Fut, nem toborozFázis nélkül
Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial
A legfontosabb adatok
- Ki végzi
- Prime Medicine, Inc.
- Hol
- Ann Arbor, Boston, Chicago, Los Angeles, New Haven, Sacramento és San Antonio (Egyesült Államok)Mind a 7 helyszín megnézése
- Azonosító
- Állapot
- Fut, nem toboroz
- Résztvevők
- 30 fő, tervezett
- Kezdés
- 2025. dec. 29.
- Várható befejezés
- 2027. dec.
- A vizsgálat típusa
- Megfigyeléses
- A regiszteradat frissítve
- 2026. május 12.
Ki vehet részt
- Életkor
- 18 év felett
- Nem
- Nők és férfiak
- Egészséges önkéntesek
- Nem vehetnek részt
A regiszter feltételei, szó szerint
Mind a 9 feltétel megnézéseKevesebb
Ki vehet részt
- Confirmed Wilson Disease (WD) as determined by the following criteria:; An established clinical diagnosis of WD; Genetic analysis confirming the presence of biallelic pathogenic variants at ATP7B, at least one of which is EITHER p.H1069Q OR p.R778L OR Participants without a confirmed genetic diagnosis may enroll only with explicit approval from the Medical Monitor
Ki nem vehet részt
- Prior history of gene therapy, liver transplantation, hepatocyte (cellular) transplantation, or active listing for liver transplantation
- Significant neurological conditions within the prior 12 months which may impact participant safety or participation in the study, including ability to complete study requirements or procedures as outlined in the clinical study protocol
- Prior or active malignancy or myeloproliferative disorder (excluding Stage 1 or lower, fully treated/excised malignant and pre-malignant disease of the skin, cervix or colon. Additionally, any other malignant and pre-malignant disease that the Investigator in consultation with the treating oncologist and study Medical Monitor deem has been fully treated/excised for > 5 years)
- In patients with psychiatric involvement, current or fluctuant clinical instability with new or changing diagnoses or substantial medication regimen changes in the past 12 months that could limit their participation, in the opinion of the Investigator
- History of cirrhotic decompensation within the past year
- Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the participant or would preclude the participant from successful study completion, including participant unable or unwilling to comply with the protocol requirements
- For individuals with known ATP7B genotype: individual does not have at least 1 ATP7B allele with either the p.H1069Q or p.R778L mutation
- Current participation in an investigational study for the treatment of WD
Helyszínek itt: Egyesült Államok
- University of Michigan MedicineAnn Arbor, Egyesült Államok
- Massachusetts General HospitalBoston, Egyesült Államok
- Northwestern UniversityChicago, Egyesült Államok
- University of California Los Angeles Medical CenterLos Angeles, Egyesült Államok
- Yale New Haven HospitalNew Haven, Egyesült Államok
- University of California Davis HealthSacramento, Egyesült Államok
- American Research CorporationSan Antonio, Egyesült Államok
A vizsgálatról
Mit akar kideríteni a vizsgálat
The aim of this study is to inform and improve future clinical trials in Wilson Disease (WD) by better understanding how patients with WD are living with and managing the disease, and by identifying key factors that shape their decisions to participate in clinical research.
A vizsgálat csoportja
- Participants with at least 1 allele with the ATP7B p.H1069Q or p.R778L mutation
Adatok és források
- Regiszteradat
- Szerepel a ClinicalTrials.gov-on.
- Hivatalos cím
- Prescreening Study to Identify Potential Participants for a Clinical Study in Wilson Disease (WD) Due to the ATP7B p.H1069Q or p.R778L Mutations
- A regiszteradat frissítve
- 2026. május 12.