Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)
A legfontosabb adatok
- Hol
- Houston (Egyesült Államok)A helyszín megnézése
- Fázis
- 1/2. fázis
Helyszínek itt: Egyesült Államok
- UTHealth HoustonHouston, Egyesült ÁllamokNo longer taking part
Kérdések a vizsgálat csapatának
- Milyen gyakran kell bejárnom, és egy-egy alkalom mennyi ideig tart?
- Megtérítik az utazási költséget?
- Megtarthatom a saját orvosomat?
- Mi történik, ha ki akarok lépni?
Contact the study team: see the registry record → Contacts and Locations
Oldal nyomtatása az orvosomnakA vizsgálat adatai
- Ki végzi
- n-Lorem Foundation
- Azonosító
- NCT07226297
- A regiszteradat frissítve
- 2025. november 10.
- Eredeti leírás
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Charcot-Marie-Tooth disease type 2D (CMT2D) due to a pathogenic, de novo deletion mutation in GARS1A teljes eredeti leírásKevesebb
An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Charcot-Marie-Tooth Type 2D Due to GARS1 Genetic Mutation
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Charcot-Marie-Tooth disease type 2D (CMT2D) due to a pathogenic, de novo deletion mutation in GARS1
Bevonási feltételek: Informed consent/assent provided by the participant (when appropriate), and/or the participant's parent(s) or legally authorized representative(s); Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records; Genetically confirmed GARS1 genetic variant.
Kizáró feltételek: Participant has any condition that, in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.