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CPI-613 in Treating Patients With Myelodysplastic Syndromes Who Failed Previous Therapy

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ClinicalTrials.gov Identifier: NCT01902381
Recruitment Status : Suspended (Lack of accrual)
First Posted : July 18, 2013
Last Update Posted : July 23, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Wake Forest University Health Sciences

Tracking Information
First Submitted Date  ICMJE July 15, 2013
First Posted Date  ICMJE July 18, 2013
Last Update Posted Date July 23, 2019
Actual Study Start Date  ICMJE August 2013
Estimated Primary Completion Date September 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 15, 2013)
Response rate (RR), defined as the combined rate of complete remission (CR), marrow CR, partial remission (PR), or stable disease (SD), as described by Cheson, et al. (2006) [ Time Frame: Up to 5 years ]
Proportion of RR (along with a 95% confidence interval) of patients who respond will be presented.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01902381 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 15, 2013)
  • Safety profile of CPI-613, based on evaluation of symptoms, vital signs, ECOG performance status and survival, clinical chemistry, hematology, and coagulation, assessed by National Cancer Institute Common Terminology Criteria for Adverse Events v 4.0 [ Time Frame: Up to 1 month post-treatment ]
    Toxicities will be examined by looking at each toxicity identified by grade.
  • Progression-free survival (PFS) [ Time Frame: Up to 5 years ]
    Survival curves for PFS using Kaplan-Meier techniques will be estimated. In addition, the 6 month and 1-year PFS rates for these participants will be estimated.
  • Overall survival (OS) [ Time Frame: Up to 5 years ]
    Survival curves for OS using Kaplan-Meier techniques will be estimated. In addition, the 6 month and 1-year OS rates for these participants will be estimated.
  • Number of patients who achieve a reduction in blood transfusion requirements [ Time Frame: Up to 5 years ]
  • Number of patients who achieve hematologic improvement (HI), as defined by Cheson, et al. (2006) [ Time Frame: Up to 5 years ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE CPI-613 in Treating Patients With Myelodysplastic Syndromes Who Failed Previous Therapy
Official Title  ICMJE A Pilot Study of CPI-613 in Patients With Myelodysplastic Syndrome Who Have Failed Previous Therapy
Brief Summary This pilot clinical trial studies 6, 8-bis (benzylthio) octanoic acid (CPI-613) in treating patients with myelodysplastic syndromes who failed previous therapy. Sometimes when chemotherapy or biological therapy is given, it does not stop the growth of tumor cells. The tumor is said to be resistant to treatment. 6, 8-bis (benzylthio) octanoic acid may interfere with the growth of tumor cells and may be an effective treatment for myelodysplastic syndromes that did not respond to previous therapy.
Detailed Description

PRIMARY OBJECTIVES:

I. To evaluate the safety and anti-cancer activities of CPI-613 in myelodysplastic syndrome (MDS) patients who have failed previous agents (such as decitabine [Dacogen], azacitidine [Vidaza], growth factors or lenalidomide).

OUTLINE:

Patients receive 6, 8-bis (benzylthio) octanoic acid intravenously (IV) over 2 hours on days 1 and 4 of weeks 1-3. Courses repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up for 5 years.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Previously Treated Myelodysplastic Syndromes
Intervention  ICMJE Drug: 6,8-bis(benzylthio)octanoic acid
Given IV
Other Names:
  • alpha-lipoic acid analogue CPI-613
  • CPI-613
Study Arms  ICMJE Experimental: Treatment (6, 8-bis(benzylthio) octanoic acid)
Patients receive treatment 6,8-bis(benzylthio)octanoic acid IV over 2 hours on days 1 and 4 of weeks 1-3. Courses repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Intervention: Drug: 6,8-bis(benzylthio)octanoic acid
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Suspended
Estimated Enrollment  ICMJE
 (submitted: July 15, 2013)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 2019
Estimated Primary Completion Date September 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Histologically or cytologically documented MDS of any risk group that has failed previous therapy (therapy failure is defined as patients who have been sufficiently treated with previous agents without response in the opinion of the treating physician, or whose disease has progressed or relapsed while on a hypomethylating agent)
  • Eastern Cooperative Oncology Group (ECOG) performance status of =< 3
  • Expected survival > 2 months
  • Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) must use accepted contraceptive methods (abstinence, intrauterine device [IUD], oral contraceptive or double barrier device) during the study, and must have a negative serum or urine pregnancy test within 1 week prior to treatment initiation
  • Fertile men must practice effective contraceptive methods during the study, unless documentation of infertility exists
  • Patients must have fully recovered from the acute, non-hematological, non-infectious toxicities of any prior treatment with cytotoxic drugs, radiotherapy or other anti-cancer modalities; patients with persisting, non-hematologic, non-infectious toxicities from prior treatment =< grade 2 are eligible, but must be documented as such
  • Aspartate aminotransferase (AST/serum glutamic oxaloacetic transaminase [SGOT]) =< 3 x upper normal limit (UNL)
  • Alanine aminotransferase (ALT)/serum glutamate pyruvate transaminase (SGPT) =< 3 x UNL (=< 5x ULN if liver metastases present)
  • Bilirubin =< 1.5 x UNL
  • Serum creatinine =< 1.5 mg/dL or 133 umol/L
  • International normalized ratio (or INR) must be < 1.5
  • Albumin >= 2.0 g/dL or >= 20 g/L
  • Mentally competent, ability to understand and willingness to sign an Institutional Review Board (IRB)-approved written informed consent form
  • Have access via central line (e.g., portacath)

Exclusion Criteria:

  • Serious medical illness, such as significant cardiac disease (e.g. symptomatic congestive heart failure, unstable angina pectoris, coronary artery disease, myocardial infarction within the past 3 months, uncontrolled cardiac arrhythmia, pericardial disease or New York Heart Association class III or IV), or severe debilitating pulmonary disease, that would potentially increase patients' risk for toxicity
  • Patients with active central nervous system (CNS) or epidural tumor
  • Any active uncontrolled bleeding or bleeding diathesis (e.g., active peptic ulcer disease)
  • Any condition or abnormality which may, in the opinion of the investigator, compromise his or her safety
  • Pregnant women, or women of child-bearing potential not using reliable means of contraception
  • Fertile men unwilling to practice contraceptive methods during the study period
  • Lactating females
  • Life expectancy less than 2 months
  • Unwilling or unable to follow protocol requirements
  • A history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, family history of long QT syndrome, etc.)
  • Evidence of active infection or serious infection within the past month
  • Requirement for immediate palliative treatment of any kind including surgery
  • Prior illicit drug addiction
  • Patients with large and recurrent pleural or peritoneal effusions requiring frequent drainage (e.g. weekly)
  • Patients with any amount of clinically significant pericardial effusion
  • Patients with known human immunodeficiency virus (HIV) infection; (Note: patients with known HIV infection are excluded because patients with an immune deficiency are at increased risk of lethal infections when treated with marrow-suppressive therapy, and because there may be unknown or dangerous drug interactions between CPI-613 and anti-retroviral agents used to treat HIV infections)
  • Patients who have received radiotherapy, surgery, treatment with cytotoxic agents (except CPI-613), treatment with biologic agents, immunotherapy, or any other anti-cancer therapy of any kind, or any other standard or investigational treatment for their cancer, or any other investigational agent for any indication, within the past 2 weeks prior to initiation of CPI-613 treatment
  • Patients that have received a chemotherapy regimen with stem cell support in the previous 6 months
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01902381
Other Study ID Numbers  ICMJE IRB00024007
NCI-2013-01312 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
P30CA012197 ( U.S. NIH Grant/Contract )
CCCWFU 29113 ( Other Identifier: Wake Forest University Health Sciences )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Wake Forest University Health Sciences
Study Sponsor  ICMJE Wake Forest University Health Sciences
Collaborators  ICMJE National Cancer Institute (NCI)
Investigators  ICMJE
Principal Investigator: Timothy Pardee Wake Forest University Health Sciences
PRS Account Wake Forest University Health Sciences
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP