Myelodysplastic SyndromesPhase 1NCT06297941
Study of REM-422 in Patients With AML or Higher Risk MDS
A Phase 1 study testing REM-422 in people with Myelodysplastic Syndromes and Higher Risk Myelodysplastic Syndromes, run by Remix Therapeutics at 9 sites in 2 countries.
Trial summary
The goal of this study is to determine the safety and antitumor effects of REM-422, a MYB mRNA degrader, in people with Higher Risk MDS and relapsed/refractory AML Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes, Higher Risk Myelodysplastic Syndromes, Acute Myeloid Leukemia
- What is being tested?
- REM-422
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 9 sitesin 2 countries
- Study dates
- 26 Apr 2024to 30 Mar 2028
- 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
- Be able to provide informed consent.
- Be 18 or older at the time of informed consent.
- Disease criteria:
- Histologically confirmed diagnosis of either:
- R/R AML, defined as relapse after transplantation, second or later relapse, refractory to initial induction or reinduction treatment or to initial treatment with hypomethylating (HMA)-based combinations, relapse after initial treatment, or otherwise considered relapsed or refractory in the opinion of the Investigator.
- High-risk and very-high-risk (VHR) MDS (higher-risk) per the International Prognostic Scoring System-Revised (IPSS-R) and/or International Prognostic Scoring System-Molecular (IPSS-M).
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- Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
- Has agreed to undergo serial blood and bone marrow sampling.
- Participants must have completed systemic non-investigational therapy at least 14 days prior to initiating REM-422. Hydroxyurea is permissible for controlling peripheral leukemic blasts prior to enrollment and for up to 28 days following initiation of REM-422.
- Toxicities from prior therapy must be either stable or recovered to ≤ Grade 1.
- Participants must be able to swallow and retain oral medications.
- Oxygen saturation > 92% on room air or up to 2 L/min supplemental oxygen by nasal cannula with ≤ Grade 1 dyspnea.
- People of childbearing potential (POCBP) must have a negative serum beta-human chorionic gonadotropin test result.
- POCBP must agree to use acceptable, effective methods of contraception and not donate ova from screening until 6 months after discontinuation of REM-422. Women who have undergone surgical or ablative sterilization or who have been postmenopausal for ≥ 2 years are not considered to be of childbearing potential.
- Men must agree to use acceptable, effective methods of contraception and must agree not to donate sperm from the start of receiving REM-422 until 6 months after discontinuation of REM-422.
- Adequate organ function and laboratory parameters
Who cannot join
- Active central nervous system (CNS) leukemia or a confirmed diagnosis of CNS leukemia.
- Has undergone hematopoietic stem cell transplantation (HSCT) within 60 days of the first dose of REM-422 or is receiving immunosuppressive therapy post HSCT at the time of screening, or has GVHD requiring systemic treatment (topical steroids for ongoing skin GVHD is permitted).
- Has immediate, life-threatening, severe complications of leukemia, such as uncontrolled bleeding, pneumonia with hypoxia or sepsis, and/or disseminated intravascular coagulation.
- Known hypersensitivity or contraindication to any component of REM-422 or to drugs chemically related to REM-422 or its excipients.
- Clinically significant active infection. Note: Patients with simple urinary tract infection or uncomplicated bacterial pharyngitis responding to active treatment are permitted. Note: Patients receiving intravenous (IV) antibiotics ≤ 7 days prior to enrollment are excluded (prophylactic antibiotics, antivirals, or antifungals are permitted).
- Evidence of active HIV infection.
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- Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection.
- Primary immunodeficiency.
- Current or expected need for daily systemic corticosteroid therapy ≥ 10 mg of prednisone equivalent.
- Note: Patients who are receiving topical or inhaled corticosteroids with minimal systemic absorption are eligible for enrollment and may continue with minimal corticosteroid use as long as they are on a stable dose.
- Live vaccine ≤ 2 weeks prior to the start of REM-422.
- Use of moderate or strong cytochrome P450 (CYP)3A inhibitors (including azoles that are moderate to strong CYP3A inhibitors) or CYP3A inducers < 5 half-lives or 14 days, whichever is shorter, prior to initiating REM-422 administration and during the study.
- Drugs that reduce gastric acidity, such as H2-receptor antagonists (eg, ranitidine, famotidine) and proton pump inhibitors (eg, omeprazole, esomeprazole) within 7 days prior to the initiation of REM-422 administration or during the study.
- Currently pregnant, have intentions to become pregnant during the study duration, or are currently lactating.
- Has dysphagia, short-gut syndrome, gastroparesis, or any other condition that limits the ingestion or gastrointestinal absorption of orally administered drugs.
- Current use of prohibited medication ≤ 1 week before starting REM-422.
- Clinically significant cardiovascular disease
- Has undergone major surgery (opening a mesenchymal barrier such as the pleural cavity, peritoneum, or meninges or surgical procedures requiring general anesthesia) < 4 weeks prior to enrollment.
- History of organ transplant that requires use of immunosuppressive agents.
- History or current autoimmune disease requiring systemic treatment (eg, Crohn's disease, ulcerative colitis, rheumatoid arthritis, systemic lupus).
- Radiation therapy ≤ 7 days prior to the start of REM-422.
- Concurrent or previous other malignancy ≤ 2 years of enrollment, except curatively treated malignancies including basal or squamous cell skin cancer, breast cancer, prostate intraepithelial neoplasm, and carcinoma in situ of the cervix.
- Unwillingness or inability to follow protocol requirements.
- Any condition that, in the opinion of the Investigator, would interfere with evaluation of REM-422 or interpretation of the participant's safety or study results.
Trial locations and contacts
9 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
9 sites in 2 countries
United States5 sites, 5 recruiting
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
Amir Fathi, MD
- Recruiting
City of Hope
Duarte, California, 91010
Anthony S Stein, MD
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
Courtney D DiNardo, MD
- Recruiting
Memorial Sloan Kettering
New York, New York, 10065
Eytan M Stein, MD
- Recruiting
Moffitt Cancer Center
Tampa, Florida, 33612
Onyee Chan, MD
France4 sites, 4 recruiting
- Recruiting
Centre Hospitalier Universitaire (CHU) de Bordeaux
Bordeaux
Arnaud Pigneux, MD
- Recruiting
AP-HP - Hôpital Saint-Louis
Paris
Raphael Itzykson, MD
- Recruiting
IUCT-Oncopole
Toulouse
Pierre Bories, MD
- Recruiting
Institut de Cancerologie Gustave-Roussy
Villejuif
Stephane De Botton, MD
About this study
Official title: A Phase 1, Multicenter, Open-Label Study of REM-422, an MYB mRNA Degrader, in Patients With Relapsed/Refractory AML or Higher-Risk MDS
The goal of this study is to determine the safety and antitumor effects of REM-422, a MYB mRNA degrader, in people with Higher Risk MDS and relapsed/refractory AML
Read the detailed description
This is a Phase 1, open-label, non-randomized, multicenter study investigating REM-422, a potent, selective, and oral small molecule mRNA degrader that reduces expression of the MYB transcription factor for patients with higher risk MDS or relapsed/refractory AML.
This study includes a Dose Escalation Phase and a Dose Expansion Phase. The purpose of the Dose Escalation Phase is to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) of REM-422 in patients with higher risk MDS or relapsed/refractory AML. The purpose of Dose Expansion is to further evaluate the safety and anti-tumor activity of the RP2D carried forward from Dose Escalation.
Participation in this study will continue until disease progression, therapy intolerance, or participant withdrawal.
- Conditions
- Myelodysplastic Syndromes, Higher Risk Myelodysplastic Syndromes, Acute Myeloid Leukemia, Acute Myeloid Leukemia Refractory
- Being tested
- REM-422
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
REM-422Drug
REM-422 is a first in class, small molecule mRNA inhibitor that reduces expression of the MYB transcription factor
REM-422 will be administered orally once daily
| Study group | Role | What they receive |
|---|---|---|
| REM-422 | Experimental | Dose Escalation: Participants will receive escalating doses of REM-422 to determine Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D)-422, oral capsule administered once daily
Dose Expansion: Participants will receive REM-422 at the identified RP2D Treatment will continue until disease progression, therapy intolerance, or participant withdrawal Safety evaluation will continue until 30 days of last administration of REM-422 Receives: Drug: REM-422 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency and severity of Treatment Emergent Adverse Events (TEAEs)
Frequency and severity of Treatment Emergent Adverse Events (TEAEs) will be evaluated according to the NCI-CTCAE version 5.0 and number of participants with Dose Limiting Toxicities will be assessed to determine Safety and Tolerability of REM-422 from the date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to approximately 24 months.
Measured: 24 months
Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D)
Frequency and severity of Treatment Emergent Adverse Events (TEAEs) will be evaluated according to the NCI-CTCAE version 5.0 and the number of participants with Dose Limiting Toxicities will be assessed from the date of first dose of REM-422 until the date of first documented progression or date of death from any cause, whichever came first, assessed up to approximately 24 months
Measured: Assessed at the end of Cycle 1 (each cycle is 28 days) for each participant for approximately 24 months
Secondary outcomes (14)
AML: Rate of Complete Response (remission) (CR)
Rate of Complete Response (CR) will be measured based on Investigator assessment per the modified International Working Group (IWG) response criteria 2003 from the date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to approximately 24 months.
Measured: 24 months
AML: Rate of CR with partial hematologic recovery (CRh)
Rate of Complete Response (CR) with partial hematologic recovery (CRh) will be measured based on Investigator assessment per the modified International Working Group (IWG) response criteria 2003 from the date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to approximately 24 months.
Measured: 24 months
AML: Duration of CR
Duration of CR will be evaluated per the modified International Working Group (IWG) response criteria 2003 following treatment with REM-422 from the date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to approximately 24 months.
Measured: 24 months
AML: Duration of CRh
Duration of CRh will be evaluated per the modified International Working Group (IWG) response criteria 2003 following treatment with REM-422 from the date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to approximately 24 months.
Measured: 24 months
AML: Overall response rate (ORR) (CR + CRh + CRi + PR) with incomplete hematologic recovery [CRi] + partial response [PR]) per modified IWG response criteria 2003 based on Investigator assessment
Duration of Overall Response Rate (ORR) will be evaluated per the modified International Working Group (IWG) response criteria 2003 following treatment with REM-422 from the date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to approximately 24 months.
Measured: 24 months
AML: Duration of response (DOR)
Duration of Response (DoR) will be evaluated following treatment with REM-422 based on Investigator assessment for participants who achieve a response per IWG response criteria 2003, defined as the time from when response criteria are first met for CR, CRi, CRh, or PR until progressive disease (PD) per IWG response criteria 2003 is documented or death, whichever occurs first
Measured: 24 months
MDS: Rate of Compete Response (CR)
Rate of CR will be evaluated based on Investigator assessment per IWG 2023 response criteria following treatment with REM-422 for participants who achieve a Complete Response (CR), defined as the time from when criteria are first met for CR until the date of first documented progression or date of death from any cause, whichever came first, assessed up to approximately 24 months.
Measured: 24 months
MDS: Duration of Complete Response (CR)
Duration of CR will be evaluated per IWG 2023 response criteria following treatment with REM-422 based on Investigator assessment for participants who achieve a CR per IWG response criteria 2003, defined as the time from when response criteria are first met for CR until progressive disease (PD) per IWG response criteria 2003 is documented or death, whichever occurs first
Measured: 24 months
MDS: Overall Response Rate (ORR)
ORR will be evaluated per IWG 2023 response criteria based on Investigator assessment following treatment with REM-422 from the date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to approximately 24 months.
Measured: 24 months
MDS: Duration of Response (DOR)
DOR will be evaluated following treatment with REM-422 based on Investigator assessment for participants who achieve a response per IWG 2023 response criteria, defined as the time from when criteria are first met for CR, CRi, CRh, or PR until PD per IWG 2023 or death, whichever occurs first
Measured: 24 months
Determine pharmacokinetic profile (Cmax) of REM-422
Measure Maximal concentration (Cmax) of REM-422
Measured: 24 months
Determine pharmacokinetic profile (Cmin) of REM-422
Measure Minimal concentration (Cmin) of REM-422
Measured: 24 months
Determine pharmacokinetic profile (Tmax) of REM-422
Measure Time to peak drug concentration (Tmax) of REM-422
Measured: 24 months
Determine pharmacokinetic profile (AUC) of REM-422
Measure Area Under the Curve (AUC) of REM-422
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2024
- Study start26 Apr 2024
- Primary completion31 Jul 2027 (estimated)
- Study completion30 Mar 2028 (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.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Remix Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mythili Koneru, MD, Study chair, Remix Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06297941
- Sponsor's protocol ID
- REM-422-102
- Status last verified
- Oct 2026
- First submitted
- 23 Feb 2024
- Last updated
- 29 Oct 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 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Multicenter, Open-Label Study of REM-422, an MYB mRNA Degrader, in Patients With Relapsed/Refractory AML or Higher-Risk MDS
- ClinicalTrials.gov ID
- NCT06297941
- Acronym / sponsor ID
- REM-422-102
- Conditions
- Myelodysplastic Syndromes, Higher Risk Myelodysplastic Syndromes, Acute Myeloid Leukemia, Acute Myeloid Leukemia Refractory
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Remix TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 26 Apr 2024 (actual)
- Primary completion
- 31 Jul 2027 (estimated)
- Study completion
- 30 Mar 2028 (estimated)
- First posted
- 7 Mar 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.
The goal of this study is to determine the safety and antitumor effects of REM-422, a MYB mRNA degrader, in people with Higher Risk MDS and relapsed/refractory AML
Detailed description
This is a Phase 1, open-label, non-randomized, multicenter study investigating REM-422, a potent, selective, and oral small molecule mRNA degrader that reduces expression of the MYB transcription factor for patients with higher risk MDS or relapsed/refractory AML.
This study includes a Dose Escalation Phase and a Dose Expansion Phase. The purpose of the Dose Escalation Phase is to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) of REM-422 in patients with higher risk MDS or relapsed/refractory AML. The purpose of Dose Expansion is to further evaluate the safety and anti-tumor activity of the RP2D carried forward from Dose Escalation.
Participation in this study will continue until disease progression, therapy intolerance, or participant withdrawal.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| REM-422 | Experimental | Drug: REM-422Dose Escalation: Participants will receive escalating doses of REM-422 to determine Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D)-422, oral capsule administered once daily Dose Expansion: Participants will receive REM-422 at the identified RP2D Treatment will continue until disease progression, therapy intolerance, or participant withdrawal Safety evaluation will continue until 30 days of last administration of REM-422 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Be able to provide informed consent.
- Be 18 or older at the time of informed consent.
- Disease criteria:
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
- Has agreed to undergo serial blood and bone marrow sampling.
Key exclusion criteria (24 listed)
- Active central nervous system (CNS) leukemia or a confirmed diagnosis of CNS leukemia.
- Has undergone hematopoietic stem cell transplantation (HSCT) within 60 days of the first dose of REM-422 or is receiving immunosuppressive therapy post HSCT at the time of screening, or has GVHD requiring systemic treatment (topical steroids for ongoing skin GVHD is permitted).
- Has immediate, life-threatening, severe complications of leukemia, such as uncontrolled bleeding, pneumonia with hypoxia or sepsis, and/or disseminated intravascular coagulation.
- Known hypersensitivity or contraindication to any component of REM-422 or to drugs chemically related to REM-422 or its excipients.
- Clinically significant active infection. Note: Patients with simple urinary tract infection or uncomplicated bacterial pharyngitis responding to active treatment are permitted. Note: Patients receiving intravenous (IV) antibiotics ≤ 7 days prior to enrollment are excluded (prophylactic antibiotics, antivirals, or antifungals are permitted).
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 |
|---|---|---|
| REM-422REM-422 is a first in class, small molecule mRNA inhibitor that reduces expression of the MYB transcription factor REM-422 will be administered orally once daily | Drug | REM-422 |
Primary outcome
Frequency and severity of Treatment Emergent Adverse Events (TEAEs)
Time frame: 24 months
Frequency and severity of Treatment Emergent Adverse Events (TEAEs) will be evaluated according to the NCI-CTCAE version 5.0 and number of participants with Dose Limiting Toxicities will be assessed to determine Safety and Tolerability of REM-422 from the date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to approximately 24 months.
Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D)
Time frame: Assessed at the end of Cycle 1 (each cycle is 28 days) for each participant for approximately 24 months
Frequency and severity of Treatment Emergent Adverse Events (TEAEs) will be evaluated according to the NCI-CTCAE version 5.0 and the number of participants with Dose Limiting Toxicities will be assessed from the date of first dose of REM-422 until the date of first documented progression or date of death from any cause, whichever came first, assessed up to approximately 24 months
14 secondary outcomes
AML: Rate of Complete Response (remission) (CR)
Time frame: 24 months
AML: Rate of CR with partial hematologic recovery (CRh)
Time frame: 24 months
AML: Duration of CR
Time frame: 24 months
AML: Duration of CRh
Time frame: 24 months
AML: Overall response rate (ORR) (CR + CRh + CRi + PR) with incomplete hematologic recovery [CRi] + partial response [PR]) per modified IWG response criteria 2003 based on Investigator assessment
Time frame: 24 months
AML: Duration of response (DOR)
Time frame: 24 months
MDS: Rate of Compete Response (CR)
Time frame: 24 months
MDS: Duration of Complete Response (CR)
Time frame: 24 months
MDS: Overall Response Rate (ORR)
Time frame: 24 months
MDS: Duration of Response (DOR)
Time frame: 24 months
Determine pharmacokinetic profile (Cmax) of REM-422
Time frame: 24 months
Determine pharmacokinetic profile (Cmin) of REM-422
Time frame: 24 months
Determine pharmacokinetic profile (Tmax) of REM-422
Time frame: 24 months
Determine pharmacokinetic profile (AUC) of REM-422
Time frame: 24 months
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 29 Oct 2026.
- Individual participant dataSharing planned: No
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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.
Mythili Koneru, MD study chair
Remix Therapeutics
9 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing REM-422 in people with Myelodysplastic Syndromes and Higher Risk Myelodysplastic Syndromes, run by Remix Therapeutics at 9 sites in 2 countries.
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 16 inclusion and 24 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 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, France. 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 Remix Therapeutics (781-827-0902, ClinicalTrials@remixtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency and severity of Treatment Emergent Adverse Events (TEAEs) (time frame: 24 months). The study lists 2 primary outcomes in all. 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 29 Oct 2026.