RECRUITING

Study of REM-422 in Patients with AML or Higher Risk MDS

Conditions

Myelodysplastic Syndromes Higher Risk Myelodysplastic Syndromes Acute Myeloid Leukemia Acute Myeloid Leukemia Refractory

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

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

Quick Facts

Study Start:2024-04-26

Study Completion:2027-06-15

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06297941

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Be able to provide informed consent. 2. Be 18 or older at the time of informed consent. 3. Disease criteria: 1. 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. 2. 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). 4. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1. 5. Has agreed to undergo serial blood and bone marrow sampling. 6. 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. 7. Toxicities from prior therapy must be either stable or recovered to ≤ Grade 1. 8. Participants must be able to swallow and retain oral medications. 9. Oxygen saturation \> 92% on room air or up to 2 L/min supplemental oxygen by nasal cannula with ≤ Grade 1 dyspnea. 10. People of childbearing potential (POCBP) must have a negative serum beta-human chorionic gonadotropin test result. 11. 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. 12. 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. 13. Adequate organ function and laboratory parameters	1. Active central nervous system (CNS) leukemia or a confirmed diagnosis of CNS leukemia. 2. 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). 3. Has immediate, life-threatening, severe complications of leukemia, such as uncontrolled bleeding, pneumonia with hypoxia or sepsis, and/or disseminated intravascular coagulation. 4. Known hypersensitivity or contraindication to any component of REM-422 or to drugs chemically related to REM-422 or its excipients. 5. 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). 6. Evidence of active HIV infection. 7. Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection. 8. Primary immunodeficiency. 9. Current or expected need for daily systemic corticosteroid therapy ≥ 10 mg of prednisone equivalent. 10. Live vaccine ≤ 6 weeks prior to the start of REM-422. 11. Use of strong CYP3A inhibitors (except azole antifungals) or CYP3A inducers 12. 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. 13. Currently pregnant, have intentions to become pregnant during the study duration, or are currently lactating. 14. Has dysphagia, short-gut syndrome, gastroparesis, or any other condition that limits the ingestion or gastrointestinal absorption of orally administered drugs. 15. Current use of prohibited medication ≤ 1 week before starting REM-422. 16. Clinically significant cardiovascular disease: 17. 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. 18. History of organ transplant that requires use of immunosuppressive agents. 19. History or current autoimmune disease requiring systemic treatment (eg, Crohn's disease, ulcerative colitis, rheumatoid arthritis, systemic lupus). 20. Radiation therapy ≤ 7 days prior to the start of REM-422. 21. 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. 22. Receiving any other investigational treatment for any indication ≤ 3 weeks prior to enrollment. 23. Unwillingness or inability to follow protocol requirements. 24. 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.

Inclusion Criteria

Exclusion Criteria

1. Be able to provide informed consent.
2. Be 18 or older at the time of informed consent.
3. Disease criteria:
1. 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.
2. 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).
4. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
5. Has agreed to undergo serial blood and bone marrow sampling.
6. 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.
7. Toxicities from prior therapy must be either stable or recovered to ≤ Grade 1.
8. Participants must be able to swallow and retain oral medications.
9. Oxygen saturation \> 92% on room air or up to 2 L/min supplemental oxygen by nasal cannula with ≤ Grade 1 dyspnea.
10. People of childbearing potential (POCBP) must have a negative serum beta-human chorionic gonadotropin test result.
11. 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.
12. 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.
13. Adequate organ function and laboratory parameters

1. Active central nervous system (CNS) leukemia or a confirmed diagnosis of CNS leukemia.
2. 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).
3. Has immediate, life-threatening, severe complications of leukemia, such as uncontrolled bleeding, pneumonia with hypoxia or sepsis, and/or disseminated intravascular coagulation.
4. Known hypersensitivity or contraindication to any component of REM-422 or to drugs chemically related to REM-422 or its excipients.
5. 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).
6. Evidence of active HIV infection.
7. Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection.
8. Primary immunodeficiency.
9. Current or expected need for daily systemic corticosteroid therapy ≥ 10 mg of prednisone equivalent.
10. Live vaccine ≤ 6 weeks prior to the start of REM-422.
11. Use of strong CYP3A inhibitors (except azole antifungals) or CYP3A inducers
12. 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.
13. Currently pregnant, have intentions to become pregnant during the study duration, or are currently lactating.
14. Has dysphagia, short-gut syndrome, gastroparesis, or any other condition that limits the ingestion or gastrointestinal absorption of orally administered drugs.
15. Current use of prohibited medication ≤ 1 week before starting REM-422.
16. Clinically significant cardiovascular disease:
17. 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.
18. History of organ transplant that requires use of immunosuppressive agents.
19. History or current autoimmune disease requiring systemic treatment (eg, Crohn's disease, ulcerative colitis, rheumatoid arthritis, systemic lupus).
20. Radiation therapy ≤ 7 days prior to the start of REM-422.
21. 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.
22. Receiving any other investigational treatment for any indication ≤ 3 weeks prior to enrollment.
23. Unwillingness or inability to follow protocol requirements.
24. 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.

Contacts and Locations

Study Contact

Remix Therapeutics

CONTACT

781-827-0902

ClinicalTrials@remixtx.com

Rosalie Jiang

CONTACT

781-584-9390

rjiang@remixtx.com

Principal Investigator

Christopher Bowden, MD

STUDY_CHAIR

Remix Therapeutics

Study Locations (Sites)

City of Hope

Duarte, California, 91010

United States

Moffitt Cancer Center

Tampa, Florida, 33612

United States

Massachusetts General Hospital

Boston, Massachusetts, 02114

United States

Memorial Sloan Kettering

New York, New York, 10065

United States

MD Anderson Cancer Center

Houston, Texas, 77030

United States

Collaborators and Investigators

Sponsor: Remix Therapeutics

Christopher Bowden, MD, STUDY_CHAIR, Remix Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-04-26

Study Completion Date2027-06-15

Study Record Updates

Study Start Date2024-04-26

Study Completion Date2027-06-15

Terms related to this study

Additional Relevant MeSH Terms

Myelodysplastic Syndromes
Higher Risk Myelodysplastic Syndromes
Acute Myeloid Leukemia
Acute Myeloid Leukemia Refractory