NCT03397173

TET2 Mutations in Myelodysplastic Syndromes and Acute Myeloid Leukemia With Azacitidine + Ascorbic Acid

Completed Phase 2 Last updated 11 August 2021

What this trial tests

Phase 2 trial testing Azacitidine in Myelodysplastic Syndromes in 12 participants. Completed in 21 January 2021.

Timeline

16 March 2018

Primary endpoint
3 January 2021

21 January 2021

Quick facts

Lead sponsor	Case Comprehensive Cancer Center
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	12
Start date	16 March 2018
Primary completion	3 January 2021
Estimated completion	21 January 2021
Sites	1 location across United States

Drugs / interventions tested

• Azacitidine (azacitidine) — full drug profile →
• Ascorbic acid (ASCORBIC ACID) — full drug profile →

Conditions studied

• Myelodysplastic Syndromes — all drugs for Myelodysplastic Syndromes →
• Myeloproliferative Neoplasm — all drugs for Myeloproliferative Neoplasm →
• Acute Myeloid Leukemia — all drugs for Acute Myeloid Leukemia →

Sponsor

Case Comprehensive Cancer Center — full company profile →

Who can join

18 and older, any sex, with Myelodysplastic Syndromes or Myeloproliferative Neoplasm. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of this study is to evaluate the efficacy of treatment with azacitidine (an FDA approved drug for the treatment of MDS) and high dose ascorbic acid in patients with TET2 mutations. This approach is intended to enhance the enzymatic activity of TET2 protein, which in term may help to improve counts and symptoms, related to Myelodysplastic Syndromes and Acute Myeloid Leukemia. This combination is specific to individuals who carry this mutation.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. TET (Ten-eleven translocation) family proteins: structure, biological functions and applications.
   Zhang X, Zhang Y, Wang C, Wang X. · · 2023 · cited 150× · PMID 37563110 · DOI 10.1038/s41392-023-01537-x
2. Epigenetics-targeted drugs: current paradigms and future challenges.
   Dai W, Qiao X, Fang Y, Guo R, et al · · 2024 · cited 131× · PMID 39592582 · DOI 10.1038/s41392-024-02039-0
3. Methylation across the central dogma in health and diseases: new therapeutic strategies.
   Liu R, Zhao E, Yu H, Yuan C, et al · · 2023 · cited 79× · PMID 37620312 · DOI 10.1038/s41392-023-01528-y
4. Nutritional perspectives for the prevention and mitigation of COVID-19.
   Akhtar S, Das JK, Ismail T, Wahid M, et al · · 2021 · cited 47× · PMID 33570583 · DOI 10.1093/nutrit/nuaa063
5. Translating recent advances in the pathogenesis of acute myeloid leukemia to the clinic.
   Bewersdorf JP, Abdel-Wahab O. · · 2022 · cited 36× · PMID 35318270 · DOI 10.1101/gad.349368.122
6. Combination Therapy with Vitamin C Could Eradicate Cancer Stem Cells.
   Satheesh NJ, Samuel SM, Büsselberg D. · · 2020 · cited 33× · PMID 31947879 · DOI 10.3390/biom10010079
7. Epigenetic modulators provide a path to understanding disease and therapeutic opportunity.
   Honer MA, Ferman BI, Gray ZH, Bondarenko EA, et al · · 2024 · cited 21× · PMID 38914477 · DOI 10.1101/gad.351444.123
8. Risk-Adapted, Individualized Treatment Strategies of Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML).
   Bewersdorf JP, Zeidan AM. · · 2021 · cited 21× · PMID 33807279 · DOI 10.3390/cancers13071610