Subjects with a divergent PGx phenotype experience more side effects and/or reduced drug efficacy compared to those with a PGx phenotype in normal range.
ID
Bron
Verkorte titel
Aandoening
- Bewegingsstoornissen (incl. parkinsonisme)
Aandoening
Huntington's Disease
Betreft onderzoek met
Ondersteuning
Onderzoeksproduct en/of interventie
Uitkomstmaten
Primaire uitkomstmaten
Observed prescription changes in HD gene carriers.
Achtergrond van het onderzoek
Huntington's disease (HD) is a hereditary, neurodegenerative disorder characterized by motor, cognitive and psychiatric symptoms. Currently, HD can only be managed symptomatically, including a large variety in prescribed drugs. Many HD patients experience negative medication effects (e.g, side-effects or non-response). Pharmacogenetic (PGx) studies show how individual genetic differences affect medication efficacy and toxicity, and holds the potential to explain and resolve these negative medication effects. This study aims to characterize which part of negative medication effects can be resolved by implementing PGx testing. The ultimate goal is to better guide the long-term pharmacological management of HD in a more personalized manner by predicting the individual patient's risk of negative medication effects.
Doel van het onderzoek
Subjects with a divergent PGx phenotype experience more side effects and/or reduced drug efficacy compared to those with a PGx phenotype in normal range.
Onderzoeksopzet
Inclusion period: 2.5 years Follow-up period: 1 year
Publiek
Wetenschappelijk
Leeftijd
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
In order to be eligible to participate in this study, a subject must meet all of the following criteria: - A capacitated individual, aged ≥ 18 years. - Genetically confirmed CAG-repeat expansion of ≥ 36 in the HTT gene. - Either about to start with prescribed medication related to HD or already using one or more HD related drugs. HD-related drugs are drugs considered to treat symptoms that either are related to manifest HD or are prescribed in pre-motor manifest or prodromal HD stage for symptoms that may be attributed to HD. - Sufficient knowledge of the Dutch language to understand the subject information letter and sign the informed consent form.
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
A potential subject who meets any of the following criteria will be excluded from participation in this study: - Any medical condition, in the view of the investigator, which might endanger subject’s safety and/or satisfactory participation in the study.
Opzet
Deelname
Voornemen beschikbaar stellen Individuele Patiënten Data (IPD)
metc-ldd@lumc.nl
Opgevolgd door onderstaande (mogelijk meer actuele) registratie
Andere (mogelijk minder actuele) registraties in dit register
Geen registraties gevonden.
In overige registers
| Register | ID |
|---|---|
| NTR-new | NL8251 |
| Ander register | Medical Ethical Committee LDD (Leiden - Den Haag - Delft) : METC058 |
| CCMO | NL70391.058.19 |
| OMON | NL-OMON55123 |