CPX-351 in Higher Risk Myelodysplastic Syndromes

  • STATUS
    Recruiting
  • participants needed
    65
  • sponsor
    Groupe Francophone des Myelodysplasies
Updated on 19 February 2024
cardiovascular disease
direct bilirubin
heart failure
prednisone
cytarabine
lenalidomide
arrhythmia
nephropathy
leukemia
conjugated bilirubin
platelet transfusion
systemic steroids
chronic myelomonocytic leukemia
blood cell count
serum total bilirubin
myelodysplastic syndrome
central nervous system leukemia

Summary

Study of the efficacy of CPX-351 treatment in patients with higher risk myelodysplastic syndromes : as first line treatment or after hypomethylating agents failure

Description

A phase I/II study of the efficacy of CPX-351 treatment in patients with higher risk myelodysplastic syndromes : as first line treatment or after hypomethylating agents failure.

CPX-351 is an advanced liposomal formulation of daunorubicin and cytarabine encapsulated at a 1:5 ratio.

Patients will receive induction treatment with CPX-351. Patients in response (complete response (CR), complete response with incomplete hematologic improvement (CRi), partial response (PR)) after induction will receive monthly courses of consolidation therapy with CPX-351.

Details
Condition Preleukemia, MYELODYSPLASTIC SYNDROME
Age 18years - 70years
Treatment CPX-351 in cohort A, CPX-351 in cohort B
Clinical Study IdentifierNCT04273802
SponsorGroupe Francophone des Myelodysplasies
Last Modified on19 February 2024

Eligibility

Yes No Not Sure

Inclusion Criteria

Myelodysplastic syndrome (WHO 2016 classified) including CMML (even if white blood cell count (WBC) > 13000/mm3)
For COHORT A: untreated patients except by erythropoiesis stimulating agents, Lenalidomide or non-chemotherapy during a phase of lower risk MDS; For COHORT B: absence of response (CR, CRi, PR or HI according to international working group (IWG) 2006 for MDS) after a minimum of 6 cycles of single hypomethylating agent or relapse after a response
For COHORT A: less than 20% of marrow blasts. For COHORT B: less than doubling of marrow blasts compared with onset of hypomethylating agent
Classical international prognostic scoring system (IPSS) int-2 or high risk score
For COHORT A: age between 18 and 70 years; For COHORT B: age 18 years
For COHORT A: Performance status (ECOG grading) 1; For COHORT B: Performance status 2\
Eligible for standard intensive chemotherapy
Absence of concomitant severe cardiovascular disease which would make intensive chemotherapy impossible, i.e. arrhythmias requiring chronic treatment, congestive heart failure or symptomatic ischemic heart disease, reduced left ventricular function assessed by multigated acquisition (MUGA) scan or echocardiogram
Patient must have adequate organ function as indicated by the following laboratory values: Renal: Serum creatinine < 2 mg/dl or calculated creatinine clearance 60 mL/min by MDRD (modification of the diet in renal disease) or CKD-EPI (chronic kidney disease epidemiology collaboration) equation for patients with creatinine levels > 1.5xULN ; Hepatic: Serum total bilirubin 2.5xULN OR direct bilirubin ULN for patients with total bilirubin levels 2 mg/dL, aspartate aminotransferase (ALT) and alanine aminotransferase (ALT) 2.5xULN, Alkaline Phosphatase 5xULN (if > 2.5xULN, then liver fraction should be 2.5xULN)
Patients not known to be refractory to platelet transfusions
Female subjects of child-bearing potential must agree to undergo medically supervised pregnancy test prior to starting study drug. The first pregnancy test will be performed at screening (within 7 days prior to first study drug administration), and on the day of the first study drug administration and confirmed negative prior to dosing and Day 1 before dosing all subsequent cycles. Female patient is not actively breastfeeding at the time of study entry
Female patients are either post-menopausal, free from menses for > 2 years, surgically sterilized or willing to use 2 adequate barrier methods of contraception to prevent pregnancy or agree to abstain from becoming pregnant throughout the study, starting with Visit 1. Females of reproductive potential as well as fertile men and their partners who are female of reproductive potential must agree to abstain from sexual intercourse or to use two highly effective forms of contraception from the time of giving informed consent, during the study and for 6 months (females and males) following the last dose of CPX-351
Male patients agree to use an adequate method of contraception for the duration of the study. Men should be advised not to father a child while receiving CPX-351 and for 6 months post study
Patients are available for regular blood sampling, study related assessments, and appropriate clinical management at the treating institution for the duration of the study
Patients have the ability to understand and willingness to sign an informed consent form indicating the investigational nature of the study

Exclusion Criteria

Active and uncontrolled infection
Last dose of hypomethylating agent given more than 4 months before entering the trial
Uncontrolled intercurrent illness or circumstances that could limit compliance with the study, including but not limited to the following: symptomatic congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmia, pancreatitis, or psychiatric or social conditions that may interfere with patient compliance
Current participation or participation in a study with an investigational compound or device within 30 days of initial dosing with study drug
Known human immunodeficiency virus (HIV) infection or HIV-related malignancy
Clinically active hepatitis B or hepatitis C infection
Known allergy or hypersensitivity to any component of CPX-351
Currently active" second malignancy, other than non-melanoma skin cancer and in situ carcinoma of the cervix
Subjects with a history of Wilson's disease or other copper-related disorder
Treatment with growth factors such as erythropoietin alfa (EPO) or granulocyte colony-stimulating factor (G-CSF) or cytotoxic and non-cytotoxic agents (including low dose oral chemotherapy with the exception of hydroxyurea) in the 30 days before inclusion
Treatment with systemic steroids that has not been stabilized to the equivalent of 10 mg/day prednisone during the 4 weeks prior to the start of the study drugs
Clinical evidence of central nervous system leukemia
Pregnancy or breastfeeding during the projected duration of the study
Absence of social security
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact

site

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Loading...

Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

Study AnnotationsStudy Notes

Notes added here are public and can be viewed by anyone. Notes added here are only available to you and those who you share with.

user name

Added by • 

 • 

Private

Reply by • Private
Loading...

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.
Loading...

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note
  • abc Select a piece of text.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.