trialsTrialActive

A Study to Assess the Safety and Efficacy of Two Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) Harboring IDH Mutations Who Are Not Candidates to Receive Intensive Induction Chemotherapy

A phase 1/2 trial of Azacitidine in acute myeloid leukaemia, run by Celgene, active and no longer recruiting.

A Study to Assess the Safety and Efficacy of Two Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) Harboring IDH Mutations Who Are Not Candidates to Receive Intensive Induction Chemotherapy is a phase 1/2 interventional study registered as NCT02677922 by Celgene, with 130 participants enrolled, started 2016-06-03 and due to reach its primary completion in 2018-08-02. Interventions recorded: AG-120, Azacitidine and AG-221. Results have been posted on the registry.

Setting

A Phase 1b/2 Open-Label, Randomized Study of 2 Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine: Oral AG-120 Plus Subcutaneous Azacitidine and Oral AG-221 Plus SC Azacitidine in Subjects With Newly Diagnosed Acute Myeloid Leukemia Harboring an IDH1 or an IDH2 Mutation, Respectively, Who Are Not Candidates to Receive Intensive Induction Chemotherapy

Phase

Phase 1/2

Sponsor

Celgene

Registry

Started

2016-06-03

Reported

2022

Enrolled

130 enrolled

Outcomes

In plain words

What these results mean for people, not percentages

130 enrolled

Overall Response Rate: Phase 2 (Randomized Stage)primaryresponse endpoint

73.5 vs 36.4 out of 100 had their tumour shrink with Phase 2 Randomized Stage: AG-221 (100mg) + AZA compared with Phase 2 Randomized Stage: AZA Monotherapy; 37.1 more per 100.
Roughly one extra person helped for every 3 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.

The p-value (0.0003) says a difference this large would rarely happen by chance; it does not say how large or how useful the difference is.
A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.

Overall Survival: Phase 2 (Randomized Stage)survival endpoint

Median 22 vs 22.3 months with Phase 2 Randomized Stage: AG-221 (100mg) + AZA compared with Phase 2 Randomized Stage: AZA Monotherapy; about 0.3 months shorter for half the group.
A median is a midpoint: half the people did better than this and half did worse, so it is not a prediction for any one person.

The p-value (0.9720) means the difference could plausibly be due to chance.
Overall survival counts deaths from any cause, so it is the most direct measure of whether a treatment helps people live longer.

Caveats

These results apply to the people the trial enrolled: A Phase 1b/2 Open-Label, Randomized Study of 2 Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine: Oral AG-120 Plus Subcutaneous Azacitidine and Oral AG-221 Plus SC Azacitidine in Subjects With Newly Diagnosed Acute Myeloid Leukemia Harboring an IDH1 or an IDH2 Mutation, Respectively, Who Are Not Candidates to Receive Intensive Induction Chemotherapy. People in a different situation may not see the same effect.
The trial selected people by a biomarker (IDH); the result should not be assumed for people whose cancer does not have it.

Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.

130 enrolled.

Overall Response Rate: Phase 2 (Randomized Stage)primary

· p = 0.0003

Phase 2 Randomized Stage: AG-221 (100mg) + AZA73.5 of 100

n = 68

Phase 2 Randomized Stage: AZA Monotherapy36.4 of 100

n = 33

Download

95% CI 61.4 to 83.5; Figures from the ClinicalTrials.gov results section (first posted 2022-11-22), not from a publication · 95% CI 20.4 to 54.9

Overall Survival: Phase 2 (Randomized Stage)

· p = 0.9720

Download

95% CI 14.6 to NA; Figures from the ClinicalTrials.gov results section (first posted 2022-11-22), not from a publication · 95% CI 11.9 to NA

Endpoint	Arm	n	Value	HR (95% CI)	p	Source
Overall Response Rate: Phase 2 (Randomized Stage)primary	Phase 2 Randomized Stage: AG-221 (100mg) + AZA	68	73.5%	-	0.0003	link
Overall Response Rate: Phase 2 (Randomized Stage)primary	Phase 2 Randomized Stage: AZA Monotherapy	33	36.4%	-	0.0003	link
Overall Survival: Phase 2 (Randomized Stage)	Phase 2 Randomized Stage: AG-221 (100mg) + AZA	68	22 months	-	0.9720	link
Overall Survival: Phase 2 (Randomized Stage)	Phase 2 Randomized Stage: AZA Monotherapy	33	22.3 months	-	0.9720	link

Connected

Similar pages

Trial
A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Best Supportive Care as Maintenance Therapy in Japanese Participants With Acute Myeloid Leukemia (AML) in Complete Remission
Shares Azacitidine, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Bristol Myers Squibb, Acute myeloid leukaemia and the tag pipeline.
Trial
PBSS1113 in Combination With Azacitidine to Treat Patients With AML/MDS
Shares Azacitidine, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Acute myeloid leukaemia, Cytotoxic chemotherapy and the tag pipeline.
Trial
A Pivotal Study of APG-2575 (Lisaftoclax) Combined With Azacitidine in the Treatment of Acute Myeloid Leukemia
Shares Azacitidine, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Acute myeloid leukaemia, Cytotoxic chemotherapy and the tag pipeline.
Trial
A Phase 2 Study of MAX-40279 Combined With Venetoclax and Azacitidine in Unfit Newly Diagnosed Acute Myeloid Leukemia
Shares Azacitidine, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Acute myeloid leukaemia, Cytotoxic chemotherapy and the tag pipeline.
Trial
FCN-338 in Combination With Azacitidine or Chemotherapy in Myeloid Neoplasms
Shares Azacitidine, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Cytotoxic chemotherapy and the tag pipeline.
Trial
An Open-label Phase 3b Study of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy.
Shares Azacitidine, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Acute myeloid leukaemia, Cytotoxic chemotherapy and the tag pipeline.
Trial
An Efficacy and Safety Study of Oral Azacitidine (CC-486) as Maintenance Therapy in Chinese Participants With Acute Myeloid Leukemia in Complete Remission
Shares Bristol Myers Squibb, Acute myeloid leukaemia and the tag pipeline.
Trial
A Study of Gilteritinib, Venetoclax and Azacitidine as a Combined Treatment for People Newly Diagnosed With Acute Myeloid Leukemia
Shares Azacitidine, Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Acute myeloid leukaemia, Cytotoxic chemotherapy and the tag pipeline.

cancers

Acute myeloid leukaemia IDH1- and IDH2-mutated acute myeloid leukaemia

technologies

Cytotoxic chemotherapy Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)

drugs

ApprovedSmall-molecule hypomethylating agent (cytotoxic)

Azacitidine · Vidaza / Onureg (oral)

companies

Bristol Myers Squibb