Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.

Phase 3 Recruiting 24 enrolled Elpida Therapeutics SPC Hospital Sant Joan de Deu · collab University of Texas Southwestern Medical Center · collab

Non-randomizedParallel-groupOpen-labelTreatment

Summary

Phase 3, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of MELPIDA in individuals with Hereditary Spastic Paraplegia Type 50 (SPG50).

Timeline

Start: 2026-04-01
Primary completion: 2032-02-28
Completion: 2032-06-01

Publications

Agianda HAP, Kim HM, Battaglia N, Rong J, Tam A, Gonzalez Saez-Diez E, Boerkoel CF, Saffari A, Quiroz V, Schierbaum L, Zaman Z, Bernardi K, Ebrahimi-Fakhari D. Diagnostic Utility of the ATG9A Ratio in AP-4-Associated Hereditary Spastic Paraplegia. Ann Clin Transl Neurol. 2026 Apr;13(4):834-839. doi: 10.1002/acn3.70308. Epub 2026 Jan 5.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	MELPIDA	Gene therapy (AAV / viral vector)	—	Intrathecal

Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.

Summary

Timeline

Publications

Drugs

Indications