Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss
A legfontosabb adatok
- Hol
- Allentown, Bethesda, Boston és Oklahoma City (Egyesült Államok)Mind a 4 helyszín megnézése
- Fázis
- 1/2. fázis
Helyszínek itt: Egyesült Államok
- Lehigh Valley Hospital-Cedar CrestAllentown, Egyesült ÁllamokContact: Ravi N. Samy, MD, FACSRésztvevőt keres
- National Institutes of Health Clinical CenterBethesda, Egyesült ÁllamokContact: Maame Akumatey (Study Coordinator)Résztvevőt keres
- Massachusetts Eye and Ear Infirmary(MEEI)Boston, Egyesült ÁllamokContact: Michael Cheung, MSc, CCRP, Clinical Research Project ManagerRésztvevőt keres
- Hearts for Hearing-Oklahoma CityOklahoma City, Egyesült ÁllamokContact: René Gifford, PhDRésztvevőt keres
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
- Skylark Bio Inc.
- Azonosító
- NCT07627971
- A regiszteradat frissítve
- 2026. augusztus 27.
- Eredeti leírás
Skylark Bio is conducting a study of an investigational new drug called SKY-GJB2 and its delivery device, the SKY-CAT. SKY-GJB2 is a gene therapy that is being developed to treat children who have hearing loss due to changes in the GJB2 gene (also referred to as Connexin 26). The SKY-CAT is a device that delivers the gene therapy.A teljes eredeti leírásKevesebb
A Phase 1/2, Open-label, Single Dose Study of SKY-GJB2 Given as a Single Unilateral Intracochlear Administration in Pediatric Subjects With GJB2-mediated Hearing Loss
Skylark Bio is conducting a study of an investigational new drug called SKY-GJB2 and its delivery device, the SKY-CAT. SKY-GJB2 is a gene therapy that is being developed to treat children who have hearing loss due to changes in the GJB2 gene (also referred to as Connexin 26). The SKY-CAT is a device that delivers the gene therapy.
The purpose of this study is to:
Learn about the safety and tolerability (does not cause ongoing discomfort) of SKY-GJB2 Evaluate the efficacy of SKY-GJB2 (how well is works)
Bevonási feltételek: Male or female aged 9 months to 7 years at the time of gene therapy administration; Subjects must have at least two (biallelic) pathogenic or likely pathogenic variants in the GJB2 gene; Bilateral sensorineural hearing loss as assessed by ABR. Hearing loss at ≥85 dB HL for at least one of the frequencies (500-4000 Hz) in the study treatment ear; Subject's parent(s)/guardian(s) provide informed consent before the initiation of study-related procedures; Subject is able and willing to comply (or provide assent if old enough) will all study requirements.
Kizáró feltételek: Bilateral Cochlear Implants; Subject has non-GJB2 mediated hearing loss including genetic, syndromic, or non-syndromic hearing loss that is not associated with GJB2 mutations; Subject has autosomal dominant nonsyndromic hearing loss due to GJB2 mutation; No response on ABR testing.