2 weeks since prior radiotherapy
- Concurrent radiotherapy to localized lesions allowed
- At least 2 weeks since prior retinoids Prior isotretinoin or 9-cis-retinoic acid allowed
Trial locations and contacts
26 sites in 2 countries.
26 sites in 2 countries
United States25 sites
University of Michigan Comprehensive Cancer Center
Ann Arbor, Michigan, 48109-0752
Children's Hospital Medical Center - Cincinnati
Cincinnati, Ohio, 45229-3039
Children's Hospital of Columbus
Columbus, Ohio, 43205-2696
Cancer Center and Beckman Research Institute, City of Hope
Duarte, California, 91010-3000
University of Texas - MD Anderson Cancer Center
Houston, Texas, 77030-4009
Indiana University Cancer Center
Indianapolis, Indiana, 46202-5289
Children's Mercy Hospital
Kansas City, Missouri, 64108
Long Beach Memorial Medical Center
Long Beach, California, 90806
Children's Hospital Los Angeles
Los Angeles, California, 90027-0700
Jonsson Comprehensive Cancer Center, UCLA
Los Angeles, California, 90095-1781
University of Wisconsin Comprehensive Cancer Center
Madison, Wisconsin, 53792-6164
University of Minnesota Cancer Center
Minneapolis, Minnesota, 55455
Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, 37232-6838
Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901
NYU School of Medicine's Kaplan Comprehensive Cancer Center
New York, New York, 10016
Memorial Sloan-Kettering Cancer Center
New York, New York, 10021
Herbert Irving Comprehensive Cancer Center
New York, New York, 10032
Children's Hospital of Orange County
Orange, California, 92868
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Mayo Clinic Cancer Center
Rochester, Minnesota, 55905
Primary Children's Medical Center
Salt Lake City, Utah, 84113
UCSF Cancer Center and Cancer Research Institute
San Francisco, California, 94143-0128
Children's Hospital and Regional Medical Center - Seattle
Seattle, Washington, 98105
Children's National Medical Center
Washington D.C., District of Columbia, 20010-2970
Australia1 site
Princess Margaret Hospital for Children
Perth, Western Australia, 6001
About this study
Official title: A Phase I Study of Fenretinide (NSC #374551) in Children With High Risk Solid Tumors
Phase I trial to study the effectiveness of fenretinide in treating children who have solid tumors that have not responded to standard therapy. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.
Read the detailed description
OBJECTIVES:
- Determine the maximum tolerated dose of fenretinide (HPR) in children with high risk solid tumors.
II. Determine the toxicities of HPR in these patients. III. Determine the pharmacokinetics of HPR in these patients. IV. Determine the CSF level of HPR in patients whom cerebrospinal fluid is obtained for routine purposes while on this study.
- Determine the effect of HPR on plasma retinol levels in these patients. VI. Determine the activity of HPR in these patients. VII. Determine the antitumor activity of HPR on minimal residual bone marrow disease in neuroblastoma.
OUTLINE: This is a dose escalation study.
Patients receive oral fenretinide 3 times a day on days 1-7. Treatment repeats every 3 weeks for up to 8 courses. Patients may receive an additional 22 courses of therapy in the presence of stable or responding residual tumor. Patients with recurrent neuroblastoma, after prior myeloablative therapy with no measurable disease, will stop treatment after 8 courses. Cohorts of 3-6 patients receive escalating doses of fenretinide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose limiting toxicity.
Patients are followed until death.
- Being tested
- fenretinide
- Keywords
- recurrent neuroblastoma, unspecified childhood solid tumor, protocol specific
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
fenretinideDrug
| Study group | Role | What they receive |
|---|---|---|
| Arm I | Experimental | Patients receive oral fenretinide 3 times a day on days 1-7. Treatment repeats every 3 weeks for up to 8 courses. Patients may receive an additional 22 courses of therapy in the presence of stable or responding residual tumor. Patients with recurrent neuroblastoma, after prior myeloablative therapy with no measurable disease, will stop treatment after 8 courses. Cohorts of 3-6 patients receive escalating doses of fenretinide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose limiting toxicity. Receives: Drug: fenretinide |
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2003 (estimated)
- Study startMar 1998
- Primary completionMar 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for NeuroblastomaStudies 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
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Judith G. Villablanca, MD, Study chair, Children's Hospital Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00003191
- Sponsor's protocol ID
- NCI-2012-02262
- Other IDs
- CCG-09709, CDR0000066023
- Status last verified
- Aug 2006
- First submitted
- 1 Nov 1999
- Last updated
- 7 Feb 2013
- Results posted
- No
Publications
Children's Oncology Group (CCG 09709); Villablanca JG, Krailo MD, Ames MM, Reid JM, Reaman GH, Reynolds CP. Phase I trial of oral fenretinide in children with high-risk solid tumors: a report from the Children's Oncology Group (CCG 09709). J Clin Oncol. 2006 Jul 20;24(21):3423-30. doi: 10.1200/JCO.2005.03.9271.
PubMed 16849757Villablanca JG, Ames MW, Reid JM, et al.: Phase I trial of oral [N-(-4-hydroxyphenyl)retinamide] (4-HPR) in children with resistant/recurrent solid tumors: a children's cancer group study (CCG 09709). [Abstract] Proceedings of the American Society of Clinical Oncology 21: A-1588, 2002.
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of Fenretinide (NSC #374551) in Children With High Risk Solid Tumors
- ClinicalTrials.gov ID
- NCT00003191
- Acronym / sponsor ID
- NCI-2012-02262
- Keywords
- recurrent neuroblastoma, unspecified childhood solid tumor, protocol specific
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- Mar 1998
- Primary completion
- Mar 2005 (actual)
- First posted
- 26 Nov 2003 (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.
Phase I trial to study the effectiveness of fenretinide in treating children who have solid tumors that have not responded to standard therapy. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.
Detailed description
OBJECTIVES:
- Determine the maximum tolerated dose of fenretinide (HPR) in children with high risk solid tumors.
II. Determine the toxicities of HPR in these patients. III. Determine the pharmacokinetics of HPR in these patients. IV. Determine the CSF level of HPR in patients whom cerebrospinal fluid is obtained for routine purposes while on this study.
- Determine the effect of HPR on plasma retinol levels in these patients. VI. Determine the activity of HPR in these patients. VII. Determine the antitumor activity of HPR on minimal residual bone marrow disease in neuroblastoma.
OUTLINE: This is a dose escalation study.
Patients receive oral fenretinide 3 times a day on days 1-7. Treatment repeats every 3 weeks for up to 8 courses. Patients may receive an additional 22 courses of therapy in the presence of stable or responding residual tumor. Patients with recurrent neuroblastoma, after prior myeloablative therapy with no measurable disease, will stop treatment after 8 courses. Cohorts of 3-6 patients receive escalating doses of fenretinide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose limiting toxicity.
Patients are followed until death.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm I | Experimental | Drug: fenretinidePatients receive oral fenretinide 3 times a day on days 1-7. Treatment repeats every 3 weeks for up to 8 courses. Patients may receive an additional 22 courses of therapy in the presence of stable or responding residual tumor. Patients with recurrent neuroblastoma, after prior myeloablative therapy with no measurable disease, will stop treatment after 8 courses. Cohorts of 3-6 patients receive escalating doses of fenretinide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose limiting toxicity. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- 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 |
|---|---|---|
| fenretinide | Drug | Arm I |
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 7 Feb 2013.
- Results publication: PubMed 16849757Children's Oncology Group (CCG 09709); Villablanca JG, Krailo MD, Ames MM, Reid JM, Reaman GH, Reynolds CP. Phase I trial of oral fenretinide in children with high-risk solid tumors: a report from the Children's Oncology Group (CCG 09709). J Clin Oncol. 2006 Jul 20;24(21):3423-30. doi: 10.1200/JCO.2005.03.9271.
- Results publicationVillablanca JG, Ames MW, Reid JM, et al.: Phase I trial of oral [N-(-4-hydroxyphenyl)retinamide] (4-HPR) in children with resistant/recurrent solid tumors: a children's cancer group study (CCG 09709). [Abstract] Proceedings of the American Society of Clinical Oncology 21: A-1588, 2002.
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Neuroblastoma
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.
Judith G. Villablanca, MD study chair
Children's Hospital Los Angeles
26 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing fenretinide in people with Neuroblastoma and Unspecified Childhood Solid Tumor, Protocol Specific, run by National Cancer Institute (NCI) at 26 sites in 2 countries.
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 7 Feb 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States, Australia. 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?
On the ClinicalTrials.gov record for NCT00003191, which also lists 2 linked publications.
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 7 Feb 2013.