Polish Registry of SMA Patients - Role in Trial Recruitment

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Miłosz Duda
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1 Polish Registry of SMA Patients - Role in Trial Recruitment A. Lusakowska 1, A.Kostera Pruszczyk 1, M.Jedrzejowska 2, B.Ryniewicz 1 A. Kaminska 1 1 Department of Neurology, Medical University of Warsaw, Poland 2 Institute of Experimental and Clinical Medicine, Polish Academy of Science Warsaw, Poland

2 How did we learn about TREAT-NMD? 2007, EAMDA meeting in Warsaw invitation to participate in TREAT-NMD activities as a member 2008 The neuromuscular group from Department of Neurology of Medical University of Warsaw started to gather patients for The Polish Registry of Patients with Neuromuscular Disorders 2010 The project Clinical and genetic characteristics of neuromuscular diseases for future application of gene therapy funded by the Ministry of Science and Higher Education, from January 2010 to January 2012 (No. 641/N- TREAT/09/2010/0).

3 TREAT-NMD support

4 How did we develop the Registry? Identification of patients with DMD/ BMD and SMA treated in Department of Neurology or in our Out-patient Clinic at present or in the past Sources of information: pedigrees, case records, biopsy results, genetic test results - from late sixties up to now

5 How did we increase the number of the patients in the Registry? Collaboration with patients organisations (3 in Poland) Information on the website of Department of Neurology Information sent to patients in files of Department of Neurology Information given during lectures for doctors from all over Poland specializing in neurology (on basic and advanced levels), for family doctors and pediatricians and students of Medical University Information ( posters, oral presentations) given during Congresses of Polish Society of Child Neurologists and Polish Neurological Society Poster information sent out to all Genetic Counseling Centers

kontakt dr Anna Łusakowska tel. 22-599 27 70 dr Anna Kostera-Pruszczyk tel. 22-599 29 85 dr Barbara Ryniewicz tel. 22-599 29 86 dr Maria Jędrzejowska tel.

dystrofią miotoniczną (DM1 i DM2) realizowany jest w ramach projektu Kliniczno - genetyczna charakterystyka chorób nerwowo mięśniowych w celu przyszłego zastosowania leczenia przy pomocy terapii

6 kontakt dr Anna Łusakowska tel dr Anna Kostera-Pruszczyk tel dr Barbara Ryniewicz tel dr Maria Jędrzejowska tel (Zespół Nerwowo-Mięśniowy IMDiK PAN) Polski Rejestr Pacjentów z Chorobami Nerwowo - Mięśniowymi dystrofią mięśniową Duchenne a i Beckera (DMD i BMD) rdzeniowym zanikiem mięśni (SMA) dystrofią miotoniczną (DM1 i DM2) realizowany jest w ramach projektu Kliniczno - genetyczna charakterystyka chorób nerwowo mięśniowych w celu przyszłego zastosowania leczenia przy pomocy terapii genowej, będącego częścią międzynarodowej inicjatywy TREAT - NMD (Translational Research in Europe Treatment and Assessment of Neuromuscular Diseases - Europejska Sied Badawcza - Diagnostyka i Leczenie Chorób Nerwowo-Mięśniowych Celem Rejestru jest poprawa standardów opieki i leczenia pacjentów z chorobami nerwowo - mięśniowymi umożliwienie polskim pacjentom udziału w próbach klinicznych z zastosowaniem nowych terapii Szczegółowe informacje o Rejestrze znajdą Paostwo na stronie Koordynator projektu: Katedra i Klinika Neurologii Warszawskiego Uniwersytetu Medycznego

7 Polish Registry of DMD/BMD and SMA Patients October 2011

8 Population of SMA patients in Polish Registry

9 What are our plans? Registry of DM1 and DM2 patients - under construction Population of identified patients in Neurological Department of Warsaw

10 Clinical trial in Poland concerning SMA in the past EUROSMART (acetyl-l-carnitine) 2003/2004.

11 Ongoing SMA clinical trial with olesoxime sponsored by TROPHOS TITLE: Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled Study to assess Safety and Efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients (SMA 2 and SMA 3 non ambulant) (Belgium, France, Germany, Italy, Netherlands, Poland, United Kingdom) 160 patients were recruited in 10 months Primary Outcome Measures: Motor Function Measure (MFM) D1+D2 score Secondary Outcome Measures: HFMS ( Hammersmith functional scale), electromyography (CMAP-compound Muscle Action Potential and MUNE- Motor Unit Number), Olesoxime cholesterol- like molecule, mitochondrial pore modulator (mptp). Olesoxime promotes the function and survival of motorneurons.

12 SMA clinical trial with olesoxime sponsored by TROPHOS January/2010 Polish Registry was invited to participate in feasibility enquiry prepared by TROPHOS. The feasibility enquiry were reviewed and approved by Global Database Oversigh Committee March /2010 Anonymized and aggregate data on SMA patients was sent back to TREAT-NMD May /2010 Acceptance of participation in clinical trial sponsored by Trophos 20 patients could be enrolled Febuary/2011 Annoucement of clinical trial on Department of Neurology website 05 Apr 2011 first patient enrollment 17 Aug 2011 last patient enrollment 4 months recruitment All patients were enrolled to the trial in the Department of Neurology in Warsaw (trial site)

13 Distribution of the recruited patients in Poland into the Trophos trial Warsaw

14 Recruitment for trial using SMA Registry Step I selection of patients from database fitting to the basic inclusion criteria (age, type of SMA, non-ambulant SMA type 3 patients, genetically confirmed) Step II telephone contact with patients: preliminary evaluation of possibilty of participation in the trial (verification of qualified patients according to further criteria, willingness to participate) Step III - screening visit (V-1) verification according to all inclusion and exlusion criteria definitive selection CRUCIAL ROLE OF THE REGISTRY IN STEP I AND STEP II

15 Inclusion and exclusion criteria of Trophos trial in relation to the Registry information Inclusion criteria 11 inclusion criteria only 3 covered by database Exclusion criteria 13 major exclusion criteria None was covered by database

16 Conclusions Registry plays a very important role for patients recruitment into clinical trials - accelerates and facilitates identification of the eligible patients The data contained in registry are sufficient for preliminary selection of the patients for the clinical trial Since the inclusion and exclusion criteria are specific for a given trial, the definitive recruitment of patients needs an individual assesment

17 Thank you for your attention!

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