Relapsed Acute Myeloid LeukemiaPhase 1IMAGINENCT07320235

Imetelstat Combinations in Relapsed AML

A Phase 1 study testing Imetelstat, Azacitidine and Venetoclax in people with Relapsed Acute Myeloid Leukemia, run by Douglas Tremblay at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Relapsed Acute Myeloid Leukemia
What is being tested?: Imetelstat, Azacitidine and Venetoclax
Type of treatment: Pill or oral medicine, Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 36 participants
Where: 1 siteUnited States
Study dates: 2 Jan 2026to 15 Jun 2035
Phase: Phase 1
Are there results?: Not yet: the study is under way

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

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.
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).

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Gabriela Bello contact

(212) 241-0463

gabriela.bello@mssm.edu

Rashmi Unawane contact

212-824-2385

rashmi.unawane@mssm.edu

United States1 site, 1 recruiting

Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Gabriela Bello, (212) 241-0463, gabriela.bello@mssm.edu
Rashmi Unawane, 212-824-2385, Rashmi.Unawane@mssm.edu
Recruiting

Other trials in United States

About this study

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

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.

Conditions: Relapsed Acute Myeloid Leukemia
Being tested: Imetelstat, Azacitidine, Venetoclax
Keywords: IMAGINE Trial, Acute Myeloid Leukemia, AML, Relapsed Acute Myeloid Leukemia, Imetelstat, Imetelstat and Azacitidine
Medical subject headings: Leukemia, Myeloid, Acute

What participants receive

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

ImetelstatDrug
Also called GRN163L
Three 3 dose levels administered on Day 1 of each 28-day cycle for Safety Run-in Phase and optimal dose to be administered on Day 1 of each 28-day cycle
AzacitidineDrug
75mg/m2 IV or subcutaneous (SQ) once daily for Days 1 (+/- 1 day) through 7 (+/- 1 day) of each 28-day cycle. Azacitidine can be administered locally as long as documentation of administration is provided to the study team.
VenetoclaxDrug
Venetoclax 400mg oral once daily for Days 1 (+/- 1 day) through 14 (+/- 1 day) of each 28-day cycle.
Venetoclax reduced dosey should be substituted if concomitant posaconazole, or if other concomitant strong CYP3A4 inhibitor (e.g. voriconazole) or 200mg for moderate CYP3A4 inhibitor (e.g. isavuconazole).
For Cycle 1, participants will receive a venetoclax ramp-up dose on day 1, day 2, and days 3-14 of venetoclax,.
If the participant is being treated with concomitant posaconazole, or other concomitant strong CYP3A4 inhibitor, or with a concomitant moderate CYP3A4 inhibitor or a pg-p inhibitor, participants will receive a lower ramp-up dose on day 1, day 2, and days 3-14 of venetoclax.

Study groups in NCT07320235
Study group	Role	What they receive
Part A Safety Run-In phase: Imetelstat in combination with Azacitidine	Active comparator	Part A combination therapy: * Imetelstat will be administered on Day 1 of each 28-day cycle * Azacitidine once daily for Days 1 (+/- 1 day) through 7 (+/- 1 day) of each 28-day cycle. Receives: Drug: Imetelstat; Drug: Azacitidine
Part B Combination Therapy: Imetelstat in combination with Azacitidine with or without Venetoclax	Active comparator	* Imetelstat will be administered on Day 1 of each 28-day cycle * Azacitidine once daily for Days 1 (+/- 1 day) through 7 (+/- 1 day) of each 28-day cycle. * Venetoclax once daily for Days 1 (+/- 1 day) through 14 (+/- 1 day) of each 28-day cycle.

What is being measured

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

Dose Limiting Toxicity (DLT)
The DLT-evaluable population includes participants in the Part A portion of the trial who received at least one dose of imetelstat and azacitidine and either experienced a Dose-Limiting Toxicity (DLT) during the first-cycle DLT assessment period or completed their first-cycle DLT assessment.
Measured: Cycle 1 Day 1 up to Cycle 1 Day 28 (each cycle is 28 days), for 6 cycles

Secondary outcomes (4)

Optimal Biological Dose (OBD)
The OBD-evaluable population includes participants in the Part B portion of the trial who received at least one dose of imetelstat, azacitidine, and venetoclax and either experienced a Dose-Limiting Toxicity (DLT) during the first-cycle DLT assessment period or completed their first-cycle DLT assessment.
Optimal Biological Dose (OBD) is defined as the lowest safe dose providing the highest rate of efficacy.
Measured: Cycle 1 Day 1 up to Cycle 1 Day 28 (each cycle is 28 days), for 6 cycles
Disease-Free Survival (DFS)
Disease-Free Survival (DFS) is defined as the time from the first occurrence of CR, CRh, or CRi, based on ELN 2022 response criteria (which, in this study, can only be assessed at cycles 2, 4, or 6), until the first documented evidence of relapsed disease or death from any cause, whichever occurs first.Participants who do not achieve CR, CRh, or CRi at one of their cycle 2, 4, or 6 assessments will be excluded from this analysis.
Measured: Cycle 1 Day 1 (each cycle is 28 days) until the first documented evidence of relapsed disease or death from any cause, or up to 6 months. whichever occurs first
Duration of Response (DOR)
Duration of Response (DOR) is defined as the time from the first occurrence of CR, CRh, CRi, MLFS, or PR based on ELN 2022 response criteria (which, in this study, can only be assessed at D22 of cycles 2, 4, or 6), until the first documented evidence of relapsed disease or death from any cause, whichever occurs first. Participants who do not achieve CR, CRh, Cri, MLFS, or PR at one of their cycle 2, 4, or 6 assessments will be excluded from this analysis.
Measured: Cycle 1 Day 1 (each cycle is 28 days) until the first documented evidence of relapsed disease or death from any cause, or up to 6 months whichever occurs first
Overall Survival (OS)
Overall Survival (OS) is defined as the time from C1D1 to the date of death, regardless of the actual cause of the participant's death. For participants who are still alive at the time of data analysis or who are lost to follow-up, OS time will be censored at the last recorded date that the participant is known to be alive as of the data cut-off date for the analysis.
Measured: Cycle 1 Day 1 (each cycle is 28 days) to the date of death, regardless of the actual cause of death, or or up to 5 years, whichever comes first

Timeline

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

2 Jan 202615 Jun 2035

First posted6 Jan 2026
Study start2 Jan 2026
Primary completion17 Jun 2030 (estimated)
Study completion15 Jun 2035 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (25)Papers the sponsor has linked to this study.
Other trials for Relapsed Acute Myeloid LeukemiaStudies of the same condition, open and under way.
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 1
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Model description: 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.
Masking: None (open label)
Enrolment: 36 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT07320235
Sponsor's protocol ID: STUDY-25-01212
Other IDs: PRMC-25-108
Acronym: IMAGINE
Status last verified: Apr 2026
First submitted: 9 Dec 2025
Last updated: 22 Apr 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No: IPD sharing plan is Undecided.

Publications

Zeidan AM, Platzbecker U, Bewersdorf JP, Stahl M, Ades L, Borate U, Bowen D, Buckstein R, Brunner A, Carraway HE, Daver N, Diez-Campelo M, de Witte T, DeZern AE, Efficace F, Garcia-Manero G, Garcia JS, Germing U, Giagounidis A, Griffiths EA, Hasserjian RP, Hellstrom-Lindberg E, Iastrebner M, Komrokji R, Kulasekararaj AG, Malcovati L, Miyazaki Y, Odenike O, Santini V, Sanz G, Scheinberg P, Stauder R, van de Loosdrecht AA, Wei AH, Sekeres MA, Fenaux P. Consensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes. Blood. 2023 Apr 27;141(17):2047-2061. doi: 10.1182/blood.2022018604.
PubMed 36724453
Hayano RS, Adachi R. Estimation of the total population moving into and out of the 20 km evacuation zone during the Fukushima NPP accident as calculated using "Auto-GPS" mobile phone data. Proc Jpn Acad Ser B Phys Biol Sci. 2013;89(5):196-9. doi: 10.2183/pjab.89.196.
PubMed 23666090
Antar AI, Otrock ZK, Jabbour E, Mohty M, Bazarbachi A. FLT3 inhibitors in acute myeloid leukemia: ten frequently asked questions. Leukemia. 2020 Mar;34(3):682-696. doi: 10.1038/s41375-019-0694-3. Epub 2020 Jan 9.
PubMed 31919472
DiNardo CD, Wei AH. How I treat acute myeloid leukemia in the era of new drugs. Blood. 2020 Jan 9;135(2):85-96. doi: 10.1182/blood.2019001239.
PubMed 31765470
DiNardo CD, Jonas BA, Pullarkat V, Thirman MJ, Garcia JS, Wei AH, Konopleva M, Dohner H, Letai A, Fenaux P, Koller E, Havelange V, Leber B, Esteve J, Wang J, Pejsa V, Hajek R, Porkka K, Illes A, Lavie D, Lemoli RM, Yamamoto K, Yoon SS, Jang JH, Yeh SP, Turgut M, Hong WJ, Zhou Y, Potluri J, Pratz KW. Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia. N Engl J Med. 2020 Aug 13;383(7):617-629. doi: 10.1056/NEJMoa2012971.
PubMed 32786187
Maiti A, Rausch CR, Cortes JE, Pemmaraju N, Daver NG, Ravandi F, Garcia-Manero G, Borthakur G, Naqvi K, Ohanian M, Short NJ, Alvarado Y, Kadia TM, Takahashi K, Yilmaz M, Jain N, Kornblau S, Montalban Bravo G, Sasaki K, Andreeff M, Bose P, Ferrajoli A, Issa GC, Jabbour EJ, Masarova L, Thompson PA, Wang S, Konoplev S, Pierce SA, Ning J, Qiao W, Welch JS, Kantarjian HM, DiNardo CD, Konopleva MY. Outcomes of relapsed or refractory acute myeloid leukemia after frontline hypomethylating agent and venetoclax regimens. Haematologica. 2021 Mar 1;106(3):894-898. doi: 10.3324/haematol.2020.252569. No abstract available.
PubMed 32499238
Feld J, Tremblay D, Dougherty M, Czaplinska T, Sanchez G, Brady C, Kremyanskaya M, Bar-Natan M, Keyzner A, Marcellino BK, Gabrilove J, Navada SC, Silverman LR, El Jamal SM, Mascarenhas J, Shih AH. Safety and Efficacy: Clinical Experience of Venetoclax in Combination With Hypomethylating Agents in Both Newly Diagnosed and Relapsed/Refractory Advanced Myeloid Malignancies. Hemasphere. 2021 Mar 9;5(4):e549. doi: 10.1097/HS9.0000000000000549. eCollection 2021 Apr.
PubMed 33718803
Pollyea DA, Bixby D, Perl A, Bhatt VR, Altman JK, Appelbaum FR, de Lima M, Fathi AT, Foran JM, Gojo I, Hall AC, Jacoby M, Lancet J, Mannis G, Marcucci G, Martin MG, Mims A, Neff J, Nejati R, Olin R, Percival ME, Prebet T, Przespolewski A, Rao D, Ravandi-Kashani F, Shami PJ, Stone RM, Strickland SA, Sweet K, Vachhani P, Wieduwilt M, Gregory KM, Ogba N, Tallman MS. NCCN Guidelines Insights: Acute Myeloid Leukemia, Version 2.2021. J Natl Compr Canc Netw. 2021 Jan 6;19(1):16-27. doi: 10.6004/jnccn.2021.0002.
PubMed 33406488
HAYFLICK L, MOORHEAD PS. The serial cultivation of human diploid cell strains. Exp Cell Res. 1961 Dec;25:585-621. doi: 10.1016/0014-4827(61)90192-6. No abstract available.
PubMed 13905658
Bodnar AG, Ouellette M, Frolkis M, Holt SE, Chiu CP, Morin GB, Harley CB, Shay JW, Lichtsteiner S, Wright WE. Extension of life-span by introduction of telomerase into normal human cells. Science. 1998 Jan 16;279(5349):349-52. doi: 10.1126/science.279.5349.349.
PubMed 9454332
Harley CB, Futcher AB, Greider CW. Telomeres shorten during ageing of human fibroblasts. Nature. 1990 May 31;345(6274):458-60. doi: 10.1038/345458a0.
PubMed 2342578
Diebold J, Raphael M, Prevot S, Audouin J. Lymphomas associated with HIV infection. Cancer Surv. 1997;30:263-93.
PubMed 9547997
McEachern MJ, Krauskopf A, Blackburn EH. Telomeres and their control. Annu Rev Genet. 2000;34:331-358. doi: 10.1146/annurev.genet.34.1.331.
PubMed 11092831
Harley CB. Telomerase is not an oncogene. Oncogene. 2002 Jan 21;21(4):494-502. doi: 10.1038/sj.onc.1205076.
PubMed 11850774
Wright WE, Piatyszek MA, Rainey WE, Byrd W, Shay JW. Telomerase activity in human germline and embryonic tissues and cells. Dev Genet. 1996;18(2):173-9. doi: 10.1002/(SICI)1520-6408(1996)18:23.0.CO;2-3.
PubMed 8934879

Interested in taking part?

Gabriela Bello

(212) 241-0463

gabriela.bello@mssm.edu

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See sites and contacts

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All Relapsed Acute Myeloid Leukemia trials

Study at a glance

From the registry record, last updated by the sponsor on 22 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: IMetelstat and Azacitidine With or Without Venetoclax GIveN in rElapsed Acute Myeloid Leukemia (IMAGINE Trial)
ClinicalTrials.gov ID: NCT07320235
Acronym / sponsor ID: IMAGINE / STUDY-25-01212
Conditions: Relapsed Acute Myeloid Leukemia
Keywords: IMAGINE Trial, Acute Myeloid Leukemia, AML, Relapsed Acute Myeloid Leukemia, Imetelstat, Imetelstat and Azacitidine
Phase: Phase 1
Study type: Interventional
Recruitment status: Recruiting
Enrolment: 36 participants (estimated)
Lead sponsor: Douglas TremblayUniversity, hospital or other
Collaborators: Geron Corporation
Study start: 2 Jan 2026 (actual)
Primary completion: 17 Jun 2030 (estimated)
Study completion: 15 Jun 2035 (estimated)
First posted: 6 Jan 2026 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 2
Model description: 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.

Arm	Type	Receives
Part A Safety Run-In phase: Imetelstat in combination with Azacitidine	Active comparator	Drug: Imetelstat, Drug: AzacitidinePart A combination therapy: * Imetelstat will be administered on Day 1 of each 28-day cycle * Azacitidine once daily for Days 1 (+/- 1 day) through 7 (+/- 1 day) of each 28-day cycle.
Part B Combination Therapy: Imetelstat in combination with Azacitidine with or without Venetoclax	Active comparator	–* Imetelstat will be administered on Day 1 of each 28-day cycle * Azacitidine once daily for Days 1 (+/- 1 day) through 7 (+/- 1 day) of each 28-day cycle. * Venetoclax once daily for Days 1 (+/- 1 day) through 14 (+/- 1 day) of each 28-day cycle.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

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
ImetelstatAlso: GRN163LThree 3 dose levels administered on Day 1 of each 28-day cycle for Safety Run-in Phase and optimal dose to be administered on Day 1 of each 28-day cycle	Drug	Part A Safety Run-In phase: Imetelstat in combination with Azacitidine
Azacitidine75mg/m2 IV or subcutaneous (SQ) once daily for Days 1 (+/- 1 day) through 7 (+/- 1 day) of each 28-day cycle. Azacitidine can be administered locally as long as documentation of administration is provided to the study team.	Drug	Part A Safety Run-In phase: Imetelstat in combination with Azacitidine
VenetoclaxVenetoclax 400mg oral once daily for Days 1 (+/- 1 day) through 14 (+/- 1 day) of each 28-day cycle. Venetoclax reduced dosey should be substituted if concomitant posaconazole, or if other concomitant strong CYP3A4 inhibitor (e.g. voriconazole) or 200mg for moderate CYP3A4 inhibitor (e.g. isavuconazole). For Cycle 1, participants will receive a venetoclax ramp-up dose on day 1, day 2, and days 3-14 of venetoclax,. If the participant is being treated with concomitant posaconazole, or other concomitant strong CYP3A4 inhibitor, or with a concomitant moderate CYP3A4 inhibitor or a pg-p inhibitor, participants will receive a lower ramp-up dose on day 1, day 2, and days 3-14 of venetoclax.	Drug	–

Primary outcome

Dose Limiting Toxicity (DLT)
Time frame: Cycle 1 Day 1 up to Cycle 1 Day 28 (each cycle is 28 days), for 6 cycles
The DLT-evaluable population includes participants in the Part A portion of the trial who received at least one dose of imetelstat and azacitidine and either experienced a Dose-Limiting Toxicity (DLT) during the first-cycle DLT assessment period or completed their first-cycle DLT assessment.

4 secondary outcomes

Optimal Biological Dose (OBD)
Time frame: Cycle 1 Day 1 up to Cycle 1 Day 28 (each cycle is 28 days), for 6 cycles
Disease-Free Survival (DFS)
Time frame: Cycle 1 Day 1 (each cycle is 28 days) until the first documented evidence of relapsed disease or death from any cause, or up to 6 months. whichever occurs first
Duration of Response (DOR)
Time frame: Cycle 1 Day 1 (each cycle is 28 days) until the first documented evidence of relapsed disease or death from any cause, or up to 6 months whichever occurs first
Overall Survival (OS)
Time frame: Cycle 1 Day 1 (each cycle is 28 days) to the date of death, regardless of the actual cause of death, or or up to 5 years, whichever comes first

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2026.
Background: PubMed 36724453Zeidan AM, Platzbecker U, Bewersdorf JP, Stahl M, Ades L, Borate U, Bowen D, Buckstein R, Brunner A, Carraway HE, Daver N, Diez-Campelo M, de Witte T, DeZern AE, Efficace F, Garcia-Manero G, Garcia JS, Germing U, Giagounidis A, Griffiths EA, Hasserjian RP, Hellstrom-Lindberg E, Iastrebner M, Komrokji R, Kulasekararaj AG, Malcovati L, Miyazaki Y, Odenike O, Santini V, Sanz G, Scheinberg P, Stauder R, van de Loosdrecht AA, Wei AH, Sekeres MA, Fenaux P. Consensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes. Blood. 2023 Apr 27;141(17):2047-2061. doi: 10.1182/blood.2022018604.
Background: PubMed 23666090Hayano RS, Adachi R. Estimation of the total population moving into and out of the 20 km evacuation zone during the Fukushima NPP accident as calculated using "Auto-GPS" mobile phone data. Proc Jpn Acad Ser B Phys Biol Sci. 2013;89(5):196-9. doi: 10.2183/pjab.89.196.
Background: PubMed 31919472Antar AI, Otrock ZK, Jabbour E, Mohty M, Bazarbachi A. FLT3 inhibitors in acute myeloid leukemia: ten frequently asked questions. Leukemia. 2020 Mar;34(3):682-696. doi: 10.1038/s41375-019-0694-3. Epub 2020 Jan 9.
Background: PubMed 31765470DiNardo CD, Wei AH. How I treat acute myeloid leukemia in the era of new drugs. Blood. 2020 Jan 9;135(2):85-96. doi: 10.1182/blood.2019001239.
Background: PubMed 32786187DiNardo CD, Jonas BA, Pullarkat V, Thirman MJ, Garcia JS, Wei AH, Konopleva M, Dohner H, Letai A, Fenaux P, Koller E, Havelange V, Leber B, Esteve J, Wang J, Pejsa V, Hajek R, Porkka K, Illes A, Lavie D, Lemoli RM, Yamamoto K, Yoon SS, Jang JH, Yeh SP, Turgut M, Hong WJ, Zhou Y, Potluri J, Pratz KW. Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia. N Engl J Med. 2020 Aug 13;383(7):617-629. doi: 10.1056/NEJMoa2012971.
Background: PubMed 32499238Maiti A, Rausch CR, Cortes JE, Pemmaraju N, Daver NG, Ravandi F, Garcia-Manero G, Borthakur G, Naqvi K, Ohanian M, Short NJ, Alvarado Y, Kadia TM, Takahashi K, Yilmaz M, Jain N, Kornblau S, Montalban Bravo G, Sasaki K, Andreeff M, Bose P, Ferrajoli A, Issa GC, Jabbour EJ, Masarova L, Thompson PA, Wang S, Konoplev S, Pierce SA, Ning J, Qiao W, Welch JS, Kantarjian HM, DiNardo CD, Konopleva MY. Outcomes of relapsed or refractory acute myeloid leukemia after frontline hypomethylating agent and venetoclax regimens. Haematologica. 2021 Mar 1;106(3):894-898. doi: 10.3324/haematol.2020.252569. No abstract available.
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Individual participant dataSharing planned: No. IPD sharing plan is Undecided.

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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.

Gabriela Bello contact

(212) 241-0463

gabriela.bello@mssm.edu

Rashmi Unawane contact

212-824-2385

rashmi.unawane@mssm.edu

Douglas Tremblay, MD principal investigator

Icahn School of Medicine at Mount Sinai

John Mascarenhas, MD study chair

Icahn School of Medicine at Mount Sinai

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Imetelstat, Azacitidine and Venetoclax in people with Relapsed Acute Myeloid Leukemia, run by Douglas Tremblay 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?

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: looking for participants now, as of the sponsor's last update on 22 Apr 2026. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Gabriela Bello ((212) 241-0463, gabriela.bello@mssm.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Dose Limiting Toxicity (DLT) (time frame: Cycle 1 Day 1 up to Cycle 1 Day 28 (each cycle is 28 days), for 6 cycles). 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 NCT07320235, which also lists 25 linked publications.

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 22 Apr 2026.