NeuroblastomaPhase 2NCT00808899
Neuroblastoma Protocol 2008: Therapy for Children With Advanced Stage High Risk Neuroblastoma
A Phase 2 study testing Temsirolimus, Irinotecan and Surgical Resection of Primary Tumor in people with Neuroblastoma, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
A Phase II study of temsirolimus in combination with standard chemotherapy (irinotecan; cyclophosphamide, doxorubicin and etoposide (CAE); cisplatin and etoposide (HiPE) and topotecan (TPT) followed by and additional six courses of induction chemotherapy and then intensification with autologous hematopoietic stem cell… Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Temsirolimus, Irinotecan and Surgical Resection of Primary Tumor
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2008to Jul 2009
- Phase
- Phase 2
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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients <18 years old with newly diagnosed, advanced stage, high-risk neuroblastoma defined as one of the following:
- Children < 1 yo with International Neuroblastoma Staging System (INSS) stage 2a, 2b, 3, 4 or 4S disease and MYCN amplification (>10 copies, or greater than four-fold increase in MYCN signal as compared to reference signal)
- INSS 2a or 2b disease and MYCN amplification, regardless of age or additional biologic features
- INSS stage 3 and:
- MYCN amplification (>10 copies, or greater than four-fold increase in MYCN signal as compared to reference signal, regardless of age or additional biologic features
- Age > 18 mo (> 547 days) with unfavorable pathology, regardless of MYCN status
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- INSS stage 4 and:
- MYCN amplification, regardless of age or additional biologic features
- Age > 18 months (> 547 days) regardless of biologic features
- Age 12 - 18 months (365 - 547 days) with any of the following three unfavorable biologic features (MYCN amplification, unfavorable pathology and/or DNA index =1) or any biologic feature that is indeterminant/unknown
- Children less than or equal to 365 days initially diagnosed with: INSS stage 1, 2, 4S who progressed to a stage 4 without interval chemotherapy.
- Histologic proof of neuroblastoma or positive bone marrow for tumor cells with increased urine catecholamines.
- Adequate renal and hepatic function (serum creatinine <3 x upper limit of normal for age, (AST) aspartate aminotransferase < 3 x upper limit of normal).
- No prior therapy, unless an emergency situation requires local tumor treatment (discuss with PI)
- Written, informed consent according to institutional guidelines
Who cannot join
- Any evidence, as judged by the investigator, of severe or uncontrolled systemic disease (e.g., unstable or uncompensated respiratory, cardiac, hepatic, or renal disease).
- Pregnant or breast feeding (women of child-bearing potential).
- Children with INSS 4 disease, age <12 months with all 3 favorable biologic features (non-amplified MYCN, favorable pathology and DNA index >1).
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
A Phase II study of temsirolimus in combination with standard chemotherapy (irinotecan; cyclophosphamide, doxorubicin and etoposide (CAE); cisplatin and etoposide (HiPE) and topotecan (TPT) followed by and additional six courses of induction chemotherapy and then intensification with autologous hematopoietic stem cell transplantation. The first five courses of induction chemotherapy will also evaluate the feasibility of combining weekly temsirolimus with these standard chemotherapy combinations. This will be followed by 16 months of oral maintenance therapy with eight months of 13-cis-retinoic acid and then eight months of oral topotecan.
Read the detailed description
All children will receive fixed doses of intravenous temsirolimus (50 mg/m2 weekly 6 times ) concomitantly with two courses of fixed dosages of irinotecan (20 mg/m2 intravenously daily 5 times ,2 days off, repeated daily 5 times .If these initial dosages are not tolerable then subsequent patients will be given a reduced dosage of temsirolimus (25 mg/m2 weekly 6 times) with 20 mg/m2 of irinotecan.If this dosage combination is not tolerable, the irinotecan dosage will be decreased to 15 mg/m2 .If this dosage combination is not tolerable then further enrollment to the initial six week treatment will be terminated.The second course of irinotecan will begin on day 22 and response will be determined after six weeks (two courses). Resection of primary tumor will be attempted after this initial therapy, whenever possible.
Following initial treatment children will undergo alternating courses of induction chemotherapy with cyclophosphamide, doxorubicin, etoposide, topotecan, and cisplatin (Block 2). The first cohort of 17 patients will receive Block 2 with temsirolimus (50mg/m2) for all three courses, weekly 2 times. If this is not tolerated subsequent patients will receive Block 2 chemotherapy with reduced dosages of temsirolimus (25mg/m2).
- Conditions
- Neuroblastoma
- Being tested
- Temsirolimus, Irinotecan, Surgical Resection of Primary Tumor, Cyclophosphamide, Doxorubicin
- Keywords
- Neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TemsirolimusDrug
Temsirolimus
IrinotecanDrug
Irinotecan
Surgical Resection of Primary TumorProcedure or surgery
Surgical Resection of Primary Tumor
CyclophosphamideDrug
Cyclophosphamide
DoxorubicinDrug
Doxorubicin
EtoposideDrug
Etoposide
CisplatinDrug
Cisplatin
TopotecanDrug
Topotecan
PBSCProcedure or surgery
Peripheral Blood Stem Cell Harvest
Radiation TherapyRadiation
Radiation Therapy
13-cis-retinoic acidDrug
13-cis-retinoic acid
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Fixed doses of IV temsirolimus concomitantly with two courses of fixed dosages of irinotecan, 2 days off, repeated daily 5 times.If initial dosages are not tolerable, subsequent patients will be given a reduced dosage of temsirolimus with irinotecan.If this dosage combination is not tolerable,irinotecan dosage will be decreased.If this dosage combination is not tolerable.Further enrollment to initial six week treatment will be terminated.Second course of irinotecan will begin on day 22, response will be determined after six weeks. Resection of primary tumor will be attempted after initial therapy.Following initial treatment children will undergo alternating courses of induction chemotherapy with cyclophosphamide,doxorubicin,etoposide,topotecan, and cisplatin.First cohort of 17 patients will receive Block 2 with temsirolimus for all three courses, weekly 2 times.If this is not tolerated subsequent patients will receive Block 2 chemotherapy with reduced dosages of temsirolimus. Receives: Drug: Temsirolimus; Drug: Irinotecan; Procedure: Surgical Resection of Primary Tumor; Drug: Cyclophosphamide; Drug: Doxorubicin; Drug: Etoposide; Drug: Cisplatin; Drug: Topotecan; Procedure: PBSC; Radiation: Radiation Therapy; Drug: 13-cis-retinoic acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Response Plus Partial Response
The objective was to measure the efficacy and feasability of Temsirolimus and Irinotecan as measured by the objective response rate and toxicity rate.
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2008 (estimated)
- Study startDec 2008
- Primary completionJul 2009
- Study completionJul 2009
Why it stopped: Voluntarily closed and terminated by the PI due to lack of feasibility
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Wayne L Furman, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00808899
- Sponsor's protocol ID
- NB2008
- Status last verified
- May 2010
- First submitted
- 11 Dec 2008
- Last updated
- 30 May 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No