- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01803152
Dendritic Cell Vaccine for Children and Adults With Sarcoma
June 30, 2023 updated by: Macarena De La Fuente, MD
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.
Study Overview
Status
Active, not recruiting
Conditions
Study Type
Interventional
Enrollment (Actual)
19
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Florida
-
Miami, Florida, United States, 33136
- University of Miami
-
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 year to 100 years (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- 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
- 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.
Exclusion Criteria:
- 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.
- 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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Part 1-DC Vaccine/Lysate
|
Subjects will receive DC Vaccine administered once weekly, via intradermal injection, for 4 weeks for a total of four vaccinations, per study protocol.
Other Names:
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.
Other Names:
Subjects will self-apply Imiquimod topically to each designated vaccine site before and after scheduled administrations of DC Vaccine or Lysate, per study protocol.
Other Names:
Baseline, post-surgery blood draw via catheter to obtain peripheral blood mononuclear cells (PBMCs) from which Dendritic cells will be obtained.
Other Names:
|
Active Comparator: Part 2-Gemcitabine/DC Vaccine/Lysate
|
Subjects will receive DC Vaccine administered once weekly, via intradermal injection, for 4 weeks for a total of four vaccinations, per study protocol.
Other Names:
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.
Other Names:
Subjects will self-apply Imiquimod topically to each designated vaccine site before and after scheduled administrations of DC Vaccine or Lysate, per study protocol.
Other Names:
Baseline, post-surgery blood draw via catheter to obtain peripheral blood mononuclear cells (PBMCs) from which Dendritic cells will be obtained.
Other Names:
Post-surgery, Leukapheresis and clearance of subject.
Gemcitabine 1000 mg/m2 IV will be administered once weekly for 3 weeks per study protocol.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
From Day 1 to 30 Days Post-Treatment, about 9 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Measurement levels of Myeloid Derived Supressor Cells before and after treatment
Time Frame: From Baseline to 3 Months Post-Treatment, up to 12 months
|
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.
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From Baseline to 3 Months Post-Treatment, up to 12 months
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Progression-free survival
Time Frame: Up to 24 months Post-Treatment
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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.
|
Up to 24 months Post-Treatment
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Overall Survival
Time Frame: Up to 5 years Post-Treatment
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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.
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Up to 5 years Post-Treatment
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The rate of Complete Response (CR) or Partial Response (PR) in subjects receiving treatment
Time Frame: Up to 24 months Post-Treatment
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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.
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Up to 24 months Post-Treatment
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Measurement levels of Myeloid Derived Supressor Cells after Gemcitabine treatment
Time Frame: From Baseline to End of Treatment, about 10 months
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To determine if gemcitabine is effective in the inhibition and depletion of Myeloid Derived Supressor Cells in the study patients.
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From Baseline to End of Treatment, about 10 months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Gina D'Amato, MD, University of Miami
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
January 6, 2014
Primary Completion (Actual)
September 10, 2019
Study Completion (Estimated)
July 23, 2024
Study Registration Dates
First Submitted
February 27, 2013
First Submitted That Met QC Criteria
February 28, 2013
First Posted (Estimated)
March 4, 2013
Study Record Updates
Last Update Posted (Actual)
July 5, 2023
Last Update Submitted That Met QC Criteria
June 30, 2023
Last Verified
June 1, 2023
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Neoplasms, Connective and Soft Tissue
- Neoplasms by Histologic Type
- Neoplasms
- Sarcoma
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Antimetabolites, Antineoplastic
- Antimetabolites
- Antineoplastic Agents
- Immunologic Factors
- Adjuvants, Immunologic
- Interferon Inducers
- Vaccines
- Imiquimod
- Gemcitabine
Other Study ID Numbers
- 20110462
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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