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

• Status: Recruiting
• Trial end date: 2024-02-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: First Affiliated Hospital of Jinan University
• Collaborator: Ruijin Hospital
• Treatments: Antimony Sodium Gluconate

Criteria

Inclusion Criteria:

1. Pathologically confirmed myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML).

2. 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.

3. Life expectancy >12 weeks.

4. ECOG Performance status < 3.

5. Aged from 18 to 75.

6. Active bone marrow hyperplasia indicated by morphology.

7. Normal liver and renal function, bilirubin ≤35μmol/L, ASL/ALT lower than 2xULN, creatinine level ≤150μmol/L.

8. Normal cardiac function.

9. Lung function: dyspnea ≤ CTC AE grade 1 and SaO2≥ 92% in indoor air environment.

10. Written Informed consent.

Exclusion Criteria:

1. Confirmed CNS involvement.

2. Severe cardiac diseases including myocardial infarction or heart insufficiency.

3. QT interval ≥450ms on ECG.

4. With other visceral malignancy.

5. Active tuberculosis or HIV(+).

6. Patients with pregnancy or lactation.

7. Allergic or significantly contraindicated to any drugs involved in intervention.

8. Previous intolerance or allergy history to similar drugs.

9. Participation at same time in another study in which investigational drugs are used.

10. Any other conditions interfering the study.

11. Abnormal liver function which does not meet the inclusion criteria.

12. ECOG performance status ≥3, CCI >1, ADL <100.

13. Aged <18 years or >75years