NeuroblastomaPhase 1NCT00002748
Gene Therapy in Treating Children With Refractory or Recurrent Neuroblastoma
A Phase 1 study testing gene-modified tumor cell vaccine therapy and interleukin-2 gene in people with Neuroblastoma, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
RATIONALE: Inserting the gene for interleukin-2 into a person's neuroblastoma cells may make the body build an immune response and kill tumor cells. PURPOSE: Phase I trial to study the effectiveness of using interleukin-2 gene-modified neuroblastoma cells in treating children who have refractory or recurrent… Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- gene-modified tumor cell vaccine therapy and interleukin-2 gene
- Type of treatment
- Vaccines
- How many people
- 38 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 1991to Aug 2007
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS: Histologically proven high risk neuroblastoma at the completion of planned primary therapy No rapidly progressing disease Allogeneic transduced cell line available Demonstrated production of at least 150 picograms of interleukin-2 per 10 to the 6th cells per day
PATIENT CHARACTERISTICS: Age: Under 21 at diagnosis Performance status: ECOG 0-2 Life expectancy: At least 8 weeks Hematopoietic: (unless marrow replaced by tumor) Absolute neutrophil count greater than 500/mm3 Platelet count greater than 50,000/mm3 Hepatic: Bilirubin less than 1.5 mg/dL AST no greater than 2 times normal PT normal Renal: Creatinine less than 1.5 mg/dL OR Creatinine clearance greater than 80 mL/min Urinalysis normal Metabolic: Electrolytes (including calcium, phosphate) normal Glucose normal Weight greater than 10th percentile for age Albumin greater than 3 g/dL Other: No active infection HIV negative Not pregnant or nursing
PRIOR CONCURRENT THERAPY: See Disease Characteristics Recovered from prior chemotherapy No concurrent antibiotics except prophylactic trimethoprim/sulfamethoxazole No concurrent drugs other than analgesics
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105-2794
About this study
Official title: Phase I Study of Cytokine-Gene Modified Autologous or Partially Matched Allogeneic Neuroblastoma Cells for Treatment of Relapsed/Refractory Neuroblastoma
RATIONALE: Inserting the gene for interleukin-2 into a person's neuroblastoma cells may make the body build an immune response and kill tumor cells.
PURPOSE: Phase I trial to study the effectiveness of using interleukin-2 gene-modified neuroblastoma cells in treating children who have refractory or recurrent neuroblastoma.
Read the detailed description
OBJECTIVES: I. Determine the safety in children of recurrent neuroblastoma of two weekly subcutaneous injections of autologous, or partially HLA-matched allogeneic, neuroblastoma cells that have been modified by insertion of the interleukin-2 gene introduced by a retroviral vector. II. Determine whether multiple histocompatibility-restricted or unrestricted antitumor immune responses are induced by this treatment and the cell dose required to produce these effects. III. Obtain preliminary data on the antitumor effects of this regimen.
OUTLINE: Autologous or partially HLA-matched allogeneic neuroblastoma cells are transduced with a human gene for interleukin-2 production. Patients receive subcutaneous injections of the gene-modified cells on days 1 and 8, with the second injection containing 10 times more cells than the first injection. After a 3-4 week rest, stable and responding patients may receive additional weekly injections at the second dose. Cohorts of 3-6 patients will be entered at increasing cell doses until the maximum tolerated dose is estimated. Multiple injection sites may be used at the higher cell-dose levels. Patients are followed every week for 6 weeks, every other week for 6 weeks, and monthly for 1 year. Additional visits may be required as clinically indicated.
PROJECTED ACCRUAL: Approximately 12 patients each will be entered into the autologous and the partially HLA-matched allogeneic tumor cell treatment groups. Accrual is expected to require 4 years for the autologous tumor cell group and 2 years for the partially HLA-matched allogenic tumor cell group.
- Conditions
- Neuroblastoma
- Being tested
- gene-modified tumor cell vaccine therapy, interleukin-2 gene
- 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.
gene-modified tumor cell vaccine therapyBiological
interleukin-2 geneBiological
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2004 (estimated)
- Study startDec 1991
- Primary completionAug 2007
- Study completionAug 2007
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.
- More trials run by St. Jude Children's Research HospitalThe 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
- Treatment
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- Gregory Hale, MD, Study chair, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00002748
- Sponsor's protocol ID
- CDR0000064681
- Other IDs
- U01CA058211, P30CA021765, SJCRH-CYGENE, SJCRH-CYGNE2, NCI-H96-0005
- Status last verified
- Oct 2011
- First submitted
- 1 Nov 1999
- Last updated
- 4 Oct 2011
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Oct 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Cytokine-Gene Modified Autologous or Partially Matched Allogeneic Neuroblastoma Cells for Treatment of Relapsed/Refractory Neuroblastoma
- ClinicalTrials.gov ID
- NCT00002748
- Acronym / sponsor ID
- CDR0000064681
- Conditions
- Neuroblastoma
- Keywords
- recurrent neuroblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Dec 1991
- Primary completion
- Aug 2007 (actual)
- Study completion
- Aug 2007 (actual)
- First posted
- 22 Jan 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: Inserting the gene for interleukin-2 into a person's neuroblastoma cells may make the body build an immune response and kill tumor cells.
PURPOSE: Phase I trial to study the effectiveness of using interleukin-2 gene-modified neuroblastoma cells in treating children who have refractory or recurrent neuroblastoma.
Detailed description
OBJECTIVES: I. Determine the safety in children of recurrent neuroblastoma of two weekly subcutaneous injections of autologous, or partially HLA-matched allogeneic, neuroblastoma cells that have been modified by insertion of the interleukin-2 gene introduced by a retroviral vector. II. Determine whether multiple histocompatibility-restricted or unrestricted antitumor immune responses are induced by this treatment and the cell dose required to produce these effects. III. Obtain preliminary data on the antitumor effects of this regimen.
OUTLINE: Autologous or partially HLA-matched allogeneic neuroblastoma cells are transduced with a human gene for interleukin-2 production. Patients receive subcutaneous injections of the gene-modified cells on days 1 and 8, with the second injection containing 10 times more cells than the first injection. After a 3-4 week rest, stable and responding patients may receive additional weekly injections at the second dose. Cohorts of 3-6 patients will be entered at increasing cell doses until the maximum tolerated dose is estimated. Multiple injection sites may be used at the higher cell-dose levels. Patients are followed every week for 6 weeks, every other week for 6 weeks, and monthly for 1 year. Additional visits may be required as clinically indicated.
PROJECTED ACCRUAL: Approximately 12 patients each will be entered into the autologous and the partially HLA-matched allogeneic tumor cell treatment groups. Accrual is expected to require 4 years for the autologous tumor cell group and 2 years for the partially HLA-matched allogenic tumor cell group.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- Up 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 |
|---|---|---|
| gene-modified tumor cell vaccine therapy | Biological | – |
| interleukin-2 gene | Biological | – |
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 4 Oct 2011.
- St. Jude Children's Research HospitalLink provided by the sponsor.
Related trials for 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 recruitingObservational study
Genomic Structural Variation in Cancer Susceptibility
Breast Cancer, Colon Cancer
RecruitingPhase 2
Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors
Metastatic Chondrosarcoma, Locally Advanced Chondrosarcoma
RecruitingObservational study
Prospective Comprehensive Molecular Analysis of Endocrine Neoplasms
Endocrine Tumors, Thyroid Neoplasms
RecruitingPhase 1
PEACH TRIAL- Precision Medicine and Adoptive Cellular Therapy
Neuroblastoma, Diffuse Intrinsic Pontine Glioma
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.
Gregory Hale, MD study chair
St. Jude Children's Research Hospital
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 gene-modified tumor cell vaccine therapy and interleukin-2 gene in people with Neuroblastoma, run by St. Jude Children's Research Hospital 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?
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 4 Oct 2011. 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 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 4 Oct 2011.