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Not yet recruiting NCT07753148

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

Phase I Interventional Clonal Cytopenia of Undetermined Significance (CCUS) Low-Risk Myelodysplastic Syndrome (LR-MDS)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: REGN17235.
Who it may be relevant to
Registry conditions: Clonal Cytopenia of Undetermined Significance (CCUS), Low-Risk Myelodysplastic Syndrome (LR-MDS). Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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

Interventions

  • Drug REGN17235
    Administered per the protocol

Primary outcome measures

  • 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 outcome measures (4)
  • Hematologic improvement per International Working Group (IWG) 2018 [Time frame: Up to 5 years]
  • Concentration of REGN17235 in serum [Time frame: Up to 5 years]
  • Occurrence of Anti-Drug Antibodies (ADA) to REGN17235 in serum [Time frame: Up to 5 years]
  • Magnitude of ADA to REGN17235 in serum [Time frame: Up to 5 years]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07753148 · R17235-HM-24125 · 2026-525464-18-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗