Palermo DEEP-3 study

DEEP-3 study
Palermo, 06 ottobre 2014
Palermo 15 Dicembre 2012 Do#. ssa Angela Vitrano U.O.C “Ematologia e Mala7e Rare del Sangue e degli Organi Ematopoie=ci “ A.O. “Riuni= Villa Soﬁa Cervello”-‐ Palermo WHAT IS DEEP-‐3? DEEP-3 is an observational safety study to evaluate the
nature and incidence of adverse effects of deferiprone
treatment in patients with beta-thalassaemia major aged
from 1 month to less than 18 years.
CONFIDENTIAL Product: DEFERIPRONE
Protocol No.: DEEP-3
Sponsor: Universitätsklinikum Erlangen
Maximiliansplatz
91054 Erlangen, Germany
Trial Coordinating Investigator: Dr. Maria Caterina Putti
Azienda Ospedaliera di Padova
Via Giustiniani, 35128 Padova, Italy
Funder: European Commission
(FP7 Framework Research Program “HEALTH- 2010.4.2-1: Off-patent
medicines for children”)
RECRUITMENT SITES AND PRINCIPAL INVESTIGATORS Town Country Ins=tu=on / address Principal Inves=gator Cairo Egypt Cairo University Faculty of Medicine Al Orman Guiza, Giza 12613 Prof. Amal El-‐
Beshlawy Athena Greece NaMonal And Kapodistrian University Of Athens CHRISTOU LADA, 6 -‐ 10561 Athena Prof. Antonis KaUamis Tirane Albania Qendra Spitalore Universitare "Nene Tereza" Tirane RR Dribes 370, Tirana Dr. Eleni Nastas Nicosia Cyprus Cyprus Ministry of Health, Nicosia Thalassaemia Center, Koritsas Street, 6 1474 Nicosia Dr. Soteroula Christou Tunis Tunisia Centre naMonal de Greﬀe de Moelle Osseuse, Rue Jebel Lakhdar, Bab Saadoun, 1006 Tunis Prof. Mohamed Bejaoui Napoli Italy Azienda Ospedaliera di Rilievo Nazionale "Antonio Cardarelli", Via A. Cardarelli, 9 -‐ 80131 Napoli Dr. Aldo Filosa Palermo Italy Azienda ospedaliera Ospedali RiuniM Villa Soﬁa-‐ Cervello, Viale Strasburgo 233 -‐ 90146 Palermo Prof. Aurelio Maggio RECRUITMENT SITES AND PRINCIPAL INVESTIGATORS Town Country Ins=tu=on / address Principal Inves=gator Padova Italy Azienda Ospedaliera di Padova, Via GiusMniani,1 -‐ 35128 Padova Dr. Maria Caterina Pub Bari Italy Azienda Ospedaliero-‐Universitaria, Consorziale Policlinico di Bari Piazza Giulio Cesare -‐ 70124 Bari Dr. Giovanni Carlo Del Vecchio Palermo Italy ARNAS "CIVICO-‐DI CRISTINA-‐BENFRATELLI" P.O. CIVICO-‐PALERMO, U.O.C. Ematologia Piazza N. LeoUa, 4 90100 Palermo Dr. Liana Cuccia Cosenza Italy " Presidio Ospedaliero Azienda Ospedaliera Cosenza Stabil. "Annunziata“, Centro di Studi della Microcitemia U.O.C. EmatologiaVia Felice Migliori – 87100 Cosenza Dr. Mariagrazia Bisconte LenMni SR Italy Ospedale Civile di LenMni, Centro di Thalassaemia, Via Ospedale 96016 LenMni Dr. Francesca Commendatore Modena Italy Policlinico di Modena, Clinica Pediatrica Via del Pozzo, 71 41100 Modena Dr. Giovanni Palazzi Sassari Italy Azienda Mista Ospedaliera-‐Universitaria di Sassari Clinica Pediatrica Università -‐ ASL 1 D.H per Talassemia Viale S. Pietro, 12 07100 Sassari Dr. Carlo Cosmi Cagliari Italy Ospedale Regionale per le Microcitemie-‐ASL8 Cagliari Via Jenner 09121 Cagliari Dr.ssa Raﬀaella Origa Firenze Italy Azienda Ospedaliero-‐Universitaria Meyer Viale Pieraccini, 24 50139 Firenze Dr Tommaso Casini OBJECTIVE(S) Primary
To investigate serious adverse reactions related to deferiprone treatment in
children aged 1 month - 18 years diagnosed with beta-thalassaemia major.
Secondary
To investigate:
•  non-serious adverse reactions related to deferiprone treatment in children
aged 1 month - 18 years diagnosed with beta-thalassaemia major
•  risk factors for ADRs related to deferiprone use
INVESTIGATIONAL PLAN Ø  Study Design/Schematic
This is a multi-centre, multi-national, observational cohort study. Patients
will be identified and data collected both retrospectively and prospectively.
Ø  Study Duration
Observation period is defined from the start of deferiprone treatment up to
conclusion or October 2015 whatever comes first.
Ø  Dosage, treatment regimen, route of administration
Any dose of deferiprone is acceptable. For prospective data collection
only authorised dosage are acceptable.
Ø  study population
All patients with beta-thalassaemia major treated with deferiprone for iron
overload and being aged 1 month to <18 years at the time of initiation of
the therapy will be included in the study.
ELIGIBILITY CRITERIA Inclusion Criteria:
1.  Patient has beta-thalassaemia major;
2. Patient has received deferiprone, either as monotherapy or in combination with
deferoxamine (DFO);
3. Patient is aged 1 month to <18 years at the start of therapy with deferiprone;
4. If data are collected retrospectively a minimum set of data (as defined in next
slide) must be available.
ELIGIBILITY CRITERIA Minimum data set
A minimum data set to be available and recorded for data collection is defined as
follows:
1. Demographic data
2. Exact dose and type of deferiprone treatment (including also name of the
product, start and end date)
3. HIV and hepatitis C, B status
4. Spleen size: splenectomised, normal, splenomegaly (once/year)
5. Annual amount of transfused PRBC (blood units or cc PRBC /kg/year)
6. Complete blood cell count and biochemical profile (creatinine, glucose, AST,
ALT,GGT, leukocytes (WBC), neutrophils, haemoglobin, platelets) at least 4
values/year
7. Ferritin levels at least once/year
ELIGIBILITY CRITERIA Exclusion Criteria
1.  Previous therapy and medical records cannot be obtained from the clinicians
(for the retrospective data collection).
2. A minimum set of data cannot be obtained
3. Patient has participated in an investigational study during the time of deferiprone
therapy and no consent to use data has been obtained from the sponsor of the
investigational study
4. Inability or unwillingness to sign consent form from parents/legal representatives
and/or the patients. (for prospective data collection)
5. Patient has received deferiprone as off-label use (for prospective data
collection)
STUDY ASSESSMENTS Retrospective data
Data for patients which already commenced deferiprone treatment at the time
the study commences will be collected retrospectively using intensive chart
review. All medical and therapy records of these patients will be screened
manually by trained researchers locally at each study site. A unique
anonymised identifier will be given to each individual patient to protect patients’
confidentiality. If the patients continue taking deferiprone at the time the study
commences data collection will be continued prospectively.
Prospective data
Prospective data will be collected by the treating clinician supported by
research staff. Prospective data concern data from patients newly starting on
deferiprone before October 2014 and data from patients which continue to take
deferiprone. For each consequent visit of the patient, clinicians will be asked to
report any adverse drug event (ADE) in patients under their care. Clinicians will
be reminded to collect data at each routine visit.
DATA TO BE COLLECTED Demographic
• Patient identifier
• Date of birth
• Genotype
• Gender
• Ethnicity
Baseline data
• Height and Weight before starting therapy
• Tanner stage, Spleen size, Body mass index before starting therapy
• Transfusion therapy (amount, frequency, annual blood cell consumption)
• Chelation therapy (age/year of initiation of therapy, type and dosage of
previous therapy)
• HIV and Hepatitis C, B status
DATA TO BE COLLECTED Ø  Diagnosis
All chronic and acute diagnoses will be obtained and classified using ICD 10. The
information to be collected comprise of the following
•  Date of diagnosis, Diagnosis Text, and Diagnosis Code ICD 10
Ø  Medication
All chronically used medications (taken for >1 month) prescribed along with
deferiprone will be documented and classified using ATC terminology.
Any dose alterations will also be documented. Medication chart are updated at
each visit.
.
DATA TO BE COLLECTED Ø  Laboratory test results
Haematology
• Haemoglobin*, Haematocrit
• Erythrocytes (RBC)
• Erythroblasts
• Leukocytes (WBC)*
• Neutrophils*
• Platelets*
Biochemistry
•
•
•
•
•
Sodium, Potassium, Serum creatinine*, Alkaline phosphatase (AP)
Aspartate aminotransferase (AST, SGOT)*
Alanine aminotransferase (ALT, SGPT)*
Gamma-Glutamyl transpeptidase (GGT)*
Total bilirubin, Total protein, Albumin, Uric acid, Urea, Glucose*,
Serum ferritin*
DATA TO BE COLLECTED Ø  Adverse Event Data
•  Type of ADR using MedDRA
•  Start date (and time) of onset of reaction
•  Stop date (and time) or duration of reaction
•  Additional comments (e.g. clinical notes to explain the cause of ADR,
consequences of ADR)
•  Severity
Data Management
v  Data entry into the e-CRF
ARRUOLAMENTO DEEP-‐3 Studio Retrospettivo
Attivare Agreement
Monitor dell’ AOR Villa Sofia-Cervello provvede alla
raccolta dati presso il Centro arruolante
Somma di 900,00 Euro per paziente erogata dall’AOR
Villa Sofia-Cervello vs Centro arruolante
Thank you

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