Safety and Effectiveness Study of CPI-613 to Treat Refractory or Relapsed Leukemia and Myelodysplastic Syndrome

NCT01520805 | Withdrawn Phase 2

• 1 other identifier: CL-CPI-613-022
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine whether CPI-613 is effective and safe in either patients with refractory or relapsed acute myeloid leukemia (AML) or patients with myelodysplastic syndrome (MDS) who have failed therapy with a hypomethylating agent (such as decitabine \[Vidaza\] and azacitidine \[AZA\]).

Conditions

Acute Myeloid Leukemia (AML); Myelodysplastic Syndrome (MDS)

Keywords

acute myeloid leukemia; myelodysplastic syndrome; refractory; relapsed; hypomethylating agents

Outcome Measures

Primary Outcomes (1)

• Overall Survival (OS)

  Monitored until participants passed away, for an expected average of 6 months.

Secondary Outcomes (6)

• Overall Remission Rate

  Monitored at the end of every 4-week treatment cycle during treatment with CPI-613, for an expected average of 20 weeks.

• Response Rate

  Monitored at the end of every 4-week treatment cycle during treatment with CPI-613, for an expected average of 20 weeks.

• Duration of Overall Remission

  Monitored at the end of every 4-week treatment cycle during treatment with CPI-613, for an expected average of 20 weeks.

• Progression Free Survival (PFS)

  Monitored during treatment with CPI-613 and until participants passed away, which will be an expected average of 6 months.

• Quality of Life (QOL)

  Monitored before, during and 1 week after treatment with CPI-613, for an expected average of 20 weeks.

Study Arms (1)

CPI-613

EXPERIMENTAL

CPI-613 will be intravenously infused over 2 hours, given twice weekly for 3 weeks followed by a week of rest.

Drug: CPI-613

Interventions

CPI-613 DRUG

CPI-613 drug product, provided in concentrated form at 50 mg/mL, must be diluted with D5W prior to administration. CPI-613 is to be infused intravenously (IV) via a central venous catheter. The dose of CPI-613 will be either the Maximum Tolerated Dose (MTD) or the highest No-Significant- Adverse-Effects-Dose-Level (NOAEL), as determined from the nearly completed Phase 1 dose-escalation clinical trial in patients with hematologic malignancies (i.e., Cornerstone Study# CL-CPI-613-009 or Wake Forest Study# CCCWFU 29109, under IND 107,800).

Also known as: 6,8-bis-benzylsulfanyloctanoic acid, 6,8-bis(benzylthio)octanoic acid, 6,8-bis-benzylsulfonyloctanoic acid, Bylantra

CPI-613

Eligibility Criteria

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

You may qualify if:

• Have either documented refractory or relapsed AML, or documented MDS of any risk group that has failed a hypomethylating agent (such as decitabine \[Vidaza\] and azacitidine \[AZA\]). (Therapy failure with a hypomethylating agent is defined as patients who have been sufficiently treated with hypomethylating agents without response in the opinion of the treating physician, or whose disease has progressed or relapsed while on a hypomethylating agent.) Has never been treated with CPI-613.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
• Expected survival \>2 months.
• years of age and older of both genders.
• 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.
• No radiotherapy, surgery or hormonal therapy for any kind of within 2 weeks prior to participating in this study. Patients must have fully recovered from the acute toxicities of any prior treatment with any anti-cancer drugs (including hypomethylating agents in MDS patients), radiotherapy or other anti-cancer modalities (i.e., returned to baseline status as noted before most recent treatment) for any tumors. Patients with persisting, stable chronic toxicities from such prior treatment ≤Grade 1 are eligible, but must be documented as such.
• Recombinant erythropoietin or G-CSF is not allowed, since CPI-613 does not induce myelosuppression.
• No evidence of active or serious infection of any kind within the past month. No systemic fungal, bacterial, viral or other infection not controlled, defined as exhibiting ongoing signs/symptoms related to the infection and without improvement, despite appropriate antibiotics or other treatment.
• Signed informed consent form.

You may not qualify if:

• Serious medical illness, such as significant cardiac disease (e.g. symptomatic congestive heart failure, unstable angina pectoris, myocardial infarction within the past 6 months, uncontrolled cardiac arrhythmia, or New York Heart Association Class III or IV), or severe debilitating pulmonary disease, that would potentially increase patients' risk for toxicity.
• Active heart disease including myocardial infarction within the previous 6 months, symptomatic coronary artery disease, abnormal ECG, or symptomatic congestive heart failure.
• Any active uncontrolled bleeding, or any patients with a bleeding diathesis (e.g., active peptic ulcer disease).
• Dyspnea with minimal to moderate exertion. Patients with large pleural, pericardial, or peritoneal effusions.
• Evidence of active infection, or serious infection within the past month.
• Patients with active central nervous system (CNS) or epidural solid or hematologic tumors.
• Patients receiving any standard or investigational therapy for any tumor indication within the past 2 weeks, or any investigational agent for any indication within the past 4 weeks, prior to the study.
• Patients who have received immunotherapy of any type for any indications within the past 4 weeks prior to the study.
• Ongoing oral corticosteroids are not permitted. However, topical and inhaled corticosteroids are permitted, and prophylactic steroids are allowed for transfusion reactions.
• Life expectancy less than 2 months.
• Pregnant women, or women of child-bearing potential not using reliable means of contraception.
• Lactating females.
• Fertile men unwilling to practice contraceptive methods during the study period.
• Unwillingness or inability to follow protocol requirements.

Sponsors & Collaborators

• Cornerstone Pharmaceuticals: lead

Study Sites (1)

Cornerstone Pharmaceuticals, Inc

Cranbury, New Jersey, 08512, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Recurrence

Interventions

devimistat

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• King C Lee, Ph.D.

  Cornerstone Pharmaceuticals

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: January 20, 2012
• First Posted: January 30, 2012
• Study Start: January 1, 2016
• Primary Completion: December 1, 2018
• Study Completion: December 1, 2018
• Last Updated: December 29, 2016

Record last verified: 2016-12

Locations

US (1)