Epigenetics, Vitamin C, and Abnormal Blood Cell Formation - Vitamin C in Patients With Low-Risk Myeloid Malignancies

Status unconfirmed · Not applicable · Has a placebo group

Conditions studied: Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia-1, Cytopenia

In brief

The primary purpose of this multi-centre, randomized, placebo-controlled, double-blind phase II study is to investigate if oral vitamin C may change the biology of low-risk myeloid malignancies; i.e., clonal cytopenia of undetermined significance (CCUS), low-risk myelodysplastic syndromes (MDS), and chronic myelomonocytic leukemia (CMML)-0/1 by reversing the epigenetic changes characteristic of these disease entities. The epigenetic regulator TET2 is the gene most often affected in CCUS. Preclinical studies have shown that active demethylation by the TET enzymes is dependent on vitamin C, and the investigators and collaborators have shown that plasma vitamin C levels are exceedingly low in hematological cancer patients but are easily corrected by oral vitamin C. This study is part of an array of EVITA studies aimed at clarifying whether the standard of care of patients with myeloid malignancies should be changed and oral vitamin C supplement added to the treatment recommendations.

Key facts

Study ID

NCT03682029

Run by

Rigshospitalet, Denmark

People needed

109

Starts

2017-11-21

Expected to finish

2025-09-27

Last updated by the study team

2024-04-17

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• A diagnosis of CCUS:
• Persistent cytopenia for > 6 months defined as hgb < 11.3 g/dL (7 mmol/L) in women and hgb < 12.9 g/dL (8 mmol/L) in men, thrombocyte count < 150 x 10\^9/L or neutrophil count < 1.8 x 10\^9/L
• Normal cytogenetics (with the exception of deletion of the Y chromosome which can be accepted)
• A bone marrow morphology that is not diagnostic of MDS or any other malignancy
• Other common causes of cytopenia (vitamin or other deficiencies, virus infection, etc.) have been ruled out
• Hematolytic conditions have been ruled out
• The presence of a detectable mutation in genes recurrently affected in myeloid malignancy representing a clonal marker (excluding germline mutations)
• OR
• A diagnosis of MDS as according to World Health Organization (WHO) 2016 diagnostic criteria
• Revised international prognostic scoring system (IPSS-R) risk score ≤ 3 AND bone marrow blast percentage < 5 defining low-risk
• OR
• A diagnosis of CMML-0 or -1 as according to WHO 2016 diagnostic criteria
• AND
• (All diagnostic categories) The presence of a detectable mutation in genes recurrently affected in myeloid malignancy representing a clonal marker (excluding germline mutations)

You may not qualify if…

• Unwillingness to discontinue any and all use of vitamin C medication/supplementation including multivitamin at least 24 hours prior to Baseline investigations and sampling
• Lack of ability to understand the information given, or lack of willingness to sign a written informed consent document
• Treatment with chemotherapy within the past 6 months
• Patients receiving active treatment for their myeloid malignancy, including investigational agents, with the exception of granulocyte colony-stimulating factor (G-CSF) and erythropoietin
• History of allergic reactions to ascorbic acid
• Unwillingness to comply with all aspects of the protocol

Where it is running

• Keck Hospital of University of Southern California — Los Angeles, California, United States
• Rigshospitalet — Copenhagen, N/A = Not Applicable, Denmark
• Aalborg University Hospital — Aalborg, Denmark
• Herlev University Hospital — Copenhagen, Denmark
• Odense University Hospital — Odense, Denmark

Full record on ClinicalTrials.gov

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