Treatment of High Risk Myelodysplastic Syndromes (MDS) Not Candidates for Allogeneic Transplantation of Hematopoietic Progenitors (ALO-HSCT)

An observational, non-interventional, prospective and multicenter study of Azacitidine in newly diagnosed High Risk Myelodysplastic Syndromes.

Primary objectives are to asses mutational status of target genes by Next Generation Sequencing, to evaluate prognostic value of geriatric assessment scales and to evaluate overall survival.

The main hypothesis is that mutation status of target genes and geriatric scales have statistical significant impact on overall survival.

Study time points will be at diagnosis, 6, 12, 18 and 24 months, always taking into account the routine clinical practice, when sample to assess mutational status will be collected. Geriatric assessment will only be performed at diagnosis.

Upon the signature of informed consent and the checking of inclusion criteria, patients will receive treatment with Azacitidine 75 mg/sqm on a 28 days based cycles (both 7-0-0 and 5-0-2 regimens are allowed) until disease progression, unacceptable toxicity or investigator decision.

150 patients are expected to be recruited at study sites.

Study Overview

Status

Unknown

Study Type

Observational

Enrollment (Anticipated)

150

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Joan Bargay, MD PhD
  • Phone Number: 34 871 202158
  • Email: jbargay@hsll.es

Study Contact Backup

Study Locations

    • Islas Baleares
      • Palma, Islas Baleares, Spain, 07198
        • Recruiting
        • Son Llatzer
        • Contact:
        • Principal Investigator:
          • Joan Bargay, MD PhD
        • Principal Investigator:
          • Lurdes Zamora, PhD
        • Sub-Investigator:
          • Marta Cabezon, PhD
        • Sub-Investigator:
          • Jose Borras, Bsc

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Newly diagnosed High-Risk Myelodysplastic Syndrome

Description

Inclusion Criteria:

  • Patients diagnosed with myelodysplastic syndrome (MDS) according to WHO criteria, including non-proliferative chronic myelomonocytic leukemia (CMML) that is considered high risk, and not candidates for transplantation.
  • High-risk MDS: (following the Grupo Español Síndrome Mielodisplásico, GESDM MDS guides) and the recommendations of Valcárcel, D. et al).

Median expected OS less than 30 months. Intermediate-2 or high risk IPSS and / or high or very high risk WPSS and / or high or very high risk IPSSS-R.

IPSS of intermediate risk and / or WPSS of intermediate risk and / or IPSS-R of intermediate risk that present at least one of the following characteristics:

Cytogenetic abnormality of the IPSS-R high or very high cytogenetic risk group Platelet count <30 x10E9/l Neutrophil count <0.5 x 10E9 / l Presence of dense and diffuse myelofibrosis, with or without collagen formation (grades 2-3 of the European consensus) Category 3 of the LRSS score (MD Anderson low risk score system)

  • Patient diagnosed with LAM de novo according to WHO criteria, with 20-30% blasts count in bone marrow, trilineal dysplasia and more than 70 years and not candidate for intensive chemotherapy.
  • Willing to provide voluntary written informed consent

Exclusion Criteria:

  • Serious active medical condition, not related to MDS, that could limit patient compliance and follow-up or that, in the Investigator's opinion,could compromise the patient's safety.
  • Presence of other active malignant disease
  • Organic function parameters: (except when the alteration is due to MDS) Hepatic: Bilirubin> 2 x upper limit of normal (ULN) Renal: Creatinine> 2 x upper limit of normal (ULN)
  • Ejection fraction (<40%) and / or symptomatic heart failure.
  • Leukaemia secondary to Myeloproliferative Syndrome.
  • Serious psychiatric or neurological disease.
  • Positive Serology for HIV.
  • Proliferative CMML

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
High Risk MDS
New diagnosed patients treated with Azacitidine 75 mg/sqm SC QD on a 28 days based cycles (both 7-0-0 and 5-0-2 regimens are allowed) until disease progression, unacceptable toxicity, death or investigator decision

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Overall Survival (OS)
Time Frame: 5- year overall survival
5- year overall survival
Progression Free Survival (PFS)
Time Frame: 5-year progression free survival
5-year progression free survival
Overall Response Rate (OOR)
Time Frame: 12 months OOR
12 months OOR
Treatment-Emergent Adverse Events Rate (TEAE)
Time Frame: 12 Months TEAE rate
12 Months TEAE rate

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: Joan Bargay, MD PhD, Hospital Son Llàtzer
  • Principal Investigator: Lurdes Zamora, PhD, Germans Trias I Pujol Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 12, 2016

Primary Completion (Anticipated)

November 1, 2020

Study Completion (Anticipated)

June 30, 2021

Study Registration Dates

First Submitted

October 20, 2020

First Submitted That Met QC Criteria

October 20, 2020

First Posted (Actual)

October 26, 2020

Study Record Updates

Last Update Posted (Actual)

October 26, 2020

Last Update Submitted That Met QC Criteria

October 20, 2020

Last Verified

October 1, 2020

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

IPD Plan Description

There is not a confirmed IPD sharing plan. It will be confirmed by Principal Investigators

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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