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Acute Myeloid Leukemia (AML)Phase 2NCT01520805

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

A Phase 2 study testing CPI-613 in people with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), run by Cornerstone Pharmaceuticals at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial 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]). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
CPI-613
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
1 siteUnited States
Study dates
Jan 2016to Dec 2018
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.
  • 18 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.
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  • 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.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Cornerstone Pharmaceuticals, Inc

    Cranbury, New Jersey, 08512

Other trials in United States

About this study

Official title: A Phase 2a Open-Label Clinical Trial Evaluating Efficacy & Safety of CPI-613 in Patients With Refractory/Relapsed Acute Myeloid Leukemia (AML), and in Patients With Myelodysplastic Syndrome (MDS) Who Failed Hypomethylating Agents

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]).

Read the detailed description

A new therapy for AML is necessary because, although there are several treatment options for patients with AML, these treatments are very toxic and not available to all AML patients or only useful for acute promyelocytic leukemia (APL). Also, there is essentially no treatment for patients with refractory or relapsed AML outside of bone marrow transplant. Accordingly, there is a great medical need for a safe and effective therapy for AML, especially refractory and relapsed AML. Also, although hypomethylating agents have been found to be effective against MDS, these agents are toxic. Furthermore, after relapsing from a hypomethylating agent, there is no treatment for this disease.

A nearly completed clinical trial of CPI-613 (Cornerstone Study# CL-CPI-613-009 or Wake Forest Study# CCCWFU 29109, under IND# 107,800) shows that CPI-613 is well tolerated at doses as high as 3,000 mg/m2. Results from this nearly completed trial also suggest that CPI- 613 may be effective against refractory and relapsed AML, as well as against MDS that is relapsed from a hypomethylating agent. Therefore, CPI-613 may be a suitable treatment option for refractory/relapsed AML and MDS relapsed from a hypomethylating agent. The promising preliminary efficacy data from Study# CL-CPI-613-009 (Wake Forest Study# CCCWFU 29109, under IND# 107,800) is the basis on which Cornerstone is conducting the current Phase 2a trial to further assess the efficacy of CPI-613 against these diseases.

Being tested
CPI-613
Keywords
acute myeloid leukemia, myelodysplastic syndrome, refractory, relapsed, hypomethylating agents
Medical subject headings
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Recurrence

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CPI-613Drug

    Also called 6,8-bis-benzylsulfanyloctanoic acid, 6,8-bis(benzylthio)octanoic acid, 6,8-bis-benzylsulfonyloctanoic acid, Bylantra

    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).

Study groups in NCT01520805
Study groupRoleWhat they receive
CPI-613ExperimentalCPI-613 will be intravenously infused over 2 hours, given twice weekly for 3 weeks followed by a week of rest.
Receives: Drug: CPI-613

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Survival (OS)

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

Secondary outcomes (6)

  1. Overall Remission Rate

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

  2. Response Rate

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

  3. Duration of Overall Remission

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

  4. Progression Free Survival (PFS)

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

  5. Quality of Life (QOL)

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

  6. Safety

    Safety assessment will be based on clinical signs, vital signs, blood work, adverse events, AEs, etc.

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Jan 2012 (estimated)
  2. Study startJan 2016
  3. Primary completionDec 2018 (estimated)
  4. Study completionDec 2018 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01520805
Sponsor's protocol ID
CL-CPI-613-022
Other IDs
CL-CPI-613-022
Status last verified
Dec 2016
First submitted
20 Jan 2012
Last updated
29 Dec 2016
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing CPI-613 in people with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), run by Cornerstone Pharmaceuticals at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 29 Dec 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Survival (OS) (time frame: Monitored until participants passed away, for an expected average of 6 months.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01520805.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Dec 2016.

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