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Advanced CancerPhase 1STMN1-LPNCT01505153

Phase I Intratumoral Pbi-shRNA STMN1 LP in Advanced and/or Metastatic Cancer

A Phase 1 study testing pbi-shRNA STMN1 LP in people with Advanced Cancer and Metastatic Cancer, run by Gradalis, Inc. at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a Phase I safety trial of bifunctional shRNA-STMN1 (pbi-shRNA™STMN1) BIV (bilamellar invaginated vesicle) lipoplex (LP), pbi-shRNA™ STMN1 LP administered by a single intratumoral (IT) injection. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Advanced Cancer, Metastatic Cancer, Solid Tumors
What is being tested?
pbi-shRNA STMN1 LP
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
1 siteUnited States
Study dates
Feb 2012to 13 Apr 2017
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Histologically confirmed advanced and/or metastatic cancer, and, if limited to a single lesion, not considered a candidate for curative surgery or radiation therapy).
  • Biopsy accessible lesion.
  • Per cohort dose/volume, the volume of the lesion to be injected must be 3x volume of the injectate.
  • Subjects that have completed all acceptable therapies with curative potential that are the current standard of care for their respective diseases.
  • Recovered from all toxicities (≤ Grade 1) related to prior therapies except for alopecia.
  • 1 measurable or evaluable lesion; ≥ 1.8 cm diameter for cohort 1 (see Table 10); injection and biopsy accessible.
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  • Age ≥18 years.
  • ECOG performance status (PS) 0-2.
  • Organ and marrow function as defined below:
  • Absolute granulocyte count ≥ 1,500/mm^3 Platelets ≥ 100,000/mm^3 Total bilirubin ≤ 1.5x institutional ULN Creatinine ≤ 2.0 mg/dL
  • Ability to understand and the willingness to sign a written informed consent document including permission for pre- and Days 1 and 2 post- injection biopsy and Day 8 injected lesion excision.
  • Negative pregnancy test.

Who cannot join

  • Surgery involving general anesthesia, chemotherapy, radiotherapy, or immunotherapy within 3 weeks prior to entering the study.
  • Patient must not have received any other investigational agents within 4 weeks prior to study entry.
  • Patients with known brain metastases unless treated with whole brain radiation and stable for >/= 2 months or treated with stereotactic radiotherapy only and stable for >/=1 month.
  • Short term (<30 days) concurrent systemic steroids ≤0.125 mg/kg prednisone per day (maximum 7.5 mg/day) and bronchodilators (inhaled steroids) are permitted; other steroid regimens and/or immunosuppressives are excluded.
  • Prior malignancy (excluding nonmelanoma carcinomas of the skin) unless in remission for >/= 2 years.
  • Kaposi's Sarcoma.
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  • Uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Patients who are pregnant or nursing.
  • Patients with known HIV.
  • Patients with chronic Hepatitis B and C infection.
  • Patients with uncontrolled diseases.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Mary Crowley Cancer Research Centers

    Dallas, Texas, 75230

Other trials in United States

About this study

Official title: Phase I Trial of Intratumoral Bi-functional shRNA Stathmin 1-knockdown Lipoplex in Patients With Advanced and/or Metastatic Cancer

This is a Phase I safety trial of bifunctional shRNA-STMN1 (pbi-shRNA™STMN1) BIV (bilamellar invaginated vesicle) lipoplex (LP), pbi-shRNA™ STMN1 LP administered by a single intratumoral (IT) injection. Patients with superficially accessible advanced cancer following prior therapies will be entered into the study following a modified dose escalation design based on the demonstrated safety of our previous clinical experience (BB-IND 13744) with the same liposome and vector DNA backbone expressing a different transgene (of which doses up to 7 mg DNA IV/single dose have been administered). Patients will accrue in 4-patient escalation cohorts using a modified Fibronacci escalation schema (100%-50%-33%-33%) at a starting intratumoral dose of 0.010 mg/kg of DNA through a dose of 0.053 mg/kg DNA intratumoral / single dose. Should a single, but not more than two (2), ≥ Grade 3 Dose Limiting Toxicity (DLT) occur in any cohort, following mandated review (see below) an additional two (2) patients will be accrued at that dose (total of six). If more than one ≥ Grade 3 toxicity occurs in any cohort, the preceding dose cohort will be expanded to six (from four) and if < 2/6 patients experience ≥ Grade 3 toxicity, that dose will be the Phase II recommended dose. Should no ≥ Grade 3 toxicity occur in any cohort (other than Grade 3 local injection site reaction), an additional two (2) patients will be treated at 0.053 mg/kg DNA intratumoral / single dose.

Being tested
pbi-shRNA STMN1 LP
Keywords
Advanced cancer, Metastatic cancer, solid tumors, Melanoma
Medical subject headings
Neoplasm Metastasis, Melanoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • pbi-shRNA STMN1 LPBiological

    This is a Phase I safety trial of bifunctional shRNA-STMN1 (pbi-shRNA™STMN1) BIV (bilamellar invaginated vesicle) lipoplex (LP), pbi-shRNA™ STMN1 LP administered by a single intratumoral (IT) injection. Patients will accrue in 4-patient escalation cohorts using a modified Fibronacci escalation schema at a starting intratumoral dose of 0.010 mg/kg of DNA through a dose of 0.053 mg/kg DNA intratumoral / single dose.

Study groups in NCT01505153
Study groupRoleWhat they receive
pbi-shRNA STMN1 LPExperimentalpbi-shRNA™ STMN1 LP administered by a single intratumoral (IT) injection.
Receives: Biological: pbi-shRNA STMN1 LP

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To determine the safety of intratumoral administration of pbi-shRNA™ STMN1 LP

    To determine the safety of intratumoral administration of pbi-shRNA™ STMN1 LP in patients with superficial advanced and/or metastatic cancer who have no acceptable form of standard therapy.

    Measured: 1 month

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Jan 2012 (estimated)
  2. Study startFeb 2012
  3. Primary completionMar 2017
  4. Study completion13 Apr 2017

Resources

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
20 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01505153
Sponsor's protocol ID
CL-PTL 103
Acronym
STMN1-LP
Status last verified
Feb 2018
First submitted
4 Jan 2012
Last updated
22 Feb 2018
Results posted
No
Data monitoring committee
Yes

Publications

  • Phadke AP, Jay CM, Wang Z, Chen S, Liu S, Haddock C, Kumar P, Pappen BO, Rao DD, Templeton NS, Daniels EQ, Webb C, Monsma D, Scott S, Dylewski D, Frieboes HB, Brunicardi FC, Senzer N, Maples PB, Nemunaitis J, Tong AW. In vivo safety and antitumor efficacy of bifunctional small hairpin RNAs specific for the human Stathmin 1 oncoprotein. DNA Cell Biol. 2011 Sep;30(9):715-26. doi: 10.1089/dna.2011.1240. Epub 2011 May 25.

    PubMed 21612405

  • Rao DD, Maples PB, Senzer N, Kumar P, Wang Z, Pappen BO, Yu Y, Haddock C, Jay C, Phadke AP, Chen S, Kuhn J, Dylewski D, Scott S, Monsma D, Webb C, Tong A, Shanahan D, Nemunaitis J. Enhanced target gene knockdown by a bifunctional shRNA: a novel approach of RNA interference. Cancer Gene Ther. 2010 Nov;17(11):780-91. doi: 10.1038/cgt.2010.35. Epub 2010 Jul 2.

    PubMed 20596090

  • Rana S, Maples PB, Senzer N, Nemunaitis J. Stathmin 1: a novel therapeutic target for anticancer activity. Expert Rev Anticancer Ther. 2008 Sep;8(9):1461-70. doi: 10.1586/14737140.8.9.1461.

    PubMed 18759697

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing pbi-shRNA STMN1 LP in people with Advanced Cancer and Metastatic Cancer, run by Gradalis, Inc. 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 12 inclusion and 11 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 22 Feb 2018. 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?

To determine the safety of intratumoral administration of pbi-shRNA™ STMN1 LP (time frame: 1 month). 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 NCT01505153, which also lists 3 linked publications.

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 22 Feb 2018.

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