Recurrent NeuroblastomaPhase 2NCT00053326
Fenretinide in Treating Children With Recurrent or Resistant Neuroblastoma
A Phase 2 study testing fenretinide and pharmacological study in people with Recurrent Neuroblastoma, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
This phase II trial is studying how well fenretinide works in treating children with recurrent or resistant neuroblastoma. 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
- Recurrent Neuroblastoma
- What is being tested?
- fenretinide and pharmacological study
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteUnited States
- Study dates
- May 2003to Mar 2006
- Phase
- Phase 2
- 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
Who may be able to join
- Diagnosis of recurrent or resistant/refractory high-risk neuroblastoma by one or both of the following:
- Histological confirmation
- Demonstration of tumor cells in bone marrow with increased urinary catecholamines
- Stratum I:
- At least 1 unidimensionally measurable lesion*
- At least 20 mm by MRI and/or CT scan OR at least 10 mm by spiral CT scan
Show all 48Show fewer
- Stratum II: Meets one or both of the following criteria:
- At least 1 site with positive uptake on meta-iodobenzylguanidine (MIBG) I 123 scan
- Tumor in bilateral bone marrow aspirate/biopsy by routine morphology (no NSE staining only)
- Stratum III:
- At least 5 tumor cells/10^6 mononuclear cells in the bone marrow by immunocytology only (on 2 successive bone marrows performed from 1 day to 4 weeks apart)
- Patients in first response (i.e., patients with persistent tumor at end of frontline therapy, but who have never had disease relapse or progression) must have histological* or morphological (by bone marrow) confirmation** of viable tumor on CT scan, MRI, or MIBG scan after completion of myeloablative therapy (for strata I and II)
- No catecholamine elevation only
- Performance status - 0-2
- At least 2 months
- Hemoglobin greater than 7.5 g/dL (transfusion allowed)
- Bilirubin no greater than 1.5 times normal
- SGPT and SGOT less than 2.5 times normal
- Creatinine normal for age
- No hematuria or proteinuria greater than 1+ on urinalysis
- Calcium less than 11.6 mg/dL
- Triglycerides less than 300 mg/dL
- Not pregnant
- Negative pregnancy test
- Fertile patients must use effective contraception
- No seizure disorders unless on anticonvulsants and well controlled
- No skin toxicity greater than grade 1
- Must be able to consume entire intact study capsule in the dosage prescribed for body surface area
- Recovered from prior immunotherapy
- At least 7 days since prior anticancer biologic therapy
- At least 2 days since prior growth factors
- Prior autologous stem cell transplantation allowed
- No prior allogeneic stem cell transplantation
- No concurrent immunomodulating agents
- At least 2 weeks since prior myelosuppressive chemotherapy (4 weeks for nitrosoureas) and recovered
- No concurrent anticancer chemotherapy
- No concurrent steroids
- Recovered from prior radiotherapy
- At least 4 weeks since prior radiotherapy to target lesion
- Prior radiotherapy to non target lesions allowed
- No concurrent radiotherapy to sole measurable lesion for symptom relief
- Concurrent palliative radiotherapy to non target or localized painful lesions allowed
- Prior tretinoin or isotretinoin allowed
- At least 2 weeks since other prior retinoids
- No prior fenretinide
- No concurrent supplemental oral or IV vitamin A, ascorbic acid, or vitamin E (except if contained in routine total parenteral nutrition [TPN] vitamin supplements)
- No concurrent drugs suspected of causing pseudotumor cerebri (e.g., tetracycline, nalidixic acid, nitrofurantoin, phenytoin, sulfonamides, lithium, amiodarone, or vitamin A [except as part of routine TPN supplements])
- No other concurrent anticancer agents
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Oncology Group
Arcadia, California, 91006-3776
About this study
Official title: A Phase II Study of Fenretinide (NSC# 374551, IND# 40294) in Children With Recurrent/Resistant High Risk Neuroblastoma
This phase II trial is studying how well fenretinide works in treating children with recurrent or resistant neuroblastoma. 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 response rate in pediatric patients with recurrent or resistant high-risk neuroblastoma treated with fenretinide.
Determine the toxic effects of this drug in these patients. Determine the proportion of patients with disease detected only by bone marrow immunocytology, who clear all evidence of disease during treatment with this drug.
Determine minimal residual disease response by marrow and meta-iodobenzylguanidine (MIBG) I 123 scan in patients treated with this drug.
OUTLINE: Patients are stratified according to presence of measurable disease on CT scan/MRI (yes vs no). A third stratum of patients with tumor cells in bone marrow by immunocytology only is enrolled but is not evaluated for response.
Patients receive oral fenretinide 3 times daily (or 2 times daily if over 18 years of age) on days 1-7. Treatment repeats every 3 weeks for up to 30 courses in the absence of disease progression or unacceptable toxicity. Patients in stratum III who fail to achieve a complete response after 8 courses of therapy are removed from study.
Patients are followed monthly until blood counts and visual acuity are stable or normalized and then every 6 months for 2 years and annually for 3 years.
PROJECTED ACCRUAL: A total of 70 patients (25 each for strata I and II, 20 for stratum III) will be accrued for this study within 1-2 years.
- Conditions
- Recurrent Neuroblastoma
- Being tested
- fenretinide, pharmacological study
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
fenretinideDrug
Given orally
Optional correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (fenretinide) | Experimental | Patients receive oral fenretinide 3 times daily (or 2 times daily if over 18 years of age) on days 1-7. Treatment repeats every 3 weeks for up to 30 courses in the absence of disease progression or unacceptable toxicity. Receives: Drug: fenretinide; Other: pharmacological study |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response rate
A responder is defined to be a patient who achieves a best overall response of complete response (CR), very good partial response (VGPR) or partial response (PR).
Measured: Up to 8 courses of therapy
Toxicity as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 3.0
Assessed via a descriptive tabulation of the toxicity rates, overall and by stratum.
Measured: Up to 5 years
Secondary outcomes (3)
Levels of fenretinide
Assessed via descriptive analysis of the steady state levels of fenretinide overall and by stratum
Measured: At baseline and during courses 1, 2, and 5
Plasma retinol levels
Assessed via descriptive analysis of the plasma retinol levels overall and by stratum.
Measured: At baseline and during courses 1, 2, and 5
Minimal residual disease (MRD) (Stratum 3)
Assessed by descriptive calculation of the proportion of responders.
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2003 (estimated)
- Study startMay 2003
- Primary completionMar 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recurrent 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Judith Villablanca, Principal investigator, Children's Oncology Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00053326
- Sponsor's protocol ID
- NCI-2012-01802
- Other IDs
- NCI-2012-01802, COG-ADVL0024, COG-A0996, CDR0000269408, COG-ANBL0321, ADVL0024, ANBL0321, U10CA098543
- Status last verified
- Oct 2013
- First submitted
- 27 Jan 2003
- Last updated
- 8 Oct 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Fenretinide (NSC# 374551, IND# 40294) in Children With Recurrent/Resistant High Risk Neuroblastoma
- ClinicalTrials.gov ID
- NCT00053326
- Acronym / sponsor ID
- NCI-2012-01802
- Conditions
- Recurrent Neuroblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- May 2003
- Primary completion
- Mar 2006 (actual)
- First posted
- 28 Jan 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.
This phase II trial is studying how well fenretinide works in treating children with recurrent or resistant neuroblastoma. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.
Detailed description
OBJECTIVES:
Determine the response rate in pediatric patients with recurrent or resistant high-risk neuroblastoma treated with fenretinide.
Determine the toxic effects of this drug in these patients. Determine the proportion of patients with disease detected only by bone marrow immunocytology, who clear all evidence of disease during treatment with this drug.
Determine minimal residual disease response by marrow and meta-iodobenzylguanidine (MIBG) I 123 scan in patients treated with this drug.
OUTLINE: Patients are stratified according to presence of measurable disease on CT scan/MRI (yes vs no). A third stratum of patients with tumor cells in bone marrow by immunocytology only is enrolled but is not evaluated for response.
Patients receive oral fenretinide 3 times daily (or 2 times daily if over 18 years of age) on days 1-7. Treatment repeats every 3 weeks for up to 30 courses in the absence of disease progression or unacceptable toxicity. Patients in stratum III who fail to achieve a complete response after 8 courses of therapy are removed from study.
Patients are followed monthly until blood counts and visual acuity are stable or normalized and then every 6 months for 2 years and annually for 3 years.
PROJECTED ACCRUAL: A total of 70 patients (25 each for strata I and II, 20 for stratum III) will be accrued for this study within 1-2 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (fenretinide) | Experimental | Drug: fenretinide, Other: pharmacological studyPatients receive oral fenretinide 3 times daily (or 2 times daily if over 18 years of age) on days 1-7. Treatment repeats every 3 weeks for up to 30 courses in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (48 listed)
- Diagnosis of recurrent or resistant/refractory high-risk neuroblastoma by one or both of the following:
- Stratum I:
- Stratum II: Meets one or both of the following criteria:
- Stratum III:
- Patients in first response (i.e., patients with persistent tumor at end of frontline therapy, but who have never had disease relapse or progression) must have histological* or morphological (by bone marrow) confirmation** of viable tumor on CT scan, MRI, or MIBG scan after completion of myeloablative therapy (for strata I and II)
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| fenretinideGiven orally | Drug | Treatment (fenretinide) |
| pharmacological studyOptional correlative studies | Other | Treatment (fenretinide) |
Primary outcome
Response rate
Time frame: Up to 8 courses of therapy
A responder is defined to be a patient who achieves a best overall response of complete response (CR), very good partial response (VGPR) or partial response (PR).
Toxicity as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 3.0
Time frame: Up to 5 years
Assessed via a descriptive tabulation of the toxicity rates, overall and by stratum.
3 secondary outcomes
Levels of fenretinide
Time frame: At baseline and during courses 1, 2, and 5
Plasma retinol levels
Time frame: At baseline and during courses 1, 2, and 5
Minimal residual disease (MRD) (Stratum 3)
Time frame: Up to 5 years
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 8 Oct 2013.
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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.
Judith Villablanca principal investigator
Children's Oncology Group
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing fenretinide and pharmacological study in people with Recurrent Neuroblastoma, run by National Cancer Institute (NCI) at 1 site 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?
The sponsor lists 48 inclusion and 0 exclusion criteria. 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 8 Oct 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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.
What does the primary outcome measure?
Response rate (time frame: Up to 8 courses of therapy). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
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 8 Oct 2013.