drugset

drugset / Trial / NCT02341807

Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations

NCT02341807 ↗

Phase 1/2 Completed 15 enrolled Spark Therapeutics, Inc.

Non-randomizedParallel-groupOpen-labelTreatment

Summary

This clinical study evaluates the safety and tolerability of AAV2-hCHM in participants with Choroideremia gene mutations.

Timeline

Start: 2015-01-15
Primary completion: 2022-10-12
Completion: 2022-10-12

Publications

Aleman TS, Huckfeldt RM, Serrano LW, Pearson DJ, Vergilio GK, McCague S, Marshall KA, Ashtari M, Doan TM, Weigel-DiFranco CA, Biron BS, Wen XH, Chung DC, Liu E, Ferenchak K, Morgan JIW, Pierce EA, Eliott D, Bennett J, Comander J, Maguire AM. Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial. Ophthalmology. 2022 Oct;129(10):1177-1191. doi: 10.1016/j.ophtha.2022.06.006. Epub 2022 Jun 15.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	AAV2-hCHM	Gene therapy (AAV / viral vector)	5e+10 vg	Other
Subject	AAV2-hCHM	Gene therapy (AAV / viral vector)	1e+11 vg	Other

Indications