Clinical Trials

A clinical trial is currently evaluating therapeutic strategies targeting 5-Methylcytosine dynamics in hematologic malignancies, including myelodysplastic syndromes, acute myeloid leukemia, and chronic myelomonocytic leukemia. Sponsored by academic and clinical consortia—including investigator Kirsten Grønbæk, the Van Andel Institute - Stand Up To Cancer Epigenetics Dream Team, and European university hospitals—this actively recruiting Phase II study assesses DNA hypomethylation approaches. Ultimately, the research seeks to establish the safety and efficacy of combining active and passive hypomethylation modalities to restore normal transcriptional regulation.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03999723 Recruiting
Myelodysplastic Syndromes|Acute Myeloid Leukemia|Chronic Myelomonocytic Leukemia
Kirsten Grønbæk|Van Andel Institute - Stand Up To Cancer Epigenetics Dream Team|Karolinska University Hospital|Skane University Hospital|Sahlgrenska University Hospital Sweden|University of Southern California|Imperial College London|University of Copenhagen|Zealand University Hospital|Aalborg University Hospital|Odense University Hospital|Technical University of Denmark|Aarhus University Hospital|Uppsala University Hospital|Rigshospitalet Denmark
 2019-09-11 Phase 2

(data from https://clinicaltrials.gov, updated on 2024-05-22)

Check the 5-Methylcytosine product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

5-Methylcytosine functions as a key epigenetic mark in nuclear DNA, binding methyl-CpG-binding proteins to recruit repressor complexes that suppress downstream gene transcription. Disruption of normal cytosine methylation dynamics impairs cell differentiation and epigenetic stability, contributing to the pathogenesis of hematologic conditions such as myelodysplastic syndromes, acute myeloid leukemia, and chronic myelomonocytic leukemia.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.