NeuroblastomaPhase 1NCT01241162
Decitabine Followed by a Cancer Antigen Vaccine for Patients With Neuroblastoma and Sarcoma
A Phase 1 study testing Autologous dendritic cell vaccine with adjuvant in people with Neuroblastoma and Ewings Sarcoma, run by University of Louisville at 1 site in United States.
Trial summary
This treatment study for relapsed high-risk neuroblastoma, Ewings sarcoma, osteogenic sarcoma, rhabdomyosarcoma or synovial sarcoma involves an autologous cancer testis (CT) antigen specific dendritic cell (DC) vaccine preceded by decitabine as a demethylating chemotherapy. Read more about this study
- Who is it for?
- Ages 1 year to 24 years
- Conditions the study is for
- Neuroblastoma, Ewings Sarcoma, Osteogenic Sarcoma
- What is being tested?
- Autologous dendritic cell vaccine with adjuvant
- Type of treatment
- Vaccines, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2010to Oct 2016
- 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 24 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Confirmed diagnosis of relapsed high-risk neuroblastoma,Ewings sarcoma, osteogenic sarcoma, rhabdomyosarcoma, synovial sarcoma
- Patient may have gross tumor that has been treated with multi-agent chemotherapy prior study entry, but does not need to have gross tumor prior to study entry.
- Patients must have had a diagnosis of neuroblastoma or sarcoma either by histological verification of neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines.
- Have received standard chemotherapy and/or SCT, and are at least 6 months post-transplant.
- Age: Patients must be 1 - < 25 years of age when registered on study.
- Organ Function Requirements: All patients must have adequate organ function defined as:
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- Hematological Function: ANC ≥ 500; Platelet count ≥ 75.
- Renal Function: Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m2 OR A maximum serum creatinine (mg/dL) based on age/gender as follows: 1YO M\&F = 0.6, 2-5YO M\&F = 0.8, 6-9YO M\&F = 1, 10-12YO M\&F = 1.2, 13-15YO M = 1.5, 13-15YO F = 1.4, 16+ M = 1.7, 16+ F = 1.4
- Cardiac Function: Patient must have normal cardiac function documented by Ejection fraction (> 55%) documented by echocardiogram or radionuclide MUGA evaluation OR Fractional shortening (≥ 28%) documented by echocardiogram
- Liver Function: Total bilirubin ≤ 1.5 x normal for age, AND SGPT (ALT) and SGOT (AST) ≤ 3 x normal for age.
- Room air pulse oximetry >94%.
- Male and female sexually active patients of reproductive age who wish to participate must agree to use acceptable contraception.
- Lansky performance scale > 70, ECOG < 2 (Appendix I).
Who cannot join
- Patient is pregnant.
- Patients with a positive result for any of the following diagnostic tests: Hep B Ag, Hep B Core Ab, Hep C Ab, HIV-1 Ab, HIV-2 Ab, HTLV-1 Ab, HTLV-2 Ab, RPR.
- Patient has a history of autoimmune disease, specifically inflammatory bowel disease, systemic lupus erythematosis, or rheumatoid arthritis.
- Patient is receiving concurrent systemic steroid therapy.
- Patient has a known systemic hypersensitivity to DAC, Hiltonol, or any vaccine component.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Louisville, Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, 40202
About this study
Official title: A Phase I Trial Combining Decitabine and Vaccine Therapy for Patients With Relapsed Neuroblastoma and Sarcoma.
This treatment study for relapsed high-risk neuroblastoma, Ewings sarcoma, osteogenic sarcoma, rhabdomyosarcoma or synovial sarcoma involves an autologous cancer testis (CT) antigen specific dendritic cell (DC) vaccine preceded by decitabine as a demethylating chemotherapy.
Read the detailed description
For vaccine production, mature DC will be pulsed with overlapping peptides mixes derived from full-length NY-ESO-1, MAGE-A1, and MAGE-A3.
- Being tested
- Autologous dendritic cell vaccine with adjuvant
- Keywords
- Neuroblastoma, Ewings sarcoma, osteogenic sarcoma, rhabdomyosarcoma, synovial sarcoma, high-risk, relapsed, children
- Medical subject headings
- Neuroblastoma, Sarcoma, Ewing, Osteosarcoma, Rhabdomyosarcoma, Sarcoma, Synovial, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous dendritic cell vaccine with adjuvantBiological
Also called Decitabine (trade name Dacogen or 5-aza-2'-deoxycytidine/DAC, Hiltonol
Week 1:
Decitabine (DAC): 10 mg/m2/day IV (Mon-Fri)
Weeks 2 and 3:
Vaccine: 3-5 E6 peptide pulsed DC (Mon)
Hiltonol applied topically to vaccine site before and after vaccination
| Study group | Role | What they receive |
|---|---|---|
| Single arm study | Experimental | Biological/Vaccine: Autologous dendritic cell vaccine with adjuvant Receives: Biological: Autologous dendritic cell vaccine with adjuvant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tolerance of study treatment
Tolerance to DAC, at at least 50% dosing, and 3 of the 4 planned vaccinations during the first two cycles.
Measured: 2 years
Secondary outcomes (2)
Tumor Response
Assessment of tumor responses--complete or partial remission, stable disease, and disease progression--by CT/PET/MIBG or MRI after cycles 2 and 4.
Measured: 2 years
Immune Response
Assessment of post-vaccination T cell responses to MAGE-A1, MAGE-A3, and NY-ESO-1 by immunoassays.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Nov 2010 (estimated)
- Study startAug 2010
- Primary completionOct 2016
- Study completionOct 2016
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 University of LouisvilleThe 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
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Louisville
- Sponsor type
- University, hospital or other
- Collaborators
- Solving Kids' Cancer
- Responsible party
- Sponsor
- Investigators
- Kenneth G Lucas, MD, Principal investigator, University of Louisville, Kosairs Charities Pediatric Clinical Research Unit
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01241162
- Sponsor's protocol ID
- 34183
- Status last verified
- May 2017
- First submitted
- 15 Nov 2010
- Last updated
- 9 May 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Trial Combining Decitabine and Vaccine Therapy for Patients With Relapsed Neuroblastoma and Sarcoma.
- ClinicalTrials.gov ID
- NCT01241162
- Acronym / sponsor ID
- 34183
- Keywords
- Neuroblastoma, Ewings sarcoma, osteogenic sarcoma, rhabdomyosarcoma, synovial sarcoma, high-risk, relapsed, children
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- University of LouisvilleUniversity, hospital or other
- Collaborators
- Solving Kids' Cancer
- Study start
- Aug 2010
- Primary completion
- Oct 2016 (actual)
- Study completion
- Oct 2016 (actual)
- First posted
- 16 Nov 2010 (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.
This treatment study for relapsed high-risk neuroblastoma, Ewings sarcoma, osteogenic sarcoma, rhabdomyosarcoma or synovial sarcoma involves an autologous cancer testis (CT) antigen specific dendritic cell (DC) vaccine preceded by decitabine as a demethylating chemotherapy.
Detailed description
For vaccine production, mature DC will be pulsed with overlapping peptides mixes derived from full-length NY-ESO-1, MAGE-A1, and MAGE-A3.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm study | Experimental | Biological: Autologous dendritic cell vaccine with adjuvantBiological/Vaccine: Autologous dendritic cell vaccine with adjuvant |
Who may qualify
- Ages
- 1 year to 24 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (13 listed)
- Confirmed diagnosis of relapsed high-risk neuroblastoma,Ewings sarcoma, osteogenic sarcoma, rhabdomyosarcoma, synovial sarcoma
- Patient may have gross tumor that has been treated with multi-agent chemotherapy prior study entry, but does not need to have gross tumor prior to study entry.
- Patients must have had a diagnosis of neuroblastoma or sarcoma either by histological verification of neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines.
- Have received standard chemotherapy and/or SCT, and are at least 6 months post-transplant.
- Age: Patients must be 1 - < 25 years of age when registered on study.
Key exclusion criteria (5 listed)
- Patient is pregnant.
- Patients with a positive result for any of the following diagnostic tests: Hep B Ag, Hep B Core Ab, Hep C Ab, HIV-1 Ab, HIV-2 Ab, HTLV-1 Ab, HTLV-2 Ab, RPR.
- Patient has a history of autoimmune disease, specifically inflammatory bowel disease, systemic lupus erythematosis, or rheumatoid arthritis.
- Patient is receiving concurrent systemic steroid therapy.
- Patient has a known systemic hypersensitivity to DAC, Hiltonol, or any vaccine component.
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 |
|---|---|---|
| Autologous dendritic cell vaccine with adjuvantAlso: Decitabine (trade name Dacogen or 5-aza-2'-deoxycytidine/DAC, HiltonolWeek 1: Decitabine (DAC): 10 mg/m2/day IV (Mon-Fri) Weeks 2 and 3: Vaccine: 3-5 E6 peptide pulsed DC (Mon) Hiltonol applied topically to vaccine site before and after vaccination | Biological | Single arm study |
Primary outcome
Tolerance of study treatment
Time frame: 2 years
Tolerance to DAC, at at least 50% dosing, and 3 of the 4 planned vaccinations during the first two cycles.
2 secondary outcomes
Tumor Response
Time frame: 2 years
Immune Response
Time frame: 2 years
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 9 May 2017.
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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.
Kenneth G Lucas, MD principal investigator
University of Louisville, Kosairs Charities Pediatric Clinical Research Unit
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 Autologous dendritic cell vaccine with adjuvant in people with Neuroblastoma and Ewings Sarcoma, run by University of Louisville 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?
The sponsor lists 13 inclusion and 5 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 9 May 2017. 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.
What does the primary outcome measure?
Tolerance of study treatment (time frame: 2 years). 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 9 May 2017.