Clinical trial NCT05776472: A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Official title: A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
Collaborators: name: Iqvia Pty Ltd
First posted: 2023-03-20
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult patients (≥18 years) with PNH, who started routine treatment with pegcetacoplan up to 12 months prior to enrollmentor at enrollment

Brief summary: This is a 36-month, long-term, multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH. Patients meeting the eligibility criteria will be enrolled in the study and followed prospectively for approximately 36 months. Patient data will be collected from start of pegcetacoplan treatment to end of follow-up. Retrospective data on pegcetacoplan will be captured from the time of pegcetacoplan treatment initiation. Pegcetacoplan treatment data will be collected for a minimum of approximately 36 months and up to a maximum of approximately 72 months, including retrospective period depending on when the patient started pegcetacoplan treatment. After pegcetacoplan treatment discontinuation, patients will remain in the study for 8 weeks to capture any AEs. The scope of the study is to collect both retrospective and prospective data. Baseline is defined as start of pegcetacoplan treatment. The main part of the study will be prospective,collecting data on effectiveness, safety (all AEs), patient- and clinician-reported outcomes and health care resource use.

Detailed description: As pegcetacoplan is a new product on the market, with a new mechanism of action, there is an urgent need to provide data to treaters, payers and the PNH community on the real-world usage and effectiveness of pegcetacoplan. This study aims to fill part of that knowledge gap and to add to the knowledge base regarding the use of pegcetacoplan in routine medical practice. Another important rationale for this study is to provide information on RBC transfusions and health care resource utilization pre and post pegcetacoplan treatment initiation.

The study plans to include approximately 200 patients at 70 sites in Europe, Middle East, Canada and Australia. Additional countries may be added in the study if necessary. Patients meeting the eligibility criteria will be enrolled in the study and followed prospectively for approximately 36 months. Patient data will be collected from start of pegcetacoplan treatment to end of follow-up. Retrospective data on pegcetacoplan will be captured from the time of pegcetacoplan treatment initiation. Pegcetacoplan treatment data will be collected for a minimum of approximately 36 months and up to a maximum of approximately 72 months, including retrospective period depending on when the patient started pegcetacoplan treatment. After pegcetacoplan treatment discontinuation, patients will remain in the study for 8 weeks to capture any AEs. Patients will come to their routine visits and the available data from each visit will be collected.

The scope of the study is to collect both retrospective and prospective data. The main part of the study will be prospective, collecting data on effectiveness, safety, patient- and clinician-reported outcomes and health care resource use. The study will also collect retrospective data before pegcetacoplan treatment start, which will consist of information on PNH treatment, blood transfusions and healthcare resource use. Data will be collected for up to 12 months prior to pegcetacoplan treatment start. As patients may have been treated with pegcetacoplan for up to 12 months prior to enrollment, retrospective data may be collected for up to 24 months. This means that the total data collection period including both the retrospective and the prospective part is up to 48 (+/- 3) months.

Eligibility criteria: Inclusion Criteria:

* Patients ≥18 years of age with a documented PNH diagnosis.
* Patient started routine treatment with pegcetacoplan for PNH up to 12 months before enrollment or prescribed pegcetacoplan at enrollment. Decision to initiate treatment shall be made by the treating physician and independently from the decision to include the patient in the study.
* Patient is willing and able to provide written informed consent to participate in the study in a manner approved by the Institutional Review Board/Independent Ethics Committee and local regulations.

Exclusion Criteria:

* Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to the start of the current pegcetacoplan treatment.
* Initiated current treatment with pegcetacoplan in an interventional study.

Primary outcomes:
measure: Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months
timeFrame: 6 months
Secondary outcomes:
measure: Change of LDH values from initiation of pegcetacoplan treatment to 6 months
timeFrame: 6 months
measure: Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months
timeFrame: 6 months
measure: Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months
timeFrame: 6 months
measure: Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months
timeFrame: 6 months
measure: Change in Ferritin from initiation of pegcetacoplan treatment to 6 months
timeFrame: 6 months
measure: Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 6 months
measure: Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 12 months
measure: Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 18 months
measure: Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 24 months
measure: Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 30 months
measure: Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 36 months
measure: Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 6 months
measure: Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 12 months
measure: Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 18 months
measure: Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 24 months
measure: Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 30 months
measure: Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 36 months
measure: Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 6 months
measure: Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 12 months
measure: Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 18 months
measure: Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 24 months
measure: Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 30 months
measure: Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 36 months
measure: Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 6 months
measure: Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 12 months
measure: Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 18 months
measure: Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 24 months
measure: Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 30 months
measure: Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 36 months
measure: Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 6 months
measure: Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 12 months
measure: Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 18 months
measure: Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 24 months
measure: Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 30 months
measure: Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 36 months
measure: Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 6 months
measure: Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 12 months
measure: Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 18 months
measure: Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 24 months
measure: Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 30 months
measure: Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 36 months
measure: Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 6 months
measure: Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 12 months
measure: Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 18 months
measure: Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 24 months
measure: Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 30 months
measure: Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 36 months
measure: Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 6 months
measure: Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 12 months
measure: Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 18 months
measure: Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 24 months
measure: Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 30 months
measure: Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 36 months
measure: Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 6 months
measure: Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 12 months
measure: Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 18 months
measure: Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 24 months
measure: Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 30 months
measure: Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
timeFrame: 36 months
measure: Annualized number of red blood cell (RBC) transfusions during pegcetacoplan treatment until end of follow-up compared to the 12 month period before pegcetacoplan treatment
timeFrame: 12 months
measure: Annualized number of red blood cell (RBC) units during pegcetacoplan treatment until end of follow-up compared to the 12 month period before pegcetacoplan treatment
timeFrame: 12 months
measure: Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 6 months
measure: Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 12 months
measure: Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 18 months
measure: Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 24 months
measure: Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 30 months
measure: Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 36 months
measure: Health care resource use: Annualized number of hospitalizations and emergency room visits during pegcetacoplan treatment until end of follow-up compared to the 12-month period before pegcetacoplan treatment.
timeFrame: 12 months
measure: Patient treatment satisfaction every 6 months until end of follow-up
timeFrame: 6 months
measure: Patient treatment satisfaction every 6 months until end of follow-up
timeFrame: 12 months
measure: Patient treatment satisfaction every 6 months until end of follow-up
timeFrame: 18 months
measure: Patient treatment satisfaction every 6 months until end of follow-up
timeFrame: 24 months
measure: Patient treatment satisfaction every 6 months until end of follow-up
timeFrame: 30 months
measure: Patient treatment satisfaction every 6 months until end of follow-up
timeFrame: 36 months
measure: Physician treatment satisfaction every 6 months until end of follow-up
timeFrame: 6 months
measure: Physician treatment satisfaction every 6 months until end of follow-up
timeFrame: 12 months
measure: Physician treatment satisfaction every 6 months until end of follow-up
timeFrame: 18 months
measure: Physician treatment satisfaction every 6 months until end of follow-up
timeFrame: 24 months
measure: Physician treatment satisfaction every 6 months until end of follow-up
timeFrame: 30 months
measure: Physician treatment satisfaction every 6 months until end of follow-up
timeFrame: 36 months
measure: Adverse events (AE), including serious adverse events (SAE)
timeFrame: 24 hours
measure: Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 6 months
measure: Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 12 months
measure: Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 18 months
measure: Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 24 months
measure: Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 30 months
measure: Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 36 months
measure: Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 6 months
measure: Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 12 months
measure: Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 18 months
measure: Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 24 months
measure: Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 30 months
measure: Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 36 months
measure: Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 6 months
measure: Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 12 months
measure: Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 18 months
measure: Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 24 months
measure: Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 30 months
measure: Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 36 months
measure: Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 6 months
measure: Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 12 months
measure: Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 18 months
measure: Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 24 months
measure: Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 30 months
measure: Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 36 months
measure: Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 6 months
measure: Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 12 months
measure: Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 18 months
measure: Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 24 months
measure: Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 30 months
measure: Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 36 months
measure: estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 6 months
measure: estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 12 months
measure: estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 18 months
measure: estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 24 months
measure: estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 30 months
measure: estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 36 months
measure: Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 6 months
measure: Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 12 months
measure: Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 18 months
measure: Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 24 months
measure: Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 30 months
measure: Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 36 months
measure: Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 6 months
measure: Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 12 months
measure: Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 18 months
measure: Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 24 months
measure: Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 30 months
measure: Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 36 months
measure: Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 6 months
measure: Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 12 months
measure: Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 18 months
measure: Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 24 months
measure: Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 30 months
measure: Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
timeFrame: 36 months
Other outcomes:
Locations:
city: Liverpool
country: Australia
facility: Liverpool Hospital
state: New South Wales
city: Waratah
country: Australia
facility: Calvary Mater Newcastle
state: New South Wales
city: Birtinya
country: Australia
facility: Sunshine Coast University Hospital
state: Queensland
city: Brugge
country: Belgium
facility: AZ Sint-Jan
city: Bruxelles
country: Belgium
facility: CHU Brugmann-Site Horta
city: Liège
country: Belgium
facility: Centre Hospitalier Universitaire de Liege
city: Turnhout
country: Belgium
facility: AZ Turnhout - Campus Sint-Elisabeth
city: Toronto
country: Canada
facility: University Health Network
state: Ontario
city: Montréal
country: Canada
facility: Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-Montréal
state: Quebec
city: Brno
country: Czechia
facility: Fakultni nemocnice Brno
city: Praha 2
country: Czechia
facility: Ustav hematologie a krevni transfuze
city: Helsinki
country: Finland
facility: Helsingin yliopistollinen keskussairaala
city: Nice
country: France
facility: CHU Nice - Hôpital de l'Archet 1
state: Alpes Maritimes
city: Pessac cedex
country: France
facility: CHU de Bordeaux - Hôpital Haut-Lévêque
state: Gironde
city: Suresnes Cedex
country: France
facility: Hopital Foch
state: Hauts De Seine
city: Grenoble
country: France
facility: CHU de Grenoble - Hôpital Albert Michallon
state: Isere
city: Nantes cedex 1
country: France
facility: CHU de Nantes - Hotel Dieu
state: Loire Atlantique
city: Vandœuvre-lès-Nancy
country: France
facility: CHU de Nancy - Hôpital de Brabois Adultes
state: Meurthe Et Moselle
city: Lille cedex
country: France
facility: Hopital Claude Huriez - CHRU Lille
state: Nord
city: Paris La Defense
country: France
facility: Hôpital Saint-Louis
state: Paris
city: Pierre-Bénite
country: France
facility: Centre Hospitalier Lyon Sud
state: Rhone
city: Poitiers
country: France
facility: CHU Poitiers
city: Goettigen
country: Germany
facility: Alb-Fils-Kliniken GmbH
state: Baden Wuerttemberg
city: Aschaffenburg
country: Germany
facility: Studienzentrum Aschaffenburg
state: Bayern
city: Frankfurt
country: Germany
facility: Universitaetsklinikum Frankfurt Goethe-Universitaet
state: Hessen
city: Gießen
country: Germany
facility: Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen
state: Hessen
city: Offenbach
country: Germany
facility: Dres Ballo und Dr. Böck
state: Hessen
city: Essen
country: Germany
facility: Universitaetsklinikum Essen
state: Nordrhein Westfalen
city: Leipzig
country: Germany
facility: Universitaetsklinikum Leipzig AoeR
state: Sachsen
city: Saalfeld
country: Germany
facility: Praxis Fenchel Saalfeld
state: Thueringen
city: Koeln
country: Germany
facility: MV Zentrum für Onkologie und Hämatologie
city: Athens
country: Greece
facility: Evangelismos Hospital
city: Athens
country: Greece
facility: University General Hospital "Attikon"
city: Larissa
country: Greece
facility: University General Hospital of Larissa
city: Thessaloniki
country: Greece
facility: General Hospital of Thessaloniki "IPPOKRATEIO"
city: Thessaloníki
country: Greece
facility: G Papanikolaou General Hospital
city: Budapest
country: Hungary
facility: DPC - Orszagos Hematologiai es Infektologiai Intezet
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Budapest
country: Hungary
facility: Semmelweis University
city: Bari
country: Italy
facility: Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
city: Bassano Del Grappa
country: Italy
facility: Ospedale San Bassiano
city: Bologna
country: Italy
facility: Irccs Aou Di Bologna- Pol. Di S.Orsola
city: Cagliari
country: Italy
facility: Ospedale Oncologico Armando Businco
city: Firenze
country: Italy
facility: Azienda Ospedaliera Universitaria Careggi
city: Milano
country: Italy
facility: Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
city: Milano
country: Italy
facility: Ospedale San Raffaele
city: Novara
country: Italy
facility: Ospedale Maggiore della Carità
city: Reggio Calabria
country: Italy
facility: Grande Ospedale Metropolitano - Presidio Morelli
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
city: Rome
country: Italy
facility: Università di Roma La Sapienza
city: Torino
country: Italy
facility: Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
city: Białystok
country: Poland
facility: Uniwersytecki Szpital Kliniczny w Bialymstoku
city: Bydgoszcz
country: Poland
facility: Szpital Uniwersytecki nr 2 im. dr J. Biziela w Bydgoszczy
city: Gdańsk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
city: Kraków
country: Poland
facility: SPZOZ Szpital Uniwersytecki w Krakowie
city: Lublin
country: Poland
facility: Uniwersytecki Szpital Kliniczny nr 1 w Lublinie
city: Warszawa
country: Poland
facility: Warszawski Uniwersytet Medyczny
city: Łódź
country: Poland
facility: WWCOiT in Lodz, Oddział Hematologii i Transplantologii
city: Buraydah
country: Saudi Arabia
facility: Prince Faisal Bin Bandar Cancer Center
city: Jeddah
country: Saudi Arabia
facility: King Faisal Specialist Hospital & Research Center
city: Riyadh
country: Saudi Arabia
facility: King Faisal Specialist Hospital and Research Center - Madinah
city: Riyadh
country: Saudi Arabia
facility: King Faisal Specialist Hospital and Research Center - Riyadh
city: Badalona
country: Spain
facility: ICO Badalona - Hospital Universitari Germans Trias i Pujol
city: Barakaldo
country: Spain
facility: Hospital de Cruces
city: Barcelona
country: Spain
facility: Hospital Clinic de Barcelona
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Bilbao
country: Spain
facility: Hospital de Basurto
city: El Palmar
country: Spain
facility: Hospital Universitario Virgen de la Nieves
city: Las Palmas De Gran Canaria
country: Spain
facility: Hospital Universitario de Gran Canaria Dr. Negrin
city: Lugo
country: Spain
facility: Hospital Lucus Augusti
city: Malaga
country: Spain
facility: Hospital Regional Universitario de Malaga
city: Oviedo
country: Spain
facility: Hospital Universitario Central de Asturias
city: San Sebastian
country: Spain
facility: Hospital Universitario Donostia
city: Santiago De Compostela
country: Spain
facility: Complejo Hospitalario Universitario de Santiago
city: London
country: United Kingdom
facility: King's College Hospital
Study officials:
affiliation: Swedish Orphan Biovitrum
name: Study Physician +46 08-697-20 00, medical.info@sobi.com
role: STUDY_DIRECTOR
References: