Clinical trial NCT03110432: Prospective German Very High Cardiovascular Risk Patients Dyslipidemia Treatment Indication Registry
Official title: Prospective German Very High Cardiovascular Risk Patients Dyslipidemia Treatment Indication Registry
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Dyslipoproteinemias
Hypercholesterolemia, Familial
Familial Hypercholesterolemia - Homozygous
Familial Combined Hyperlipidemia
Keywords: Cardiovascular
LDL Cholesterol
Drug utlisation
high risk
secondary prevention
PCSK9 inhibition
Lead sponsor: GWT-TUD GmbH
Sponsor class: OTHER
First posted: 2017-04-12
Enrollment: 1695
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with dyslipidemia and very high cardiovascular risk ("highest risk patients" according to G-BA stipulation for PCSK9i use)

Brief summary: This is a prospective German registry for patients with dyslipidemia with very high cardiovascular risk who principally meet the Gemeinsamer Bundesausschuss (G-BA) stipulations for Proprotein convertase subtilisin/kexin like type 9 inhibitor (PCSK9i) use, and are treated by office-based cardiologists or in lipid ambulances.

Detailed description: The study is purely observational, and will document data from the patient charts only. Treatment of patients will not be changed by this study, and all clinical decisions (including on frequency of visits) will be upon the discretion of the physician. No blood samples must be taken solely for the purpose of the study.

The registry will include two types of centers: 1) office-based cardiologists and 2) specialized lipid ambulances (outpatient departments).

Data on patient disease and treatment history will be collected a first documentation (retrospectively).

The documentation time is 3 years per patient. After the baseline visit, patients are followed-up every 6 ± 2months (last visit at month 36). This interval is considered narrow enough not to miss important events (safety reporting, cardiovascular events, hospitalizations).

Patients with stable (maintenance) lipid-lowering therapy (including those with existing PCSK9i therapy) or those with any therapy changes (including newly initiated PCSK9i treatment) will be documented in this study. Compared to the former group with stable drug treatment, the latter group will likely have major LDL-C changes during the first few weeks, which will be accounted for by (retrospective) monthly documentation in the first 3 months (data will be documented at the 6-month visit.

The documentation periods will be substantially longer than in the controlled studies of the PCSK9i (endpoints were as early as 3 months), and thus will provide much-needed information about the long-term effects on LDL-C and other lipid parameters, safety, and drug retention rates. Longer follow-up periods would likely be compromised by high rates of (administrative) discontinuation rates.

Eligibility criteria: Inclusion Criteria:

* • with familial, homozygous hypercholesterolemia, in whom pharmaceutical and diet options for lipid lowering have proved insufficient, or

  * with confirmed familial, heterozygous hypercholesterolemia under consideration of the total familial risk, or
  * with heterozygous familial or non- familial hypercholesterolemia or mixed dyslipidemia with

    * therapy refractory course
    * maximal dietary and pharmaceutical lipid lowering therapy - in any case documented over a 12-month period
    * unsatisfactorily lowered LDL-C value (and thus with an indication for LDL apheresis)
    * confirmed vascular disease
    * other risk factors for cardiovascular events

Exclusion Criteria:

* Concurrent participation of the patient in a clinical randomised study.

Primary outcomes:
measure: LDL cholesterol goal achievement
timeFrame: 3 years
Secondary outcomes:
measure: LDL cholesterol reduction
timeFrame: up top 3 years
measure: Number of treatment changes
timeFrame: up to 3 years
Other outcomes:
measure: Change in quality of life
timeFrame: up to 3 years
Locations:
city: Leipzig
country: Germany
facility: Klinik und Poliklinik für Kardiologie, Universitätsklinikum
city: Tübingen
country: Germany
facility: Innere Medizin IV - Diabetologie, Endokrinologie und Nephrologie am Universitaetsklinikum
city: Villingen-Schwenningen
country: Germany
facility: Nephrologisches Zentrum
Study officials:
affiliation: GWT-TUD GmbH, Germany
name: David Pittrow, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Innere Medizin IV - Diabetologie, Endokrinologie und Nephrologie am Universitaetsklinikum Tuebingen, Germany
name: Andreas Birkenfeld, MD, PhD
role: STUDY_CHAIR
affiliation: Nephrologisches Zentrum Villingen-Schwenningen, Germany
name: Bernd Hohenstein, MD, PhD
role: STUDY_CHAIR
affiliation: Klinik für Innere Medizin III, Universität des Saarlandes, Germany
name: Ulrich Laufs, MD, PhD
role: STUDY_CHAIR
affiliation: Nephrologisches Zentrum Göttingen GbR, Germany
name: Volker JJ Schettler, MD, PhD
role: STUDY_CHAIR
affiliation: Lipid Clinic, Charité Universitaetsmedizin, Berlin, Germany
name: Elisabeth Steinhagen-Thiessen, MD, PhD
role: STUDY_CHAIR
affiliation: Ludwig Maximilian University, Medizinische Klinik und Poliklinik IV, Muenchen, Germany
name: Klaus G Parhofer, MD, PhD
role: STUDY_CHAIR
References:
citation: Parhofer KG, Pittrow D, Birkenfeld AL, Fraass U, Hohenstein B, Siegert C, Klotsche J, Steinhagen-Thiessen E, Dexl S, Schettler VJJ, Laufs U. Determinants of lipid lowering therapy intensification in very high risk patients with dyslipidaemia eligible for PCSK9 monoclonal antibodies: 1-year outcomes of the PERI-DYS study. Acta Cardiol. 2025 Apr 24:1-11. doi: 10.1080/00015385.2025.2490381. Online ahead of print.
citation: Laufs U, Birkenfeld AL, Fraass U, Hohenstein B, Siegert C, Klotsche J, Steinhagen-Thiessen E, Pittrow D, Dexl S, Salmen S, Schettler VJJ, Parhofer KG; Collaborators in the PERI-DYS Study. Novel Insights into the Management of Patients with Very High Cardiovascular Risk Eligible for PCSK9 Inhibitor Treatment: Baseline Findings from the PERI-DYS Study. Cardiovasc Drugs Ther. 2024 Feb;38(1):119-129. doi: 10.1007/s10557-022-07386-0. Epub 2022 Sep 30.