Last reviewed · How we verify

NCT01983969

Aza-SAHA-GBM With AutoSCT for Refractory Lymphoma

Completed Phase 1, PHASE2 Results posted Last updated 27 January 2020
What this trial tests

Phase 1, PHASE2 trial testing Azacitidine in Advanced Cancers in 61 participants. Completed in 22 November 2017.

Timeline
7 November 2013
Primary endpoint
22 November 2017
22 November 2017

Quick facts

Lead sponsorM.D. Anderson Cancer Center
PhasePhase 1, PHASE2
StatusCompleted
Study typeINTERVENTIONAL
Designsequential
Maskingnone
Primary purposetreatment
Enrollment61
Start date7 November 2013
Primary completion22 November 2017
Estimated completion22 November 2017
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

M.D. Anderson Cancer Center — full company profile →

Who can join

Adults 15 to 65, any sex, with Advanced Cancers or Lymphoma. 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.

Frequency of DLT Primary · Enrollment up to day 30 post transplant for each dosing cohort

Maximum tolerated dose (MTD) of azacitidine based on DLT was defined as any Grade 4 nonhematologic and noninfectious toxicity or any grade 3 mucositis or skin toxicity lasting \> 3 days at peak severity. For dose finding, the continunal reassessment method was used with a target DLT probability per cohort of 25%. Azacitidine doses were chosen adaptively for sucessive cohorts with a minimum size of 2 patients. Toxicity scoring followed the National Cancer Institute Common Toxicity Criteria, version 3.

GroupValue95% CI
Azacitidine Dose Level 116
Azacitidine Dose Level 228
Azacitidine Dose Level 340
Participants With Event-free Survival (EFS) Primary · Enrollment up to 100 days post transplant.

EFS is defined as the time from transplantation to either relapse, second tumors, or death, whichever occurred first, or last contact. EFS was analzyed by the individual disease groups rather than the cohort dose levels.

GroupValue95% CI
DLBCL17
Hodgkin Lymphoma16
T-cell NHL7
Other B-cell Lymphoma5

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 100 Days post autologous transplant.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Azacitidine Dose Level 1
Serious: 0/37 (0%)
Deaths: 1/37
Azacitidine Dose Level 2
Serious: 0/18 (0%)
Deaths: 0/18
Azacitidine Dose Level 3
Serious: 0/5 (0%)
Deaths: 1/5
Other adverse events (15 terms — click to expand)

ReactionSystemAzacitidine Dose Level 1Azacitidine Dose Level 2Azacitidine Dose Level 3
Neutropenic FeverInfections and infestations
MucositisGastrointestinal disorders
NauseaGastrointestinal disorders
DiarrheaGastrointestinal disorders
TransaminitisHepatobiliary disorders
Fluid OverloadGeneral disorders
Skin RashSkin and subcutaneous tissue disorders
Elevated BilirubinHepatobiliary disorders
InfectionInfections and infestations
Elevated CreatinineRenal and urinary disorders
FeverGeneral disorders
PneumonitisRespiratory, thoracic and mediastinal disorders
HypertensionCardiac disorders
DVTCardiac disorders
Hemorrhagic CystitisRenal and urinary disorders

Data from ClinicalTrials.gov NCT01983969 adverse events section.

Sponsor's own description

The goal of this clinical research study is to find the highest tolerable dose of azacitidine that can be given with vorinostat, gemcitabine, busulfan, and melphalan, with a stem cell transplant, and with or without rituximab. Researchers also want to learn about the safety and level of effectiveness of this combination.

Publications & conference data

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

  1. Epigenetic regulation in hematopoiesis and its implications in the targeted therapy of hematologic malignancies.
    Zhao A, Zhou H, Yang J, Li M, et al · · 2023 · cited 81× · PMID 36797244 · DOI 10.1038/s41392-023-01342-6
  2. Epigenetic drugs in cancer therapy.
    Suraweera A, O'Byrne KJ, Richard DJ. · · 2025 · cited 50× · PMID 40011240 · DOI 10.1007/s10555-025-10253-7
  3. Histone Deacetylase Inhibitor SAHA as Potential Targeted Therapy Agent for Larynx Cancer Cells.
    Grabarska A, Grabarska A, Łuszczki JJ, Nowosadzka E, et al · · 2017 · cited 36× · PMID 28123594 · DOI 10.7150/jca.16655
  4. Differential effects of histone deacetylase inhibitors on cellular drug transporters and their implications for using epigenetic modifiers in combination chemotherapy.
    Valdez BC, Li Y, Murray D, Brammer JE, et al · · 2016 · cited 16× · PMID 27564097 · DOI 10.18632/oncotarget.11561
  5. Improved outcomes of high-risk relapsed Hodgkin lymphoma patients after high-dose chemotherapy: a 15-year analysis.
    Nieto Y, Gruschkus S, Valdez BC, Jones RB, et al · · 2022 · cited 13× · PMID 33951890 · DOI 10.3324/haematol.2021.278311
  6. Histone Modifications and Their Targeting in Lymphoid Malignancies.
    Fernández-Serrano M, Winkler R, Santos JC, Le Pannérer MM, et al · · 2021 · cited 10× · PMID 35008680 · DOI 10.3390/ijms23010253
  7. Impact of Histone Modifications and Their Therapeutic Targeting in Hematological Malignancies.
    Markouli M, Strepkos D, Piperi C. · · 2022 · cited 5× · PMID 36362442 · DOI 10.3390/ijms232113657
  8. Epigenetic targets in B- and T-cell lymphomas: latest developments.
    Ribeiro ML, Sánchez Vinces S, Mondragon L, Roué G. · · 2023 · cited 3× · PMID 37273421 · DOI 10.1177/20406207231173485

Verify or expand the search:

Other trials of Azacitidine

Trials testing the same drug.

Other recruiting trials for Advanced Cancers

Currently open trials in the same condition.

Other M.D. Anderson Cancer Center trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01983969.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing