NeuroblastomaPhase 1NCT00006480
Biological Therapy and Gene Therapy in Treating Children With Recurrent or Refractory Neuroblastoma
A Phase 1 study testing aldesleukin, therapeutic autologous lymphocytes and chemotherapy in people with Neuroblastoma, run by Fred Hutchinson Cancer Center at 2 sites in United States.
Trial summary
RATIONALE: Biological therapies use different ways to stimulate the immune system and stop cancer cell from growing. Inserting genetic material made in the laboratory into a person's blood cells may make the body build an immune response to kill tumor cells. Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- aldesleukin, therapeutic autologous lymphocytes and chemotherapy
- How many people
- Not given
- Where
- 2 sitesUnited States
- Study dates
- May 2000to Mar 2005
- 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 17 years
- All sexes
- No healthy volunteers
- Child
DISEASE CHARACTERISTICS: Histologically and/or radiographically proven disseminated neuroblastoma Recurrent or refractory to first-line therapy as defined by less than complete response to standard induction chemotherapy combined with surgical resection Histologic verification of neuroblastoma required at original diagnosis No radiographically detectable CNS involvement No clinically evident progressive encephalopathy
PATIENT CHARACTERISTICS: Age: 1 to 17 (children only) Performance status: Not specified Life expectancy: At least 3 months Hematopoietic: Not specified Hepatic: Not specified Renal: No dialysis dependency Cardiovascular: No uncontrolled cardiac arrhythmia No hypertension requiring pressor support Pulmonary: No requirement for supplemental oxygen unless expected to resolve within 2 weeks Neurologic: See Disease Characteristics No refractory seizure disorder Other: No detectable human antimouse antibody reactivity if received prior murine antibody preparations No history of ganciclovir allergy or intolerance HIV negative Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception during and for at least 2 months after study
PRIOR CONCURRENT THERAPY: Biologic therapy: No other concurrent antibody therapy during or after study No other concurrent immunotherapy (e.g., interferons, vaccines, or other cellular products) Chemotherapy: At least 3 weeks since prior standard or experimental chemotherapy and recovered Endocrine therapy: No concurrent systemic corticosteroids unless specifically for amelioration of toxicity induced by transferred T-cell therapy Radiotherapy: Not specified Surgery: Not specified Other: At least 3 weeks since prior immunosuppressive therapies and recovered No concurrent pentoxifylline No other concurrent investigational agents No concurrent ganciclovir, any ganciclovir derivatives, or acyclovir for non-life-threatening herpes virus infections
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Cancer Center and Beckman Research Institute, City of Hope
Duarte, California, 91010-3000
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109-1024
About this study
Official title: Phase I Study to Evaluate the Safety of Cellular Immunotherapy for Recurrent/Refractory Neuroblastoma Using Genetically-Modified Autologous CD8+ T Cell Clones
RATIONALE: Biological therapies use different ways to stimulate the immune system and stop cancer cell from growing. Inserting genetic material made in the laboratory into a person's blood cells may make the body build an immune response to kill tumor cells.
PURPOSE: Phase I trial to study the effectiveness of biological therapy and gene therapy in treating children who have recurrent or refractory neuroblastoma.
Read the detailed description
OBJECTIVES: I. Determine the safety and toxicity of cellular immunotherapy using ex vivo expanded autologous CD8+ cytotoxic T-lymphocyte clones genetically modified to express the CE7R scFvFc:zeta chimeric immunoreceptor and the HyTK selection/suicide gene in children with recurrent or refractory disseminated neuroblastoma. II. Determine the antitumor activity of this regimen in these patients. III. Determine the duration of in vivo persistence of adoptively transferred clones and the effect of interleukin-2 on maintaining the in vivo persistence of these clones. IV. Screen for the development of host anti-scFvFc:zeta and HyTK immune responses in patients treated with this regimen. V. Determine the efficacy of ganciclovir in ablating transferred clones in vivo if toxicity occurs in these patients.
OUTLINE: This is a multicenter study. Patients undergo autologous peripheral blood stem cell harvest. CD8+ cytotoxic T-lymphocyte (CTL) clones are isolated, genetically modified to express the CE7R scFvFc:zeta chimeric immunoreceptor and the HyTK selection/suicide gene, and then expanded ex vivo. While the modified CTL clones are being generated, patients each receive an individualized salvage chemotherapy regimen that may consist of one of the following: cyclophosphamide and topotecan; ifosfamide, carboplatin, and etoposide; or another chemotherapy regimen chosen by the patient's primary oncologist. The first cohort of 5 patients receives escalating doses of modified CTL clones IV over 30 minutes on days 0, 14, and 28 in the absence of disease progression or unacceptable toxicity. Each patient begins the series of 3 infusions as soon as an adequate number of modified CTL clones are ready and after the acute side effects of chemotherapy have resolved. In the absence of unacceptable toxicity in the first cohort, the second cohort of 5 patients receives the same treatment as cohort 1 plus interleukin-2 subcutaneously every 12 hours on days 15-24 and 29-38. Patients with unacceptable toxicity receive ganciclovir IV every 12 hours for 14 days (or longer if symptomatic resolution is not achieved in that interval). Patients are followed at day 100 and then periodically thereafter.
PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study within 3 years.
- Conditions
- Neuroblastoma
- Being tested
- aldesleukin, therapeutic autologous lymphocytes, chemotherapy, ganciclovir
- Keywords
- disseminated neuroblastoma, 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.
aldesleukinBiological
therapeutic autologous lymphocytesBiological
chemotherapyDrug
ganciclovirDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2003 (estimated)
- Study startMay 2000
- Study completionMar 2005
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 Fred Hutchinson Cancer CenterThe 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
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- Julie R. Park, MD, Study chair, Fred Hutchinson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00006480
- Sponsor's protocol ID
- 1524.00
- Other IDs
- FHCRC-1524.00, NCI-H00-0065, CDR0000068310
- Status last verified
- Sep 2010
- First submitted
- 6 Nov 2000
- Last updated
- 21 Sep 2010
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study to Evaluate the Safety of Cellular Immunotherapy for Recurrent/Refractory Neuroblastoma Using Genetically-Modified Autologous CD8+ T Cell Clones
- ClinicalTrials.gov ID
- NCT00006480
- Acronym / sponsor ID
- 1524.00
- Conditions
- Neuroblastoma
- Keywords
- disseminated neuroblastoma, recurrent neuroblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- Fred Hutchinson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- May 2000
- Study completion
- Mar 2005 (actual)
- First posted
- 31 Dec 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: Biological therapies use different ways to stimulate the immune system and stop cancer cell from growing. Inserting genetic material made in the laboratory into a person's blood cells may make the body build an immune response to kill tumor cells.
PURPOSE: Phase I trial to study the effectiveness of biological therapy and gene therapy in treating children who have recurrent or refractory neuroblastoma.
Detailed description
OBJECTIVES: I. Determine the safety and toxicity of cellular immunotherapy using ex vivo expanded autologous CD8+ cytotoxic T-lymphocyte clones genetically modified to express the CE7R scFvFc:zeta chimeric immunoreceptor and the HyTK selection/suicide gene in children with recurrent or refractory disseminated neuroblastoma. II. Determine the antitumor activity of this regimen in these patients. III. Determine the duration of in vivo persistence of adoptively transferred clones and the effect of interleukin-2 on maintaining the in vivo persistence of these clones. IV. Screen for the development of host anti-scFvFc:zeta and HyTK immune responses in patients treated with this regimen. V. Determine the efficacy of ganciclovir in ablating transferred clones in vivo if toxicity occurs in these patients.
OUTLINE: This is a multicenter study. Patients undergo autologous peripheral blood stem cell harvest. CD8+ cytotoxic T-lymphocyte (CTL) clones are isolated, genetically modified to express the CE7R scFvFc:zeta chimeric immunoreceptor and the HyTK selection/suicide gene, and then expanded ex vivo. While the modified CTL clones are being generated, patients each receive an individualized salvage chemotherapy regimen that may consist of one of the following: cyclophosphamide and topotecan; ifosfamide, carboplatin, and etoposide; or another chemotherapy regimen chosen by the patient's primary oncologist. The first cohort of 5 patients receives escalating doses of modified CTL clones IV over 30 minutes on days 0, 14, and 28 in the absence of disease progression or unacceptable toxicity. Each patient begins the series of 3 infusions as soon as an adequate number of modified CTL clones are ready and after the acute side effects of chemotherapy have resolved. In the absence of unacceptable toxicity in the first cohort, the second cohort of 5 patients receives the same treatment as cohort 1 plus interleukin-2 subcutaneously every 12 hours on days 15-24 and 29-38. Patients with unacceptable toxicity receive ganciclovir IV every 12 hours for 14 days (or longer if symptomatic resolution is not achieved in that interval). Patients are followed at day 100 and then periodically thereafter.
PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study within 3 years.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
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 |
|---|---|---|
| aldesleukin | Biological | – |
| therapeutic autologous lymphocytes | Biological | – |
| chemotherapy | Drug | – |
| ganciclovir | 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 21 Sep 2010.
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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.
Julie R. Park, MD study chair
Fred Hutchinson Cancer Center
2 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 aldesleukin, therapeutic autologous lymphocytes and chemotherapy in people with Neuroblastoma, run by Fred Hutchinson Cancer Center at 2 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 21 Sep 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 21 Sep 2010.