Sodium Stibogluconate in the MDS/AML With One of the 65 Defined p53 Mutations

  • End date
    Feb 1, 2024
  • participants needed
  • sponsor
    First Affiliated Hospital of Jinan University
Updated on 18 June 2021
myeloid leukemia


To evaluate the safety and effectiveness of Sodium Stibogluconate in the treatment of myelodysplastic syndrome/acute myeloid leukemia (MDS/AML) with p53 mutation from a defined list. The list includes 65 p53 mutations that were experimentally confirmed to be pharmacologically restored with tumor-suppressive function by antimonials.


p53 is inactivated by over hundreds of diverse mutations in cancer. The investigator purposefully selected the phenotype-reversible temperature-sensitive (TS) p53 mutations for pharmacological rescue, and pertinently used p53 thermostability as readout to screen for TS p53 mutation rescue compounds. By this, the investigator identified antiparasitic drug antimonials efficiently thermostabilized the TS mutants by noncovalent binding. The antimonials met the three go-to criteria as a targeted drug-availability of co-crystal structure, structure model-compatible Structure-Activity Relationship, and target (p53)-specificity in cells, and consequently extended survival of xenograft mouse with TS p53 mutant.

Under the clinical antiparasitic dosage, the antimonials effectively rescued TS p53 mutant in patient-derived primary acute myeloid leukemia cells. Scan of the most frequent 815 p53 missense mutations identified 65 of them, predominantly TS mutations, as the antimonial-treatable mutations. Thus, the trial aims to evaluate the safety and effectiveness of the approved antimonial-Sodium Stibogluconate-in the treatment of myelodysplastic syndrome/acute myeloid leukemia (MDS/AML) with the 65 antimonial-treatable p53 mutants.

Condition myelodysplastic syndromes, Bone Marrow Disorder, Temperature-Sensitive p53 Mutation, P53 Mutation, Myelodysplastic Syndromes (MDS), TP53 Gene Mutation, Bone marrow disorder, acute myelogenous leukemia, Myeloid Malignancy, myelodysplastic syndrome (mds), anll, acute myeloblastic leukemia, Acute Myeloid Leukemia, Preleukemia, Myeloid Neoplasm, Acute myeloid leukemia, MYELODYSPLASTIC SYNDROME, sodium stibogluconate, Pentostam, Acute Myelogenous Leukemia (AML)
Treatment Sodium stibogluconate
Clinical Study IdentifierNCT04906031
SponsorFirst Affiliated Hospital of Jinan University
Last Modified on18 June 2021


Yes No Not Sure

Inclusion Criteria

Pathologically confirmed myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML)
Patients with one of the 65 antimonial-treatable p53 mutations with > 5% VAF: Q136P, Y234H, V272M, F270V, P278A, R213L, Y126H, T253N, T253I, R158L, Q136E, P142F, A129D, L194R, R110P, V172G, C176F, I254N, K305R, E285D, T155P, H296D, E258G, G279V, T211A, R213P, C229Y, I232F, E294K, P152R, R196P, M160T, N131S, N131H, K139N, L330H, Y220N, E298Q, D148E, L264R, E224D, H168P, N263H, K320N, S227C, E286D, K292T, V203A, M237R, F212L, K132Q, Y236S, Y126S, Q136H, E221A, I232S, Y163H, P190T, C182Y, P142L, Y163S, V218E, I195S, V272A, and S106R
Life expectancy >12 weeks
ECOG Performance status < 3\
Aged from 18 to 75
Active bone marrow hyperplasia indicated by morphology
Normal liver and renal function, bilirubin 35mol/L, ASL/ALT lower than 2xULN, creatinine level 150mol/L
Normal cardiac function
Lung function: dyspnea CTC AE grade 1 and SaO2 92% in indoor air environment
Written Informed consent

Exclusion Criteria

Confirmed CNS involvement
Severe cardiac diseases including myocardial infarction or heart insufficiency
QT interval 450ms on ECG
With other visceral malignancy
Active tuberculosis or HIV(+)
Patients with pregnancy or lactation
Allergic or significantly contraindicated to any drugs involved in intervention
Previous intolerance or allergy history to similar drugs
Participation at same time in another study in which investigational drugs are used
Any other conditions interfering the study
Abnormal liver function which does not meet the inclusion criteria
ECOG performance status 3, CCI >1, ADL <100\
Aged <18 years or >75years
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