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Clinical Trials/NCT07753148
NCT07753148Not yet recruitingPhase 1

An Open-Label, Multi-Center Phase I Study to Investigate the Safety and Tolerability of REGN17235 in Patients With Clonal Cytopenia of Undetermined Significance and Low-Risk Myelodysplastic Syndrome With SF3B1 Mutation

Regeneron Pharmaceuticals0 sites52 target enrollmentStarted: September 17, 2026Last updated:
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Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 1
Status
Not yet recruiting
Enrollment
52
Primary Endpoint
Occurrence of Treatment Emergent Adverse Events (TEAEs)
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

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

Study Design

Study Type
Interventional
Allocation
Non Randomized
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
18 Years to — (Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • •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

Exclusion Criteria

  • •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
  • •Allogeneic hematopoietic stem cell transplant within 100 days of enrollment or any signs or symptoms of ongoing Graft-Versus Host Disease (GVHD) as described in the protocol
  • •Note: Other protocol defined Inclusion/Exclusion Criteria apply

Arms & Interventions

Part 1 Dose Escalation

Experimental

Intervention: REGN17235 (Drug)

Part 2 Safety Expansion

Experimental

Intervention: REGN17235 (Drug)

Outcomes

Primary Outcomes

Occurrence of Treatment Emergent Adverse Events (TEAEs)

Time Frame: Up to 5 years

Severity of TEAEs

Time Frame: Up to 5 years

Occurrence of Serious Adverse Events (SAEs)

Time Frame: Up to 5 years

Severity of SAEs

Time Frame: Up to 5 years

Secondary Outcomes

  • Concentration of REGN17235 in serum(Up to 5 years)
  • Occurrence of Anti-Drug Antibodies (ADA) to REGN17235 in serum(Up to 5 years)
  • Magnitude of ADA to REGN17235 in serum(Up to 5 years)
  • Hematologic improvement per International Working Group (IWG) 2018(Up to 5 years)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

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