Brain and Central Nervous System TumorsPhase 1NCT00003926
Amifostine to Protect From Side Effects of PSCT in Treating Patients With Solid Tumors
A Phase 1 study testing amifostine trihydrate, busulfan and filgrastim in people with Brain and Central Nervous System Tumors and Childhood Germ Cell Tumor, run by Masonic Cancer Center, University of Minnesota at 1 site 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. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. Read more about this study
- Who is it for?
- Ages 1 year to 45 years
- Conditions the study is for
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Chordoma
- What is being tested?
- amifostine trihydrate, busulfan and filgrastim
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 1998to Aug 2003
- 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 1 year to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Histologically confirmed high-risk or relapsed solid tumors or brain tumors, including:
- Metastatic or relapsed Ewing's sarcoma
- Metastatic or relapsed rhabdomyosarcoma
- Refractory Wilms' tumor
- Diffuse anaplastic Wilms' tumor
- Stage III or IV neuroblastoma
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- Recurrent retinoblastoma
- Metastatic or relapsed germ cell tumors
- Metastatic or relapsed other soft tissue sarcomas
- Small cell ovarian sarcoma
- Metastatic or relapsed primitive neuroectodermal tumors of the bone
- Recurrent brain tumors
- Desmoplastic small round cell tumors
- Recurrent or metastatic chordomas
- Metastatic or relapsed hepatoblastoma
- Patients receive peripheral blood stem cell transplantation only if in complete remission or in very good partial remission with no disease progression
- Must have radiologic, nuclear image, or histologic verification of relapse
- Age 1 to 45
- Performance status:Karnofsky 70-100%
- Absolute neutrophil count greater than 1,000/mm^3
- Platelet count greater than 100,000/mm^3
- Hemoglobin count at least 10 g/dL
- Bilirubin less than 2 times upper limit of normal (ULN)
- SGOT or SGPT less than 2.5 times ULN
- Creatinine less than 2 times ULN
- Creatinine clearance greater than 70 mL/min
- Cardiac shortening fraction greater than 30%
- Cardiac ejection fraction greater than 45%
- At least 1 week since prior hematopoietic growth factor and recovered
- At least 3 weeks since prior chemotherapy (6 weeks for nitrosoureas) and recovered
- Recovered from any prior therapy
Who cannot join
- Osteogenic sarcoma
- Less than 4 months
- Uncontrolled bleeding
- Congestive heart failure
- Uncontrolled hypertension
- Asthma
Show all 12Show fewer
- Pregnant or nursing
- Uncontrolled metabolic disease
- Active severe infection
- Allergy to aminothiol compounds
- Prior bone marrow transplantation
- Other concurrent investigational agents
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota Cancer Center
Minneapolis, Minnesota, 55455
About this study
Official title: A Phase I Study of the Chemoprotectant Amifostine With Autologous Stem Cell Transplantation for High Risk or Relapsed Pediatric Solid Tumors and Brain Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. Chemoprotective drugs such as amifostine may protect normal cells from the side effects of high-dose chemotherapy.
PURPOSE: Phase I trial to study the effectiveness of amifostine in protecting from the side effects of peripheral stem cell transplantation in treating patients who have high-risk or relapsed solid tumors.
Read the detailed description
OBJECTIVES:
- Determine the dose-limiting toxicity of amifostine chemoprotection with peripheral blood stem cell transplantation plus chemotherapy in patients with high-risk or relapsed solid tumors or brain tumors.
- Determine response or time to disease progression in patients treated with this regimen.
OUTLINE: This is a dose-escalation study of amifostine. Patients are stratified according to age (1 to 18 vs 19 to 45 years).
All patients receive filgrastim (G-CSF) IV for 1 week. On day 6 of G-CSF administration, patients undergo peripheral blood stem cell (PBSC) harvest followed by chemotherapy.
Patients receive oral busulfan every 6 hours on days -8 to -6 followed by melphalan IV over 30 minutes on days -5 and -4 and thiotepa IV over 2 hours on days -3 and -2. Patients receive amifostine IV over 5 minutes beginning 30 minutes prior to melphalan and thiotepa administration on days -5 to -1. PBSC are reinfused on day 0.
Cohorts of 3-6 patients receive escalating doses of amifostine until the maximum tolerated dose is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.
Patients are followed on day 50; at 3, 6, and 9 months; and at 1, 2, and 3 years post PBSC transplantation.
PROJECTED ACCRUAL: A maximum of 60 patients (30 per stratum) will be accrued for this study within 3 years.
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Chordoma, Kidney Cancer, Liver Cancer, Neuroblastoma
- Being tested
- amifostine trihydrate, busulfan, filgrastim, melphalan, thiotepa
- Keywords
- soft tissue sarcoma, regional neuroblastoma, disseminated neuroblastoma, recurrent Wilms tumor, recurrent retinoblastoma, recurrent adult brain tumor, adult rhabdomyosarcoma, ovarian germ cell tumor, chordoma, ovarian sarcoma, unresectable neuroblastoma, desmoplastic small round cell tumor, rhabdomyosarcoma, Ewing sarcoma, neuroectodermal tumor, teratoma, malignant testicular germ cell tumor, malignant ovarian germ cell tumor, extragonadal germ cell tumor, malignant germ cell tumor
- Medical subject headings
- Central Nervous System Neoplasms, Chordoma, Kidney Neoplasms, Liver Neoplasms, Neuroblastoma, Ovarian Neoplasms, Retinoblastoma, Sarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
amifostine trihydrateDrug
Also called Ethyol
Patients receive amifostine intravenous (IV) over 5 minutes beginning 30 minutes prior to melphalan and thiotepa administration on days -5 to -1.
Cohorts of 3-6 patients receive escalating doses of amifostine until the maximum tolerated dose is determined.
busulfanDrug
Also called Busulfex
Patients receive oral busulfan every 6 hours on days -8 to -6.
filgrastimDrug
Also called granulocyte colony-stimulating factor, G-CSF
All patients receive filgrastim (G-CSF) IV for 1 week.
melphalanDrug
Also called Alkeran
melphalan intravenous (IV) over 30 minutes on days -5 and -4
thiotepaDrug
Also called Thioplex
thiotepa intravenous (IV) over 2 hours on days -3 and -2.
peripheral blood stem cell transplantation (PBSC)Procedure or surgery
Also called bone marrow transplant
PBSC are reinfused on day 0
| Study group | Role | What they receive |
|---|---|---|
| Solid/brain tumor patients (1-18 years) | Experimental | Patients with solid tumor or brain tumor in the 1-18 years old stratum. Receives: Drug: amifostine trihydrate; Drug: busulfan; Drug: filgrastim; Drug: melphalan; Drug: thiotepa; Procedure: peripheral blood stem cell transplantation (PBSC) |
| Solid/brain tumor patients (19-45 years) | Experimental | Patients with solid tumor or brain tumor in the 19-45 years old stratum. Receives: Drug: amifostine trihydrate; Drug: busulfan; Drug: filgrastim; Drug: melphalan; Drug: thiotepa; Procedure: peripheral blood stem cell transplantation (PBSC) |
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 May 2003 (estimated)
- Study startNov 1998
- Primary completionAug 2002
- Study completionAug 2003
Why it stopped: Withdrawn due to slow accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Brain and Central Nervous System TumorsStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe 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
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Investigators
- John P. Perentesis, MD, Study chair, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00003926
- Sponsor's protocol ID
- 1997LS053
- Other IDs
- UMN-MT-9713, UMN-9712M00074
- Status last verified
- Nov 2017
- First submitted
- 1 Nov 1999
- Last updated
- 29 Nov 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of the Chemoprotectant Amifostine With Autologous Stem Cell Transplantation for High Risk or Relapsed Pediatric Solid Tumors and Brain Tumors
- ClinicalTrials.gov ID
- NCT00003926
- Acronym / sponsor ID
- 1997LS053
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Chordoma, Kidney Cancer, Liver Cancer, Neuroblastoma
- Keywords
- soft tissue sarcoma, regional neuroblastoma, disseminated neuroblastoma, recurrent Wilms tumor, recurrent retinoblastoma, recurrent adult brain tumor, adult rhabdomyosarcoma, ovarian germ cell tumor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Withdrawn due to slow accrual
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- Nov 1998
- Primary completion
- Aug 2002 (actual)
- Study completion
- Aug 2003 (actual)
- First posted
- 2 May 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.
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. Chemoprotective drugs such as amifostine may protect normal cells from the side effects of high-dose chemotherapy.
PURPOSE: Phase I trial to study the effectiveness of amifostine in protecting from the side effects of peripheral stem cell transplantation in treating patients who have high-risk or relapsed solid tumors.
Detailed description
OBJECTIVES:
- Determine the dose-limiting toxicity of amifostine chemoprotection with peripheral blood stem cell transplantation plus chemotherapy in patients with high-risk or relapsed solid tumors or brain tumors.
- Determine response or time to disease progression in patients treated with this regimen.
OUTLINE: This is a dose-escalation study of amifostine. Patients are stratified according to age (1 to 18 vs 19 to 45 years).
All patients receive filgrastim (G-CSF) IV for 1 week. On day 6 of G-CSF administration, patients undergo peripheral blood stem cell (PBSC) harvest followed by chemotherapy.
Patients receive oral busulfan every 6 hours on days -8 to -6 followed by melphalan IV over 30 minutes on days -5 and -4 and thiotepa IV over 2 hours on days -3 and -2. Patients receive amifostine IV over 5 minutes beginning 30 minutes prior to melphalan and thiotepa administration on days -5 to -1. PBSC are reinfused on day 0.
Cohorts of 3-6 patients receive escalating doses of amifostine until the maximum tolerated dose is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.
Patients are followed on day 50; at 3, 6, and 9 months; and at 1, 2, and 3 years post PBSC transplantation.
PROJECTED ACCRUAL: A maximum of 60 patients (30 per stratum) will be accrued for this study within 3 years.
Study design
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Solid/brain tumor patients (1-18 years) | Experimental | Drug: amifostine trihydrate, Drug: busulfan, Drug: filgrastim, Drug: melphalan, Drug: thiotepa, Procedure: peripheral blood stem cell transplantation (PBSC)Patients with solid tumor or brain tumor in the 1-18 years old stratum. |
| Solid/brain tumor patients (19-45 years) | Experimental | Drug: amifostine trihydrate, Drug: busulfan, Drug: filgrastim, Drug: melphalan, Drug: thiotepa, Procedure: peripheral blood stem cell transplantation (PBSC)Patients with solid tumor or brain tumor in the 19-45 years old stratum. |
Who may qualify
- Ages
- 1 year to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (31 listed)
- Histologically confirmed high-risk or relapsed solid tumors or brain tumors, including:
- Patients receive peripheral blood stem cell transplantation only if in complete remission or in very good partial remission with no disease progression
- Must have radiologic, nuclear image, or histologic verification of relapse
- Age 1 to 45
- Performance status:Karnofsky 70-100%
Key exclusion criteria (12 listed)
- Osteogenic sarcoma
- Less than 4 months
- Uncontrolled bleeding
- Congestive heart failure
- Uncontrolled hypertension
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 |
|---|---|---|
| amifostine trihydrateAlso: EthyolPatients receive amifostine intravenous (IV) over 5 minutes beginning 30 minutes prior to melphalan and thiotepa administration on days -5 to -1. Cohorts of 3-6 patients receive escalating doses of amifostine until the maximum tolerated dose is determined. | Drug | Solid/brain tumor patients (1-18 years), Solid/brain tumor patients (19-45 years) |
| busulfanAlso: BusulfexPatients receive oral busulfan every 6 hours on days -8 to -6. | Drug | Solid/brain tumor patients (1-18 years), Solid/brain tumor patients (19-45 years) |
| filgrastimAlso: granulocyte colony-stimulating factor, G-CSFAll patients receive filgrastim (G-CSF) IV for 1 week. | Drug | Solid/brain tumor patients (1-18 years), Solid/brain tumor patients (19-45 years) |
| melphalanAlso: Alkeranmelphalan intravenous (IV) over 30 minutes on days -5 and -4 | Drug | Solid/brain tumor patients (1-18 years), Solid/brain tumor patients (19-45 years) |
| thiotepaAlso: Thioplexthiotepa intravenous (IV) over 2 hours on days -3 and -2. | Drug | Solid/brain tumor patients (1-18 years), Solid/brain tumor patients (19-45 years) |
| peripheral blood stem cell transplantation (PBSC)Also: bone marrow transplantPBSC are reinfused on day 0 | Procedure or surgery | Solid/brain tumor patients (1-18 years), Solid/brain tumor patients (19-45 years) |
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early (reason given: Withdrawn due to slow accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Nov 2017.
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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.
John P. Perentesis, MD study chair
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing amifostine trihydrate, busulfan and filgrastim in people with Brain and Central Nervous System Tumors and Childhood Germ Cell Tumor, run by Masonic Cancer Center, University of Minnesota 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 31 inclusion and 12 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: stopped early, as of the sponsor's last update on 29 Nov 2017. 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 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 was stopped early (reason given: Withdrawn due to slow accrual). 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 29 Nov 2017.