Primary Bilary Cirrhosis (PBC)
Conditions
Brief summary
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.
Detailed description
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.
Interventions
None listed
Sponsors
University of Leipzig
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Age ≥ 18 years 2. Diagnosis of PBC according to EASL criteria 3. Treatment with seladelpar 4. Written informed consent
Exclusion criteria
1. current or previous participation in a phase I to IV interventional clinical trial for seladelpar treatment of PBC 2. Pregnancy and breastfeeding
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| biochemical response according to POISE criteria | start of seladelpar therapy (week 0) until week 48 after start of seladelpar | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | start of seladelpar therapy (week 0) until week 48 after start of seladelpar | * ALP \< 1.5 x ULN * ALP \< 1.0 x ULN * bilirubin \< 1.0 x ULN * bilirubin \< 0.6 x ULN |
| Improvement in Vibration Controlled Transient Elastography (VCTE) | start of seladelpar therapy (week 0) until week 48 after start of seladelpar | 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. |
| indication for seladelpar | start of seladelpar therapy (week 0) until week 48 after start of seladelpar | The treating physician provides the indication for seladalpar as one of "biochemical response", "pruritus", "both". |
| AEs and SAEs will be classified using the Medical Dictionary for Regulatory Activities (MedDRA) | 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) |
| concomitant diseases | start of seladelpar therapy (week 0) until week 48 after start of seladelpar | concomitant diseases as documented on every visit time point (week 0-4-8-12-24-36-48) |
| concomitant medication classes | start of seladelpar therapy (week 0) until week 48 after start of seladelpar | 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. |
| problems with pruritus | start of seladelpar therapy (week 0) until week 48 after start of seladelpar | Patients will be asked to report if they have problems with pruritus. Units of Measure: mild, moderate or severe. |
| questionnaire | 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.) | Patients will be asked to filled out the Numeric Rating Scale (NRS) Pruritus (for average and worst itch assessment) questionnaire |
Countries
Germany, Switzerland
Contacts
CONTACTJohannes Wiegand, Prof. Dr.
Outcome results
None listed