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Mithramycin for Lung, Esophagus, and Other Chest Cancers

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. Read our disclaimer for details. Identifier: NCT01624090
Recruitment Status : Completed
First Posted : June 20, 2012
Last Update Posted : September 5, 2019
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

Brief Summary:


- Mithramycin is a drug that was first tested as a cancer therapy in the 1960s. It acted against some forms of cancer, but was never accepted as a treatment. Research suggests that it may be useful against some cancers of the chest, such as lung and esophageal cancer or mesothelioma. Researchers want to see if mithramycin can be used to treat these types of cancer.


- To see if mithramycin is safe and effective against different chest cancers.


- Individuals at least 18 years of age who have lung, esophagus, pleura, or mediastinum cancers.


  • Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Imaging studies and tumor tissue samples will be used to monitor the cancer before treatment.
  • Participants will receive mithramycin every day for 7 days, followed by 7 days without treatment. Each 14-day round of treatment is called a cycle.
  • Treatment will be monitored with frequent blood tests and imaging studies.
  • Participants will continue to take the drug for as long as the side effects are not severe and the tumor responds to treatment.

Condition or disease Intervention/treatment Phase
Lung Cancer Esophageal Cancer Mesothelioma Gastrointestinal Neoplasms Breast Cancer Drug: Mithramycin Phase 2

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Evaluation of Mithramycin, an Inhibitor of Cancer Stem Cell Signaling, in Patients With Malignancies Involving Lungs, Esophagus, Pleura, or Mediastinum
Actual Study Start Date : September 6, 2012
Actual Primary Completion Date : August 20, 2019
Actual Study Completion Date : August 20, 2019

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: 1/mithramycin
Single agent IV mithramcyin
Drug: Mithramycin
30 mcg/kg IV over 6 h once daily for 7 days, to be repeated every 21 days (one cycle) until disease progression or unacceptable toxicity

Primary Outcome Measures :
  1. Objective response rate [ Time Frame: Every 8 weeks until disease progression or unacceptable toxicity ]
    Changes in tumor size and occurrence of metastases.

Secondary Outcome Measures :
  1. Safety [ Time Frame: Until 30 days after last dose of study drug ]
    List and description of toxicities

  2. Pharmacokinetics [ Time Frame: 8 weeks (days 1, 2, 4, 7, 8 & 9 of the 1st 2 4 week cycles) ]
    Drug level in blood

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
  • Diagnosis: Patients with measurable inoperable, histologically confirmed primary lung and esophageal carcinomas, thymic neoplasms, germ cell tumors, malignant pleural mesotheliomas or chest wall sarcomas, as well as patients with gastric, colorectal or renal cancers and sarcomas metastatic to the thorax are eligible
  • Histologic confirmation of disease in the Laboratory of Pathology, CCR, NCI, NIH.
  • Disease amenable to biopsy via percutaneous approach or other minimally invasive procedures such as thoracoscopy, bronchoscopy, laparoscopy, or GI endoscopy
  • Age >18
  • ECOG status 0-2.
  • Patients must have had or refused first-line standard chemotherapy for their inoperable malignancies.
  • Patients must have had no chemotherapy, biologic therapy, or radiation therapy for their malignancy for at least 30 days prior to treatment. Patients may have received localized radiation therapy to non-target lesions provided that the radiotherapy is completed 14 days prior to commencing therapy, and the patient has recovered from any toxicity. At least 3 half-lives must have elapsed since monoclonal antibody treatment. At least six weeks must have elapsed between mitomycin C or nitrosourea treatment.
  • Patients must have adequate organ and marrow function as defined below:

    a) Hematologic and Coagulation Parameters:

    i. Peripheral ANC greater than or equal to 1500/mm^3

ii. Platelets greater than or equal to 100,000/ mm^3 (transfusion independent)

iii. Hemoglobin greater than or equal to 8 g/dL (PRBC transfusions permitted)

iv. PT/PTT within normal limits (patient may be eligible for trial if abnormality is deemed clinically insignificant and cleared for protocol therapy by Hematology Consult Service)

b) Hepatic Function

i. Bilirubin (total) < 1.5 times upper limit of normal (ULN)

ii. ALT (SGPT) less than or equal to 3.0 times ULN

iii. Albumin > 2 g/dL

c) Renal Function

i. Creatinine within normal institutional limits or creatinine clearance greater than or equal to 60 mL/min/1.73 m^2 for patients with creatinine levels above institutional normal.

ii. Normal ionized calcium, magnesium and phosphorus (can be on oral supplementation)

  • Cardiac Function: Left ventricular ejection fraction (EF) >40% by Echocardiogram, MUGA, or cardiac MR.
  • Ability of subject to understand, and be willing to sign informed consent.
  • Female and male patients (and when relevant their partners) must be willing to practice birth control (including abstinence) during and for two months after treatment, if of childbearing potential during sexual contact with a female of childbearing potential.
  • Patients must be willing to undergo 2 tumor biopsies


  • Patients with ABCB4, ABCB11, RALBP or CYP851 genotypes associated with mithramycin-mediated hepatotoxicity.
  • Clinically significant systemic illness (e.g. serious active infections or significant cardiac, pulmonary, hepatic or other organ dysfunction), that in the judgment of the PI would compromise the patient s ability to tolerate protocol therapy or significantly increase the risk of complications
  • Patients with cerebral metastases
  • Patients with any of the following pulmonary function abnormalities will be excluded: FEV, < 30% predicted; DLCO, < 30% predicted (post-bronchodilator); Oxygen saturation greater than 92% on room air. Arterial blood gas will be drawn if clinically indicated.
  • Patients with evidence of active bleeding, intratumoral hemorrhage or history of bleeding diatheses, unless specifically occurring as an isolated incident during reversible chemotherapy induced thrombocytopenia
  • Patients on therapeutic anticoagulation. Note: prophylactic anticoagulation (i.e. intralumenal heparin) for venous or arterial access devices is allowed
  • Patients who are concurrently receiving or requiring any of the following agents, which may increase the risk for mithramycin related toxicities, such as hemorrhage:

    • Thrombolytic agents
    • Aspirin or salicylate-containing products, which may increase risk of hemorrhage
    • Dextran
    • Dipyridamole
    • Sulfinpyrazone
    • Valproic acid
    • Clopidogrel
  • Lactating or pregnant females (due to risk to fetus or newborn, and lack of testing for excretion in breast milk)
  • Patients with history of HIV, HBV or HCV due to potentially increased risk of mithramycin toxicity in this population
  • Hypersensitivity to mithramycin
  • Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study

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): NCT01624090

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United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Cancer Institute (NCI)
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Principal Investigator: David S Schrump, M.D. National Cancer Institute (NCI)

Additional Information:
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Responsible Party: National Cancer Institute (NCI) Identifier: NCT01624090     History of Changes
Other Study ID Numbers: 120151
First Posted: June 20, 2012    Key Record Dates
Last Update Posted: September 5, 2019
Last Verified: September 3, 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) ):
Cancer Stem Cell
Thoracic Malignancies
Mythramycin Treatment
Lung Tumors
Metastatic Lung Tumors
Additional relevant MeSH terms:
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Gastrointestinal Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Gastrointestinal Diseases
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms, Mesothelial
Calcium-Regulating Hormones and Agents
Physiological Effects of Drugs
Antibiotics, Antineoplastic
Antineoplastic Agents
Protein Synthesis Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors