Thrombocythemia, EssentialNCT00567502
Safety and Pregnancy Outcomes in Thrombocythemia Patients Exposed to XAGRID® (Anagrelide Hydrochloride) Compared to Other Treatments
An observational study of Thrombocythemia, Essential, run by Shire at 164 sites in 14 countries.
Trial summary
This is an observational safety study being conducted in Europe comparing patients taking Xagrid to patients taking other cytoreductive treatments. The plan is to enrol at least 750 subjects taking Xagrid with up to 3000 subjects taking other cytoreductive therapies. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Thrombocythemia, Essential
- How many people
- 3,647 participants
- Where
- 164 sitesin 14 countries
- Study dates
- 31 May 2005to 30 Apr 2014
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients receiving cytoreductive therapy for the treatment of at-risk essential thrombocytopenia (ET).
Who may be able to join
- High risk ET patients
- Subjects who can give written informed consent.
- Subjects taking cytoreductive therapy
Who cannot join
- Contraindications listed by the product being used.
- Patient cannot be participating in another clinical trial.
Trial locations and contacts
164 sites in 14 countries.
164 sites in 14 countries
Italy41 sites
Ospedale Le Torrette
Ancona, 60020
Ospedale Arezzo
Arezzo, 52100
Ospedale Policlinico Consorziale
Bari, 70124
A.O. Spedali Civili di Brescia
Brescia, 25100
Presidio Ospedaliero Perrino
Brindisi, 72100
Ospedale Ferrarotto
Catania, 95124
Ospedale Civile
Civitanova Marche, 62012
Ospedale Santa Croce
Fano, 61132
Ospedale Careggi
Florence, 50139
Azienda Ospedaliera Ospedali Riuniti Foggia
Foggia, 71100
Ospedale S. Martino
Genova, 16132
Ospedale S.Martino
Genova, 16132
Ospedale San Martino
Genova, 16132
Ospedale San salvatore
L’Aquila, 67000
Ospedale Umberto I
Mestre, 30173
Policlinico
Modena, 41100
Azienda ospedaliera S. Gerardo
Monza, 20052
Ospedale A.Cardarelli
Naples, 80131
Ospedale Cardarelli
Naples, 80131
Universita Federico II
Naples, 80131
Ospedale Maggiore della Carita
Novara, 28100
Azienda Ospedaliera di Padova
Padova, 35128
Ospedale Maggiore
Parma, 43100
IRCCS Policlinico S. Matteo
Pavia, 27100
Policlinico Monteluce
Perugia, 06122
Ospedale S. Salvatore
Pesaro, 61100
Ospedale Civile
Piacenza, 29100
Ospedale civile
Ragusa, 97100
Ospedale Santa Maria delle Croci
Ravenna, 48100
Arcispedale S. Maria Nuova
Reggio Emilia, 42100
San camillo de Lellis
Rieti, 02100
Ospedale oncologico regionale
Rionero in Vulture, 85028
Policlinico Gemelli
Roma, 00168
Ospedale Nuova Regina Margherita
Rome, 00153
Ospedale Umberto I
Rome, 00161
Ospedale S.Spirito
Rome, 00193
Ospedale Casa Sollievo della Sofferenza
San Giovanni Rotondo, 41100
Padiglione ex-oncologico Ospedale S.Maria
Terni, 05100
Ospedale Luigi Gonzaga
Torino, 10043
Ospedale Molinette
Torino, 10126
Ospedale civile
Vigevano, 27029
Germany22 sites
Studienzentrum
Aschaffenburg, 63739
Praxis Dr. Heinrich, Dr. Brudler, Dr. Bangerter
Augsburg, 86150
Universitatsklinikum Koeln
Cologne, 50924
Praxis Schmitz and Steinmetz
Cologne, 50931
Hamatologisch-Onkologische Praxis
Dresden, 01307
Praxis Dr. Weniger und Dr. Bittrich
Erfurt, 99085
Onkologische Gemeinschaftspraxis
Frankfurt am Main, 60389
IORC GmbH
Hamburg, 22081
Study site
Hamburg, D-20095
Internistische Praxis
Hornberg, 34576
Gemeinschaftspraxis fur Hamatologie und Internistische Onkologie
Magdeburg, 39104
Hamatologisch-Onkologische Praxis
Moers, 47441
Praxis Dr. Schick
München, 81241
Ambulante Chemotherapie
München, 81679
Hamato-Onkologische Schwerpunktpraxis
Munich, 80639
Hamatologisch-Onkologische Gemeinschaftspraxis
Münster, 48149
Praxis und Tagesklinik fur Internistische Onkologie
Recklinghausen, 45657
Caritasklinik St. Theresia
Saarbrücken, 66113
Schwerpunktpraxis fur Hamatologie und Internistische Onkologie
Saarbrücken, 66113
Universitatsklinik Ulm
Ulm, 89081
Hamatologie und Onkologie
Weiden, 92637
Praxis Dr. med. Yolanda Rodemer
Wilhelmshaven, 26386
Greece22 sites
401 General Army Hospital
Athens, 11525
Henry Dunant Hospital
Athens, 11526
General Hospital of Athens G.Gennimatas
Athens, 11527
General Hospital of Athens Hippocratio
Athens, 11527
General Hospital of Athens Laiko
Athens, 11527
Laikon General Hospital of Athens
Athens, 11527
University Hospital of Athens Attikon
Athens, 12462
Hygeia Hospital
Athens, 15123
3rd Hospital of IKA
Athens
General Hospital of Athens
Athens
Metropolitan Hospital
Athens
University Hospital of Athens ¿Attikon¿
Athens
University Hospital of Heraklion
Heraklion, 71110
University Hospital of Ioannina
Ioannina, 45500
University General Hospital of Larissa
Larissa, 41110
Makarios Hospital
Nikiá
St. Andrews General Hospital of Patra
Pátrai, 26335
University Hospital of Patra
Pátrai, 26500
Study site
Piraeus
General Hospital of Thessaioniki
Thessaloniki, 54642
Ippokration Hospital D Clinic of Internal Medicine
Thessaloniki, 54642
Papageorgiou Hospital
Thessaloniki, 56403
France21 sites
Centre Hospitalier Victor Dupouy
Argenteuil, 95170
Centre Hospitalier de Beauvais
Beauvais, 60021
Hopital Avicenne
Bobigny
Hopital de la Cavale Blanche
Brest, 29609
CHU Clemenceau
Caen, 14033
Hopital Henri Mondor
Créteil, 94100
Cabinet Medical
Dijon, 21000
Hopital Simone Veil
Eaubonne, 95602
CHU de Grenoble Hopital Albert Michallon
Grenoble, 38043
Centre Hospitalier de Lens
Lens, 62307
Hopital Saint Vincent de Paul
Lille, 59020
CHRU de Lille - Hopital Claude Huriez
Lille, 59037
CHU de Limoges - Hopital Universitaire Dupuytren
Limoges, 87042
Centre Leon-Berard
Lyon, 69373
Hopital Nord
Marseille, 13915
C.H.U. de Nice Hopital de l Archet 1
Nice, 06202
Hopital Saint-Louis
Paris, 75010
Hopital Pitie Salpetriere
Paris, 75013
Hopital Hotel Dieu
Paris, 75181
Hopital Lariboisiere
Paris, 75475
Hopital Haut Leveque - CHU de Bordeaux
Pessac, 33604
Spain16 sites
Hospital San Eloy
Barakaldo, 48902
Hospital del Mar
Barcelona, 08003
Hospital Germans Trias i Pujol
Barcelona, 08916
Hospital Josep Trueta
Girona, 17007
Complexo Hospitalario Xeral Calde
Lugo, 27004
Hospital Universitario de la Princesa
Madrid, 28006
Hospital Universitario Ramon y Cajal
Madrid, 28034
Hospital Clinico San Carlos
Madrid, 28040
Hospital U. 12 de Octubre
Madrid, 28041
Hospital JM Morales Meseguer
Murcia, 30008
Hospital Clinico Universitario Salamanca
Salamanca, 37007
Hospital Universitario de Canarias
Santa Cruz de Tenerife, 38320
Hospital Clinico Universitario de Santiago
Santiago de Compostela, 15706
Hospital Clinico Universitario Valencia
Valencia, 46010
Consorcio Hospital General Universitario de Valencia
Valencia, 46014
Hospital Arnau de Vilanova
Valencia, 46015
Sweden12 sites
Falu Lasarett
Falun, 79182
Medicine Clinic
Gävle, SE 80187
Lanssjukhuset Ryhov
Jönköping, 55185
Ljungby Hospital Medical Clinic
Ljungby, 341 82
University Hospital MAS
Malmö, 20502
University Hospital
Örebro, 70185
Sodersjukhuset - Medicinkliniken
Stockholm, 11883
Karolinska University Hospital
Stockholm, 14186
Lanssjukhuset Sundsvall - Harnosand
Sundsvall, 85188
Uddevalla Hospital
Uddevalla, 45180
Akademiska Hospital Uppsala
Uppsala, 75185
Centrallasarettet - Medicinavdelningen
Västerås, 72189
United Kingdom12 sites
Royal United Hospital
Bath, BA1 3NG
Belfast City Hospital
Belfast, BT9 7AB
Birmingham Heartlands Hospital
Birmingham, B9 5SS
Avon Haematology Unit BHOC
Bristol, BS9 1BD
Darlington Memorial Hospital
Darlington, DL3 6HX
Doncaster Royal Infirmary
Doncaster, DN2 5LT
Georgina Oncology Unit
Dudley, DYI 2HQ
James Paget University Hospital
Great Yarmouth, NR31 6LA
Hull Royal Infirmary
Hull, HU3 2JZ
Guy's and Thomas' NHS Foundation Trust
London, SE17EH
North Tyneside General Hospital
North Shields, NE298NH
Royal Shrewsbury Hospital
Shrewsbury, SY3 8QR
Denmark7 sites
Rigshospitalet
Copenhagen, 2100
SUS-Esbjerg Maematologisk afdelning
Esbjerg, 6700
Herlev Hospital
Herlev, 2500
Odense University Hospital
Odense, 5000
Roskilde Sygehus
Roskilde, 4000
Vejle Sygehus
Vejle, 7000
Viborg Hospital
Viborg, 8800
Norway4 sites
Sykehuset Ostfold
Fredrikstad, 1603
Universitetssykehuset Nord-Norge Hf
Tromsø, 1919
Study site
Trondheim, 7006
Akershus University Hospital
Viken, 1474
Finland3 sites
Helsinki University Hospital
Helsinki, 00029
Jyvaskyla Central Hospital
Jyväskylä, 40620
Kuopio University Hospital
Kuopio, 70211
About this study
Official title: A Non-Interventional, Post Authorisation Safety Study, to Continuously Monitor Safety and Pregnancy Outcomes in a Cohort of At-Risk Essential Thrombocythaemia (ET) Subjects Exposed to Xagrid Compared to Other Conventional Cytoreductive Treatments
This is an observational safety study being conducted in Europe comparing patients taking Xagrid to patients taking other cytoreductive treatments. The plan is to enrol at least 750 subjects taking Xagrid with up to 3000 subjects taking other cytoreductive therapies. The study will collect follow up data for 5 years for each patient enrolled that will focus on collecting data related to pre-defined events (PDEs) and Suspected Serious Adverse Reactions (SSARs).
- Conditions
- Thrombocythemia, Essential
- Medical subject headings
- Thrombocythemia, Essential
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 1 | XAGRID® (anagrelide hydrochloride) | |
| 2 | Xagrid + Other cytoreductive | |
| 3 | Other cytoreductive |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With At Least One Pre-Defined Event (PDE), Deaths, Pregnancies
Pre-defined events (PDEs) were evaluated whenever an event occurred and was defined by a panel of independent qualified physicians, blinded to cytoreductive therapy, validated all PDEs prior to analysis (Event Validation Panel). Non-PDE death only included deaths not recorded as outcome of another PDE.
Measured: Up to 5 years
Number of Participants With Suspected Serious Adverse Reaction (SSAR) Events
SSAR: serious adverse event (SAE) that was considered related to cytoreductive therapy. SAE: any untoward medical occurrence that at any dose resulted in death, life-threatening (at the time of the event), in-patient hospitalization/prolongation of existing hospitalization (elective hospitalizations/procedures for pre-existing conditions that had not worsened were excluded), resulted in persistent or significant disability/incapacity or congenital abnormality/birth defect. Relatedness (suspected/not suspected) to XAGRID or other cytoreductive theraphy was determined by the investigator. As for SSARs, it was important to consider whether the events were related to XAGRID or other cytoreductive therapy. A participant was included in Xagrid or other treatment group based on treatment exposure, participants received Xagrid + Other was counted both in Xagrid and other treatment group.
Measured: Up to 5 years
Secondary outcomes (4)
Event Rate of Thrombohaemorrhagic Events
Event Rate of Thrombohaemorrhagic Events was calculated by dividing number of participants with events by total patient-year exposure. The reporting unit is per 100 participant-years of treatment exposure. Thrombohaemorrhagic Events is a composite endpoint of the PDEs myocardial infarction, angina, stroke, transient ischaemic attack, venous thromboembolic events, intermittent claudication/digital ischaemia, and major haemorrhagic events.
Measured: Up to 5 years
Platelet Count
Measured: Baseline, Month 6,12,18, 24, 30, 36, 42, 48, 54, 60
Duration of Exposure for Each Essential Thrombocythemia (ET) Therapy
Total duration for each participant = sum of [stop date - start date + 1] across all periods of time where the specific treatment was taken during the study, where start date = registration/consent date for treatments started before registration/consent date and/or stop date withdrawal/final date for treatments ongoing at the time of withdrawal/end of study. Where a participant has multiple records of the same therapy on the same day, the therapy is counted once for that day.
Measured: Up to 5 years
Cumulative Dose for Each Essential Thrombocythemia (ET) Therapy
Since the study is observational nature, interpreting the table is difficult due to inconsistencies in reporting the units of the dose.
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Dec 2007 (estimated)
- Study start31 May 2005
- Primary completion30 Apr 2014
- Study completion30 Apr 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Thrombocythemia, EssentialStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 3,647 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00567502
- Sponsor's protocol ID
- SPD422-401
- Status last verified
- May 2021
- First submitted
- 4 Dec 2007
- Last updated
- 9 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Birgegard G, Besses C, Griesshammer M, Gugliotta L, Harrison CN, Hamdani M, Wu J, Achenbach H, Kiladjian JJ. Treatment of essential thrombocythemia in Europe: a prospective long-term observational study of 3649 high-risk patients in the Evaluation of Anagrelide Efficacy and Long-term Safety study. Haematologica. 2018 Jan;103(1):51-60. doi: 10.3324/haematol.2017.174672. Epub 2017 Oct 27.
PubMed 29079600Gugliotta L, Besses C, Griesshammer M, Harrison C, Kiladjian JJ, Coll R, Smith J, Abhyankar B, Birgegard G. Combination therapy of hydroxycarbamide with anagrelide in patients with essential thrombocythemia in the evaluation of Xagrid(R) efficacy and long-term safety study. Haematologica. 2014 Apr;99(4):679-87. doi: 10.3324/haematol.2012.083097. Epub 2013 Dec 13.
PubMed 24334294Besses C, Kiladjian JJ, Griesshammer M, Gugliotta L, Harrison C, Coll R, Smith J, Abhyankar B, Birgegard G. Cytoreductive treatment patterns for essential thrombocythemia in Europe. Analysis of 3643 patients in the EXELS study. Leuk Res. 2013 Feb;37(2):162-8. doi: 10.1016/j.leukres.2012.11.004. Epub 2012 Nov 29.
PubMed 23199894Kiladjian JJ, Besses C, Griesshammer M, Gugliotta L, Harrison C, Coll R, Smith J, Birgegard G. Efficacy and safety of cytoreductive therapies in patients with essential thrombocythaemia aged >80 years: an interim analysis of the EXELS study. Clin Drug Investig. 2013 Jan;33(1):55-63. doi: 10.1007/s40261-012-0042-0.
PubMed 23184668