Trial · NCT01261312

SGI-110 in Participants With Myelodysplastic Syndromes (MDS) or Acute Myelogenous Leukemia (AML)

NCT01261312, Phase 1, Phase 2, Completed, Astex Pharmaceuticals, Inc.; results first posted 2025-01-23

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. SGI-110 in Participants With Myelodysplastic Syndromes (MDS) or Acute Myelogenous Leukemia (AML). Trials, record NCT01261312. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01261312

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: SGI-110 in Participants With Myelodysplastic Syndromes (MDS) or Acute Myelogenous Leukemia (AML)
NCT number: NCT01261312
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 414 participants, actual
Lead sponsor: Astex Pharmaceuticals, Inc.
Conditions: MDS, CMML, AML
Interventions: Guadecitabine
First posted: 2010-12-16
Last updated: 2025-01-23
Results first posted: 2025-01-23

Source ClinicalTrials.gov

Registered outcomes27

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Cycle 1 Day 8	DNA LINE-1 demethylation is defined as the largest percent decrease from baseline in methylation values within a participant between Day 8 and Day 22 of the first treatment cycle. BED was assessed based on DNA LINE-1 demethylation results and defined as the smallest dose that achieves the maximum biological pharmacodynamic (PD) effect (LINE-1 demethylation) in at least 3 successive dose levels.
Primary	Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Cycle 1 Day 15	DNA LINE-1 demethylation is defined as the largest percent decrease from baseline in methylation values within a participant between Day 8 and Day 22 of the first treatment cycle. BED was assessed based on DNA LINE-1 demethylation results and defined as the smallest dose that achieves the maximum biological pharmacodynamic (PD) effect (LINE-1 demethylation) in at least 3 successive dose levels.
Primary	Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Cycle 1 Day 22	DNA LINE-1 demethylation is defined as the largest percent decrease from baseline in methylation values within a participant between Day 8 and Day 22 of the first treatment cycle. BED was assessed based on DNA LINE-1 demethylation results and defined as the smallest dose that achieves the maximum biological pharmacodynamic (PD) effect (LINE-1 demethylation) in at least 3 successive dose levels.
Primary	Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Cycle 2 Day 1	DNA LINE-1 demethylation is defined as the largest percent decrease from baseline in methylation values within a participant between Day 8 and Day 22 of the first treatment cycle. BED was assessed based on DNA LINE-1 demethylation results and defined as the smallest dose that achieves the maximum biological pharmacodynamic (PD) effect (LINE-1 demethylation) in at least 3 successive dose levels.
Primary	Dose Escalation Phase-Maximum Tolerated Dose (MTD): Number of Participants With Dose Limiting Toxicity (DLT)	From the start of study treatment up to 30 days post treatment (Up to approximately 46 months)	The MTD was defined as the largest dose for which less than 33% of subjects experienced a dose limiting toxicity (DLT) during Cycle 1 of guadecitabine administration at each dose level. DLTs were defined using the Common Terminology Criteria for Adverse Events Version 4.0 (CTCAE v4.0).
Primary	Dose Expansion (DE) Phase- r/r AML, TN AML: Composite Complete Response (CRc) Rate	At end of each Cycle of 28 days (Up to approximately 38 months)	Composite complete response (CRc) rate is defined as the percentage of participants whose best response is complete remission \[CR\], CR with incomplete platelet recovery \[CRp\], or CR with incomplete hematological recovery \[CRi\]) after treatment with study drug. CR as per AML response criteria is defined as peripheral blood absolute neutrophil count (ANC) ≥1.0×10\^9/L, Platelets ≥100×10\^9/L, independence from red blood cell (RBC) and platelet transfusions over the past week, no myeloblasts and \<5% myeloblasts in bone marrow. CRp as per AML response criteria is defined as peripheral blood ANC ≥1.0×10\^9/L, Platelets \<100×10\^9/L, independence from RBC transfusions over the past week, no myeloblasts and \<5% myeloblasts in bone marrow. CRi as per AML response criteria is defined as peripheral blood ANC \<1.0×10\^9/L, no myeloblasts and \<5% myeloblasts in bone marrow.
Primary	Dose Expansion (DE) Phase- r/r MDS, TN MDS: Overall Response Rate (ORR)	At end of each Cycle of 28 days (Up to approximately 45 months)	ORR is defined as percentage of participants with complete response(CR), partial response(PR), marrow complete response(mCR) and haematological improvement(HI). CR:normal peripheral counts with persistent granulocyte count ≥1.0×10\^9/L, platelet count ≥100×10\^9/L and normal bone marrow (BM) with persistent marrow blasts ≤5%; persistent dysplasia was noted. PR:normal peripheral counts with granulocyte count ≥1.0×10\^9/L and platelet count ≥100 ×10\^9/L and normal BM with marrow blasts \>5% but were reduced by 50% or more. mCR:reduction of BM blasts to ≤5% without normalization of peripheral counts. HI is divided as erythroid response(HI-E): hemoglobin increase ≥1.5 g/dL or red blood cells transfusion independence, platelet response (HI-P): absolute increase of platelet count from \<20 to \>20×10\^9/L and by at least 100%,/if more than 20×10\^9/L, by an absolute increase of 30×10\^9/L, neutrophil response (HI-N): granulocyte increase ≥100%, and by an absolute increase ≥0.5×10\^9/L.
Secondary	Dose Escalation Phase: Response Rate in AML Participants	At end of each Cycle of 28 days (Up to approximately 23 months)	Response rate for AML participants was assessed by Modified International Working Group (IWG) 2003 response criteria with complete response (CR), complete response with incomplete platelet recovery (CRp), CR with incomplete blood count recovery(CRi), and partial response (PR). CR: absolute neutrophil count (ANC) \>1.0×10\^9/L, platelets ≥100×10\^9/L, independence from RBC and platelet transfusions, no or \<5% myeloblasts in bone marrow(BM). CRp: ANC \>1.0×10\^9/L, Platelets \<100×10\^9/L, independence from RBC transfusions, no or \<5% myeloblasts in BM. CRi: ANC \<1.0×10\^9/L, no or \<5% myeloblasts in BM. PR: ANC \>1.0×10\^9/L, Platelets ≥100×10\^9/L, no or decrease of ≥50% in myeloblasts to 5-25% in BM.
Secondary	Dose Escalation Phase: Response Rate in MDS Participants	At end of each Cycle of 28 days (Up to approximately 23 months)	Response rate for MDS participants was assessed by IWG 2006 Response Criteria with CR, PR, marrow complete response(mCR) and HI. CR: normal peripheral counts with persistent granulocyte count ≥1.0×10\^9/L, platelet count ≥100×10\^9/L; normal BM with persistent marrow blasts ≤5%; persistent dysplasia. PR: Normal peripheral counts with granulocyte count ≥1.0×10\^9/L, platelet count ≥100×10\^9/L and normal BM with blasts \>5% but reduced by 50% or more. mCR: reduction of BM blasts to ≤5% without normalization of peripheral counts.
Secondary	Dose Escalation and Dose Expansion Phase- r/r AML, TN AML: Duration of Response	At end of each Cycle of 28 days (Up to approximately 38 months)	Duration of response (in number of days) was calculated from the first time a complete response (CR, CRp, or CRi) was observed to time of relapse defined as the earliest time point whereby BM blasts or peripheral blood blasts become ≥5% and stayed at that level in subsequent visits while participants were still on study. CR:normal peripheral counts with persistent granulocyte count ≥1.0×10\^9/L, platelet count ≥100×10\^9/L and normal bone marrow (BM) with persistent marrow blasts ≤5%; persistent dysplasia was noted. CRp as per AML response criteria is defined as peripheral blood ANC ≥1.0×10\^9/L, Platelets \<100×10\^9/L, independence from RBC transfusions over the past week, no myeloblasts and \<5% myeloblasts in bone marrow. CRi as per AML response criteria is defined as peripheral blood ANC \<1.0×10\^9/L, no myeloblasts and \<5% myeloblasts in bone marrow.

1 to 10 of 27Source ClinicalTrials.gov

See all 27 registered outcomes

Outcomes27

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Cycle 1 Day 8	Pharmacodynamic (PD) analysis set included all participants who provided evaluable blood samples for analysis of at least one PD parameter or biomarker being tested. Overall number analyzed is number of participants with data available for analysis.	percent change	MEAN
PRIMARY	Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Cycle 1 Day 15	Pharmacodynamic (PD) analysis set included all participants who provided evaluable blood samples for analysis of at least one PD parameter or biomarker being tested. Overall number analyzed is number of participants with data available for analysis.	percent change	MEAN
PRIMARY	Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Cycle 1 Day 22	Pharmacodynamic (PD) analysis set included all participants who provided evaluable blood samples for analysis of at least one PD parameter or biomarker being tested. Overall number analyzed is number of participants with data available for analysis.	percent change	MEAN
PRIMARY	Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Cycle 2 Day 1	Pharmacodynamic (PD) analysis set included all participants who provided evaluable blood samples for analysis of at least one PD parameter or biomarker being tested. Overall number analyzed is number of participants with data available for analysis.	percent change	MEAN
PRIMARY	Dose Escalation Phase-Maximum Tolerated Dose (MTD): Number of Participants With Dose Limiting Toxicity (DLT)	From the start of study treatment up to 30 days post treatment (Up to approximately 46 months)	Safety data analysis set included all regimen 1, 2A and 2 B participants who received at least one dose of study drug.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Dose Expansion (DE) Phase- r/r AML, TN AML: Composite Complete Response (CRc) Rate	At end of each Cycle of 28 days (Up to approximately 38 months)	Efficacy analysis set included r/r AML and TN AML participants who received at least one dose of study drug.	percentage of participants	NUMBER
PRIMARY	Dose Expansion (DE) Phase- r/r MDS, TN MDS: Overall Response Rate (ORR)	At end of each Cycle of 28 days (Up to approximately 45 months)	Efficacy analysis set included r/r MDS and TN MDS participants who received at least one dose of study drug.	percentage of participants	NUMBER
SECONDARY	Dose Escalation Phase: Response Rate in AML Participants	At end of each Cycle of 28 days (Up to approximately 23 months)	Efficacy analysis set included regimen 1, 2A and 2B participants who received at least one dose of study drug. Overall number of participants analyzed are the number of participants with AML available for analysis.	percentage of participants	NUMBER
SECONDARY	Dose Escalation Phase: Response Rate in MDS Participants	At end of each Cycle of 28 days (Up to approximately 23 months)	Efficacy analysis set included regimen 1, 2A and 2B participants who received at least one dose of study drug. Overall number of participants analyzed are the number of participants with MDS available for analysis.	percentage of participants	NUMBER
SECONDARY	Dose Escalation and Dose Expansion Phase- r/r AML, TN AML: Duration of Response	At end of each Cycle of 28 days (Up to approximately 38 months)	Efficacy analysis set included regimen 1, 2A and 2B, r/r AML and TN AML participants who received at least one dose of study drug. Data for responders was reported for this outcome measure.	days	MEDIAN

1 to 10 of 27Source ClinicalTrials.gov

See all 27 outcomes

Results by group386

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Dose Escalation: Regimen 1 - Guadecitabine 3 mg/m^2 Daily	-16.143	Standard Deviation 12.692	percent change
Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Dose Escalation: Regimen 2A - Guadecitabine 6 mg/m^2 Once Weekly	-4.362	Standard Deviation 5.484	percent change
Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Dose Escalation: Regimen 2B - Guadecitabine 60 mg/m^2 Twice Weekly	-10.574	Standard Deviation 8.444	percent change
Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Dose Escalation: Regimen 1 - Guadecitabine 3 mg/m^2 Daily	-10.359	Standard Deviation 9.837	percent change
Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Dose Escalation: Regimen 2A - Guadecitabine 6 mg/m^2 Once Weekly	-5.312	Standard Deviation 5.796	percent change
Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Dose Escalation: Regimen 2B - Guadecitabine 60 mg/m^2 Twice Weekly	-17.540	Standard Deviation 9.207	percent change
Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Dose Escalation: Regimen 1 - Guadecitabine 3 mg/m^2 Daily	-6.325	Standard Deviation 7.477	percent change
Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Dose Escalation: Regimen 2A - Guadecitabine 6 mg/m^2 Once Weekly	-4.777	Standard Deviation 4.765	percent change
Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Dose Escalation: Regimen 2B - Guadecitabine 60 mg/m^2 Twice Weekly	-16.959	Standard Deviation 10.112	percent change
Dose Escalation Phase- Biological Effective Dose (BED): Percent Change From Baseline in DNA Long Interspersed Nucleotide Element-1 (LINE-1) Demethylation	Dose Escalation: Regimen 1 - Guadecitabine 3 mg/m^2 Daily	-2.805	Standard Deviation 4.249	percent change

1 to 10 of 386Source ClinicalTrials.gov

See all 386 results by group

Participant flow208

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Dose Escalation Phase (Day 0- Week 170)	STARTED	Dose Escalation: Regimen 1 - Guadecitabine 3 mg/m^2 Daily	44
Dose Escalation Phase (Day 0- Week 170)	STARTED	Dose Escalation: Regimen 2A - Guadecitabine 6 mg/m^2 Once Weekly	35
Dose Escalation Phase (Day 0- Week 170)	STARTED	Dose Escalation: Regimen 2B - Guadecitabine 60 mg/m^2 Twice Weekly	15
Dose Escalation Phase (Day 0- Week 170)	STARTED	Dose Expansion: r/r AML Guadecitabine 60 mg/m^2 (5-Day)	0
Dose Escalation Phase (Day 0- Week 170)	STARTED	Dose Expansion: r/r AML Guadecitabine 90 mg/m^2 (5-Day)	0
Dose Escalation Phase (Day 0- Week 170)	STARTED	Dose Expansion: r/r AML Guadecitabine 60 mg/m^2 (10-Day)	0
Dose Escalation Phase (Day 0- Week 170)	STARTED	Dose Expansion: TN AML Guadecitabine 60 mg/m^2 (5-Day)	0
Dose Escalation Phase (Day 0- Week 170)	STARTED	Dose Expansion: TN AML Guadecitabine 90 mg/m^2 (5-Day)	0
Dose Escalation Phase (Day 0- Week 170)	STARTED	Dose Expansion: TN AML Guadecitabine 60 mg/m^2 (10-Day)	0
Dose Escalation Phase (Day 0- Week 170)	STARTED	Dose Expansion: r/r MDS Guadecitabine 60 mg/m^2 (5-Day)	0

1 to 10 of 208Source ClinicalTrials.gov

See all 208 participant flow

Adverse events10,647

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Anaemia	Blood and lymphatic system disorders	Dose Escalation: Regimen 1 - Guadecitabine 3 mg/m^2 Daily	11	44
other	Anaemia	Blood and lymphatic system disorders	Dose Escalation: Regimen 2A - Guadecitabine 6 mg/m^2 Once Weekly	8	34
other	Anaemia	Blood and lymphatic system disorders	Dose Escalation: Regimen 2B - Guadecitabine 60 mg/m^2 Twice Weekly	5	15
other	Anaemia	Blood and lymphatic system disorders	Dose Expansion: r/r AML Guadecitabine 60 mg/m^2 (5-Day)	6	24
other	Anaemia	Blood and lymphatic system disorders	Dose Expansion: r/r AML Guadecitabine 90 mg/m^2 (5-Day)	6	26
other	Anaemia	Blood and lymphatic system disorders	Dose Expansion: r/r AML Guadecitabine 60 mg/m^2 (10-Day)	28	53
other	Anaemia	Blood and lymphatic system disorders	Dose Expansion: TN AML Guadecitabine 60 mg/m^2 (5-Day)	11	24
other	Anaemia	Blood and lymphatic system disorders	Dose Expansion: TN AML Guadecitabine 90 mg/m^2 (5-Day)	10	27
other	Anaemia	Blood and lymphatic system disorders	Dose Expansion: TN AML Guadecitabine 60 mg/m^2 (10-Day)	14	52
other	Anaemia	Blood and lymphatic system disorders	Dose Expansion: r/r MDS Guadecitabine 60 mg/m^2 (5-Day)	15	26

1 to 10 of 10,647Source ClinicalTrials.gov

See all 10,647 adverse events

Adverse event totals39

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Dose Escalation: Regimen 1 - Guadecitabine 3 mg/m^2 Daily	5	44
Total, all-cause mortality	Dose Escalation: Regimen 2A - Guadecitabine 6 mg/m^2 Once Weekly	4	35
Total, all-cause mortality	Dose Escalation: Regimen 2B - Guadecitabine 60 mg/m^2 Twice Weekly	0	15
Total, all-cause mortality	Dose Expansion: r/r AML Guadecitabine 60 mg/m^2 (5-Day)	4	25
Total, all-cause mortality	Dose Expansion: r/r AML Guadecitabine 90 mg/m^2 (5-Day)	3	28
Total, all-cause mortality	Dose Expansion: r/r AML Guadecitabine 60 mg/m^2 (10-Day)	10	55
Total, all-cause mortality	Dose Expansion: TN AML Guadecitabine 60 mg/m^2 (5-Day)	4	24
Total, all-cause mortality	Dose Expansion: TN AML Guadecitabine 90 mg/m^2 (5-Day)	7	27
Total, all-cause mortality	Dose Expansion: TN AML Guadecitabine 60 mg/m^2 (10-Day)	11	52
Total, all-cause mortality	Dose Expansion: r/r MDS Guadecitabine 60 mg/m^2 (5-Day)	2	26

1 to 10 of 39Source ClinicalTrials.gov

See all 39 adverse event totals

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
26296954	DERIVED	PubMed
31060979	DERIVED	PubMed
31331399	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01261312

GET https://conductscience.org/api/trials/v1/NCT01261312/registered?page=2

API reference For AI assistants