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| ID | Type | Description | Link |
|---|---|---|---|
| PBMTC ONC-032P | Other Identifier | Pediatric Blood and Marrow Tranplant Consortium |
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| Name | Class |
|---|---|
| Children's Hospitals and Clinics of Minnesota | OTHER |
| Schneider Children's Hospital | OTHER |
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The purpose of this study is to:
Find out how safe and effective (by monitoring the good and/or bad effects) treatment with high dose temozolomide, thiotepa and carboplatin with stem cell rescue followed by 13-cis-retinoic acid has on children and adolescents with recurrent/refractory brain tumors
Find out how the body uses 13-cis-retinoic acid by studying the your blood levels and proteins in the blood that break down the 13-cis-retinoic acid
Determine how well 13-cis-retinoic acid penetrates into the spinal fluid.
Researchers have used high doses of combination chemotherapy followed by a stem cell rescue to treat recurrent brain tumors with moderate success. High dose chemotherapy with stem cell rescue has resulted in long term survival of about 25% in patients with several different types of recurrent brain tumors. Stem cells are cells in the bone marrow that produce blood cells. The stem cells are collected from the blood of the patient before the high dose chemotherapy. Patients are given high doses of chemotherapy to kill every brain tumor cell, but in the process the cells of the bone marrow are also killed. The previously collected stem cells are then infused into the patient to rescue the bone marrow and allow for healthy blood cells to re-populate and grow in the bone marrow. Initial studies used the drug etoposide along with carboplatin and thiotepa for the high dose chemotherapy. Patients had severe side effects, especially severe mouth-sores, thought mainly due to the etoposide, and some patients died from these side effects.
Recent studies have shown that a new drug, temozolomide, is active against some types of brain tumors. When it was given as a single drug to children with solid tumors, the side effects were considered to be tolerable. Temozolomide is given by mouth. In this study, researchers want to give high dose chemotherapy that includes the drugs temozolomide in place of etoposide, along with thiotepa and carboplatin. Patients will then be given their own stem cells back to rescue the bone marrow from the chemotherapy. A preliminary trial using this new drug combination was performed and has shown that patients tolerate this drug combination, even at the very high doses that will be used in this protocol.
Another drug that is being used in pediatric cancer treatment is called 13-cis-retinoic acid. This drug is closely related to vitamin A. It is taken by mouth. Cancer cells are immature cells that have not "grown up" into adult cells that do work in the body. 13-cis-retinoic acid is thought to act on some types of cancer cells to make them mature into cells that function in the body. It has also been shown in the laboratory to cause some brain tumor cells to undergo apoptosis. It has been used in other types of pediatric cancers and research is just beginning to use it for treatment of recurrent brain tumors. In this study researchers want to give you 13-cis-retinoic acid for 6 months after you recover from the high dose chemotherapy with stem cell rescue.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Myeloablative Chemo-Temozolomide, Thiotepa, and Carboplatin. | Other |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| temozolomide, thiotepa, carboplatin, 13-cis-retinoic acid | Drug | 13-cis-retinoic acid, when absorbed, may be subject to first-pass metabolism and subsequent plasma (and tumor) concentrations will depend on the rate of metabolism to the inactive 4-oxo metabolite. |
| Measure | Description | Time Frame |
|---|---|---|
| Event-free Survival (EFS) | EFS will be reported in patients with recurrent or refractory medulloblastoma/ primitive neuroectodermal tumors. EFS is defined as the length of time after primary treatment for a cancer ends that the patient remains free of certain complications or events that the treatment was intended to prevent or delay. | Day +42 |
| Overall Survival (OS) | OS will be reported in patients with recurrent or refractory medulloblastoma/ primitive neuroectodermal tumors. OS is defined as the length of time from the start of treatment that patients diagnosed with disease are still alive. | Day +77 |
| Measure | Description | Time Frame |
|---|---|---|
| Toxicity of of 13-cis-retinoic Acid | To report the toxicity of of 13-cis-retinoic acid following high dose temozolomide, thiotepa and carboplatin with ASCR. | One year |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Sharon L Gardner, MD | NYU Langone Health | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | 85016 | United States | ||
| Emory University |
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| ID | Title | Description |
|---|---|---|
| FG000 | Myeloablative Chemo-Temozolomide, Thiotepa, and Carboplatin. | temozolomide, thiotepa, carboplatin, 13-cis-retinoic acid: 13-cis-retinoic acid, when absorbed, may be subject to first-pass metabolism and subsequent plasma (and tumor) concentrations will depend on the rate of metabolism to the inactive 4-oxo metabolite. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP_ICF | Yes | Yes | Yes | Study Protocol, Statistical Analysis Plan, and Informed Consent Form | Feb 18, 2011 |
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| Atlanta |
| Georgia |
| 30322 |
| United States |
| Hawaii Pacific Health | Lihue | Hawaii | 96766 | United States |
| Children's Memorial Hospital | Chicago | Illinois | 60614 | United States |
| Riley Hospital for Children | Indianapolis | Indiana | 46202 | United States |
| Children's Hospital and Clinics of Minnesota | Minneapolis | Minnesota | 55404 | United States |
| Roswell Park Cancer Institute | Buffalo | New York | 14263 | United States |
| Steven and Alexandra Cohen Children's Medical Center of New York- North Shore LIJ | New Hyde Park | New York | 11040 | United States |
| NYU Hassenfeld Center | New York | New York | 10016 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Medical Univ. of South Carolina | Charleston | South Carolina | 29425 | United States |
| Vanderbilt Univ. | Nashville | Tennessee | 37240 | United States |
| Children's Medical Center of Dallas | Dallas | Texas | 75235 | United States |
| MD Anderson Cancer Center (MDACC) | Houston | Texas | 77030 | United States |
| Virginia Commonwealth Univ. | Richmond | Virginia | 23284 | United States |
| Princess Margaret Hospital | Toronto | Ontario | M5G 2M9 | Canada |
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Myeloablative Chemo-Temozolomide, Thiotepa, and Carboplatin. | temozolomide, thiotepa, carboplatin, 13-cis-retinoic acid: 13-cis-retinoic acid, when absorbed, may be subject to first-pass metabolism and subsequent plasma (and tumor) concentrations will depend on the rate of metabolism to the inactive 4-oxo metabolite. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Event-free Survival (EFS) | EFS will be reported in patients with recurrent or refractory medulloblastoma/ primitive neuroectodermal tumors. EFS is defined as the length of time after primary treatment for a cancer ends that the patient remains free of certain complications or events that the treatment was intended to prevent or delay. | Efforts were made to contact the PI/study team members, but were unsuccessful. No study data are available | Posted | Day +42 |
|
| |||||||||||||||||||
| Primary | Overall Survival (OS) | OS will be reported in patients with recurrent or refractory medulloblastoma/ primitive neuroectodermal tumors. OS is defined as the length of time from the start of treatment that patients diagnosed with disease are still alive. | Efforts were made to contact the PI/study team members, but were unsuccessful. No study data are available | Posted | Day +77 |
|
| |||||||||||||||||||
| Secondary | Toxicity of of 13-cis-retinoic Acid | To report the toxicity of of 13-cis-retinoic acid following high dose temozolomide, thiotepa and carboplatin with ASCR. | Efforts were made to contact the PI/study team members, but were unsuccessful. No study data are available | Posted | One year |
|
|
Efforts were made to contact the PI/study team members, but were unsuccessful. No study data are available.
Efforts were made to contact the PI/study team members, but were unsuccessful. No study data are available
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Myeloablative Chemo-Temozolomide, Thiotepa, and Carboplatin. | temozolomide, thiotepa, carboplatin, 13-cis-retinoic acid: 13-cis-retinoic acid, when absorbed, may be subject to first-pass metabolism and subsequent plasma (and tumor) concentrations will depend on the rate of metabolism to the inactive 4-oxo metabolite. | 0 | 0 | 0 | 0 | 0 | 0 |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Sharon Gardner | NYU Langone | 212-263-9913 | Sharon.Gardner@nyulangone.org |
| Jun 17, 2021 |
| Prot_SAP_ICF_000.pdf |
| ID | Term |
|---|---|
| D001932 | Brain Neoplasms |
| D012008 | Recurrence |
| D004806 | Ependymoma |
| D005910 | Glioma |
| ID | Term |
|---|---|
| D016543 | Central Nervous System Neoplasms |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D018302 | Neoplasms, Neuroepithelial |
| D017599 | Neuroectodermal Tumors |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009370 | Neoplasms by Histologic Type |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009380 | Neoplasms, Nerve Tissue |
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| ID | Term |
|---|---|
| D000077204 | Temozolomide |
| D013852 | Thiotepa |
| D016190 | Carboplatin |
| D015474 | Isotretinoin |
| ID | Term |
|---|---|
| D003606 | Dacarbazine |
| D014226 | Triazenes |
| D009930 | Organic Chemicals |
| D007093 | Imidazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D013721 | Triethylenephosphoramide |
| D001388 | Aziridines |
| D001389 | Azirines |
| D056831 | Coordination Complexes |
| D012176 | Retinoids |
| D002338 | Carotenoids |
| D011090 | Polyenes |
| D000475 | Alkenes |
| D006839 | Hydrocarbons, Acyclic |
| D006838 | Hydrocarbons |
| D053138 | Cyclohexenes |
| D003510 | Cyclohexanes |
| D003516 | Cycloparaffins |
| D006840 | Hydrocarbons, Alicyclic |
| D006844 | Hydrocarbons, Cyclic |
| D013729 | Terpenes |
| D010860 | Pigments, Biological |
| D001685 | Biological Factors |
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| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|