An Extension to Study AZA PH GL 2003 CL 001 Allowing for Continuation of Azacitidine Treatment in Patients With Myelodysplastic Syndromes (MDS)

NCT ID: NCT01186939

Last Updated: 2019-11-14

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-04-01

Study Completion Date

2009-09-01

Brief Summary

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At the conclusion of study AZA PH GL 2003 CL 001 (NCT00071799), eligible participants could be enrolled in an optional extension phase in order to continue treatment with azacitidine until it became commercially available; the continued treatment was for ethical and safety reasons only and not to provide additional efficacy data.

Detailed Description

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At the conclusion of study AZA PH GL 2003 CL 001 (NCT00071799), eligible participants could be enrolled in an optional extension phase in order to continue treatment with azacitidine until it became commercially available; the continued treatment was for ethical and safety reasons only and not to provide additional efficacy data.

During the extension phase, participants were treated based on 28-day cycles and monitored for hematologic, nonhematologic, and renal toxicities. Recommended monitoring procedures included complete blood count with differential and platelets at least once each cycle prior to dosing and as needed, bone marrow biopsy and aspirate as clinically indicated, and additional tests or more frequent monitoring at the investigator's discretion based on the patient's clinical status. The azacitidine dose could be modified for toxicities. Laboratory data were not collected during the extension phase.

Conditions

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Myelodysplastic Syndromes

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Azacitidine

Azacitidine (study drug) plus best supportive care.

Group Type EXPERIMENTAL

Azacitidine

Intervention Type DRUG

Azacitidine was injected subcutaneously (SC) for 7 days. The 7-day dosing was repeated every 28 days with dose adjustments allowed. The initial dose during the primary study was 75mg/m\^2/day.

Interventions

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Azacitidine

Azacitidine was injected subcutaneously (SC) for 7 days. The 7-day dosing was repeated every 28 days with dose adjustments allowed. The initial dose during the primary study was 75mg/m\^2/day.

Intervention Type DRUG

Other Intervention Names

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AZA

Eligibility Criteria

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Inclusion Criteria

* Participants were considered eligible if they had been randomized to azacitidine treatment in the primary study and were receiving azacitidine at the time of study closure, had completed 12 months of treatment and observation in the primary study, and had signed the informed consent document for the extension phase of the study.

Exclusion Criteria

* None specific to the extension phase of the study
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Celgene

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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East Melbourne, Victoria, Australia

Site Status

Herston, , Australia

Site Status

Perth, , Australia

Site Status

Woolloongabba, , Australia

Site Status

Plovdiv, , Bulgaria

Site Status

Aulnay-sous-Bois, , France

Site Status

Berlin, , Germany

Site Status

Düsseldorf, , Germany

Site Status

Essen, , Germany

Site Status

Kiel, , Germany

Site Status

Heraklio, Crete, Greece

Site Status

Haidari, , Greece

Site Status

Budapest, , Hungary

Site Status

Bologna, , Italy

Site Status

Florence, , Italy

Site Status

Genova, , Italy

Site Status

Rome, , Italy

Site Status

Nijmegen, , Netherlands

Site Status

Lodz, , Poland

Site Status

Avda Campanar, , Spain

Site Status

León, , Spain

Site Status

London, , United Kingdom

Site Status

Countries

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Australia Bulgaria France Germany Greece Hungary Italy Netherlands Poland Spain United Kingdom

References

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Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, Schoch R, Gattermann N, Sanz G, List A, Gore SD, Seymour JF, Bennett JM, Byrd J, Backstrom J, Zimmerman L, McKenzie D, Beach C, Silverman LR; International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32. doi: 10.1016/S1470-2045(09)70003-8. Epub 2009 Feb 21.

Reference Type BACKGROUND
PMID: 19230772 (View on PubMed)

Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Gattermann N, Germing U, Sanz G, List AF, Gore S, Seymour JF, Dombret H, Backstrom J, Zimmerman L, McKenzie D, Beach CL, Silverman LR. Azacitidine prolongs overall survival compared with conventional care regimens in elderly patients with low bone marrow blast count acute myeloid leukemia. J Clin Oncol. 2010 Feb 1;28(4):562-9. doi: 10.1200/JCO.2009.23.8329. Epub 2009 Dec 21.

Reference Type BACKGROUND
PMID: 20026804 (View on PubMed)

Gore SD, Fenaux P, Santini V, Bennett JM, Silverman LR, Seymour JF, Hellstrom-Lindberg E, Swern AS, Beach CL, List AF. A multivariate analysis of the relationship between response and survival among patients with higher-risk myelodysplastic syndromes treated within azacitidine or conventional care regimens in the randomized AZA-001 trial. Haematologica. 2013 Jul;98(7):1067-72. doi: 10.3324/haematol.2012.074831. Epub 2013 Apr 12.

Reference Type BACKGROUND
PMID: 23585522 (View on PubMed)

Savona MR, Kolibaba K, Conkling P, Kingsley EC, Becerra C, Morris JC, Rifkin RM, Laille E, Kellerman A, Ukrainskyj SM, Dong Q, Skikne BS. Extended dosing with CC-486 (oral azacitidine) in patients with myeloid malignancies. Am J Hematol. 2018 Oct;93(10):1199-1206. doi: 10.1002/ajh.25216. Epub 2018 Sep 3.

Reference Type BACKGROUND
PMID: 30016552 (View on PubMed)

Seymour JF, Fenaux P, Silverman LR, Mufti GJ, Hellstrom-Lindberg E, Santini V, List AF, Gore SD, Backstrom J, McKenzie D, Beach CL. Effects of azacitidine compared with conventional care regimens in elderly (>/= 75 years) patients with higher-risk myelodysplastic syndromes. Crit Rev Oncol Hematol. 2010 Dec;76(3):218-27. doi: 10.1016/j.critrevonc.2010.04.005. Epub 2010 May 6.

Reference Type BACKGROUND
PMID: 20451404 (View on PubMed)

Garcia-Manero G, Scott BL, Cogle CR, Boyd TE, Kambhampati S, Hetzer J, Dong Q, Kumar K, Ukrainskyj SM, Beach CL, Skikne BS. CC-486 (oral azacitidine) in patients with myelodysplastic syndromes with pretreatment thrombocytopenia. Leuk Res. 2018 Sep;72:79-85. doi: 10.1016/j.leukres.2018.08.001. Epub 2018 Aug 3.

Reference Type BACKGROUND
PMID: 30114559 (View on PubMed)

Ma X, Steensma DP, Scott BL, Kiselev P, Sugrue MM, Swern AS. Selection of patients with myelodysplastic syndromes from a large electronic medical records database and a study of the use of disease-modifying therapy in the United States. BMJ Open. 2018 Jul 23;8(7):e019955. doi: 10.1136/bmjopen-2017-019955.

Reference Type BACKGROUND
PMID: 30037860 (View on PubMed)

de Lima M, Oran B, Champlin RE, Papadopoulos EB, Giralt SA, Scott BL, William BM, Hetzer J, Laille E, Hubbell B, Skikne BS, Craddock C. CC-486 Maintenance after Stem Cell Transplantation in Patients with Acute Myeloid Leukemia or Myelodysplastic Syndromes. Biol Blood Marrow Transplant. 2018 Oct;24(10):2017-2024. doi: 10.1016/j.bbmt.2018.06.016. Epub 2018 Jun 20.

Reference Type BACKGROUND
PMID: 29933073 (View on PubMed)

Wehmeyer J, Zaiss M, Losem C, Schmitz S, Niemeier B, Harde J, Hannig CV, Harich HD, Muller J, Klausmann M, Tessen HW, Potthoff K. Impact of performance status and transfusion dependency on outcome of patients with myelodysplastic syndrome, acute myeloid leukemia and chronic myelomonocytic leukemia treated with azacitidine (PIAZA study). Eur J Haematol. 2018 Dec;101(6):766-773. doi: 10.1111/ejh.13160. Epub 2018 Oct 25.

Reference Type BACKGROUND
PMID: 30091166 (View on PubMed)

Platzbecker U, Middeke JM, Sockel K, Herbst R, Wolf D, Baldus CD, Oelschlagel U, Mutherig A, Fransecky L, Noppeney R, Bug G, Gotze KS, Kramer A, Bochtler T, Stelljes M, Groth C, Schubert A, Mende M, Stolzel F, Borkmann C, Kubasch AS, von Bonin M, Serve H, Hanel M, Duhrsen U, Schetelig J, Rollig C, Kramer M, Ehninger G, Bornhauser M, Thiede C. Measurable residual disease-guided treatment with azacitidine to prevent haematological relapse in patients with myelodysplastic syndrome and acute myeloid leukaemia (RELAZA2): an open-label, multicentre, phase 2 trial. Lancet Oncol. 2018 Dec;19(12):1668-1679. doi: 10.1016/S1470-2045(18)30580-1. Epub 2018 Nov 12.

Reference Type BACKGROUND
PMID: 30442503 (View on PubMed)

Boutault R, Peterlin P, Boubaya M, Sockel K, Chevallier P, Garnier A, Guillaume T, Le Bourgeois A, Debord C, Godon C, Le Bris Y, Theisen O, Kroschinsky F, Moreau P, Bene MC, Platzbecker U, Eveillard M. A novel complete blood count-based score to screen for myelodysplastic syndrome in cytopenic patients. Br J Haematol. 2018 Dec;183(5):736-746. doi: 10.1111/bjh.15626. Epub 2018 Nov 8.

Reference Type BACKGROUND
PMID: 30406952 (View on PubMed)

Wenk C, Garz AK, Grath S, Huberle C, Witham D, Weickert M, Malinverni R, Niggemeyer J, Kyncl M, Hecker J, Pagel C, Mulholland CB, Muller-Thomas C, Leonhardt H, Bassermann F, Oostendorp RAJ, Metzeler KH, Buschbeck M, Gotze KS. Direct modulation of the bone marrow mesenchymal stromal cell compartment by azacitidine enhances healthy hematopoiesis. Blood Adv. 2018 Dec 11;2(23):3447-3461. doi: 10.1182/bloodadvances.2018022053.

Reference Type BACKGROUND
PMID: 30518537 (View on PubMed)

Zeidan AM, Klink AJ, McGuire M, Feinberg B. Treatment sequence of lenalidomide and hypomethylating agents and the impact on clinical outcomes for patients with myelodysplastic syndromes. Leuk Lymphoma. 2019 Aug;60(8):2050-2055. doi: 10.1080/10428194.2018.1551538. Epub 2019 Jan 14.

Reference Type BACKGROUND
PMID: 30636526 (View on PubMed)

Leung KK, Nguyen A, Shi T, Tang L, Ni X, Escoubet L, MacBeth KJ, DiMartino J, Wells JA. Multiomics of azacitidine-treated AML cells reveals variable and convergent targets that remodel the cell-surface proteome. Proc Natl Acad Sci U S A. 2019 Jan 8;116(2):695-700. doi: 10.1073/pnas.1813666116. Epub 2018 Dec 24.

Reference Type BACKGROUND
PMID: 30584089 (View on PubMed)

Craddock C, Slade D, De Santo C, Wheat R, Ferguson P, Hodgkinson A, Brock K, Cavenagh J, Ingram W, Dennis M, Malladi R, Siddique S, Mussai F, Yap C. Combination Lenalidomide and Azacitidine: A Novel Salvage Therapy in Patients Who Relapse After Allogeneic Stem-Cell Transplantation for Acute Myeloid Leukemia. J Clin Oncol. 2019 Mar 1;37(7):580-588. doi: 10.1200/JCO.18.00889. Epub 2019 Jan 17.

Reference Type BACKGROUND
PMID: 30653424 (View on PubMed)

Silverman LR, Fenaux P, Mufti GJ, Santini V, Hellstrom-Lindberg E, Gattermann N, Sanz G, List AF, Gore SD, Seymour JF. Continued azacitidine therapy beyond time of first response improves quality of response in patients with higher-risk myelodysplastic syndromes. Cancer. 2011 Jun 15;117(12):2697-702. doi: 10.1002/cncr.25774. Epub 2011 Jan 10.

Reference Type RESULT
PMID: 21656747 (View on PubMed)

Related Links

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Other Identifiers

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AZA PH GL 2003 CL001 E

Identifier Type: -

Identifier Source: org_study_id

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