Health Outcomes of Recently Diagnosed Myelodysplastic Syndrome (MDS)/Chronic Myelomonocytic Leukemia (CMML) Patients Depending on Treatment Strategy (Wait and See, Support, Active Treatment)

Myelodysplastic Syndrome Clinical Trial

Official title:

Post-authorization, Observational Study to Assess the Evolution in the Normal Clinical Practise of Patients With Recent Diagnosis of Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML), Depending on the Time of Active Treatment Initiated

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02085798
• Other study ID #: NIPMS-CELGENE-SP-006
• Status: Completed
• Start date: December 17, 2012
• Est. completion date: December 14, 2018

Study information

• Verified date: January 2020
• Source: Celgene
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Post-authorisation observational study to assess the evolution in normal clinical practice of patients recently diagnosed with myelodysplastic syndrome (MDS) or chronic myelomonocytic leukaemia (CMML), depending on the moment when active treatment is initiated. Subjects will be recruited from approximately 50 haematology sites in Spain.

Description:

Observational, prospective, post-authorisation multicentre study.

The study will include patients with a recent diagnosis (< 3 months) of MDS or CMML, receiving immediate active/support treatment or for whom an observation approach ("wait and see") is initially adopted, as per normal clinical practice in each participating site.

The minimum follow-up period of a participant patient will be 36 months from recruitment, until survival can be documented, differentiating between

1. Patients starting immediate active treatment: data will be collected every 3 months or every time an event arises during the active treatment phase. After the final dose of the initial treatment therapy, data collection will take place every 6 months during the post-treatment follow-up phase, regardless of the number of times the patient attends medical appointments, up to a minimum of 36 months' follow-up from the start of active treatment.

2. Patients for whom initial treatment strategy is support treatment or observation: a minimum follow-up period of 36 months will be established from the date of inclusion in the study, with data collection every 6 months or whenever an event occurs, regardless of the number of times the patient attends a medical appointment. The need for treatment for MDS (o CMML, where applicable) during this period will be considered an event, after which data will be collected every 3 months or each time an event occurs during the entire active treatment phase. After the final dose of the initial treatment therapy, data collection will take place every 6 months during the post-treatment follow-up phase, regardless of the number of times the patient attends medical appointments, up to a minimum of 36 months' follow-up from the start of active treatment.

Patients will be included consecutively, without the treatment prescription decisions affecting the decision to include the patient in the study. Indeed, in order to ensure the presence of patients with MDS of different prognoses and of patients with CMML, inclusion will be stratified into the following three cohorts, each of which will include patients receiving immediate treatment and those initially opting for observation/support:

• Group 1: Patients with low or intermediate-1 risk MDS as per the International Prognostic Scoring System (IPSS) 1.

• Group 2: Patients with intermediate-2 or high risk MDS as per IPSS.

• Group 3: Patients with any type of CMML as per the prognosis index CMML Prognostic Scoring System (CPSS) 2.

A total of 600 patients are expected to be recruited from 50 sites.

Primary objective:

To assess clinical evolution from the time of diagnosis in patients with MDS or CMML, within normal clinical practice.

The study will assess event free survival (EFS) depending on the therapeutic strategy initially adopted by the investigator after a diagnosis of MDS or CMML under normal clinical practice conditions.

EFS is defined as the period of time elapsed between diagnosis of the condition (MDS or CMML) and the appearance of one of the following events:

• Progression of the disease, or

• All-cause death, or

• Appearance of a clinically significant condition requiring a change in initial therapeutic strategy, or

• Appearance of an adverse event* requiring treatment to be suspended. *This applies to all patients included in the study, under active or support treatment.

Secondary objectives:

1. To describe the demographic, clinical (including MDS or CMML classification as per WHO 2008 (5, 19)), and analytical characteristics of patients recently diagnosed with MDS or CMML. Clinical characteristics include a health assessment as per the CIRS-G scale (Cumulative Illness Rating Scale for Geriatrics), the comorbidity rating for SMD (MDS-CI) by Della Porta and Malcovatti, 20 or other scale used across all participating sites in normal clinical practice, and the performance status of the patient as per ECOG scale.

2. To describe the therapeutic strategies initially applied for patients with MDS or CMML, based on clinical characteristics (specific cytogenetic alterations, adverse events, etc.), age, health status (CIRS-G scale), ECOG and IPSS (IPSS-R) or CPSS, in addition to the reasons for adopting different initial therapeutic strategies.

3. To analyse the response of MDS/CMML to treatment based on the IWG ( International Working Group) response criteria in myelodysplasia, modified in 2006.

4. To describe patient evolution based on time-dependent response parameters.

• Time to Progression of the disease (TTP) as per the IWG response criteria modified in 2006.

• Evolution to acute myeloblastic leukaemia (AML) (median time until transformation into AML).

• To analyse progression free survival (PFS) from inclusion in the study until documented progression of MDS or CMML or death during follow-up.

• Overall survival (OS) measured from the date of diagnosis of the disease until date of all-cause death, where applicable.

• Assessment of overall response rate of dependence on red blood cell and platelet transfusions.

5. To document the tolerance profile (safety) of the treatment administered under normal clinical practice conditions.

6. To describe the use of healthcare resources relating to the initial therapeutic strategy for MDS or CMML in normal clinical practice, which can be financially measured, and to explore the possible differences both between the treat/do not treat option and the different therapeutic regimens administered.

7. To describe clinically significant events requiring a change in initial therapeutic strategy (counting for EFS, the primary objective of the study).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 503
• Est. completion date: December 14, 2018
• Est. primary completion date: August 31, 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

-1. Male or female subjects, aged 18 years or older. 2. Subjects with documented diagose of MDS or CMML within the last 3 months prior to entering the study and naive to treatment.

3. Signed informed consent.

Exclusion Criteria:

• 1. Subjects participating in an interventional clinical trial 2. Subjects who do not agree to participate.

Related Conditions & MeSH terms

• Leukemia
• Leukemia, Myelomonocytic, Chronic
• Leukemia, Myelomonocytic, Juvenile
• Myelodysplastic Syndrome
• Myelodysplastic Syndromes
• Preleukemia
• Syndrome

Intervention

Other:
• Either Wait and See, or Supportive Treatment, or Active Treatment at physician discretion
As described above

Locations

Country	Name	City	State
Spain	Complejo Hospitalrio La Mancha Centro	Alcázar de San Juan	Ciudad Real
Spain	Hospital Universitario Fundación Alcorcón	Alcorcón	Madrid
Spain	Hospital Punta de Europa	Algeciras	Cádiz
Spain	Complejo Hospitalario Torrecárdenas	Almeria
Spain	Hospital Universitario La Ribera	Alzira	Alicante
Spain	Hospital Universitari Germans Trias I Pujol	Badalona	Barcelona
Spain	Hospital de la Santa Creu I Sant Pau	Barcelona
Spain	Hospital Duran Reynals	Barcelona
Spain	Hospital Universitario Vall d'Hebron	Barcelona
Spain	Parc de Salut Mar- Hospital del Mar	Barcelona
Spain	Hospital Universitario Puerta del Mar	Cádiz
Spain	Hospital Universitario Reina Sofía	Córdoba
Spain	Hospital Universitario Virgen de la Arrixaca	El Palmar	Murcia
Spain	Hospital Universitario Getafe	Getafe	Madrid
Spain	Hospital Universitari de Girona Josep Trueta	Girona
Spain	Hospital de Antequera	Hospital De Antequera	Málaga
Spain	Hospital Can Misses	Ibiza
Spain	CHU-Insular	Las Palmas de Gran Canaria
Spain	Hospital Universitario de Gran Canaria Doctor Negrín	Las Palmas de Gran Canaria
Spain	Hospital de León	León
Spain	Hospital Lucus Augusti	Lugo
Spain	Hospital Clínico San Carlos	Madrid
Spain	Hospital Fundación Jimenez Díaz	Madrid
Spain	Hospital Universitario Infanta Leonor	Madrid
Spain	Hospital Universitario Ramon y Cajal	Madrid
Spain	Hospital Universitario Virgen de la Victoria	Málaga
Spain	Hospital Costa del Sol	Marbella	Málaga
Spain	Hospital General Universitario Santa Lucía	Murcia
Spain	Complejo Universitario Hospitalario de Ourense	Ourense
Spain	Hospital Universitario Central de Asturias	Oviedo
Spain	Hospital Son Llàtzer	Palma de Mallorca
Spain	Complejo Hospitalrio Universitario de Pontevedra	Pontevedra
Spain	Hospital Universitario Salamanca	Salamanca
Spain	Hospital Universitario Donostia	San Sebastián	Guipuzcoa
Spain	Hospital Universitario Marqués de Valdecilla	Santander
Spain	Complejo Hospitalario Universitario de Santiago	Santiago de Compostela
Spain	Hospital General de Segovia	Segovia
Spain	Hospital de Valme	Sevilla
Spain	Hospital Universitario Virgen Macarena	Sevilla
Spain	Hospital Santa Bárbara	Soria
Spain	Hospital Nuestra Señora del Prado	Talavera de la Reina	Toledo
Spain	Hospital Arnau de Vilanova	Valencia
Spain	Hospital Clínico Universitario Valencia	Valencia
Spain	Hospital Universitario Doctor Peset	Valencia
Spain	Hospital Universitario La Fe	Valencia
Spain	Hospital Clínico Universitario de Valladolid	Valladolid
Spain	Hospital Universitario Río Hortega	Valladolid
Spain	Hospital Universitario Txagorritxu	Vitoria	Alava
Spain	Hospital Clínico Universitario Lozano Blesa	Zaragoza
Spain	Hospital Universitario Miguel Servet	Zaragoza

Sponsors (1)

Lead Sponsor	Collaborator
Celgene

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Event Free Survival	Period of time elapsed between diagnosis of the condition (MDS or CMML) and the appearance of one of an event: Progression of the disease, death (all causes), clinically significant condition requiring a change in initial therapeutic strategy, adverse event requiring treatment discontinuation	Up to a minimum of 36 months' follow-up from the start of active treatment
Secondary	Health Assesment/performance Status	Changes from baseline to end of study as per CIRS-G scale, MDS-CI, ECOG	Approximately 3 years
Secondary	Response to active treatment	Response to active treatment up to progressions	Up to end of treatment for each patient
Secondary	Patient evolution based on time-dependent response parameters	Time to progression, time to evolution to AML (median time until transformation into AML), Progression Free Survival (from inclusion into the study to documented progression or death), Overall Survival (from diagnosis to death, all causes), Overall rate of dependence on red blood cells and platelet transfusion	Approximately 3 years
Secondary	Adverse Events	Safety profile description of treatment under normal clinical practice conditions	Approximately 3 years
Secondary	Patient Description	Demography, Clinical history of MDS or CMML, Blood test, Relevant comorbidities variables	Baseline