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

NCT01902381 | Terminated Phase 2 Results FDA Drug

• 4 other identifiers: IRB00024007NCI-2013-01312P30CA012197CCCWFU 29113
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

Previously Treated Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (1)

• 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)

  Response rate (RR), defined as the combined of complete remission (CR), marrow CR, partial remission (PR), or stable disease (SD), as described by Cheson, et al. (2006). The number of patients achieving RR will be presented. Complete remission - Bone marrow: ≤ 5% myeloblasts with normal maturation of all cell lines; Marrow CR - Bone marrow: ≤ 5% myeloblasts and decrease by ≥ 50% over pretreatment Partial remission - All CR criteria if abnormal before treatment except: Bone marrow blasts decreased by ≥ 50% over pretreatment but still \> 5%, cellularity and morphology not relevant Stable disease - Failure to achieve at least PR, but no evidence of progression for \> 8 wks

  Up to 5 years

Secondary Outcomes (5)

• 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

  During treatment, a median of 4 months with a maximum of 40 months

• Progression-free Survival (PFS)

  6 and 12 months post treatment start

• Overall Survival (OS)

  6 and 12 months post treatment start

• Number of Patients Who Achieve a Reduction in Blood Transfusion Requirements

  Up to 5 years

• Number of Patients Who Achieve Hematologic Improvement (HI), as Defined by Cheson, et al. (2006)

  Up to 5 years

Study Arms (1)

Treatment (6, 8-bis(benzylthio) octanoic acid)

EXPERIMENTAL

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.

Drug: 6,8-bis(benzylthio)octanoic acid

Interventions

6,8-bis(benzylthio)octanoic acid DRUG

Given IV

Also known as: alpha-lipoic acid analogue CPI-613, CPI-613

Treatment (6, 8-bis(benzylthio) octanoic acid)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Wake Forest University Health Sciences: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

Comprehensive Cancer Center of Wake Forest University

Winston-Salem, North Carolina, 27157, United States

Location

MeSH Terms

Interventions

devimistat

Results Point of Contact

• Title: Principal Investigator
• Organization: Wake Forest Baptist Comprehensive Cancer Center

bpowell@wakehealth.edu

336-713-5440

Study Officials

• Timothy Pardee

  Wake Forest University Health Sciences

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 15, 2013
• First Posted: July 18, 2013
• Study Start: August 1, 2013
• Primary Completion: November 26, 2018
• Study Completion: November 26, 2018
• Last Updated: October 17, 2024
• Results First Posted: October 17, 2024

Record last verified: 2024-10

Locations

US (1)