NCT04906031

Brief 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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
5

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jun 2020

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2020

Completed
12 months until next milestone

First Submitted

Initial submission to the registry

May 18, 2021

Completed
10 days until next milestone

First Posted

Study publicly available on registry

May 28, 2021

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2023

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2024

Completed
Last Updated

June 9, 2021

Status Verified

June 1, 2021

Enrollment Period

2.7 years

First QC Date

May 18, 2021

Last Update Submit

June 5, 2021

Conditions

Myelodysplastic SyndromesAcute Myeloid LeukemiaMyeloid MalignancyTemperature-Sensitive p53 MutationSodium StibogluconateP53 Mutation

Keywords

Myelodysplastic SyndromesAcute Myeloid LeukemiaMyeloid MalignancyTemperature-sensitive p53 MutationsSodium Stibogluconatep53precision medicineantimonial

Outcome Measures

Primary Outcomes (1)

  • Overall response rate

    Partial response (PR) + complete response (CR) rate

    At the end of Cycle 4 (each cycle is 28 days)

Secondary Outcomes (1)

  • Adverse Event (AE)

    At the end of Cycle 4 (each cycle is 28 days)

Study Arms (1)

Sodium stibogluconate

EXPERIMENTAL

Sodium stibogluconate 900 mg/m2/day will be given on d1-5 and d15-19. 28 days per cycle.

Drug: Sodium stibogluconate

Interventions

Sodium stibogluconateDRUG

Sodium stibogluconate 900 mg/m2/day will be given on d1-5 and d15-19. 28 days per cycle.

Also known as: SSG
Sodium stibogluconate

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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 ≤35μmol/L, ASL/ALT lower than 2xULN, creatinine level ≤150μmol/L.
  • Normal cardiac function.
  • Lung function: dyspnea ≤ CTC AE grade 1 and SaO2≥ 92% in indoor air environment.
  • Written Informed consent.

You may not qualify if:

  • 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.
  • ECOG performance status ≥3, CCI \>1, ADL \<100.
  • Aged \<18 years or \>75years

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

First Affiliated Hospital of Jinan University

Guangzhou, Guangdong, 510632, China

RECRUITING

Related Publications (1)

  • Tang Y, Song H, Wang Z, Xiao S, Xiang X, Zhan H, Wu L, Wu J, Xing Y, Tan Y, Liang Y, Yan N, Li Y, Li J, Wu J, Zheng D, Jia Y, Chen Z, Li Y, Zhang Q, Zhang J, Zeng H, Tao W, Liu F, Wu Y, Lu M. Repurposing antiparasitic antimonials to noncovalently rescue temperature-sensitive p53 mutations. Cell Rep. 2022 Apr 12;39(2):110622. doi: 10.1016/j.celrep.2022.110622.

    PMID: 35417717DERIVED

MeSH Terms

Conditions

Myelodysplastic SyndromesLeukemia, Myeloid, Acute

Interventions

Antimony Sodium Gluconate

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesLeukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasms

Intervention Hierarchy (Ancestors)

Organic ChemicalsGluconatesSugar AcidsAcids, AcyclicCarboxylic AcidsHydroxy AcidsCarbohydrates

Study Officials

  • Min Lu, PHD

    Ruijin Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Hui Zeng, MD

CONTACT

+86 18002201919androps2011@hotmail.com

Huien Zhan, MD

CONTACT

+86 15084958382zhanhuien@163.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Director of Hematology Department of the First Affiliated Hospital of Jinan University

Study Record Dates

First Submitted

May 18, 2021

First Posted

May 28, 2021

Study Start

June 1, 2020

Primary Completion

February 1, 2023

Study Completion

February 1, 2024

Last Updated

June 9, 2021

Record last verified: 2021-06

Locations

CN(1)