Safety and Tolerability of REGN17235 in Adult Participants With Clonal Cytopenia of Undetermined Significance and Low-Risk Myelodysplastic Syndrome With SF3B1 Mutation

Trial statusNot yet recruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorRegeneron Pharmaceuticals

About this trial

This study will test a study drug called REGN17235 (the "study drug") to see if it can help treat Clonal Cytopenia of Undetermined Significance (CCUS) and Low-Risk Myelodysplastic Syndrome (LR-MDS) with a specific genetic mutation (SF3B1 Mutation).

The study is looking at:

* What side effects the study drug might cause * How well the study drug works * How much of the study drug is in the blood at different times * If the body makes antibodies (proteins that attach to substances your body does not recognize) against the study drug; this may cause the study drug to not work as well. * What is the best dose of the study drug to treat CCUS and LR-MDS

Eligibility criteria

Qualifiers

Presence of SF3B1 mutation in the bone marrow or peripheral blood AND diagnosis of low-risk MDS OR diagnosis of CCUS as defined by WHO 2022, 5th edition as described in the protocol

Adequate bone marrow function as described in the protocol; red blood cell transfusion dependence is permitted

Adequate hepatic and renal function as described in the protocol

Disqualifiers

Clinically significant anemia due to non-MDS or non-CCUS etiologies (eg, iron deficiency, vitamin B12 or folate deficiency, autoimmune or hereditary hemolysis, or hemorrhage) diagnosed or treated within the last 3 months prior to informed consent

Recent or uncontrolled infections as described in the protocol

Diagnosed or treated for malignancy other than MDS as described in the protocol

Prior treatment with any systemic therapy for MDS or CCUS within 5 half-lives or within 14 days prior to first administration of study drug, whichever is shorter

Trial design

Treatments tested in this trial

  • REGN17235

Treatment groups

52 Participants
are divided into 2 treatment groups

Locations

This trial has no locations

Sponsors and collaborators