RGX-111 Gene Therapy in Patients With MPS I

Phase 1/2 Suspended 21 enrolled REGENXBIO Inc.

Non-randomizedSingle-groupOpen-labelTreatment

Summary

RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I.

Timeline

Start: 2019-04-03
Primary completion: 2027-07-07
Completion: 2029-03-06

Outcome

Outcome not reported

Stopped: “The FDA placed a clinical hold on RGX-111 investigational gene therapy following an initial single case report of neoplasm (intraventricular CNS tumor) in a participant treated in its phase I/II study four years prior.”

Publications

De BP, Rosenberg JB, Selvan N, Wilson I, Yusufzai N, Greco A, Kaminsky SM, Heier LA, Ricart Arbona RJ, Miranda IC, Monette S, Nair A, Khanna R, Crystal RG, Sondhi D. Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease. Hum Gene Ther. 2023 Sep;34(17-18):905-916. doi: 10.1089/hum.2023.067.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	RGX-111	Gene therapy (AAV / viral vector)	1e+10 unknown	Other
Subject	RGX-111	Gene therapy (AAV / viral vector)	5e+10 unknown	Other