NTX-301 in MDS/AML

  • STATUS
    Recruiting
  • End date
    Mar 31, 2025
  • participants needed
    20
  • sponsor
    University of Alabama at Birmingham
Updated on 15 December 2021
graft versus host disease
myeloid leukemia
tacrolimus
anemia
mycophenolate mofetil
cyclosporine
hydroxyurea
white blood cell count
ejection fraction
leukemia
gilbert's syndrome
leukapheresis

Summary

NTX-301 is a DNMT1 inhibitor. The drug is an oral drug with preclinical data that has shown preclinical anti-leukemic efficacy. This is the first clinical trial using NTX-301 in patients with myeloid malignancies.

Details
Condition Acute Myeloid Leukemia, Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia
Treatment NTX-301
Clinical Study IdentifierNCT04167917
SponsorUniversity of Alabama at Birmingham
Last Modified on15 December 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Able to understand and comply with the study procedures, understand the risks involved in the study, and provide written informed consent before the first study-specific procedure
Men or women ≥18 years with one of the following conditions that is relapsed or refractory to at least one line of therapy
Acute myeloid leukemia as long as with myeloblast percentage in the marrow is ≤ 30% or the peripheral white blood cell count is less than 20,000 cells/μL in the absence of leukoreducing therapy (e.g., hydroxyurea, leukapheresis)
MDS classified as intermediate, high, or very high risk by International Prognostic Scoring System-Revised [IPSS-R] criteria
CMML classified as intermediate-2 or high risk per CMML-specific prognostic scoring system (CPSS) or clinical/molecular CPSS (CPSS-mol) criteria
ECOG performance status of 0, 1, or 2
Adequate organ function at screening defined as follows [reasonably minor changes pre-first dose are acceptable if deemed so by the investigator]
Hepatic
Total bilirubin ≤2 × upper limit of normal (ULN); isolated bilirubin > 2 is acceptable if participant has a diagnosis of Gilbert's syndrome
Aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) ≤3 × ULN. b) Renal
estimated glomerular filtration rate (by CKD-EPI method) ≥ 40 mL/min/1.73 m2 c)
Left ventricular ejection fraction greater than 45% or the institutional lower
Cardiac
limit of normal by either ECHO or MUGA at entry
Patients must have recovered to grade 1 or less from prior toxicity or adverse events
(exception of myelosuppression - neutropenia, anemia, thrombocytopenia - and
alopecia). Note: Participants with treatment-related toxicities that are
Able to swallow, retain, and absorb orally administered medication
unlikely to resolve per the investigator may be enrolled on a case-by-case
basis after discussion with the medical monitor
Participants with a prior history of stem cell transplant (autologous and/or allogeneic) are allowed if all of the following are met
Patients must have completed any chemotherapy, radiation therapy, or biologic therapy specific to their myeloid neoplasm ≥ 2 weeks or 5 half-lives (whichever is longer)
days or more have elapsed from the time of transplant
subject has been off systemic immunosuppressive medications (including but not limited to: cyclosporine, tacrolimus, mycophenolate mofetil, or corticosteroids) for at least 30 days prior to the first dose of NTX-301. Topical steroids are permitted
Expected life ≥ 4 months
no signs or symptoms of acute graft versus host disease, other than Grade 1 skin involvement
there are no signs or symptoms of chronic graft versus host disease requiring systemic therapy
Subjects and their partners with reproductive potential must agree to use 2 highly effective contraceptive measures during the study and must agree not to become pregnant or father a child for 3 months after the last dose of study treatment. Contraceptive measures that may be considered highly effective comprise combined hormonal contraception (oral, vaginal, or transdermal) or progestogen-only hormonal contraception (oral, injectable, implantable) associated with inhibition of ovulation, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, sexual abstinence, and surgically successful vasectomy. Abstinence is acceptable only if it is consistent with the preferred and usual lifestyle of the subject. Periodic abstinence (eg, calendar, ovulation, symptothermal, or post-ovulation methods) and withdrawal are not acceptable methods of birth control
Women of child-bearing potential (according to recommendations of the Clinical Trial
Facilitation Group [CTFG], CTFG 2014) must not be pregnant or breastfeeding
and must have a negative pregnancy test at screening. A woman is considered of
childbearing potential (ie, fertile) following menarche and until becoming
postmenopausal, unless permanently sterile. Permanent sterilization methods
include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy. A
man is considered fertile after puberty unless permanently sterile by
bilateral orchidectomy

Exclusion Criteria

Diagnosis or presence of any of the following
acute promyelocytic leukemia
core-binding factor AML in first relapse
extramedullary leukemia
symptomatic or untreated Central Nervous System (CNS) disease [note that lumbar puncture (LP) is not required for study enrollment unless there is clinical suspicion for CNS disease; patients with history of CNS disease are permitted to enroll if they have previously received appropriate therapy and CNS remission has been; participants on maintenance intrathecal chemotherapy may be enrolled and continue to receive therapy]
Patients who are receiving any other investigational agents
Pregnant women and women who are breastfeeding are excluded from this study
Patients with clinically significant illnesses which would compromise participation in the study, including, but not limited to
severe or uncontrolled infection
known HIV infection requiring protease inhibitor therapy
known Hepatitis B; defined as presence of hepatitis B surface antigen (HBsAg)
known Hepatitis C; if Hepatitis C antibody is positive, then this is defined as positive Hepatitis C RNA polymerase chain reaction (PCR) (or comparable test)
uncontrolled diabetes and/or hypertension, symptomatic congestive heart failure, unstable angina pectoris, myocardial infarction within the past 6 months, uncontrolled cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements in the opinion of the investigator
Malabsorption syndrome or other conditions that would interfere with intestinal absorption
History of prior solid organ transplant
History of prior sensitivity reaction to any cytidine derivates
History of a second malignancy, excluding non-melanoma skin cell cancer, within the
last three years. Participants with second malignancies that were indolent, in
situ or definitively treated may be enrolled even if less than three years
have elapsed since treatment. Consult the monitor if there are any queries
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