SarcomaPhase 1NCT01803152
Dendritic Cell Vaccine for Children and Adults With Sarcoma
A Phase 1 study testing Dendritic Cells Vaccine, Lysate of Tumor and Gemcitabine in people with Sarcoma and Soft Tissue Sarcoma, run by Macarena De La Fuente, MD at 1 site in United States.
Trial summary
The purpose if this study is to evaluate an investigational vaccine using patient-derived dendritic cells (DC), a type of white blood cell that helps fight infections in the body, (DC) (a vaccine made out of participants' own cells and tumor) to treat sarcoma. Read more about this study
- Who is it for?
- Ages 1 year to 100 years
- Conditions the study is for
- Sarcoma, Soft Tissue Sarcoma, Bone Sarcoma
- What is being tested?
- Dendritic Cells Vaccine, Lysate of Tumor and Gemcitabine
- Type of treatment
- Vaccines, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Jan 2014to 21 Jun 2024
- 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 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age: 1 - 100 years old.
- Histologically or cytologically confirmed sarcoma either relapsed or without known curative therapies. Both bone sarcomas and soft tissue sarcomas are eligible. Osteosarcoma, chondrosarcoma, Ewing's sarcoma and any other diagnoses of sarcoma are eligible as long as there is soft tissue that can be excised and be used to prepare lysate. Subjects presenting only with lesions that are only comprised of bone are excluded. Any number of prior therapies is allowed, including zero.
- No radiotherapy to other sites planned and/or other chemotherapy planned for the study period. No radiotherapy or chemotherapy to have been received for at least 4 weeks before first vaccine administration. To allow for better local control without introducing undue toxicity into the trial, brachytherapy at time of surgery scheduled to end by one week before first vaccination is allowed if the radioactive source is to be removed (e.g. catheters can be placed if removable but implanted seeds are not allowed). In the event of positive margins being determined after surgical resection, but not determined in time for the placement of brachytherapy catheters, external beam radiotherapy may start after the last DC vaccination is administered but before the lysate boosts begin, and radiation must be planned to be complete before the first lysate boost.
- No treatment with corticosteroids, antihistamines or salicylates for at least 1 week before first vaccination.
- Adequate organ function (to be measured at enrollment)
- Absolute neutrophil count (ANC) ≥ 0.75* 10^3/µL
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- Lymphocytes ≥ 0.5 * 10^3/µL
- Platelets ≥ 75 * 10^3/µL
- 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
- Karnofsky/Lansky score of ≥ 70% or Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
- Subjects must agree to use adequate method of contraception or abstinence throughout and up to 4 weeks after the study treatment completion.
- Life expectancy of > 3 months.
- 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.
Who cannot join
- Pregnancy
- Breast feeding females.
- Any concomitant participation in other therapeutic trials
- Virus serology known to be positive for HIV (testing is not required in the absence of clinical suspicion)
- Documented immunodeficiency or autoimmune disease
- Concomitant treatment with corticosteroids, antihistamines (H1 and H2 inhibitors) or salicylates. Patients may be eligible if the treatment is stopped at least 1 week before the first vaccination.
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- Brain metastases unless they have been stable for 3 months off of treatment directed specifically at them.
- Known allergy to gemcitabine or its formulation components. Intolerant to gemcitabine
- Does not apply to cohorts to be treated without gemcitabine
- Prior therapy with gemcitabine is allowed on all cohorts
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase I Trial of Dendritic Cell Vaccination for Children and Adults With Sarcoma
The purpose if this study is to evaluate an investigational vaccine using patient-derived dendritic cells (DC), a type of white blood cell that helps fight infections in the body, (DC) (a vaccine made out of participants' own cells and tumor) to treat sarcoma.
- Conditions
- Sarcoma, Soft Tissue Sarcoma, Bone Sarcoma
- Being tested
- Dendritic Cells Vaccine, Lysate of Tumor, Gemcitabine, Imiquimod, Leukapheresis
- Keywords
- Metastatic Sarcoma, Relapsed Sarcoma, Dendritic Cell, Myeloid Derived Suppressor Cells, MDSC, MDSC Inhibition
- Medical subject headings
- Sarcoma, Bone Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dendritic Cells VaccineBiological
Also called DC Vaccine
Subjects will receive DC Vaccine administered once weekly, via intradermal injection, for 4 weeks for a total of four vaccinations, per study protocol.
Lysate of TumorBiological
Also called Lysate, Tumor Lysate
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.
GemcitabineDrug
Also called Gemzar
Post-surgery, Leukapheresis and clearance of subject. Gemcitabine 1000 mg/m2 IV will be administered once weekly for 3 weeks per study protocol.
ImiquimodDrug
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 blood draw via catheter to obtain peripheral blood mononuclear cells (PBMCs) from which Dendritic cells will be obtained.
| Study group | Role | What they receive |
|---|---|---|
| Part 1-DC Vaccine/Lysate | Experimental | * Leukapheresis: Baseline, post-surgery;
* Dendritic Cells Vaccine (DC Vaccine): Post-Leukapheresis, administered once weekly in dose-escalation scheme for 4 weeks;
* Lysate of Tumor (Lysate): Post-DC Vaccine therapy, administered during weeks 8, 12, 16 and 20;
* Imiquimod: Self-applied topically by subject before and after scheduled DC Vaccine or Lysate administrations. Receives: Biological: Dendritic Cells Vaccine; Biological: Lysate of Tumor; Drug: Imiquimod; Procedure: Leukapheresis |
| Part 2-Gemcitabine/DC Vaccine/Lysate | Active comparator | * Leukapheresis: Baseline, post-surgery;
* Gemcitabine: Post-Leukapheresis, administered once weekly for 3 weeks;
* Dendritic Cells Vaccine (DC Vaccine): Post-Gemcitabine therapy, Recommended Phase 2 Dose (RP2D) administered once weekly for 4 weeks;
* Lysate of Tumor (Lysate): Post-DC Vaccine therapy, administered during weeks 12, 16, 20 and 32;
* Imiquimod: Self-applied topically by subject before and after scheduled DC Vaccine or Lysate administrations. Receives: Biological: Dendritic Cells Vaccine; Biological: Lysate of Tumor; Drug: Gemcitabine; Drug: Imiquimod; Procedure: Leukapheresis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
To demonstrate that DC vaccination loaded with tumor lysate is feasible and that therapy with the vaccine with topical imiquimod (as final step in vaccine maturation), with or without the inhibition of MDSC by gemcitabine pre-treatment, is safe in pediatric and adult subjects with metastatic and refractory sarcoma.
Measured: From Day 1 to 30 Days Post-Treatment, about 9 months
Secondary outcomes (5)
Measurement levels of Myeloid Derived Supressor Cells before and after treatment
To explore biomarkers of immune response. Assessment will include measurement of levels of Myeloid Derived Supressor Cells before and after treatment and T and B cell subsets before and after treatment.
Measured: From Baseline to 3 Months Post-Treatment, up to 12 months
Progression-free survival
To obtain preliminary clinical benefit by evaluating progression-free survival (PFS)in patients receiving this DC vaccine with or without gemcitabine pre-treatment. PFS is defined as the time elapsed from the start of treatment to the date of documented progression or death, whichever comes first. For surviving patients without progression who begin alternative treatment, PFS will be censored at the last date of documented progression-free status prior to starting alternative treatment. Similarly, losses to follow up will be censored at the last date of documented progression-free status.
Measured: Up to 24 months Post-Treatment
Overall Survival
To obtain preliminary clinical benefit by evaluating overall survival in patients receiving this DC vaccine with or without gemcitabine pre-treatment. 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.
Measured: Up to 5 years Post-Treatment
The rate of Complete Response (CR) or Partial Response (PR) in subjects receiving treatment
To obtain preliminary clinical benefit by evaluating response rate (RR) in patients receiving this DC vaccine with or without gemcitabine pre-treatment. Response will be assessed according to Response Evaluation Criteria in Solid Tumors (RECIST) Criteria, version 1.1.
Measured: Up to 24 months Post-Treatment
Measurement levels of Myeloid Derived Supressor Cells after Gemcitabine treatment
To determine if gemcitabine is effective in the inhibition and depletion of Myeloid Derived Supressor Cells in the study patients.
Measured: From Baseline to End of Treatment, about 10 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Mar 2013 (estimated)
- Study start6 Jan 2014
- Primary completion10 Sep 2019
- Study completion21 Jun 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for SarcomaStudies 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Macarena De La Fuente, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Macarena De La Fuente, MD, University of Miami
- Investigators
- Gina D'Amato, MD, Principal investigator, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01803152
- Sponsor's protocol ID
- 20110462
- Status last verified
- Jun 2024
- First submitted
- 27 Feb 2013
- Last updated
- 3 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Goff PH, Riolobos L, LaFleur BJ, Spraker MB, Seo YD, Smythe KS, Campbell JS, Pierce RH, Zhang Y, He Q, Kim EY, Schaub SK, Kane GM, Mantilla JG, Chen EY, Ricciotti R, Thompson MJ, Cranmer LD, Wagner MJ, Loggers ET, Jones RL, Murphy E, Blumenschein WM, McClanahan TK, Earls J, Flanagan KC, LaFranzo NA, Kim TS, Pollack SM. Neoadjuvant Therapy Induces a Potent Immune Response to Sarcoma, Dominated by Myeloid and B Cells. Clin Cancer Res. 2022 Apr 14;28(8):1701-1711. doi: 10.1158/1078-0432.CCR-21-4239.
PubMed 35115306
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Trial of Dendritic Cell Vaccination for Children and Adults With Sarcoma
- ClinicalTrials.gov ID
- NCT01803152
- Acronym / sponsor ID
- 20110462
- Conditions
- Sarcoma, Soft Tissue Sarcoma, Bone Sarcoma
- Keywords
- Metastatic Sarcoma, Relapsed Sarcoma, Dendritic Cell, Myeloid Derived Suppressor Cells, MDSC, MDSC Inhibition
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Macarena De La Fuente, MDUniversity, hospital or other
- Study start
- 6 Jan 2014 (actual)
- Primary completion
- 10 Sep 2019 (actual)
- Study completion
- 21 Jun 2024 (actual)
- First posted
- 4 Mar 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.
The purpose if this study is to evaluate an investigational vaccine using patient-derived dendritic cells (DC), a type of white blood cell that helps fight infections in the body, (DC) (a vaccine made out of participants' own cells and tumor) to treat sarcoma.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Part 1-DC Vaccine/Lysate | Experimental | Biological: Dendritic Cells Vaccine, Biological: Lysate of Tumor, Drug: Imiquimod, Procedure: Leukapheresis* Leukapheresis: Baseline, post-surgery; * Dendritic Cells Vaccine (DC Vaccine): Post-Leukapheresis, administered once weekly in dose-escalation scheme for 4 weeks; * Lysate of Tumor (Lysate): Post-DC Vaccine therapy, administered during weeks 8, 12, 16 and 20; * Imiquimod: Self-applied topically by subject before and after scheduled DC Vaccine or Lysate administrations. |
| Part 2-Gemcitabine/DC Vaccine/Lysate | Active comparator | Biological: Dendritic Cells Vaccine, Biological: Lysate of Tumor, Drug: Gemcitabine, Drug: Imiquimod, Procedure: Leukapheresis* Leukapheresis: Baseline, post-surgery; * Gemcitabine: Post-Leukapheresis, administered once weekly for 3 weeks; * Dendritic Cells Vaccine (DC Vaccine): Post-Gemcitabine therapy, Recommended Phase 2 Dose (RP2D) administered once weekly for 4 weeks; * Lysate of Tumor (Lysate): Post-DC Vaccine therapy, administered during weeks 12, 16, 20 and 32; * Imiquimod: Self-applied topically by subject before and after scheduled DC Vaccine or Lysate administrations. |
Who may qualify
- Ages
- 1 year to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (17 listed)
- Age: 1 - 100 years old.
- Histologically or cytologically confirmed sarcoma either relapsed or without known curative therapies. Both bone sarcomas and soft tissue sarcomas are eligible. Osteosarcoma, chondrosarcoma, Ewing's sarcoma and any other diagnoses of sarcoma are eligible as long as there is soft tissue that can be excised and be used to prepare lysate. Subjects presenting only with lesions that are only comprised of bone are excluded. Any number of prior therapies is allowed, including zero.
- No radiotherapy to other sites planned and/or other chemotherapy planned for the study period. No radiotherapy or chemotherapy to have been received for at least 4 weeks before first vaccine administration. To allow for better local control without introducing undue toxicity into the trial, brachytherapy at time of surgery scheduled to end by one week before first vaccination is allowed if the radioactive source is to be removed (e.g. catheters can be placed if removable but implanted seeds are not allowed). In the event of positive margins being determined after surgical resection, but not determined in time for the placement of brachytherapy catheters, external beam radiotherapy may start after the last DC vaccination is administered but before the lysate boosts begin, and radiation must be planned to be complete before the first lysate boost.
- No treatment with corticosteroids, antihistamines or salicylates for at least 1 week before first vaccination.
- Adequate organ function (to be measured at enrollment)
Key exclusion criteria (12 listed)
- Pregnancy
- Breast feeding females.
- Any concomitant participation in other therapeutic trials
- Virus serology known to be 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 Cells VaccineAlso: DC VaccineSubjects will receive DC Vaccine administered once weekly, via intradermal injection, for 4 weeks for a total of four vaccinations, per study protocol. | Biological | Part 1-DC Vaccine/Lysate, Part 2-Gemcitabine/DC Vaccine/Lysate |
| Lysate of TumorAlso: Lysate, Tumor LysatePost-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 | Part 1-DC Vaccine/Lysate, Part 2-Gemcitabine/DC Vaccine/Lysate |
| GemcitabineAlso: GemzarPost-surgery, Leukapheresis and clearance of subject. Gemcitabine 1000 mg/m2 IV will be administered once weekly for 3 weeks per study protocol. | Drug | Part 2-Gemcitabine/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. | Drug | Part 1-DC Vaccine/Lysate, Part 2-Gemcitabine/DC Vaccine/Lysate |
| LeukapheresisAlso: PheresisBaseline, post-surgery blood draw via catheter to obtain peripheral blood mononuclear cells (PBMCs) from which Dendritic cells will be obtained. | Procedure or surgery | Part 1-DC Vaccine/Lysate, Part 2-Gemcitabine/DC Vaccine/Lysate |
Primary outcome
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Time frame: From Day 1 to 30 Days Post-Treatment, about 9 months
To demonstrate that DC vaccination loaded with tumor lysate is feasible and that therapy with the vaccine with topical imiquimod (as final step in vaccine maturation), with or without the inhibition of MDSC by gemcitabine pre-treatment, is safe in pediatric and adult subjects with metastatic and refractory sarcoma.
5 secondary outcomes
Measurement levels of Myeloid Derived Supressor Cells before and after treatment
Time frame: From Baseline to 3 Months Post-Treatment, up to 12 months
Progression-free survival
Time frame: Up to 24 months Post-Treatment
Overall Survival
Time frame: Up to 5 years Post-Treatment
The rate of Complete Response (CR) or Partial Response (PR) in subjects receiving treatment
Time frame: Up to 24 months Post-Treatment
Measurement levels of Myeloid Derived Supressor Cells after Gemcitabine treatment
Time frame: From Baseline to End of Treatment, about 10 months
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 3 Jul 2024.
- Derived from this study: PubMed 35115306Goff PH, Riolobos L, LaFleur BJ, Spraker MB, Seo YD, Smythe KS, Campbell JS, Pierce RH, Zhang Y, He Q, Kim EY, Schaub SK, Kane GM, Mantilla JG, Chen EY, Ricciotti R, Thompson MJ, Cranmer LD, Wagner MJ, Loggers ET, Jones RL, Murphy E, Blumenschein WM, McClanahan TK, Earls J, Flanagan KC, LaFranzo NA, Kim TS, Pollack SM. Neoadjuvant Therapy Induces a Potent Immune Response to Sarcoma, Dominated by Myeloid and B Cells. Clin Cancer Res. 2022 Apr 14;28(8):1701-1711. doi: 10.1158/1078-0432.CCR-21-4239.
- Individual participant dataSharing planned: No
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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.
Gina D'Amato, 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 Cells Vaccine, Lysate of Tumor and Gemcitabine in people with Sarcoma and Soft Tissue Sarcoma, run by Macarena De La Fuente, MD 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 17 inclusion and 12 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 3 Jul 2024. 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?
Number of Participants with Adverse Events as a Measure of Safety and Tolerability (time frame: From Day 1 to 30 Days Post-Treatment, about 9 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01803152, which also lists 1 linked publication.
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 3 Jul 2024; AzTrials last refreshed its copy on 6 Nov 2026.