GliomaPhase 1NCT01902771
Dendritic Cell Vaccine Therapy With In Situ Maturation in Pediatric Brain Tumors
A Phase 1 study testing Dendritic Cell Vaccine, Tumor Lysate and Imiquimod in people with Glioma and Brain Cancer, run by Edward Ziga at 1 site in United States.
Trial summary
DC vaccine manufactured and partially matured using our standard operating procedures, developed in collaboration with the HGG Immuno Group, then administered through imiquimod treated skin will be safe and feasible in children with refractory brain tumors. Read more about this study
- Who is it for?
- Ages 1 year to 29 years
- Conditions the study is for
- Glioma, Brain Cancer, Brain Tumor
- What is being tested?
- Dendritic Cell Vaccine, Tumor Lysate and Imiquimod
- Type of treatment
- Vaccines, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Sep 2013to 26 Jan 2017
- 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 29 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age: ≥ 1 year and ≤ 29 years
- Relapse or progression of any central nervous system tumor initially diagnosed before the age of 21 years.
- Total or subtotal resection of tumor mass, confirmed by assessment by the neurosurgeon and by postoperative MRI scan within 72 hours after surgery. The post-operative assessment should demonstrate residual tumor less than or equal to 2 cm^3 as judged by surgeon or on MRI the tumor should only show linear contrast enhancement at the border of the resection cavity or nodule less than 2 cm^3.
- No radiotherapy and/or chemotherapy received for at least 1 month before first DC vaccination is to be administered.
- No treatment with corticosteroids or salicylates for at least 1 week before first vaccination.
- Life expectancy ≥ 3 months
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- Written consent by patient or parent(s) (if patient is < 18 years) on an institutional review board (IRB)-approved informed consent form prior to any study-specific evaluation. Assent is required from children as per University of Miami (UM) IRB guidelines. Subject must be capable of understanding the investigational nature, potential risks and benefits of the study and able to provide valid informed consent.
- Adequate organ function (to be measured at enrollment)
- Absolute neutrophil count (ANC) ≥750/L
- Lymphocytes ≥ 500/L
- Platelets ≥ 75,000/L
- Hemoglobin ≥ 9 g/dL
- Aspartate aminotransferase (AST)/Alanine transaminase (ALT) ≤ 2.5 X upper limit of normal (ULN); if liver metastases, ≤ 5 X ULN
- Serum Creatinine ≤ 1.5 X ULN
- Total Bilirubin ≤ 3 X ULN
- Albumin > 2 g/dL
- Subjects must agree to use adequate method of contraception or abstinence throughout and up to 4 weeks after the study treatment completion.
- Karnofsky score ≥ 70 or Eastern Cooperative Oncology Group (ECOG) status of 0 or 1.
Who cannot join
- Pregnancy.
- Breast feeding females.
- Any concomitant participation in other therapeutic trials.
- Virus serology positive for HIV (testing is not required in the absence of clinical suspicion).
- Documented immunodeficiency or autoimmune disease.
- Other active malignancies.
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- Refusal to use adequate contraception for fertile patients (females and males) during the study and for 30 days after the last dose of study treatment.
- Any serious or uncontrolled medical or psychiatric condition that in the opinion of the investigator makes the patient not able to participate in the study.
- Application of gliadel wafers within the prior 4 months or a plan to place Gliadel wafers at the time of resection for tumor acquisition for study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase I Study of Dendritic Cell Vaccine Therapy With In Situ Maturation for Pediatric Brain Tumors
DC vaccine manufactured and partially matured using our standard operating procedures, developed in collaboration with the HGG Immuno Group, then administered through imiquimod treated skin will be safe and feasible in children with refractory brain tumors. This will result in anti-tumor immunity that will prolong survival of subjects treated and results will be consistent with the outcomes found for subjects treated by HGG Immuno Group investigators. Study treatment will correlate with laboratory evidence of immune activation. Correlative studies will also reveal targets in the immune system which can be exploited to improve response for patients on successor trials.
- Conditions
- Glioma, Brain Cancer, Brain Tumor, Glioblastoma Multiforme, High Grade Glioma
- Being tested
- Dendritic Cell Vaccine, Tumor Lysate, Imiquimod, Leukapheresis
- Keywords
- Dendritic Cell Vaccine, DCV, Pediatric, Brain Tumor, In Situ, Lysate, Tumor Lysate, Glioblastoma Multiforme, GBM, Pheresis, Leukapheresis, High Grade Glioma, HGG
- Medical subject headings
- Glioma, Brain Neoplasms, Glioblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dendritic Cell VaccineBiological
Also called DC Vaccine
Post-Leukapheresis. Subjects will receive DC Vaccine administered once weekly, via intradermal injection, for 4 weeks for a total of four vaccinations, per study protocol.
Tumor LysateBiological
Also called Tumor Cell Lysate, Lysate of Tumor
Post-DC Vaccine therapy. Up to 1.5 mg of Lysate of tumor per dose administered via intradermal injection at intervals defined by study protocol.
ImiquimodOther
Also called Aldara
Subjects will self-apply Imiquimod topically to each designated vaccine site before and after scheduled administrations of DC Vaccine or Lysate, per study protocol.
LeukapheresisProcedure or surgery
Also called Pheresis
Baseline, post-surgery. Subjects will undergo leukapheresis procedure during baseline, after recovery from surgery to collect peripheral blood mononuclear cells (PBMCs) from which dendritic cells will be obtained, per study protocol.
| Study group | Role | What they receive |
|---|---|---|
| DC Vaccine + Lysate | Experimental | * Leukapheresis: Baseline, post-surgery;
* Dendritic Cell Vaccine (DC Vaccine): Post-Leukapheresis, administered intradermally once weekly via intradermal injection, for 4 weeks for a total of four vaccinations;
* Tumor Lysate (Lysate): Post-DC Vaccine therapy. Administered intradermally during weeks 8, 12, 16, and 28;
* Imiquimod: Self-applied topically by subject before and after scheduled DC Vaccine or Lysate administrations. Receives: Biological: Dendritic Cell Vaccine; Biological: Tumor Lysate; Other: Imiquimod; Procedure: Leukapheresis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of Toxicity in Study Participants Receiving Protocol Therapy
Rate of treatment-limiting toxicities (TLT) and/or adverse events in study participants receiving protocol therapy.
Measured: Up to 28 Weeks
Rate of Feasibility of Protocol Therapy in Study Participants
Rate of feasibility of protocol therapy in study participants. Feasibility refers to clinical feasibility - whether or not the patient can have enough monocytes removed to manufacture Dendritic Cells.
Measured: Up to 4 Weeks
Secondary outcomes (5)
Rate of Prolonged Survival or Prolonged Progression-Free Survival in Study Participants
Rate of prolonged survival or prolonged progression-free survival in study participants. Overall Survival is defined as the time elapsed from the start of treatment until death. For surviving patients, follow-up will be censored at the date of last contact. Progression-Free Survival (PFS) is defined as the time elapsed from the start of treatment to the date of documented progression or death, whichever comes first.
Measured: Up to 24 Months
Rate of Measurable Immune Response in Subjects Receiving Protocol Therapy.
Rate of measurable immune response in subjects receiving protocol therapy demonstrated by measurement levels of Myeloid Derived Suppressor Cells before and after treatment.
Measured: Up to 24 months
Comparison of clinical parameters of study participants versus associated outcomes for patients on other DC/Imiquimod studies.
A comparison of whether the clinical parameters associated with outcomes described for patients on other DC/Imiquimod protocols hold for subjects treated on this study.
Measured: Up to 24 Months
Estimation of the Proportion of Subjects with Recurrent Pediatric Brian Tumors who are able to complete DC Vaccine therapy and DC Vaccine + Lysate Therapy.
Estimation of the proportion of subjects with recurrent pediatric brain tumors who are able to receive all administrations of DC, and the proportion who are able to receive all administrations of DC and Lysate.
Measured: Up to 24 months
Identification of Parameters Associated with Poorer Activity of the Vaccine in Study Participants
Identification of parameters associated with poorer activity of the DC Vaccine in Study Participants in order to develop therapies to augment vaccine therapy.
Measured: Up to 24 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2013 (estimated)
- Study start3 Sep 2013
- Primary completion24 Oct 2016
- Study completion26 Jan 2017
Why it stopped: Lack of Accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GliomaStudies of the same condition, open and under way.
- 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
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Edward Ziga
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Edward Ziga, University of Miami
- Investigators
- Edward Ziga, MD, Principal investigator, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01902771
- Sponsor's protocol ID
- 20130136
- Status last verified
- Mar 2017
- First submitted
- 16 Jul 2013
- Last updated
- 10 Mar 2017
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of Dendritic Cell Vaccine Therapy With In Situ Maturation for Pediatric Brain Tumors
- ClinicalTrials.gov ID
- NCT01902771
- Acronym / sponsor ID
- 20130136
- Conditions
- Glioma, Brain Cancer, Brain Tumor, Glioblastoma Multiforme, High Grade Glioma
- Keywords
- Dendritic Cell Vaccine, DCV, Pediatric, Brain Tumor, In Situ, Lysate, Tumor Lysate, Glioblastoma Multiforme
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of Accrual
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Edward ZigaUniversity, hospital or other
- Study start
- 3 Sep 2013 (actual)
- Primary completion
- 24 Oct 2016 (actual)
- Study completion
- 26 Jan 2017 (actual)
- First posted
- 18 Jul 2013 (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.
DC vaccine manufactured and partially matured using our standard operating procedures, developed in collaboration with the HGG Immuno Group, then administered through imiquimod treated skin will be safe and feasible in children with refractory brain tumors. This will result in anti-tumor immunity that will prolong survival of subjects treated and results will be consistent with the outcomes found for subjects treated by HGG Immuno Group investigators. Study treatment will correlate with laboratory evidence of immune activation. Correlative studies will also reveal targets in the immune system which can be exploited to improve response for patients on successor trials.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| DC Vaccine + Lysate | Experimental | Biological: Dendritic Cell Vaccine, Biological: Tumor Lysate, Other: Imiquimod, Procedure: Leukapheresis* Leukapheresis: Baseline, post-surgery; * Dendritic Cell Vaccine (DC Vaccine): Post-Leukapheresis, administered intradermally once weekly via intradermal injection, for 4 weeks for a total of four vaccinations; * Tumor Lysate (Lysate): Post-DC Vaccine therapy. Administered intradermally during weeks 8, 12, 16, and 28; * Imiquimod: Self-applied topically by subject before and after scheduled DC Vaccine or Lysate administrations. |
Who may qualify
- Ages
- 1 year to 29 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (18 listed)
- Age: ≥ 1 year and ≤ 29 years
- Relapse or progression of any central nervous system tumor initially diagnosed before the age of 21 years.
- Total or subtotal resection of tumor mass, confirmed by assessment by the neurosurgeon and by postoperative MRI scan within 72 hours after surgery. The post-operative assessment should demonstrate residual tumor less than or equal to 2 cm^3 as judged by surgeon or on MRI the tumor should only show linear contrast enhancement at the border of the resection cavity or nodule less than 2 cm^3.
- No radiotherapy and/or chemotherapy received for at least 1 month before first DC vaccination is to be administered.
- No treatment with corticosteroids or salicylates for at least 1 week before first vaccination.
Key exclusion criteria (9 listed)
- Pregnancy.
- Breast feeding females.
- Any concomitant participation in other therapeutic trials.
- Virus serology positive for HIV (testing is not required in the absence of clinical suspicion).
- Documented immunodeficiency or autoimmune disease.
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 |
|---|---|---|
| Dendritic Cell VaccineAlso: DC VaccinePost-Leukapheresis. Subjects will receive DC Vaccine administered once weekly, via intradermal injection, for 4 weeks for a total of four vaccinations, per study protocol. | Biological | DC Vaccine + Lysate |
| Tumor LysateAlso: Tumor Cell Lysate, Lysate of TumorPost-DC Vaccine therapy. Up to 1.5 mg of Lysate of tumor per dose administered via intradermal injection at intervals defined by study protocol. | Biological | DC Vaccine + Lysate |
| ImiquimodAlso: AldaraSubjects will self-apply Imiquimod topically to each designated vaccine site before and after scheduled administrations of DC Vaccine or Lysate, per study protocol. | Other | DC Vaccine + Lysate |
| LeukapheresisAlso: PheresisBaseline, post-surgery. Subjects will undergo leukapheresis procedure during baseline, after recovery from surgery to collect peripheral blood mononuclear cells (PBMCs) from which dendritic cells will be obtained, per study protocol. | Procedure or surgery | DC Vaccine + Lysate |
Primary outcome
Rate of Toxicity in Study Participants Receiving Protocol Therapy
Time frame: Up to 28 Weeks
Rate of treatment-limiting toxicities (TLT) and/or adverse events in study participants receiving protocol therapy.
Rate of Feasibility of Protocol Therapy in Study Participants
Time frame: Up to 4 Weeks
Rate of feasibility of protocol therapy in study participants. Feasibility refers to clinical feasibility - whether or not the patient can have enough monocytes removed to manufacture Dendritic Cells.
5 secondary outcomes
Rate of Prolonged Survival or Prolonged Progression-Free Survival in Study Participants
Time frame: Up to 24 Months
Rate of Measurable Immune Response in Subjects Receiving Protocol Therapy.
Time frame: Up to 24 months
Comparison of clinical parameters of study participants versus associated outcomes for patients on other DC/Imiquimod studies.
Time frame: Up to 24 Months
Estimation of the Proportion of Subjects with Recurrent Pediatric Brian Tumors who are able to complete DC Vaccine therapy and DC Vaccine + Lysate Therapy.
Time frame: Up to 24 months
Identification of Parameters Associated with Poorer Activity of the Vaccine in Study Participants
Time frame: Up to 24 Months
Results and safety
The study was stopped early (reason given: Lack of Accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 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.
Edward Ziga, MD principal investigator
University of Miami
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 Dendritic Cell Vaccine, Tumor Lysate and Imiquimod in people with Glioma and Brain Cancer, run by Edward Ziga 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 18 inclusion and 9 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: stopped early, as of the sponsor's last update on 10 Mar 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 was stopped early (reason given: Lack of Accrual). Results are not available in the source record.
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?
Rate of Toxicity in Study Participants Receiving Protocol Therapy (time frame: Up to 28 Weeks). The study lists 2 primary outcomes in all. 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 10 Mar 2017.