RecruitingPhase 1

Imetelstat Combinations in Relapsed AML

IMetelstat and Azacitidine With or Without Venetoclax GIveN in rElapsed Acute Myeloid Leukemia (IMAGINE Trial)

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 54 months

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Total of up to 36 participants will be accrued over 54 months at Mount Sinai Hospital."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests the safety and early effects of imetelstat with azacitidine, with or without venetoclax. It is for adults with acute myeloid leukemia (AML), a blood cancer, that came back or did not respond to treatment. It will also help find the best dose of imetelstat.

IMAGINE is a two-part trial to evaluate the safety and preliminary efficacy of imetelstat in combination with azacitidine with or without venetoclax in patients with relapsed or refractory AML. The trial will consist of a safety run-in phase (Part A) employing a 3+3 design to monitor dose-limiting toxicities of imetelstat when administered in combination with a fixed dose of azacitidine. Part B will consist of a phase 1b trial employing a BOIN12 design to determine the optimal biological dose of imetelstat, starting at a lower dose level, in combination with azacitidine and venetoclax. Total of up to 36 participants will be accrued over 54 months at Mount Sinai Hospital. Estimated duration of trial is 114 months including recruitment, screening, treatment, and follow-up.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be at least 18 years old, understand the study, and freely sign a consent form.
  • Have confirmed AML, but not the type called APL. The cancer must have come back or not responded after at least one treatment, with no standard treatment available that may help.
  • AML found only outside the bone marrow, including in the skin, is allowed, but active leukemia in the brain or spinal cord is not.
  • Be expected by the study doctor to live at least 12 weeks.
  • Have an ECOG activity score of 0–3, which measures how well you can do daily tasks and care for yourself.
  • Meet the study's liver and kidney blood test limits.
  • Women who can become pregnant must have negative pregnancy tests. They and male participants must use highly effective birth control from the first dose until 6 months after the last dose.
  • Be able to follow the visit schedule and study rules.

Who can't join

  • Recent use of an experimental treatment or device within the study's waiting period; or being trial staff, sponsor staff, or their close family member without special approval.
  • Active leukemia in the brain or spinal cord, or a white blood cell count above 25 billion per liter. Medicine may be used to lower the count.
  • Active graft-versus-host disease, where donor cells attack the body, or treatment for it outside the study's allowed exceptions. An organ transplant other than a bone marrow transplant also rules out entry.
  • Another cancer being treated with radiation or chemotherapy, or not cancer-free for 2 years, unless it falls under the study's listed exceptions.
  • A heart attack or unstable chest pain within 6 months, moderate or severe heart disease, or another serious or unstable medical or mental health condition that makes joining unsafe.
  • A serious active infection, uncontrolled HIV, active hepatitis A, B, or C needing treatment, or liver scarring called cirrhosis. Some people with COVID-19 during screening may qualify after the doctor's review.
  • Being pregnant or breastfeeding.
  • For Part B only: trouble swallowing, a digestive illness, or a past digestive procedure that could affect how venetoclax is absorbed or tolerated.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria In order to be eligible for participation in this trial, * Participants must be ≥18 years of age at time of signing the Informed Consent Form (ICF). * Participants must voluntarily sign an ICF. * Participants must have WHO-confirmed non-APL AML who have not responded to or relapsed after at least one prior therapy and for whom no standard therapy that may provide clinical benefit is available. o Participants with isolated extramedullary disease (EMD), including leukemia cutis, are included but not those with active known CNS disease. * Participants must have a life expectancy of at least 12 weeks per investigator. * ECOG performance status ≤ 3. * Women of child bearing potential (WOCBP), defined as a sexually mature woman not surgically sterilized or post-menopausal for at least 24 consecutive months if ≤55 years or 12 months if \>55 years, must have a negative serum pregnancy test at screening and cycle 1 day 1 and must agree to use highly effective methods of birth control starting with the first dose of study therapy through 6 months after the last dose of study therapy. Highly effective methods of contraception include double-barrier methods (diaphragm with spermicidal gel and condoms with spermicide), partner vasectomy, and total abstinence * Male participants should agree to use a highly effective method of contraception starting with the first dose of study therapy through 6 months after the last dose of study therapy. 8. Must have adequate organ function as demonstrated by the following: * Serum total bilirubin ≤ 2.0 x upper limit of normal (ULN) unless considered due to leukemic organ involvement or Gilbert's syndrome. * Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 x ULN. * Creatinine clearance (CrCl) ≥ 30 mL/min (measured or estimated by Cockcroft-Gault formula). * Ability to adhere to the study visit schedule and all protocol requirements. * Ability to understand and the willingness to sign a written informed consent. Exclusion Criteria The participant must be excluded from participating in the trial if the participant: * Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 2 weeks or within 5 half-lives of the prior investigational agent, whichever is shorter, of the first dose of treatment. * Known clinically active central nervous system (CNS) leukemia. a. Prior CNS leukemia will be allowed if the most recent cerebral spinal fluid sample is negative prior to study initiation and there is no clinical suspicion for active CNS disease. * Has a white blood cell count \> 25 x 109/L (hydroxyurea and/or continuous cytarabine ≤1 g/m2 is permitted to meet this criterion). * Active Graft Versus Host Disease (GVHD), or any GVHD requiring treatment with immunosuppression, with the exception of topical steroids and systemic steroids at a dose equivalent to prednisone 10mg or less. Any GVHD treatment (including calcineurin inhibitors) must be discontinued at least 28 days prior to Day 1 of study treatment. * Participants with a history of other cancers must not be receiving active treatment (with radiation or chemotherapy) and must be free of disease for 2 years prior to the Screening Visit with the exception of localized prostate cancer treated with hormone therapy, breast cancer treated with hormone therapy, localized basal cell carcinoma, non-melanoma skin cancer, cervical carcinoma in situ, concurrent low-grade lymphoma, that is asymptomatic and lacks bulky disease and shows no evidence of progression, and for which the participant is not receiving any systemic therapy or radiation * Myocardial infarction or unstable angina within 6 months of Screening, or any Class 3 (moderate) or Class 4 (severe) cardiac disease as defined by the New York Heart Association Functional Classification. * Presence of active serious infection. a. If a participant is identified to have COVID-19 during the screening period, participants may be considered eligible if in the opinion of the investigator there are no COVID-19 sequelae that may place the participant at a higher risk of receiving investigational treatment * Any serious, unstable medical or psychiatric condition that would prevent, (as judged by the Investigator) the participant from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the participant at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study. * Known history of uncontrolled human immunodeficiency virus (HIV) * Active known systemic hepatitis A, B, or C infection requiring therapy or known cirrhosis. * Is or has an immediate family member (e.g., spouse, parent/legal guardian, sibling, or child) who is investigational site or pharmaceutical sponsor staff directly involved with this trial, unless prospective IRB approval (by chair or designee) is given allowing exception to this criterion for a specific participant. * Organ transplant recipients other than bone marrow transplant. * Women who are pregnant or lactating. * Participants with known gastrointestinal (GI) disease or prior GI procedure that could interfere with the oral absorption or tolerance of venetoclax, including difficulty swallowing, are not eligible (Part B only).

Study sites (1)

NY