These trials completed more than 12 months ago and should have reported results.
These trials are not yet due to report results: some are ongoing, some completed within the past 12 months.
These trials have problematic data on the registry. Details why »
|Status||Trial ID||Title||Completion date||Category|
|Exempt, with results||2017-000813-22||An Open-Label, Single Arm, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects with Leber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p....||2019-10-02||not-yet-due|
|Exempt||2017-004806-17||A first in human, double-blind, randomized, intra-subject placebo-controlled, multiple dose study of QR-313 evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subject...||not-yet-due|
|Exempt||2018-002433-38||A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene||not-yet-due|
|Exempt||2018-003500-40||An Open-Label Extension Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of QR-110 in Subjects with Leber’s Congenital Amaurosis (LCA) due to the C.2991+1655a>G Mutation (P.C...||not-yet-due|
|Other||2018-003501-25||A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety, Tolerability and Systemic Exposure of QR-110 in Subjects with Leber's Congenital Amaurosis (LCA) due to c...||not-yet-due|
|Other||2020-000535-45||An Open-Label, Dose Escalation and Double-Masked, Randomized, Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects <8 Years of Age with Leber Congenital Amauros...||not-yet-due|