Previously Treated Myelodysplastic SyndromesPhase 2NCT01902381
CPI-613 in Treating Patients With Myelodysplastic Syndromes Who Failed Previous Therapy
A Phase 2 study testing 6,8-bis(benzylthio)octanoic acid in people with Previously Treated Myelodysplastic Syndromes, run by Wake Forest University Health Sciences at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Previously Treated Myelodysplastic Syndromes
- What is being tested?
- 6,8-bis(benzylthio)octanoic acid
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2013to 26 Nov 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- 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
Show all 14Show fewer
- 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)
Who cannot join
- 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
Show all 18Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Comprehensive Cancer Center of Wake Forest University
Winston-Salem, North Carolina, 27157
About this study
Official title: A Pilot Study of CPI-613 in Patients With Myelodysplastic Syndrome Who Have Failed Previous Therapy
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.
Read the detailed description
PRIMARY OBJECTIVES:
- 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.
- Being tested
- 6,8-bis(benzylthio)octanoic acid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
6,8-bis(benzylthio)octanoic acidDrug
Also called alpha-lipoic acid analogue CPI-613, CPI-613
Given IV
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: 6,8-bis(benzylthio)octanoic acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
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. Toxicities related to the treatment will be examined by looking at each toxicity identified by grade (highest grade of each toxicity per patient so that there is only one event attributed to each patient for a given toxicity).
Measured: During treatment, a median of 4 months with a maximum of 40 months
Progression-free Survival (PFS)
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.
Disease progression: For patients with:
Less than 5% blasts: ≥ 50% increase in blasts to > 5% blasts 5%-10% blasts: ≥ 50% increase to > 10% blasts 10%-20% blasts: ≥ 50% increase to > 20% blasts 20%-30% blasts: ≥ 50% increase to > 30% blasts
Any of the following:
At least 50% decrement from maximum remission/response in granulocytes or platelets Reduction in Hgb by ≥ 2 g/dL Transfusion dependence
Survival Endpoints:
Overall: death from any cause Event free: failure or death from any cause PFS: disease progression or death from MDS DFS: time to relapse Cause-specific death: death related to MDS
Measured: 6 and 12 months post treatment start
Overall Survival (OS)
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.
Measured: 6 and 12 months post treatment start
Number of Patients Who Achieve a Reduction in Blood Transfusion Requirements
Number of patients who achieve a reduction in blood transfusion requirements.
Measured: Up to 5 years
Number of Patients Who Achieve Hematologic Improvement (HI), as Defined by Cheson, et al. (2006)
Number of patients who achieve hematologic improvement (HI), as defined by Cheson, et al. (2006).
Criteria:
Erythroid response (pretreatment, < 11 g/dL) - Hgb increase by ≥ 1.5 g/dL.
Platelet response (pretreatment, < 100 × 109/L) - Absolute increase of ≥ 30 × 109/L for patients starting with > 20 × 109/L platelets. Increase from < 20 × 109/L to > 20 × 109/L and by at least 100%
Neutrophil response (pretreatment, < 1.0 × 109/L) - At least 100% increase and an absolute increase > 0.5 × 109/L
Progression or relapse after HI - At least 1 of the following:
At least 50% decrement from maximum response levels in granulocytes or platelets Reduction in Hgb by ≥ 1.5 g/dL
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2013 (estimated)
- Study startAug 2013
- Primary completion26 Nov 2018
- Study completion26 Nov 2018
Why it stopped: Drug sponsor suspended study prematurely then study was terminated. Expected enrollment was not met.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Previously Treated Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Wake Forest University Health SciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wake Forest University Health Sciences
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Timothy Pardee, Principal investigator, Wake Forest University Health Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01902381
- Sponsor's protocol ID
- IRB00024007
- Other IDs
- NCI-2013-01312, P30CA012197, CCCWFU 29113
- Status last verified
- Oct 2024
- First submitted
- 15 Jul 2013
- Last updated
- 17 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of CPI-613 in Patients With Myelodysplastic Syndrome Who Have Failed Previous Therapy
- ClinicalTrials.gov ID
- NCT01902381
- Acronym / sponsor ID
- IRB00024007
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Drug sponsor suspended study prematurely then study was terminated. Expected enrollment was not met.
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Wake Forest University Health SciencesUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Aug 2013 (actual)
- Primary completion
- 26 Nov 2018 (actual)
- Study completion
- 26 Nov 2018 (actual)
- First posted
- 18 Jul 2013 (estimated)
- Results
- Posted 17 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (6, 8-bis(benzylthio) octanoic acid) | Experimental | Drug: 6,8-bis(benzylthio)octanoic acidPatients 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- 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
Key exclusion criteria (18 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 6,8-bis(benzylthio)octanoic acidAlso: alpha-lipoic acid analogue CPI-613, CPI-613Given IV | Drug | Treatment (6, 8-bis(benzylthio) octanoic acid) |
Primary outcome
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
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
5 secondary outcomes
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: During treatment, a median of 4 months with a maximum of 40 months
Progression-free Survival (PFS)
Time frame: 6 and 12 months post treatment start
Overall Survival (OS)
Time frame: 6 and 12 months post treatment start
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Previously Treated Myelodysplastic Syndromes
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Personalized NK Cell Therapy in CBT
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Biphenotypic Leukemia
RecruitingPhase 2
MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Timothy Pardee principal investigator
Wake Forest University Health Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 6,8-bis(benzylthio)octanoic acid in people with Previously Treated Myelodysplastic Syndromes, run by Wake Forest University Health Sciences 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 14 inclusion and 18 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: stopped early, as of the sponsor's last update on 17 Oct 2024. 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?
Yes. Results are posted in the registry (first posted 17 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Oct 2024.