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
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 Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
100
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
City of Hope
Duarte, California, United States
RECRUITINGMoffitt Cancer Center
Tampa, Florida, United States
RECRUITINGMassachusetts General Hospital
Boston, Massachusetts, United States
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.
Time frame: 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
Time frame: Assessed at the end of Cycle 1 (each cycle is 28 days) for each participant for approximately 24 months
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.
Time frame: 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.
Time frame: 24 months
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Memorial Sloan Kettering
New York, New York, United States
RECRUITINGMD Anderson Cancer Center
Houston, Texas, United States
RECRUITINGCentre Hospitalier Universitaire (CHU) de Bordeaux
Bordeaux, France
RECRUITINGAP-HP - Hôpital Saint-Louis
Paris, France
RECRUITINGIUCT-Oncopole
Toulouse, France
RECRUITINGInstitut de Cancerologie Gustave-Roussy
Villejuif, France
RECRUITINGAML: 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.
Time frame: 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.
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
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.
Time frame: 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
Time frame: 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.
Time frame: 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
Time frame: 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.
Time frame: 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
Time frame: 24 months
Determine pharmacokinetic profile (Cmax) of REM-422
Measure Maximal concentration (Cmax) of REM-422
Time frame: 24 months
Determine pharmacokinetic profile (Cmin) of REM-422
Measure Minimal concentration (Cmin) of REM-422
Time frame: 24 months
Determine pharmacokinetic profile (Tmax) of REM-422
Measure Time to peak drug concentration (Tmax) of REM-422
Time frame: 24 months
Determine pharmacokinetic profile (AUC) of REM-422
Measure Area Under the Curve (AUC) of REM-422
Time frame: 24 months