Recurrent NeuroblastomaNCT02075177
Expanded Access Study of Fenretinide Lym-X-Sorb Plus Ketoconazole in Neuroblastoma
A study testing Fenretinide Lym-X-Sorb Oral Powder and Ketoconazole in people with Recurrent Neuroblastoma and Neuroblastoma, run by South Plains Oncology Consortium at 3 sites in United States.
Trial summary
Currently there is no known effective treatment for recurrent/resistant neuroblastoma. Fenretinide is an anticancer agent that may work differently than standard chemotherapy medicines. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Recurrent Neuroblastoma, Neuroblastoma
- What is being tested?
- Fenretinide Lym-X-Sorb Oral Powder and Ketoconazole
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 3 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 Any age
- All sexes
- Child, Adult, Older adult
Who may be able to join
- diagnosis of neuroblastoma either by histologic verification and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines.
- must have high risk neuroblastoma with one of the following: 1) recurrent/progressive disease at any time, 2) refractory disease, 3) persistent disease after at least a partial response to frontline therapy, or 4) Second or greater complete remission after definitive disease progression.
- must have at least one of the following sites of disease: 1) measurable tumor on MRI, CT scan, or X-Ray; 2)MIBG scan with positive uptake in at least one site; 3) bone marrow with tumor cells seen on routine morphology.
- must have an ECOG performance status of 0, 1, or 2
- must have a life expectancy of greater than or equal to 8 weeks
- must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy
Show all 24Show fewer
- hemoglobin greater than or equal to 8.0 (may transfuse to achieve this level)
- ANC greater than or equal to 500 (must be at least 7 days after last dose of growth factor)
- platelet count greater than or equal to 50,000 (must be transfusion independent, defined as at least 1 week since last platelet transfusion)
- age-adjusted serum creatinine less than or equal to 1.5 times normal for age
- normal cardiac function documented by: ejection fraction (greater than or equal to 55%) documented by echocardiogram or radionuclide MUGA evaluation OR fractional shortening (greater than or equal to 27%) documented by echocardiogram AND EKG must demonstrate no abnormality severe enough to justify cardiac medications AND baseline QTc interval greater than or equal to 450 msecs
- total bilirubin less than or equal to 1.5 times normal for age
- ALT and AST less than or equal to 3 times normal for age (for this study, the upper limit of normal of ALT is defined as 45 U/L)
- normal prothrombin time (PT) for age
- baseline hepatitis titers without evidence of acute/active hepatitis. Patients will need to have a negative Hep B Surface Antigen (HBsAg), Hep B e Antigen (HBeAg), Anti-Hep B core Antibody IgM (Anti-HBc IgM), Anti-HAV IgM, and Anti-HCV IgM.
- Patients with CNS parenchymal or meningeal-based lesions are eligible. Patients with prior history of CNS irradiation are study eligible.
- Patients with a seizure disorder are eligible if seizures are controlled on anticonvulsants and if the specific anticonvulsant(s) is not contraindicated.
- Normal lung function as manifested by no dyspnea at rest and no oxygen requirement.
- Due to the potential teratogenic effects of retinoids, negative serum beta-HCG in females, and use of effective contraception in males and females of child-bearing potential, is required.
- skin toxicity no greater than grade 1 per CTCAE v4
- Serum triglycerides < 300mg/dL fasting or on a random plasma test.
- Serum calcium < 11.6mg/dL
- No hematuria and/or proteinuria greater than 1+ on urinalysis.
- Patients with known genetic metabolic conditions, or other ongoing serious medical issues, must be approved by the Study Chair prior to registration.
Who cannot join
- Pregnancy or breast feeding. Due to the potential teratogenic effects of retinoids, pregnant women are NOT eligible. Breast milk feeding by study patient is NOT allowed.
- Patients with history of organ and allogeneic stem cell transplantation.
- Patients with a known history of allergy to soy products.
- Patients with a known history of a severe allergy or sensitivity of wheat gluten.
- Patients requiring anti-arrhythmia cardiac medications are NOT eligible.
- Prior therapy with fenretinide, or fenretinide + ketoconazole, if DLT's were experienced.
Show all 26Show fewer
- A known history of intolerance of ketoconazole.
- Patients on other essential medications for which an interaction with ketoconazole can be expected and for which dose reductions to other essential medications cannot be made in a manner adequate to ensure patient safety.
- Patients who, in the opinion of the investigator, may not be able to comply with safety monitoring requirements of the study.
- Active hepatitis.
- Baseline cardiac QTc interval >450 msecs.
- Eligible for enrollment on other national or regional treatment protocols employing fenretinide/LXS oral power that are reasonably accessible to the patient.
- Patient must NOT receive other anti-cancer agents while on Study.
- Ceftriaxone (Rocephin®) is NOT permitted for 24 hours prior to the start of the oral fenretinide course, during the course, and for 24 hours after the completion of seven day fenretinide course due to concerns of possible adverse effects on the hepatic clearance of fenretinide. Alternative antibiotics should be used. Other cephalosporins are permitted.
- Acetaminophen (Tylenol®) is NOT permitted for 24 hours prior the start of the oral fenretinide course, during the course, and for 48 hours following the completion of the seven day fenretinide course due to concerns of possible hepatic interactions. Ibuprofen (Motrin®) should be used for antipyretic control during this time period.
- Palliative radiation is allowed.
- Patients should NOT receive supplemental Vitamin A, C, or E except as contained in routine total parenteral nutrition vitamin supplements, or in a single daily standard dose oral multivitamin supplement, because of possible interference with antitumor 4-HPR-induced, reactive oxygen species and/or ceramide, and due to the unknown effects of these drugs on retinol levels
- Patients must NOT take any drugs suspected of causing pseudotumor cerebri, which include tetracycline, nalidixic acid, nitrofurantoin, phenytoin, sulfonamides (Dapsone permitted), lithium, amiodarone, or vitamin A (except as part of routine TPN supplements or as part of a single daily standard dose oral multivitamin supplement).
- Concomitant use of herbal supplements or other alternative therapy medications IS CONTRAINDICATED due to potential adverse metabolic interactions of such supplements with fenretinide.
- Patients should NOT concurrently take medications that may potentially act as modulators of intracellular ceramide levels or ceramide cytotoxicity, sphingolipid transport, or p-glycoprotein (MDR1) or MRP1 drug/lipid transporters, such as: cyclosporine A or analogue; verapamil; tamoxifen or analogue; chlorpromazine; RU486; indomethacin; or sulfinpyrazone. Patients should NOT concurrently take medications that are known P450 inhibitors. Contact Study Chair if there are questions regarding the suitability of any medication.
- As corticosteroids may impact sphingolipid metabolism, systemic corticosteroids should NOT be used for emesis control during the course of the study. Systemic corticosteroids for asthma control are permissible but should be minimized. Inhaled corticosteroids for asthma control are allowed. Steroids for routine metabolic deficiency states are allowed. Steroids for CNS lesions are allowed.
- Because gastric acidity is necessary for the maximal dissolution and absorption of ketoconazole, when actively taking ketoconazole, patients should avoid/minimize concurrent medications that decrease gastric acid output (such as ranitidine) or increase gastric pH (such as Tums).
- Concomitant medication that may prolong cardiac QT interval, especially those with known interaction with ketoconazole (See Appendices IV, V and VI). A LIST OF THESE MEDICATIONS SHOULD BE PROVIDED TO THE PATIENT AND FAMILY - PLEASE SEE APPENDICES IV, V and VI FOR A LIST OF MEDICATIONS THAT SHOULD NOT BE USED WITH CONCURRENT KETOCONAZOLE OR THAT SHOULD BE USED WITH CAUTION. Please SEE APPENDIX IV for a partial list of medications potentially prolonging QT interval. Patients may NOT receive therapy with the medications listed as PROHIBITED in Appendix IV and V. Medications in Appendix VI may be used with cautions as noted.
- Regulatory:
- Patients and/or their parents or legal guardians must sign a written informed consent (or assent.)
- All institutional, FDA, and NCI requirements for human studies must be met.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
UT Southwestern Medical Center
Dallas, Texas, 75235
Cook Children's Hospital
Fort Worth, Texas, 76104
University of Oklahoma Health Science Center
Oklahoma City, Oklahoma, 79104
About this study
Official title: Expanded Access Study of Fenretinide (4-HPR, NSC 374551) Lym-X-Sorb (LXS) Oral Powder Plus Ketoconazole in Patients With Recurrent or Resistant Neuroblastoma (IND#68,254)
Currently there is no known effective treatment for recurrent/resistant neuroblastoma.
Fenretinide is an anticancer agent that may work differently than standard chemotherapy medicines. It may cause the buildup of wax-like substances in neuroblastoma cancer cells, called "ceramides" or other chemicals, called 'reactive oxygen species'. In laboratory studies it was found that if too much ceramide or reactive oxygen species build up in neuroblastoma cells, they may die.
Read more
In addition, researchers are testing to see if a drug called ketoconazole, commonly used to treat fungus infections, can increase fenretinide levels in the body by interfering with the body's ability to break down fenretinide.
This study is being done: 1) to allow patients with recurrent/refractory neuroblastoma patients who would otherwise not be able to access fenretinide/LXS oral powder for treatment to do so; 2) to further describe the side effects of fenretinide and ketoconazole when given by mouth for seven days every three weeks; 3) to determine if a patient's tumor gets smaller after treatment with fenretinide oral powder plus ketoconazole or fenretinide oral powder alone.
- Conditions
- Recurrent Neuroblastoma, Neuroblastoma
- Being tested
- Fenretinide Lym-X-Sorb Oral Powder, Ketoconazole
- Keywords
- neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fenretinide Lym-X-Sorb Oral PowderDrug
Also called 4-HPR, Fenretinide, 4-HPR/LXS, Fenretinide/LXS
Fenretinide Lym-X-Sorb 1500 mg/m2/day, daily for 7 days every 3 weeks
KetoconazoleDrug
Ketoconazole 6 mg/kg/day, daily for 7 days every 3 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2014 (estimated)
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.
- 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- South Plains Oncology Consortium
- Sponsor type
- Research network
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02075177
- Sponsor's protocol ID
- SPOC-2014-001
- Status last verified
- Mar 2022
- First submitted
- 27 Feb 2014
- Last updated
- 21 Mar 2022
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Study of Fenretinide (4-HPR, NSC 374551) Lym-X-Sorb (LXS) Oral Powder Plus Ketoconazole in Patients With Recurrent or Resistant Neuroblastoma (IND#68,254)
- ClinicalTrials.gov ID
- NCT02075177
- Acronym / sponsor ID
- SPOC-2014-001
- Conditions
- Recurrent Neuroblastoma, Neuroblastoma
- Keywords
- neuroblastoma
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- South Plains Oncology ConsortiumResearch network
- First posted
- 3 Mar 2014 (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.
Currently there is no known effective treatment for recurrent/resistant neuroblastoma.
Fenretinide is an anticancer agent that may work differently than standard chemotherapy medicines. It may cause the buildup of wax-like substances in neuroblastoma cancer cells, called "ceramides" or other chemicals, called 'reactive oxygen species'. In laboratory studies it was found that if too much ceramide or reactive oxygen species build up in neuroblastoma cells, they may die.
In addition, researchers are testing to see if a drug called ketoconazole, commonly used to treat fungus infections, can increase fenretinide levels in the body by interfering with the body's ability to break down fenretinide.
This study is being done: 1) to allow patients with recurrent/refractory neuroblastoma patients who would otherwise not be able to access fenretinide/LXS oral powder for treatment to do so; 2) to further describe the side effects of fenretinide and ketoconazole when given by mouth for seven days every three weeks; 3) to determine if a patient's tumor gets smaller after treatment with fenretinide oral powder plus ketoconazole or fenretinide oral powder alone.
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (24 listed)
- diagnosis of neuroblastoma either by histologic verification and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines.
- must have high risk neuroblastoma with one of the following: 1) recurrent/progressive disease at any time, 2) refractory disease, 3) persistent disease after at least a partial response to frontline therapy, or 4) Second or greater complete remission after definitive disease progression.
- must have at least one of the following sites of disease: 1) measurable tumor on MRI, CT scan, or X-Ray; 2)MIBG scan with positive uptake in at least one site; 3) bone marrow with tumor cells seen on routine morphology.
- must have an ECOG performance status of 0, 1, or 2
- must have a life expectancy of greater than or equal to 8 weeks
Key exclusion criteria (26 listed)
- Pregnancy or breast feeding. Due to the potential teratogenic effects of retinoids, pregnant women are NOT eligible. Breast milk feeding by study patient is NOT allowed.
- Patients with history of organ and allogeneic stem cell transplantation.
- Patients with a known history of allergy to soy products.
- Patients with a known history of a severe allergy or sensitivity of wheat gluten.
- Patients requiring anti-arrhythmia cardiac medications are NOT eligible.
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 Lym-X-Sorb Oral PowderAlso: 4-HPR, Fenretinide, 4-HPR/LXS, Fenretinide/LXSFenretinide Lym-X-Sorb 1500 mg/m2/day, daily for 7 days every 3 weeks | Drug | – |
| KetoconazoleKetoconazole 6 mg/kg/day, daily for 7 days every 3 weeks. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2022.
Related trials for Recurrent Neuroblastoma
RecruitingPhase 1/2
Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse
Recurrent Ganglioneuroblastoma, Recurrent Neuroblastoma
Active, not recruitingPhase 2
Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)
Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm
Active, not recruitingPhase 2
Tipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial
Malignant Solid Neoplasm, Recurrent Adrenal Gland Pheochromocytoma
Active, not recruitingPhase 2
Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma
Active, not recruitingPhase 2
Selpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial
Hematopoietic and Lymphatic System Neoplasm, Recurrent Ependymoma
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.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Fenretinide Lym-X-Sorb Oral Powder and Ketoconazole in people with Recurrent Neuroblastoma and Neuroblastoma, run by South Plains Oncology Consortium at 3 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?
The sponsor lists 24 inclusion and 26 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: status not confirmed, as of the sponsor's last update on 21 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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.
Are results available for this study?
Results are not available in the source record.
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 21 Mar 2022.