Myelodysplastic SyndromesPhase 1NCT06502145
Iadademstat With Hypomethylating Agent in Patients With Myelodysplastic Syndrome
A Phase 1 study testing Azacitidine Level -1, Azacitidine Level 0 and Azacitidine Level 1 in people with Myelodysplastic Syndromes, run by Medical College of Wisconsin at 1 site in United States.
Trial summary
This is a phase I study with a primary objective of determining the recommended phase II dose of iadademstat with azacitidine in adult subjects with myelodysplastic syndrome (MDS). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Azacitidine Level -1, Azacitidine Level 0 and Azacitidine Level 1
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Jan 2025to 31 Dec 2027
- 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
Who may be able to join
- Male or female 18 years or older.
- Patients must have a diagnosis of myelodysplastic syndrome (MDS), or MDS / myeloproliferative neoplasm (MPN), chronic myelomonocytic leukemia (CMML) as defined by the World Health Organization (WHO) criteria.
- Intermediate, high, or very-high risk by the Revised International Prognostic Scoring System (IPSS-R).
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 (0-3 if performance status is considered due to MDS).
- Patient must have a body weight of at least 50 kg.
- Patient must meet the following screening clinical laboratory values as specified below:
Show all 21Show fewer
- Hematologic: white blood cell (count) (WBC) below 30 x 109/L. Hydroxyurea can be used to achieve this level b. Hepatic: i. Total bilirubin ≤1.5 x upper limit of normal (ULN). Patients with Gilbert's syndrome can enroll if conjugated bilirubin is within normal limits.
- Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3 x ULN.
- Renal: Serum creatinine ≤1.5 x ULN.
- Patient is able to swallow oral medications.
- Female subjects who:
- Are postmenopausal for at least one year before the screening visit, OR
- Are surgically sterile, OR
- If they are of childbearing potential:
- Agree to practice one highly effective method and one additional effective (barrier) method of contraception, at the same time, from the time of signing the informed consent through six months after the last dose of study drug (female and male condoms should not be used together), OR ii. Agree to practice true abstinence, when this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [e.g., calendar, ovulation, symptothermal, post-ovulation methods], withdrawal, spermicides only, and lactational amenorrhea are not acceptable methods of contraception).
- Agree to not to donate or freeze egg(s) during the course of this study or within 180 days after receiving their last dose of study drug.
- Male subjects, even if surgically sterilized (i.e., status post vasectomy), who:
- Agree to practice effective barrier contraception during the entire study treatment period from the time of signing the informed consent through and through six months after the last dose of study drug (female and male condoms should not be used together), OR
- Agree to practice true abstinence, when this is in line with the preferred and usual lifestyle of the subject. (periodic abstinence [e.g., calendar, ovulation, symptothermal, post-ovulation methods for the female partner] withdrawal, spermicides only, and lactational amenorrhea are not acceptable methods of contraception.)
- Agree to not to donate or freeze sperm during the course of this study or within 180 days after receiving their last dose of study drug.
- Ability to provide informed consent or have a legally authorized representative provide consent.
Who cannot join
- A potential study subject who meets any of the following exclusion criteria is ineligible to participate in the study:
- Prior therapy with hypomethylating agent or Lysine-specific histone demethylase 1A (LSD1) inhibitor. If patients were treated with any other agents having KDM1A/LSD1 inhibitory activity (such as tranylcypromine or phenelzine or similar drugs), they are only allowed if treatment finalized at least 3 weeks prior to first dose on study.
- Patient will require treatment while on study with concomitant drugs that target the 5- hydroxytryptamine2B (5-HT2B) receptor or the sigma nonspecific receptor (e.g., escitalopram, fluoxetine, sertraline) except for drugs that are considered absolutely essential for the care of the patient and with appropriate treatment monitoring.
- Treatment with systemic antineoplastic chemotherapy within 14 days or 5 half-lives from the last dose - whichever is sooner - before Cycle 1, Day 1 of therapy. Radiation within 14 days before Cycle 1, Day 1 of therapy. The use of hydroxyurea for leukoreduction is permitted. Similarly, prior ESA, luspatarcept, or lenalidomide is allowed. Subjects must have recovered from the side effects of prior therapy per the treating physician's discretion.
- Patient is on treatment with any investigational products within 3 weeks prior to the first dose of study treatment.
- Patient has had major surgery within 4 weeks prior to the first study dose.
Show all 18Show fewer
- Patients with uncontrolled hypertension (i.e., systolic blood pressure >180 mm Hg, diastolic blood pressure >95 mm Hg). Use of anti-hypertensive agents to control hypertension before Cycle 1, Day 1 is allowed.
- Patients with known poorly controlled diabetes (glycosylated hemoglobin (HbA1c) ≥8%) unless actively managed with appropriate therapy; patients with a history of transient glucose intolerance due to corticosteroid administration may be enrolled in this study if all other inclusion/exclusion criteria are met.
- Patient has known active congestive heart failure New York Heart Association (NYHA) class 3 or 4 or patients with a history of congestive heart failure NYHA class 3 or 4 in the past, unless a screening echocardiogram performed within 1 month prior to study entry demonstrates a left ventricular ejection fraction that is ≥40%.
- Patients with known long QT Syndrome at screening.
- History of allogeneic stem cell transplantation or CAR-T therapy within past 60 days.
- Patient has evidence of active uncontrolled viral, bacterial, or systemic fungal infection (i.e., no signs of severe systemic inflammatory response that makes patient clinically unstable in the opinion of the investigator, and patient is hemodynamically stable, with sustained body temperature under 38°C for 48-72 hours before starting study treatment and does not need oxygen supplementation or pressors to maintain blood pressure). Patients with uncontrolled infection shall not be enrolled until the infection is treated and brought under control.
- Manifestations of malabsorption due to prior gastrointestinal (GI) surgery or GI disease that may alter the absorption of iadademstat.
- Pregnant or lactating / breastfeeding women.
- Patient has any condition which, in the investigator's opinion, makes the patient unsuitable for study participation. For example, any inter-current illness or social situation that will limit compliance with study requirements. Any serious underlying medical or psychiatric condition (e.g., alcohol or drug abuse), dementia or altered mental status or any issue that would impair the ability of the patient to understand the informed consent form or that in the opinion of the investigator would contraindicate the patient's participation in the study or confound the results of the study.
- Patient presents a known contraindication to any of the treatment drug excipients.
- Patient has known active hepatic disorder or is known to be positive for hepatitis B or C infection with the exception of those with undetectable viral load within 3 months (Hepatitis B or C testing is not required for eligibility assessment).
- Patient is known to have human immunodeficiency virus infection. (HIV testing is not required for eligibility assessment).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Medical College of Wisconsin Cancer Center Clinical Trials Office contact
United States1 site, 1 recruiting
- Recruiting
Froedtert Hospital & the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Guru Subramanian Guru Murthy, MD, MS, gmurthy@mcw.edu
Guru Subramanian Guru Murthy, MD, MS
About this study
This is a phase I study with a primary objective of determining the recommended phase II dose of iadademstat with azacitidine in adult subjects with myelodysplastic syndrome (MDS).
Read the detailed description
This is a single-arm, open-label phase 1 study designed to evaluate the safety of iadademstat with azacitidine therapy. The trial will follow a 3+3 phase 1 dose-escalation design.
First, three participants are given a low dose of the experimental treatment and monitored for pre-specified toxicity events. If 0 participants experience one of these toxicity events, then the next group of three participants is enrolled at a higher dose. If two or three participants experience toxicity, then the next group of three participants is enrolled at a lower dose (or the study ends). If one participant experiences toxicity, another group of three participants is enrolled at the same dose (hence the name, 3+3): if one or more of those participants experience toxicity, then the dose is lowered for the next group of the study ends. Otherwise, if 0 of the additional participants experience toxicity, the next group is enrolled at a higher dose.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Azacitidine Level -1, Azacitidine Level 0, Azacitidine Level 1, Iadademstat Level -1, Iadademstat Level 0
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Azacitidine Level -1Drug
Also called Vidaza, Onureg
Intravenous (IV) or subcutaneous (SC) 75 mg / m^2 days 1-7 X 28 days.
Azacitidine Level 0Drug
Also called Vidaza, Onureg
Intravenous (IV) or subcutaneous (SC) 75 mg / m^2 days 1-7 X 28 days.
Azacitidine Level 1Drug
Also called Vidaza, Onureg
Intravenous (IV) or subcutaneous (SC) 75 mg / m^2 days 1-7 X 28 days.
Iadademstat Level -1Drug
75 µg by mouth (PO) 5 days on, 2 days off for 2 weeks every 28 days.
Iadademstat Level 0Drug
75 µg PO 5 days on, 2 days off for 3 weeks every 28 days.
Iadademstat Level 1Drug
100 µg PO 5 days on, 2 days off for 3 weeks every 28 days.
| Study group | Role | What they receive |
|---|---|---|
| Dose level -1 | Experimental | Dose level -1 will be considered only if there are dose-limiting toxicities at dose level 0. This is a regimen of azacitidine and iadademstat. Receives: Drug: Azacitidine Level -1; Drug: Iadademstat Level -1 |
| Dose level 0 | Experimental | This is a regimen of azacitidine and iadademstat. Receives: Drug: Azacitidine Level 0; Drug: Iadademstat Level 0 |
| Dose level 1 | Experimental | This is a regimen of azacitidine and iadademstat. Receives: Drug: Azacitidine Level 1; Drug: Iadademstat Level 1 |
| Maximum Tolerated Dose (MTD) | Experimental | The MTD is the highest dose level at which no more than 1 of 6 treated participants, experiences a dose-limiting toxicity. Receives: Drug: Iadademstat Level -1; Drug: Iadademstat Level 0; Drug: Iadademstat Level 1 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recommended phase II dose of Iadademstat.
The dose identified in this trial that will be used in future clinical trials.
Measured: Up to two years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2024
- Study start10 Jan 2025
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Medical College of WisconsinThe sponsor's other registered studies.
- 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
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Medical College of Wisconsin
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Guru Subramanian Guru Murthy, Medical College of Wisconsin
- Investigators
- Guru Subramanian Guru Murthy, MD, MS, Principal investigator, Medical College of Wisconsin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06502145
- Sponsor's protocol ID
- PRO00053184
- Status last verified
- Mar 2026
- First submitted
- 9 Jul 2024
- Last updated
- 3 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Iadademstat With Hypomethylating Agent in Patients With Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT06502145
- Acronym / sponsor ID
- PRO00053184
- Conditions
- Myelodysplastic Syndromes
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Medical College of WisconsinUniversity, hospital or other
- Study start
- 10 Jan 2025 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 16 Jul 2024 (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.
This is a phase I study with a primary objective of determining the recommended phase II dose of iadademstat with azacitidine in adult subjects with myelodysplastic syndrome (MDS).
Detailed description
This is a single-arm, open-label phase 1 study designed to evaluate the safety of iadademstat with azacitidine therapy. The trial will follow a 3+3 phase 1 dose-escalation design.
First, three participants are given a low dose of the experimental treatment and monitored for pre-specified toxicity events. If 0 participants experience one of these toxicity events, then the next group of three participants is enrolled at a higher dose. If two or three participants experience toxicity, then the next group of three participants is enrolled at a lower dose (or the study ends). If one participant experiences toxicity, another group of three participants is enrolled at the same dose (hence the name, 3+3): if one or more of those participants experience toxicity, then the dose is lowered for the next group of the study ends. Otherwise, if 0 of the additional participants experience toxicity, the next group is enrolled at a higher dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Dose level -1 | Experimental | Drug: Azacitidine Level -1, Drug: Iadademstat Level -1Dose level -1 will be considered only if there are dose-limiting toxicities at dose level 0. This is a regimen of azacitidine and iadademstat. |
| Dose level 0 | Experimental | Drug: Azacitidine Level 0, Drug: Iadademstat Level 0This is a regimen of azacitidine and iadademstat. |
| Dose level 1 | Experimental | Drug: Azacitidine Level 1, Drug: Iadademstat Level 1This is a regimen of azacitidine and iadademstat. |
| Maximum Tolerated Dose (MTD) | Experimental | Drug: Iadademstat Level -1, Drug: Iadademstat Level 0, Drug: Iadademstat Level 1The MTD is the highest dose level at which no more than 1 of 6 treated participants, experiences a dose-limiting toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- Male or female 18 years or older.
- Patients must have a diagnosis of myelodysplastic syndrome (MDS), or MDS / myeloproliferative neoplasm (MPN), chronic myelomonocytic leukemia (CMML) as defined by the World Health Organization (WHO) criteria.
- Intermediate, high, or very-high risk by the Revised International Prognostic Scoring System (IPSS-R).
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 (0-3 if performance status is considered due to MDS).
- Patient must have a body weight of at least 50 kg.
Key exclusion criteria (18 listed)
- A potential study subject who meets any of the following exclusion criteria is ineligible to participate in the study:
- Prior therapy with hypomethylating agent or Lysine-specific histone demethylase 1A (LSD1) inhibitor. If patients were treated with any other agents having KDM1A/LSD1 inhibitory activity (such as tranylcypromine or phenelzine or similar drugs), they are only allowed if treatment finalized at least 3 weeks prior to first dose on study.
- Patient will require treatment while on study with concomitant drugs that target the 5- hydroxytryptamine2B (5-HT2B) receptor or the sigma nonspecific receptor (e.g., escitalopram, fluoxetine, sertraline) except for drugs that are considered absolutely essential for the care of the patient and with appropriate treatment monitoring.
- Treatment with systemic antineoplastic chemotherapy within 14 days or 5 half-lives from the last dose - whichever is sooner - before Cycle 1, Day 1 of therapy. Radiation within 14 days before Cycle 1, Day 1 of therapy. The use of hydroxyurea for leukoreduction is permitted. Similarly, prior ESA, luspatarcept, or lenalidomide is allowed. Subjects must have recovered from the side effects of prior therapy per the treating physician's discretion.
- Patient is on treatment with any investigational products within 3 weeks prior to the first dose of study treatment.
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 |
|---|---|---|
| Azacitidine Level -1Also: Vidaza, OnuregIntravenous (IV) or subcutaneous (SC) 75 mg / m^2 days 1-7 X 28 days. | Drug | Dose level -1 |
| Azacitidine Level 0Also: Vidaza, OnuregIntravenous (IV) or subcutaneous (SC) 75 mg / m^2 days 1-7 X 28 days. | Drug | Dose level 0 |
| Azacitidine Level 1Also: Vidaza, OnuregIntravenous (IV) or subcutaneous (SC) 75 mg / m^2 days 1-7 X 28 days. | Drug | Dose level 1 |
| Iadademstat Level -175 µg by mouth (PO) 5 days on, 2 days off for 2 weeks every 28 days. | Drug | Dose level -1, Maximum Tolerated Dose (MTD) |
| Iadademstat Level 075 µg PO 5 days on, 2 days off for 3 weeks every 28 days. | Drug | Dose level 0, Maximum Tolerated Dose (MTD) |
| Iadademstat Level 1100 µg PO 5 days on, 2 days off for 3 weeks every 28 days. | Drug | Dose level 1, Maximum Tolerated Dose (MTD) |
Primary outcome
Recommended phase II dose of Iadademstat.
Time frame: Up to two years
The dose identified in this trial that will be used in future clinical trials.
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 3 Apr 2026.
- Individual participant dataSharing planned: No
Related trials for Myelodysplastic Syndromes
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
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.
Medical College of Wisconsin Cancer Center Clinical Trials Office contact
Guru Subramanian Guru Murthy, MD, MS principal investigator
Medical College of Wisconsin
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 Azacitidine Level -1, Azacitidine Level 0 and Azacitidine Level 1 in people with Myelodysplastic Syndromes, run by Medical College of Wisconsin 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 21 inclusion and 18 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: looking for participants now, as of the sponsor's last update on 3 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 Medical College of Wisconsin Cancer Center Clinical Trials Office (866-680-0505 ext. 8900, cccto@mcw.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recommended phase II dose of Iadademstat. (time frame: Up to two years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Apr 2026.