NeuroblastomaPhase 1NCT00002730
Chemotherapy in Treating Children With Neuroblastoma
A Phase 1 study testing buthionine sulfoximine and melphalan in people with Neuroblastoma, run by New Approaches to Neuroblastoma Therapy Consortium at 4 sites 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. Some tumors become resistant to chemotherapy drugs. Combining buthionine sulfoximine with chemotherapy may reduce resistance to the drug and allow more tumor cells to be killed. Read more about this study
- Who is it for?
- Ages 0 to 21 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- buthionine sulfoximine and melphalan
- How many people
- Not given
- Where
- 4 sitesUnited States
- Study dates
- Jun 1996to not given
- 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 0 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS: Neuroblastoma histologically confirmed at initial diagnosis or demonstration of malignant, small, round cell tumor with elevated catecholamine metabolites Refractory to conventional therapy and other higher priority therapy
PATIENT CHARACTERISTICS: Age: No greater than 21 at diagnosis Performance status: 0-2 Life expectancy: At least 2 months Hematopoietic: Cytopenias from marrow involvement eligible with study chairman approval ANC at least 1,000 Platelets at least 100,000 (transfusion independent) Counts between 70,000-100,000 allowed provided: Autologous bone marrow or peripheral stem cells available for rescue Study chairman approves entry Hemoglobin at least 8 g/dL (may transfuse) Hepatic: Bilirubin no greater than 1.5 times normal AST/ALT less than 2.5 times normal Renal: Creatinine no greater than 1.5 times normal OR Creatinine clearance or radioisotope GFR at least 70 mL/min per 1.73 square meters Pulmonary: No history of dyspnea at rest No exercise intolerance Other: No active infection requiring hospitalization No pregnant or nursing women Negative pregnancy test required of fertile women Effective contraception required of fertile patients during and for 2 months after study Patients unable to receive blood products due to religious reasons may receive buthionine sulfoximine alone
PRIOR CONCURRENT THERAPY: At least 6 months since myeloablative therapy with bone marrow transplantation Recovered from toxic effects of prior therapy Biologic therapy: Not specified Chemotherapy: At least 3 weeks since chemotherapy (6 weeks since mitomycin or nitrosourea) Endocrine therapy: Not specified Radiotherapy: At least 6 weeks since radiotherapy to any extremity site or significant marrow-containing compartment At least 6 months since the following: More than 24 Gy craniospinal irradiation Total abdominopelvic plus lung irradiation Mantle plus Y-port irradiation Total-body irradiation No palliative radiotherapy to bony lesions within 1 month after entry Surgery: Not specified
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Children's Hospital Los Angeles
Los Angeles, California, 90027-0700
University of Minnesota Cancer Center
Minneapolis, Minnesota, 55455
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
UCSF Cancer Center and Cancer Research Institute
San Francisco, California, 94115-0128
About this study
Official title: PILOT STUDY OF BUTHIONINE SULFOXIMINE (BSO) IN COMBINATION WITH MELPHALAN FOR HIGH RISK NEUROBLASTOMA PATIENTS
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Some tumors become resistant to chemotherapy drugs. Combining buthionine sulfoximine with chemotherapy may reduce resistance to the drug and allow more tumor cells to be killed.
PURPOSE: Phase I trial to study the effectiveness of melphalan, buthionine sulfoximine, and G-CSF in treating children with progressive neuroblastoma that has not responded to previous therapy.
Read the detailed description
OBJECTIVES: I. Describe the toxic effects of combined chemotherapy with buthionine sulfoximine (BSO) and melphalan (L-PAM) in pediatric patients with progressive neuroblastoma. II. Determine the pharmacokinetics of BSO/L-PAM in pediatric patients. III. Assess the ability of BSO to deplete glutathione by at least 90% in tumor metastatic to bone marrow, in normal marrow, and in peripheral blood lymphocytes. IV. Estimate the response rate in these patients treated with BSO/L-PAM within the confines of a pilot study.
OUTLINE: The following acronyms are used: BSO Buthionine sulfoximine, NSC-326231 L-PAM Melphalan, NSC-8806 G-CSF Granulocyte Colony-Stimulating Factor, NSC-614629 Single-Agent Chemotherapy with Drug Resistance Inhibition. L-PAM/BSO.
PROJECTED ACCRUAL: At least 18 patients will be entered to provide an adequate number of patients with marrow involvement; if the BSO dose is increased to achieve adequate GSH depletion in the marrow, an additional 12 patients will be entered. If less than 50% of patients have tumor metastatic to marrow at entry, there will be a proportional increase in the total number of patients required.
- Conditions
- Neuroblastoma
- Being tested
- buthionine sulfoximine, melphalan
- Keywords
- recurrent neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
buthionine sulfoximineDrug
melphalanDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2004 (estimated)
- Study startJun 1996
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
Sponsor and investigators
- Lead sponsor
- New Approaches to Neuroblastoma Therapy Consortium
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- C. Patrick Reynolds, MD, PhD, Study chair, Children's Hospital Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00002730
- Sponsor's protocol ID
- CHLA-95-083
- Other IDs
- CDR0000064620, NCI-T95-00920
- Status last verified
- Nov 2024
- First submitted
- 1 Nov 1999
- Last updated
- 20 Nov 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PILOT STUDY OF BUTHIONINE SULFOXIMINE (BSO) IN COMBINATION WITH MELPHALAN FOR HIGH RISK NEUROBLASTOMA PATIENTS
- ClinicalTrials.gov ID
- NCT00002730
- Acronym / sponsor ID
- CHLA-95-083
- Conditions
- Neuroblastoma
- Keywords
- recurrent neuroblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- New Approaches to Neuroblastoma Therapy ConsortiumUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jun 1996
- First posted
- 1 Sep 2004 (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. Some tumors become resistant to chemotherapy drugs. Combining buthionine sulfoximine with chemotherapy may reduce resistance to the drug and allow more tumor cells to be killed.
PURPOSE: Phase I trial to study the effectiveness of melphalan, buthionine sulfoximine, and G-CSF in treating children with progressive neuroblastoma that has not responded to previous therapy.
Detailed description
OBJECTIVES: I. Describe the toxic effects of combined chemotherapy with buthionine sulfoximine (BSO) and melphalan (L-PAM) in pediatric patients with progressive neuroblastoma. II. Determine the pharmacokinetics of BSO/L-PAM in pediatric patients. III. Assess the ability of BSO to deplete glutathione by at least 90% in tumor metastatic to bone marrow, in normal marrow, and in peripheral blood lymphocytes. IV. Estimate the response rate in these patients treated with BSO/L-PAM within the confines of a pilot study.
OUTLINE: The following acronyms are used: BSO Buthionine sulfoximine, NSC-326231 L-PAM Melphalan, NSC-8806 G-CSF Granulocyte Colony-Stimulating Factor, NSC-614629 Single-Agent Chemotherapy with Drug Resistance Inhibition. L-PAM/BSO.
PROJECTED ACCRUAL: At least 18 patients will be entered to provide an adequate number of patients with marrow involvement; if the BSO dose is increased to achieve adequate GSH depletion in the marrow, an additional 12 patients will be entered. If less than 50% of patients have tumor metastatic to marrow at entry, there will be a proportional increase in the total number of patients required.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 0 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 |
|---|---|---|
| buthionine sulfoximine | Drug | – |
| melphalan | Drug | – |
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 20 Nov 2024.
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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.
C. Patrick Reynolds, MD, PhD study chair
Children's Hospital Los Angeles
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing buthionine sulfoximine and melphalan in people with Neuroblastoma, run by New Approaches to Neuroblastoma Therapy Consortium at 4 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?
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 20 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 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.
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?
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 20 Nov 2024.