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Study of Intralesional Injection of M4N in Patients With Refractory Malignant Tumors of the Head and Neck

This study has been completed.
Duke University
Medical University of South Carolina
Information provided by (Responsible Party):
Erimos Pharmaceuticals Identifier:
First received: April 3, 2003
Last updated: February 20, 2016
Last verified: January 2006
The purpose of this study will be to determine the safety and tolerability of intratumoral M4N. Patients suffering from cancer of the head and neck that is recurrent after primary treatment with surgery, radiation therapy, and/or chemotherapy may be eligible. The design is a Phase 1 dose escalation study of M4N administered intratumorally once weekly, initially for three weeks. Dose will be escalated on the starting schedule to a target of 20 mg/cm3 tumor volume and then, new patient cohorts will have their schedule extended to weekly M4N for 4 weeks. Dose escalation will continue, assuming tolerability, so that cohorts will be treated for 6 weeks, and finally, 8 weeks.

Condition Intervention Phase
Head and Neck Neoplasms
Drug: M4N
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Open-Label Phase 1 Dose Escalation Study of Intralesional Injection of M4N in Patients With Refractory Malignant Tumors of the Head and Neck

Resource links provided by NLM:

Further study details as provided by Erimos Pharmaceuticals:

Enrollment: 9
Study Start Date: January 2003
Study Completion Date: December 2003
Primary Completion Date: December 2003 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Intratumoral M4N
The initial dose was 5 mg/cm3 of tumor volume on Days 1, 8, and 15. Dose escalation in cohorts on this schedule took place up to 20 mg/cm3 tumor volume. The dose per lesion was based upon the volume of tumor, and the total dose did not exceed 1197 mg M4N/m2 body surface area.
Drug: M4N
Dose escalation study of M4N administered intratumorally on days 1, 8 and 15. Dose escalated in cohorts on three weeks schedule to a target of 20 mg/cm3 tumor volume.
Other Name: Tetra-O-Methyl Nordihydroguaiaretic Acid or EM-1421

Detailed Description:
Study data collected will include pre-study demographics and measurements, treatment compliance, efficacy determined by tumor measurements, M4N levels in blood for pharmacokinetics, serum and biochemical determinations, hematology determinations, and patient vital signs.

Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male and female patients ≥ 18 years of age.
  • Patients with documented histologic evidence of head and neck cancer with clinically measurable disease. Head and neck cancer could have been recurrent after primary treatment with surgery, radiation therapy and/or chemotherapy.
  • Measurable tumor by direct inspection, photography, or by imaging (CT scan or MRI).
  • Life expectancy of at least 3 months in the Investigators' opinion.
  • Negative pregnancy test if in women of childbearing potential within 1 week of starting therapy.
  • Patients had provided written informed consent to participate in study.
  • ECOG Performance Status of 0, 1, or 2.
  • Absolute neutrophil ≥ 1500/uL, hemoglobin ≥ 8 gm%, platelets ≥ 50,000/uL,
  • ALT/AST ≤ 3 x ULN (upper limit of the normal range), bilirubin ≤ 1.5 x ULN and creatinine ≤ 1.5 x ULN, PT and PTT within normal limits.

Exclusion Criteria:

  • Women who were pregnant or nursing.
  • Women of child bearing potential who were unwilling to use an adequate method of contraception during the course of the study.
  • Treatment with prior investigational agent within 30 days of entering the study.
  • Patients who are unable to comply with the study requirements.
  • Patients with known sensitivity to any of the study medication components.
  • Patients not consenting to photography.
  • Patients with tumors that have enveloped the carotid artery or whose anatomy had been distorted such that in the Investigator's opinion present high-risk of perforation or compromise during injection.
  • Prior chemotherapy, radiation therapy, or surgery for the primary carcinoma within 30 days of dosing and/or had not recovered from prior therapy toxicities.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00057512

United States, South Carolina
Charleston, South Carolina, United States, 29475
Sponsors and Collaborators
Erimos Pharmaceuticals
Duke University
Medical University of South Carolina
  More Information

Responsible Party: Erimos Pharmaceuticals Identifier: NCT00057512     History of Changes
Other Study ID Numbers: M4N001 
Study First Received: April 3, 2003
Last Updated: February 20, 2016
Health Authority: United States: Food and Drug Administration
Individual Participant Data  
Plan to Share IPD: No

Additional relevant MeSH terms:
Head and Neck Neoplasms
Neoplasms by Site
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Lipoxygenase Inhibitors
Enzyme Inhibitors
Cyclooxygenase Inhibitors
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Sensory System Agents
Peripheral Nervous System Agents
Anti-Inflammatory Agents
Antirheumatic Agents processed this record on October 25, 2016