Ewing SarcomaNCT04956198
Drug Sensitivity and Mutation Profiling
An observational study of Ewing Sarcoma and Osteosarcoma, run by Florida International University at 1 site in United States.
Trial summary
This study is a prospective, non-randomized observational study. Freshly isolated tumor cells will be tested for chemosensitivity to the standard of care drugs as single agents and in combinations using state-of-the-art viability assay designed for ex-vivo high-throughput drug sensitivity testing (DST). Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Ewing Sarcoma, Osteosarcoma, Rhabdomyosarcoma
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Nov 2020to 31 Dec 2022
- Phase
- Observational
- 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 Up to 21 years
- All sexes
- Child, Adult
Study population: Patients with newly diagnosed as well as relapsed/refractory sarcomas.
Who may be able to join
- Patients aged 21 years or younger at the time of enrollment on this study of any gender, race, or ethnicity.
- Subjects with suspected or confirmed diagnosis of all types of sarcomas.
- Subjects who are scheduled for or have recently had biopsy or tumor excised (solid tumors) or bone marrow aspirate (blood cancers).
- Subjects are willing to have a blood draw or buccal swab done for the purposes of genetic testing.
- Subjects or their parents or legal guardians willing to sign informed consent.
- Subjects aged 7 to 17 willing to sign assent.
Who cannot join
- Subjects who do not have malignant tissue available and accessible.
- The amount of excised malignant tissue is not sufficient for ex vivo drug testing and/or genetic profiling.
- Patients with other types of tumors and tumors that have a high (>90%) cure rate with safe standard therapy.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Drug Sensitivity Testing and Mutation Profiling in Childhood Sarcomas
This study is a prospective, non-randomized observational study. Freshly isolated tumor cells will be tested for chemosensitivity to the standard of care drugs as single agents and in combinations using state-of-the-art viability assay designed for ex-vivo high-throughput drug sensitivity testing (DST). In addition, the genetic profile of the tumor will be obtained from the medical records and correlated with drug response.
Read the detailed description
The excised tumors or a biopsy will be interrogated for sensitivity or resistance to FDA-approved and/or available investigational agents. In addition, normal samples (blood or buccal swab) will be collected for genetic analysis of germline mutations and cancer predisposition markers. The timeframe between the sample acquisition and ex vivo DST results return will be approximately 5-10 working days. All drugs tested in the DST assay will be assigned a hybrid score reflecting the tumor's sensitivity and drug toxicity.
This is an observational study and not a treatment protocol. It will assess how ex vivo drug testing and mutation profiling may predict clinical outcomes (response, survival, or relapse). The treating physician will decide which of the standard treatment options is appropriate independent of the DST results. The results of DST will not be available to the treating physician at the time of decision on the treatment regimen. DST will include all drugs from the standard treatment regimens available for all types of sarcomas
- Conditions
- Ewing Sarcoma, Osteosarcoma, Rhabdomyosarcoma, Wilms Tumor, Sarcoma, Spindle Cell, Sarcoma Rhabdoid
- Keywords
- ex vivo drug sensitivity assay, genomic profiling
- Medical subject headings
- Sarcoma, Ewing, Osteosarcoma, Rhabdomyosarcoma, Wilms Tumor, Sarcoma, Rhabdoid Tumor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Patients with newly diagnosed as well as relapsed/refractory sarcomas. | The investigators intend to enroll newly diagnosed or refractory/relapsed pediatric patients with all types of sarcomas where tumor tissue would be available for ex vivo drug screening and genomic profiling. This observational study will assess how ex vivo drug testing and mutation profiling may predict clinical outcomes (response, survival, or relapse). The treating physician will decide which of the standard treatment options is appropriate independent of the DST results. The results of DST will not be available to the treating physician at the time of decision on the treatment regimen. DST will include all drugs from the standard treatment regimens available for all types of sarcomas |
What is being measured
The primary outcome is the main question the study is designed to answer.
To correlate results of drug sensitivity and mutation profiles with clinical outcomes in response to therapy.
Response to therapy (RTT): an event of achieving "partial response" or "complete response" during the study period, based on the best response of the corresponding enrolled patient.
Measured: Up to 4 years
To correlate results of drug sensitivity and mutation profiles with clinical outcomes in progression-free survival.
Progression-free survival (PFS): a composite end point defined as the censored event time from enrollment to either disease relapse or mortality. This will be evaluated retrospectively at the end of the study. The investigators will use the two-sample long-rank test to assess the hazard ratio of PFS events between the two groups classified as drug sensitive and insensitive by the DST.The study will enroll 15 patients and it is assumed 50% of these subjects will be classified as drug sensitive based on the DST at the threshold value of 10.
Measured: Up to 4 years
Secondary outcomes (2)
To assess the predictive value of personalized approach in predicting RTT.
Classification accuracy of DST for predicting the RTT
Measured: Up to 4 years
To assess the predictive value of personalized approach in predicting PFS.
Classification accuracy of DST for predicting the PFS
The DTS test results will be treated as the continuous classifier, and the binary RTT status for each subject along with the binary PFS status by the end of the study will be used as the binary reference status (with and without RTT/PFS endpoint). The area under the curve (AUC) of the resulting ROC curve will be used a metric for the overall classification accuracy
Measured: Up to 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2021
- Study start17 Nov 2020
- Primary completion31 Dec 2022
- Study completion31 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ewing 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Florida International University
- Sponsor type
- University, hospital or other
- Collaborators
- Nicklaus Children's Hospital f/k/a Miami Children's Hospital, Cornelia T. Bailey Foundation
- Responsible party
- Principal investigator, Diana Azzam, PhD, Florida International University
- Investigators
- Diana Azzam, PhD, Principal investigator, Florida International UniversityDaria Salyakina, PhD, Principal investigator, Nicklaus Children's HospitalMaggie Fader, MD, Principal investigator, Nicklaus Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04956198
- Sponsor's protocol ID
- 1294510
- Status last verified
- Feb 2023
- First submitted
- 25 Jun 2021
- Last updated
- 14 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Drug Sensitivity Testing and Mutation Profiling in Childhood Sarcomas
- ClinicalTrials.gov ID
- NCT04956198
- Acronym / sponsor ID
- 1294510
- Conditions
- Ewing Sarcoma, Osteosarcoma, Rhabdomyosarcoma, Wilms Tumor, Sarcoma, Spindle Cell, Sarcoma Rhabdoid
- Keywords
- ex vivo drug sensitivity assay, genomic profiling
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Florida International UniversityUniversity, hospital or other
- Collaborators
- Nicklaus Children's Hospital f/k/a Miami Children's Hospital, Cornelia T. Bailey Foundation
- Study start
- 17 Nov 2020 (actual)
- Primary completion
- 31 Dec 2022 (actual)
- Study completion
- 31 Dec 2022 (actual)
- First posted
- 9 Jul 2021 (actual)
- 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 study is a prospective, non-randomized observational study. Freshly isolated tumor cells will be tested for chemosensitivity to the standard of care drugs as single agents and in combinations using state-of-the-art viability assay designed for ex-vivo high-throughput drug sensitivity testing (DST). In addition, the genetic profile of the tumor will be obtained from the medical records and correlated with drug response.
Detailed description
The excised tumors or a biopsy will be interrogated for sensitivity or resistance to FDA-approved and/or available investigational agents. In addition, normal samples (blood or buccal swab) will be collected for genetic analysis of germline mutations and cancer predisposition markers. The timeframe between the sample acquisition and ex vivo DST results return will be approximately 5-10 working days. All drugs tested in the DST assay will be assigned a hybrid score reflecting the tumor's sensitivity and drug toxicity.
This is an observational study and not a treatment protocol. It will assess how ex vivo drug testing and mutation profiling may predict clinical outcomes (response, survival, or relapse). The treating physician will decide which of the standard treatment options is appropriate independent of the DST results. The results of DST will not be available to the treating physician at the time of decision on the treatment regimen. DST will include all drugs from the standard treatment regimens available for all types of sarcomas
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with newly diagnosed as well as relapsed/refractory sarcomas. | – | –The investigators intend to enroll newly diagnosed or refractory/relapsed pediatric patients with all types of sarcomas where tumor tissue would be available for ex vivo drug screening and genomic profiling. This observational study will assess how ex vivo drug testing and mutation profiling may predict clinical outcomes (response, survival, or relapse). The treating physician will decide which of the standard treatment options is appropriate independent of the DST results. The results of DST will not be available to the treating physician at the time of decision on the treatment regimen. DST will include all drugs from the standard treatment regimens available for all types of sarcomas |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- Patients with newly diagnosed as well as relapsed/refractory sarcomas.
Key inclusion criteria (6 listed)
- Patients aged 21 years or younger at the time of enrollment on this study of any gender, race, or ethnicity.
- Subjects with suspected or confirmed diagnosis of all types of sarcomas.
- Subjects who are scheduled for or have recently had biopsy or tumor excised (solid tumors) or bone marrow aspirate (blood cancers).
- Subjects are willing to have a blood draw or buccal swab done for the purposes of genetic testing.
- Subjects or their parents or legal guardians willing to sign informed consent.
Key exclusion criteria (3 listed)
- Subjects who do not have malignant tissue available and accessible.
- The amount of excised malignant tissue is not sufficient for ex vivo drug testing and/or genetic profiling.
- Patients with other types of tumors and tumors that have a high (>90%) cure rate with safe standard therapy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To correlate results of drug sensitivity and mutation profiles with clinical outcomes in response to therapy.
Time frame: Up to 4 years
Response to therapy (RTT): an event of achieving "partial response" or "complete response" during the study period, based on the best response of the corresponding enrolled patient.
To correlate results of drug sensitivity and mutation profiles with clinical outcomes in progression-free survival.
Time frame: Up to 4 years
Progression-free survival (PFS): a composite end point defined as the censored event time from enrollment to either disease relapse or mortality. This will be evaluated retrospectively at the end of the study. The investigators will use the two-sample long-rank test to assess the hazard ratio of PFS events between the two groups classified as drug sensitive and insensitive by the DST.The study will enroll 15 patients and it is assumed 50% of these subjects will be classified as drug sensitive based on the DST at the threshold value of 10.
2 secondary outcomes
To assess the predictive value of personalized approach in predicting RTT.
Time frame: Up to 4 years
To assess the predictive value of personalized approach in predicting PFS.
Time frame: Up to 4 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 14 Feb 2023.
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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.
Diana Azzam, PhD principal investigator
Florida International University
Daria Salyakina, PhD principal investigator
Nicklaus Children's Hospital
Maggie Fader, MD principal investigator
Nicklaus Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Ewing Sarcoma and Osteosarcoma, run by Florida International University 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 6 inclusion and 3 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 14 Feb 2023. 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.
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?
To correlate results of drug sensitivity and mutation profiles with clinical outcomes in response to therapy. (time frame: Up to 4 years). 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 14 Feb 2023.