Safety and Efficacy Study in Recurrent Grade IV Glioma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02704858
Recruitment Status : Recruiting
First Posted : March 10, 2016
Last Update Posted : August 28, 2018
ClinDatrix, Inc.
Information provided by (Responsible Party):
Neonc Technologies, Inc.

Brief Summary:
NEO100-01 is a Phase 1/2A study of the monoterpene, perillyl alcohol (NEO100)in patients with recurrent glioblastoma. NEO100 is delivered four times a day by intranasal administration using a nebulizer and nasal mask for up to 6 months. Four escalating doses will be evaluated for tolerability, and the maximum tolerated dose will be extended in the 2A phase for a total of 25 patients. There is no concurrent control. This is the first nasal administration in the US, after prior oral studies with perillyl alcohol proved ineffective. Nasal administration of perillyl alcohol is on-going in Brazil

Condition or disease Intervention/treatment Phase
Glioblastoma Multiforme Drug: Perillyl alcohol Phase 1 Phase 2

Detailed Description:
Perillyl alcohol has previously been tested in 15 clinical studies in > 600 subjects This includes 13 studies in 255 subjects using oral administration sponsored by the National Cancer Institute and two studies in > 350 subjects using intranasal administration in Brazil. NEO100 is a highly purified (>99%) form of perillyl alcohol. Studies in Brazil suggest improved survival for patients with recurrent glioblastoma. Doses of 96 mg qid, 144mg qid, 192mg qid, and 288 mg qid administered intranasally to patients with recurrent GBM for up to 6 months, disease progression or death. From 3 to 6 patients will be evaluated after first cycle (28 days) until MTD is reached. MRI with gadolinium will be at base line, and at the beginning of even cycles. A total of 25 patients will be treated at the MTD. PK studies will be conducted during Phase 1 at first dosing, and after first dose of 3rd cycle.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 43 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Phase 1/2A Dose Escalation Study of Safety and Efficacy of NEO100 in Recurrent Grade IV Glioma
Study Start Date : April 2016
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : October 2020

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: NEO100
Intranasal delivery of NEO100 (perillyl alcohol) four times a day, escalation up to four different doses to determine maximum tolerated dose. Followed by treatment of total of 25 patients at maximum tolerated dose
Drug: Perillyl alcohol
Intranasal administration
Other Name: NEO100

Primary Outcome Measures :
  1. Phase 1 Primary Outcome: Number of Participants with Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment [ Time Frame: Up to 6 months ]
  2. Phase 2 Primary Outcome: Number of Participants Who Are Alive Each Month For 6 Months [ Time Frame: Up to 6 months ]
  3. Phase 2 Primary Outcome: Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related To Treatment [ Time Frame: 6 months ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Radiographically-confirmed progression of, or recurrent, primary or secondary Grade IV glioma, and must be on a stable or decreasing dose of steroid for at least five days prior to the date of informed consent.
  • Must have failed previous radiation treatment or combined treatment with temozolomide and radiation.
  • If progression of disease occurs within three months of conformal radiation, it must be outside of the radiation field or proven by biopsy/resection.
  • Must have an ECOG performance status of 0 - 2, or KPS ≥ 60.
  • Must have an expected survival of at least three months.
  • Must be willing to provide blood samples for pharmacokinetic study
  • Must have adequate organ and marrow function
  • Female patients of child-bearing potential and male patients must agree to use adequate contraception
  • Must have the ability to understand, and the willingness to sign, a written informed consent.

Exclusion Criteria:

  • The size of the Grade IV glioma tumor is multi-focal and > 30mm in size, as assessed at the baseline (pre-study) MRI evaluation.
  • Patient has completed chemo-radiation within the last three months, unless new contrast enhancement is outside of radiation field, or there is tissue proven recurrence or progression.
  • Patient has had surgery within seven days prior to the date of informed consent.
  • Patient has had chemotherapy within 28 days prior to first administration of study drug.
  • Patient has not recovered from adverse events due to chemotherapy, immunotherapy, or radiation therapy administered more than 28 days prior to first administration of study drug.
  • Patient has had prior treatment with bevacizumab, a chemotherapy wafer implant (Gliadel), or any other FDA- approved chemotherapy except temozolomide.
  • Patient has had more than one recurrence or progression of their tumors.
  • Patient is receiving any other investigational agents.
  • Patient has a history of allergic reactions attributed to perillyl alcohol.
  • Patient has uncontrolled intercurrent illness
  • Patient has a history of new diagnosis or treatment of cancer other than malignant glioma within five years prior to start of the study, except for basal cell carcinoma or squamous cell carcinoma.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02704858

Contact: Laurie Mellott 949-428-6650
Contact: Audrey DeRamos, MD 949-428-6660

United States, California
University of Southern California Recruiting
Los Angeles, California, United States, 90033
Contact: Aida Lozada    323-865-3980   
Principal Investigator: Naveed Wagle, MD         
United States, Florida
Cleveland Clinic Florida Not yet recruiting
Weston, Florida, United States, 33331
Contact: Abel Rodriguez    954-487-2258   
Principal Investigator: Bruno Bastos, MD         
United States, Ohio
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44333
Contact: Teresa Allison    216-444-6459   
Contact: Theresa Naska    216-444-8923   
Principal Investigator: David Peereboom, MD         
United States, Washington
University of Washington Seattle Cancer Care Alliance Recruiting
Seattle, Washington, United States, 98109
Contact: Ana Valeria Martin    206-221-1538   
Contact: Bryan Kim    206-543-4069   
Principal Investigator: Lynne Taylor, MD         
United States, Wisconsin
University of Wisconsin Hospital and Clinic Recruiting
Madison, Wisconsin, United States, 53792
Contact: Diana Trask    608-263-9528   
Contact: Belinda BuehlSoppe    608-265-9958   
Principal Investigator: Steve Howard, MD         
Sponsors and Collaborators
Neonc Technologies, Inc.
ClinDatrix, Inc.
Study Chair: Tom Chen, MD, PhD NeOnc Technologies
Study Director: Vincent F Simmon, PhD NeOnc Technologies
Study Director: Patrick Walters NeOnc Technologies

Responsible Party: Neonc Technologies, Inc. Identifier: NCT02704858     History of Changes
Other Study ID Numbers: NEO100-01
First Posted: March 10, 2016    Key Record Dates
Last Update Posted: August 28, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Neonc Technologies, Inc.:
recurrent glioblastoma
recurrent GBM

Additional relevant MeSH terms:
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Perillyl alcohol
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action