NeuroblastomaPhase 2NCT00793845
Tandem High-dose Chemotherapy and Autologous Stem Cell Rescue in Patients With High-risk Neuroblastoma
A Phase 2 study testing Cyclophosphamide, Etoposide and Carboplatin in people with Neuroblastoma, run by Samsung Medical Center at 1 site in South Korea.
Trial summary
The purpose of this study is to evaluate the efficacy and toxicity of tandem HDCT/ASCR in children with high-risk neuroblastoma. In the present study, a single arm trial of tandem HDCT/ASCR will be carried out. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Cyclophosphamide, Etoposide and Carboplatin
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteSouth Korea
- Study dates
- Aug 2008to Jan 2018
- Phase
- Phase 2
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with high-risk neuroblastoma
- Patients with intermediate-risk neuroblastoma if gross tumor remained after surgery
Who cannot join
- Patients with progressive disease before high-dose chemotherapy
- Patients whose parents want to stop or change the planned treatment
- Patients with organ toxicities of NCI grade >2 before high-dose chemotherapy
Trial locations and contacts
1 site in 1 country.
About this study
The purpose of this study is to evaluate the efficacy and toxicity of tandem HDCT/ASCR in children with high-risk neuroblastoma. In the present study, a single arm trial of tandem HDCT/ASCR will be carried out. In the present study, the investigators will investigate whether tandem HDCT/ASCR might improve the survival of patients with high-risk neuroblastoma with acceptable toxicity.
Read the detailed description
The prognosis of high-risk neuroblastoma after conventional chemoradiotherapy is generally poor. Therefore, a strategy using high-dose chemotherapy and autologous stem cell rescue (HDCT/ASCR) has been explored to improve the prognosis of patients with high-risk neuroblastoma. This strategy is based on the hypothesis that dose escalation might improve the survival of children with high-risk neuroblastoma. The results of randomized trials comparing HDCT/ASCR with chemotherapy alone showed a better event-free survival (EFS) in the HDCT/ASCR arm than in the continuous chemotherapy arm. However, the overall EFS was unsatisfactory.
In this context, investigators have examined the efficacy of double or triple tandem HDCT/ASCR to further improve the outcome of high-risk neuroblastoma patients. George et al. carried out a single arm trial of tandem transplantation as consolidation therapy, and reported improved long-term survival (5-year progression-free survival 47%) with acceptable toxicity. Kletzel et al. also conducted a single arm trial of triple tandem transplantation and reported improved survival (3-year EFS 57%). They demonstrated that further dose escalation using sequential HDCT/ASCR might result in further improvements in the survival of patients with high-risk neuroblastoma.
Investigators in the present study also carried out tandem transplantation as consolidation therapy, and reported improved long-term survival (5-year progression-free survival 62%) with acceptable toxicity. However, throughout our previous study, multiple modifications were made in the treatment plan, which resulted in significant variability over time between patients. This variability may create doubt as to whether tandem HDCT/ASCR itself resulted in the improved outcome. In addition, toxic death rate was relatively high (15.4%), although final survival rate was very high (best survival rate ever reported). Therefore, prospective study is needed to evaluate the efficacy and toxicity of tandem HDCT/ASCR.
- Conditions
- Neuroblastoma
- Being tested
- Cyclophosphamide, Etoposide, Carboplatin, Thiotepa, Melphalan
- Keywords
- tandem high-dose chemotherapy for high-risk neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
First HDCT
EtoposideDrug
First HDCT
CarboplatinDrug
First HDCT
ThiotepaDrug
Second HDCT
MelphalanDrug
Second HDCT
Total body irradiationRadiation
Second HDCT
| Study group | Role | What they receive |
|---|---|---|
| High risk neuroblastoma | Experimental | 1. Conventional chemotherapy (9 cycles)
2. Surgery conventional chemotherapy (after 6 cycles of chemotherapy)
3. Tandem HDCT/autoSCT
* First HDCT (cyclophosphamide, etoposide, carboplatin)
* Second HDCT (total body irradiation, thiotepa, melphalan)
4. Local radiotherapy
5. Retinoic acid, interleukin-2 Receives: Drug: Cyclophosphamide; Drug: Etoposide; Drug: Carboplatin; Drug: Thiotepa; Drug: Melphalan; Radiation: Total body irradiation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival and event-free survival, short-term and long-term toxicity of tandem high-dose chemotherapy and autologous stem cell transplantation
Measured: from 1 year after second high-dose chemotherapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2008 (estimated)
- Study startAug 2008
- Primary completionJan 2015
- Study completionJan 2018
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 Samsung Medical 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Samsung Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ki Woong Sung, MD, Principal investigator, Samsung Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00793845
- Sponsor's protocol ID
- 2008-07-002
- Status last verified
- Sep 2018
- First submitted
- 17 Nov 2008
- Last updated
- 18 Sep 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tandem High-dose Chemotherapy and Autologous Stem Cell Rescue in Patients With High-risk Neuroblastoma
- ClinicalTrials.gov ID
- NCT00793845
- Acronym / sponsor ID
- 2008-07-002
- Conditions
- Neuroblastoma
- Keywords
- tandem high-dose chemotherapy for high-risk neuroblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Samsung Medical CenterUniversity, hospital or other
- Study start
- Aug 2008
- Primary completion
- Jan 2015 (actual)
- Study completion
- Jan 2018 (actual)
- First posted
- 19 Nov 2008 (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.
The purpose of this study is to evaluate the efficacy and toxicity of tandem HDCT/ASCR in children with high-risk neuroblastoma. In the present study, a single arm trial of tandem HDCT/ASCR will be carried out. In the present study, the investigators will investigate whether tandem HDCT/ASCR might improve the survival of patients with high-risk neuroblastoma with acceptable toxicity.
Detailed description
The prognosis of high-risk neuroblastoma after conventional chemoradiotherapy is generally poor. Therefore, a strategy using high-dose chemotherapy and autologous stem cell rescue (HDCT/ASCR) has been explored to improve the prognosis of patients with high-risk neuroblastoma. This strategy is based on the hypothesis that dose escalation might improve the survival of children with high-risk neuroblastoma. The results of randomized trials comparing HDCT/ASCR with chemotherapy alone showed a better event-free survival (EFS) in the HDCT/ASCR arm than in the continuous chemotherapy arm. However, the overall EFS was unsatisfactory.
In this context, investigators have examined the efficacy of double or triple tandem HDCT/ASCR to further improve the outcome of high-risk neuroblastoma patients. George et al. carried out a single arm trial of tandem transplantation as consolidation therapy, and reported improved long-term survival (5-year progression-free survival 47%) with acceptable toxicity. Kletzel et al. also conducted a single arm trial of triple tandem transplantation and reported improved survival (3-year EFS 57%). They demonstrated that further dose escalation using sequential HDCT/ASCR might result in further improvements in the survival of patients with high-risk neuroblastoma.
Investigators in the present study also carried out tandem transplantation as consolidation therapy, and reported improved long-term survival (5-year progression-free survival 62%) with acceptable toxicity. However, throughout our previous study, multiple modifications were made in the treatment plan, which resulted in significant variability over time between patients. This variability may create doubt as to whether tandem HDCT/ASCR itself resulted in the improved outcome. In addition, toxic death rate was relatively high (15.4%), although final survival rate was very high (best survival rate ever reported). Therefore, prospective study is needed to evaluate the efficacy and toxicity of tandem HDCT/ASCR.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| High risk neuroblastoma | Experimental | Drug: Cyclophosphamide, Drug: Etoposide, Drug: Carboplatin, Drug: Thiotepa, Drug: Melphalan, Radiation: Total body irradiation1. Conventional chemotherapy (9 cycles) 2. Surgery conventional chemotherapy (after 6 cycles of chemotherapy) 3. Tandem HDCT/autoSCT * First HDCT (cyclophosphamide, etoposide, carboplatin) * Second HDCT (total body irradiation, thiotepa, melphalan) 4. Local radiotherapy 5. Retinoic acid, interleukin-2 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Patients with high-risk neuroblastoma
- Patients with intermediate-risk neuroblastoma if gross tumor remained after surgery
Key exclusion criteria (3 listed)
- Patients with progressive disease before high-dose chemotherapy
- Patients whose parents want to stop or change the planned treatment
- Patients with organ toxicities of NCI grade >2 before high-dose chemotherapy
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 |
|---|---|---|
| CyclophosphamideFirst HDCT | Drug | High risk neuroblastoma |
| EtoposideFirst HDCT | Drug | High risk neuroblastoma |
| CarboplatinFirst HDCT | Drug | High risk neuroblastoma |
| ThiotepaSecond HDCT | Drug | High risk neuroblastoma |
| MelphalanSecond HDCT | Drug | High risk neuroblastoma |
| Total body irradiationSecond HDCT | Radiation | High risk neuroblastoma |
Primary outcome
Overall survival and event-free survival, short-term and long-term toxicity of tandem high-dose chemotherapy and autologous stem cell transplantation
Time frame: from 1 year after second high-dose chemotherapy
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 18 Sep 2018.
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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.
Ki Woong Sung, MD principal investigator
Samsung Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cyclophosphamide, Etoposide and Carboplatin in people with Neuroblastoma, run by Samsung Medical Center at 1 site in South Korea.
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 2 inclusion and 3 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: finished, as of the sponsor's last update on 18 Sep 2018. 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 South Korea. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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.
What does the primary outcome measure?
Overall survival and event-free survival, short-term and long-term toxicity of tandem high-dose chemotherapy and autologous stem cell transplantation (time frame: from 1 year after second high-dose chemotherapy). It is what the study was designed to measure, not a finding.
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 18 Sep 2018.