NCT03603964

Guadecitabine Extension Study

Terminated Phase 2 Results posted Last updated 27 August 2024

What this trial tests

Phase 2 trial testing Guadecitabine in Acute Myeloid Leukemia in 35 participants. Terminated before completion.

Timeline

18 July 2018

Primary endpoint
4 October 2021

4 October 2021

Quick facts

Lead sponsor	Astex Pharmaceuticals, Inc.
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	35
Start date	18 July 2018
Primary completion	4 October 2021
Estimated completion	4 October 2021
Sites	21 locations across Italy, Denmark, Japan, Austria, Taiwan, South Korea, Canada, United States

Drugs / interventions tested

• Guadecitabine — full drug profile →

Conditions studied

• Acute Myeloid Leukemia — all drugs for Acute Myeloid Leukemia →
• Myeloid Dysplastic Syndrome — all drugs for Myeloid Dysplastic Syndrome →

Sponsor

Astex Pharmaceuticals, Inc. — full company profile →

Who can join

18 and older, any sex, with Acute Myeloid Leukemia or Myeloid Dysplastic Syndrome. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Treatment Emergent Adverse Events (TEAEs) Primary · From the start of study treatment until 30 days after the last dose of study treatment or prior to the participant receiving alternative anticancer therapy, whichever occurs first.

Any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE can therefore be any unfavorable and unintended sign (including a clinically significant abnormal finding in laboratory tests or other diagnostic procedures), symptom, or disease temporally associated with the use of a drug, without any judgment about causality. TEAEs are defined as events that occur or worsen on or after the date of the first study treatment (Cycle 1 Day 1 {C1D1}) until 30 days after the last dose of study treatment or the start of an alternative anti-can

Group	Value	95% CI
Guadecitabine	31

Overall Survival Secondary · From randomization in the prior study to the date of death

Overall survival was defined as the number of days from the time of randomization in the prior study to the date of death (regardless of cause). Participants without a documented death date at the time of analysis were censored at the last date known alive. Survival time in days = (earliest of date of death or censoring) - (randomization date in the prior study).

Group	Value	95% CI
Guadecitabine	2583.0	1422.0 – NA

Adverse events — posted to ClinicalTrials.gov

Time frame: From the start of study treatment until 30 days after the last dose of study treatment or prior to the participant receiving alternative anticancer therapy, whichever occurs first.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Guadecitabine

Serious: 20/35 (57%)

Deaths: 9/35

Serious adverse events (18 terms)

Reaction	System	Guadecitabine
Febrile neutropenia	Blood and lymphatic system disorders	—
Pneumonia	Infections and infestations	—
Sepsis	Infections and infestations	—
Upper gastrointestinal haemorrhage	Gastrointestinal disorders	—
Asthenia	General disorders	—
Anaphylactic reaction	Immune system disorders	—
Bronchopulmonary aspergillosis	Infections and infestations	—
Clostridium difficile colitis	Infections and infestations	—
Gastroenteritis	Infections and infestations	—
Herpes zoster	Infections and infestations	—
Infection	Infections and infestations	—
Pneumonia viral	Infections and infestations	—
Soft tissue infection	Infections and infestations	—
Patella fracture	Injury, poisoning and procedural complications	—
Hepatic neoplasm	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—
Cerebrovascular accident	Nervous system disorders	—
Cognitive disorder	Nervous system disorders	—
Interstitial lung disease	Respiratory, thoracic and mediastinal disorders	—

Most-reported serious reactions: Febrile neutropenia, Pneumonia, Sepsis, Upper gastrointestinal haemorrhage, Asthenia, Anaphylactic reaction, Bronchopulmonary aspergillosis, Clostridium difficile colitis.

Data from ClinicalTrials.gov NCT03603964 adverse events section.

Sponsor's own description

This is a multicenter, open-label extension study for participants who participated in a previous Astex-sponsored guadecitabine clinical study \[including but not limited to SGI-110-01 (NCT01261312), SGI-110-04 (NCT02348489), SGI-110-06 (NCT02920008), and SGI-110-07 (NCT02907359)\].

Publications & conference data

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

1. 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
2. Epigenetics of Bladder Cancer: Where Biomarkers and Therapeutic Targets Meet.
   Martinez VG, Munera-Maravilla E, Bernardini A, Rubio C, et al · · 2019 · cited 30× · PMID 31850055 · DOI 10.3389/fgene.2019.01125
3. The Role of the DNA Methyltransferase Family and the Therapeutic Potential of DNMT Inhibitors in Tumor Treatment.
   Kim DJ. · · 2025 · cited 24× · PMID 39996888 · DOI 10.3390/curroncol32020088
4. How metabolism bridles cytotoxic CD8⁺ T cells through epigenetic modifications.
   Van Acker HH, Ma S, Scolaro T, Kaech SM, et al · · 2021 · cited 24× · PMID 33867272 · DOI 10.1016/j.it.2021.03.006
5. Epigenetics in a Spectrum of Myeloid Diseases and Its Exploitation for Therapy.
   Maher M, Diesch J, Le Pannérer MM, Buschbeck M. · · 2021 · cited 10× · PMID 33917538 · DOI 10.3390/cancers13071746