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Myelodysplastic Syndrome (MDS)Phase 2NCT03957876

CPX-351 Therapy for MDS After Hypomethylating Agent Failure

A Phase 2 study testing CPX-351 in people with Myelodysplastic Syndrome (MDS), run by Case Comprehensive Cancer Center at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

The purpose of this study is to evaluate the efficacy of treatment with CPX-351 (an FDA approved drug for the treatment of AML) in individuals with MDS while using a new stratification tool to predict outcomes of participants following HMA failure. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndrome (MDS)
What is being tested?
CPX-351
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
4 participants
Where
1 siteUnited States
Study dates
25 Jul 2019to 20 Dec 2022
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

  • Patients must give voluntary written consent before performance of any study related procedures not part of standard medical care
  • Diagnosis of MDS or MDS/MPN according to 2016 WHO criteria12
  • Primary therapy failure with either hypomethylating agents (decitabine or azacitidine) defined as:
  • Progression (according to 2006 IWG criteria)13 after initiation of azacitidine or decitabine treatment; or
  • Failure to achieve complete or partial response or hematological improvement (according to 2006 IWG)13 after at least 4-6 cycles (4-weeks cycle) of azacitidine or decitabine; or
  • Relapse after initial complete or partial response or hematological improvement (according to 2006 IWG criteria)13 observed after at least 4 cycles of azacitidine or decitabine.
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  • Eastern Cooperative Oncology Group (ECOG) Performance Status < 2
  • Subjects must have normal organ and marrow function defined as:
  • If total bilirubin < 2x upper limit of normal (</= 3 x ULN if considered to be due to leukemic involvement or Gilbert's syndrome) at the discretion of the treating physician following discussion with PI)
  • Calculated creatinine clearance value of > 30ml/min AND a serum creatinine < 1.5mg/dL
  • LVEF >/= 50%
  • Female patients who:
  • Are postmenopausal for at least 1 year before the screening visit, OR
  • Are surgically sterile, OR
  • Agree to practice true abstinence from heterosexual contact or agree to use effective contraception without interruption during the study therapy and 90 days after the last dose
  • Male patients who:
  • Are surgically sterile, OR
  • Agree to practice true abstinence from heterosexual contact or agree to use effective contraception without interruption during the study therapy and 90 days after the last dose

Who cannot join

  • Prior treatment with CPX-351, or known hypersensitivity to CPX-351 or its components.
  • Prior treatment with intensive chemotherapy.
  • Any serious medical condition, laboratory abnormality, or psychiatric illness that, in the view of the treating physician, would place the participant at an unacceptable risk if he or she were to participate in the study or would prevent that person from giving informed consent.
  • Any active malignancy (unrelated, non-hematological malignancy) diagnosed within the past 6 months of starting the study drug (other than curatively treated carcinoma-in-situ of the cervix or non-melanoma skin cancer).
  • Patients with uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Known history of HIV or active hepatitis B or C.
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  • Major surgery within 2 weeks prior to study enrollment.
  • Pregnant or lactating females
  • Male and female patients who are fertile who do not agree to use an effective barrier methods of birth control (i.e. abstinence or 2 forms of contraception) to avoid pregnancy while receiving study treatment.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Cleveland Clinic Taussig Cancer institute, Case Comprehensive Cancer Center

    Cleveland, Ohio, 44195

Other trials in United States

About this study

Official title: A Phase II Study of CPX-351 as a Novel Therapeutic Approach for Patients With Myelodysplastic Syndromes (MDS) After Hypomethylating Agent Failure

The purpose of this study is to evaluate the efficacy of treatment with CPX-351 (an FDA approved drug for the treatment of AML) in individuals with MDS while using a new stratification tool to predict outcomes of participants following HMA failure. This approach is intended to gain a better understanding and insight into identifying new opportunities for drug approvals in this setting.

Read the detailed description

This study will evaluate efficacy of treatment with CPX-351in participants with MDS while using a new stratification tool to predict outcomes of patients following HMA failure in order to gain a better understanding and insight into identifying new opportunities for drug approvals in this setting.

CPX-351is an investigational (experimental) drug for the indication of myelodysplastic syndrome that works by delivering two chemotherapy medications (daunorubicin and cytarabine) together which are then concentrated into the bone marrow (the part of the body that makes blood cells). CPX-351 is experimental because it is not approved by the Food and Drug Administration (FDA) for the indication of myelodysplastic syndrome. This drug is approved by theFDA for the indication of acute myeloid leukemia. One or more of the Investigators conducting this study serve as consultants for the company that makes products used in this study. These financial interests are within permissible limits established by the local institutional Conflict of Interest Policy.

Prior to beginning treatment, all eligible participants will be grouped into low risk versus high risk based on a stratification tool used to determine their disease. Group 1 will be low risk participants and group 2 will be high risk participants. All study participants will get the same study drug, CPX-351. Participants will receive the CPX-351 on days 1, 3 and 5 of the first 28 day cycle. After participants finish the first round of treatment and based on their response they may be eligible for another 4 cycles of treatment. The participant's doctor will inform them if this is an available option when the time comes. Once participant have finished treatment, their doctor will continue observe for side effects and follow their condition for 1 year.

Being tested
CPX-351
Medical subject headings
Myelodysplastic Syndromes

What participants receive

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

  • CPX-351Drug

    Also called (daunorubicin and cytarabine)

    CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin.

Study groups in NCT03957876
Study groupRoleWhat they receive
intravenous CPX-351 with potential maintenance therapyExperimentalSingle agent CPX-351 administered at the standard FDA approved dose of 44 mg/m2 intravenously on days 1, 3, 5 of the induction cycle. If participants achieve complete remission (CR), complete remission with incomplete count recovery (CRi) or partial remission (PR), they will be eligible to continue on to maintenance therapy, which will consist of CPX351 at a dose of 15.4 mg/m2 every 28 days. Participants can receive up to 4 cycles of maintenance therapy.
Receives: Drug: CPX-351

What is being measured

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

  1. Efficacy of CPX-351 as Measured by Overall Response Rate (ORR)

    Efficacy of CPX as measured by ORR as defined by IWG 2006 criteria for MDS participants at end of induction. IWG 2006 responses that must be for at least 4 weeks include Complete Remission (CR), Partial Remission (PR), Marrow CR, Stable Disease (SD), Failure, Relapse after CR or PR (PD), or cytogenetic response. Hematologic Improvement (HI), which must be for at least 8 weeks, includes erythroid response (pretreatment, < 11 g/dL), Platelet response (pretreatment, < 100x109/L), Neutrophil response (pretreatment, < 1 x109/L), or Progression or relapse after HI.

    Measured: day 28 +/- 7 days of induction

Secondary outcomes (4)

  1. Time to Response (TTR) Associated With CPX-351

    TTR associated with CPX-351 in participant with MDS at the end of induction. TTR defined by the time between starting the treatment and the time of achieving best response.

    Measured: day 28 +/- 7 days of induction

  2. Duration of Response (DOR) in Participants Achieving a Response

    DOR in participants achieving a response defined by the time between first response (day C1 D28 +/-7 days from induction) and the day of loss of response

    Measured: up to 1 year after end of treatment

  3. Event-free Survival (EFS)

    EFS probability of all participants enrolled in this trial from start of treatment and up to 1 year after the end of treatment.

    Measured: up to 1 year after end of treatment

  4. Overall Survival (OS)

    OS probability of all participants enrolled in this trial from start of treatment and up to 1 year after the end of treatment.

    Measured: up to 1 year after end of treatment

Timeline

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

  1. First posted21 May 2019
  2. Study start25 Jul 2019
  3. Primary completion20 Dec 2022
  4. Study completion20 Dec 2022

Why it stopped: Slow Accrual

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
4 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03957876
Sponsor's protocol ID
CASE2918
Status last verified
Aug 2024
First submitted
10 Apr 2019
Last updated
15 Aug 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: The study team does not plan to share IPD collected in this study

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing CPX-351 in people with Myelodysplastic Syndrome (MDS), run by Case Comprehensive Cancer Center 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 18 inclusion and 9 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 15 Aug 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 15 Aug 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?

Efficacy of CPX-351 as Measured by Overall Response Rate (ORR) (time frame: day 28 +/- 7 days of induction). 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 NCT03957876.

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 15 Aug 2024.

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