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RNA Interference

Cohort 3 for Ewing Sarcoma

Phase 1

Waitlist Available

Research Sponsored by Gradalis, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline and quarterly thereafter until progressive disease is noted, up to approximately 12 months.

Awards & highlights

Study Summary

This trial is a phase 1 study to test the safety of a new drug for patients with Ewing's sarcoma that has progressed after treatment with standard therapies.

Eligible Conditions

Ewing's Sarcoma
Rare Diseases
Ewing Sarcoma
Ewing's Tumor
Sarcoma

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline and quarterly thereafter until progressive disease is noted, up to approximately 12 months.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline and quarterly thereafter until progressive disease is noted, up to approximately 12 months.

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline and quarterly thereafter until progressive disease is noted, up to approximately 12 months. for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Determine the maximum tolerated dose of intravenous administration of lipoplex

Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE v4.0

Secondary outcome measures

To assess ctDNA (EWSR-FLI1) levels and compare to tumor burden and disease response prior to and following pbi-shRNA™ EWS/FLI1 Type 1 lipoplex administration.

To assess disease response for different cohorts following pbi-shRNA™ EWS/FLI1 Type 1 lipoplex administration at different dose(s).

To assess the pharmacokinetics of pbi-shRNA™ EWS/FLI1 Type 1 plasmid.

Trial Design

8Treatment groups

Experimental Treatment

Group I: Cohort 6Experimental Treatment1 Intervention

0.156mg/kg pbi-shRNA™ EWS/FL/I1 Type 1 Lipoplex

Group II: Cohort 5Experimental Treatment1 Intervention

0.125mg/kg pbi-shRNA™ EWS/FL/I1 Type 1 Lipoplex

Group III: Cohort 4Experimental Treatment1 Intervention

0.10mg/kg pbi-shRNA™ EWS/FL/I1 Type 1 Lipoplex

Group IV: Cohort 3Experimental Treatment1 Intervention

0.08mg/kg pbi-shRNA™ EWS/FL/I1 Type 1 Lipoplex

Group V: Cohort 2Experimental Treatment1 Intervention

0.06mg/kg pbi-shRNA™ EWS/FL/I1 Type 1 Lipoplex

Group VI: Cohort 1Experimental Treatment1 Intervention

0.04mg/kg pbi-shRNA™ EWS/FL/I1 Type 1 Lipoplex

Group VII: Cohort 0Experimental Treatment1 Intervention

0.03mg/kg pbi-shRNA™ EWS/FL/I1 Type 1 Lipoplex

Group VIII: Cohort -1Experimental Treatment1 Intervention

0.02mg/kg pbi-shRNA™ EWS/FL/I1 Type 1 Lipoplex

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

pbi-shRNA™ EWS/FLI1 Type 1 Lipoplex

2016

Completed Phase 1

~10

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Gradalis, Inc.Lead Sponsor

15 Previous Clinical Trials

478 Total Patients Enrolled

2 Trials studying Ewing Sarcoma

32 Patients Enrolled for Ewing Sarcoma

John Nemunaitis, MDStudy DirectorGradalis, Inc.

11 Previous Clinical Trials

485 Total Patients Enrolled

2 Trials studying Ewing Sarcoma

32 Patients Enrolled for Ewing Sarcoma

Luisa Manning, MDStudy DirectorGradalis, Inc.

7 Previous Clinical Trials

173 Total Patients Enrolled

2 Trials studying Ewing Sarcoma

32 Patients Enrolled for Ewing Sarcoma

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is PBI-shRNA™ EWS/FLI1 Type 1 LPX safe to be administered to humans?

"As this is a Phase 1 clinical trial, there are only preliminary data supporting the safety and efficacy of pbi-shRNA™ EWS/FLI1 Type 1 LPX; thus it attained a score of 1."

Answered by AI

Are there any openings remaining in this clinical trial at the present time?

"According to clinicaltrials.gov, the recruitment period for this trial has concluded as it was last updated on March 14th 2022. Initially posted on October 1st 2016, no further participants are being sought out at present time; however, there are 2768 other trials actively enrolling new members currently."

Answered by AI

Recent research and studies

Pharmaceutical ResearchJournal

RNA Interference and Cancer Therapy

Wang, Zhaohui, Donald D. Rao, Neil Senzer, and John Nemunaitis. 2011. “RNA Interference and Cancer Therapy”. Pharmaceutical Research. Springer Science and Business Media LLC. doi:10.1007/s11095-011-0604-5.

Molecular TherapyJournal

Phase 1 Trial of Bi-shrna STMN1 BIV in Refractory Cancer

Barve, Minal, Zhaohui Wang, Padmasini Kumar, Christopher M Jay, Xiuquan Luo, Cynthia Bedell, Robert G Mennel, et al.. 2015. “Phase 1 Trial of Bi-shrna STMN1 BIV in Refractory Cancer”. Molecular Therapy. Elsevier BV. doi:10.1038/mt.2015.14.

Molecular TherapyJournal

Phase I Trial of “bi-shrnaifurin/gmcsf Dna/autologous Tumor Cell

Senzer, Neil, Minal Barve, Joseph Kuhn, Anton Melnyk, Peter Beitsch, Martin Lazar, Samuel Lifshitz, et al.. 2012. “Phase I Trial of “bi-shrnaifurin/gmcsf Dna/autologous Tumor Cell” Vaccine (FANG) in Advanced Cancer”. Molecular Therapy. Elsevier BV. doi:10.1038/mt.2011.269.

Molecular TherapyJournal