The seladelpar registry will collect real-world data of patients with PBC diagnosis treated with seladelpar in the real-life scenario in Germany and Switzerland.
The seladelpar registry will offer data acquisition at weeks 0 - 4 - 8 - 12 - 24 - 36 - 48 of seladelpar treatment. Patients can be recruited either retro- or prospectively. The seladelpar registry will be an interim project until the start of the PBC 2.0 registry. Patients within the seladelpar registry will be characterized in detail with drug specific aspects, before it will be offered to transfer data for a long-term follow-up in the German PBC 2.0 registry if patients agree to this procedure in an upcoming informed consent process once the PBC 2.0 registry will be initiated.
Study Type
OBSERVATIONAL
Enrollment
100
biochemical response according to POISE criteria
The endpoint "POISE criteria" is reached if at the 48 week visit, ALP \< 1.67 x ULN and at least 15% lower than week 0 value from the start of seladelpar
Time frame: start of seladelpar therapy (week 0) until week 48 after start of seladelpar
alternative definitions of response at the 48 week visit for patients with ALP levels > 1.5 or > 1.0 x ULN or bilirubin levels > 1.0 or > 0.6 x ULN at week 0 of seladelpar therapy
* ALP \< 1.5 x ULN * ALP \< 1.0 x ULN * bilirubin \< 1.0 x ULN * bilirubin \< 0.6 x ULN
Time frame: start of seladelpar therapy (week 0) until week 48 after start of seladelpar
Improvement in Vibration Controlled Transient Elastography (VCTE)
Improvement in Vibration Controlled Transient Elastography (VCTE) as a surrogate for fibrosis. The mean individual relative change in VCTE values from week 0 to 48 weeks will be calculated. Furthermore, for patients with values above the cut-offs 8, 10 and 15 kPa at week 0, the proportion who then fall below these cut-offs will be provided.
Time frame: start of seladelpar therapy (week 0) until week 48 after start of seladelpar
indication for seladelpar
The treating physician provides the indication for seladalpar as one of "biochemical response", "pruritus", "both".
Time frame: start of seladelpar therapy (week 0) until week 48 after start of seladelpar
AEs and SAEs will be classified using the Medical Dictionary for Regulatory Activities (MedDRA)
AEs and SAEs will be classified using the Medical Dictionary for Regulatory Activities (MedDRA)
Time frame: start of seladelpar therapy (week 0) until week 48 after start of seladelpar
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Charité - Universitätsmedizin Berlin Med. Klinik für Gastroenterologie, Infektiologie und Rheumatologie (CBF)
Berlin, Germany
DRK Kliniken Berlin Mitte Klinik für Innere Medizin - Gastroenterologie, Hepatologie, Diabetologie, Angiologie und Abhängigkeitserkrankungen
Berlin, Germany
Universitätsklinikum Frankfurt Medizinische Klinik I, Haus 11/Gastroenterologie
Frankfurt, Germany
Asklepios Klinik St. Georg, Leberzentrum, IFI-Institut
Hamburg, Germany
Klinik für Gastroenterologie, Hepatologie und Endokrinologie; Medizinische Hochschule Hannover
Hanover, Germany
Universitätsklinikum Heidelberg; Medizinische Universitätsklinik Heidelberg, Innere Medizin IV
Heidelberg, Germany
Gastroenterologische Gemeinschaftspraxis Dres. Felten/Hinz/Mittrop/Sandmann/Wallner
Herne, Germany
Universitätsklinikum des Saarlandes, Klinik für Innere Medizin II, Gastroenterologie und Endokrinologie
Homburg, Germany
Universitätsklinikum Jena; Klinik für Innere Medizin IV - Gastroenterologie, Hepatologie, Infektiologie
Jena, Germany
GHZ - gastroenterologisch - hepatologisches MVZ Kiel GmbH
Kiel, Germany
...and 11 more locations
concomitant diseases
concomitant diseases as documented on every visit time point (week 0-4-8-12-24-36-48)
Time frame: start of seladelpar therapy (week 0) until week 48 after start of seladelpar
concomitant medication classes
The following concomitant medication classes will be documented: fibrates, statins, anti-pruritic therapy. Details on particular medication and dosage may be collected and this list may be expanded.
Time frame: start of seladelpar therapy (week 0) until week 48 after start of seladelpar
problems with pruritus
Patients will be asked to report if they have problems with pruritus. Units of Measure: mild, moderate or severe.
Time frame: start of seladelpar therapy (week 0) until week 48 after start of seladelpar
questionnaire
Patients will be asked to filled out the Numeric Rating Scale (NRS) Pruritus (for average and worst itch assessment) questionnaire
Time frame: start of seladelpar therapy (week 0) until week 48 after start of seladelpar (Questionnaires can only be collected prospectively. The number of questionnaires depends on the time of patient recruitment.)
questionnaire
Patients will be asked to filled out the PROMIS Fatigue Questionnaire questionnaire
Time frame: start of seladelpar therapy (week 0) until week 48 after start of seladelpar (Questionnaires can only be collected prospectively. The number of questionnaires depends on the time of patient recruitment.)
questionnaire
Patients will be asked to filled out the PBC-40 questionnaire
Time frame: start of seladelpar therapy (week 0) until week 48 after start of seladelpar (Questionnaires can only be collected prospectively. The number of questionnaires depends on the time of patient recruitment.)
questionnaire
Patients will be asked to filled out the Euro Quol (EQ)-5D questionnaire
Time frame: start of seladelpar therapy (week 0) until week 48 after start of seladelpar (Questionnaires can only be collected prospectively. The number of questionnaires depends on the time of patient recruitment.)