CompletedPhase 1

Pbi-shRNA™ EWS/FLI1 Type 1 LPX in Subjects With Advanced Ewing's Sarcoma

United States7 participantsStarted 2016-10

Plain-language summary

The purpose of this study is to determine the safety and the maximum tolerated dose of of pbi-shRNA™ EWS/FLI1 Type 1 lipoplex in patients with advanced Ewing's sarcoma.

Who can participate

Age range8 Years

SexALL

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Inclusion criteria

✓. Histologically confirmed Ewing's Sarcoma Family of Tumors (ESFT).
✓. Age ≥8 years.
✓. Evidence of EWS translocation fusion by FISH or RT-PCR or NGS.
✓. Evidence of Type 1 fusion by molecular diagnostics.
✓. Refractory or intolerant to standard of care. Subjects must have failed surgery (if resectable), radiation (if no function-preserving surgical approach at primary site, unresectable primary following induction chemotherapy, residual microscopic or gross disease after surgery or inadequate margins), and the following chemotherapy agents: doxorubicin, vincristine, cyclophosphamide, ifosfamide, etoposide.
✓. ECOG performance status (PS) = 0-2, or Karnofsky PS ≥60% or Lansky PS ≥60%.
✓. Normal organ and marrow function as defined below:
✓. Normal pulmonary function as defined as FEV1/FVC greater than 70% in adults or greater than 80% in individuals between 8 and 18 years of age.

Exclusion criteria

✕. Anti-cancer chemotherapy, biologic therapy or immunotherapy within 3 weeks or radiation therapy within 2 weeks of first infusion.
✕. Known history of other malignancy unless having undergone curative intent therapy without evidence of that disease for ≥ 3 years except cutaneous squamous cell and basal cell skin cancer, superficial bladder cancer, in situ cervical cancer or other in situ cancers are allowed if definitively resected.
✕. Patients with PET avid disease only will be excluded.
✕. Brain metastases unless treated with curative intent (gamma knife or surgical resection) and without evidence of progression for ≥ 2 months.
✕. History of or current evidence of thrombosis.
✕. History of or current evidence of any condition (including medical, psychiatric or substance abuse disorder), therapy, or laboratory abnormality that might confound the results of the study, interfere with the subject's participation for the full duration of the study, or is not in the best interest of the subject to participate, in the opinion of the Investigator.
✕. Known HIV or chronic Hepatitis B or C infection.
✕. Have signs and symptoms consistent with an active infection.

What they're measuring

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

Timeframe: From first dose and up to 60 days following the last treatment, up to approximately 13 months.

Determine the maximum tolerated dose of intravenous administration of lipoplex

Timeframe: From first subject, first dose and after an additional 6 subjects have been treated at the MTD, up to 4 weeks.

Trial details

NCT IDNCT02736565

SponsorGradalis, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2019-08-12

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