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A Phase IIb, Non-Profit, Open-label Trial for the Intrathecal Administration of AAV9/AP4M1 for Hereditary Spastic Paraplegia Type 50 (SPG50).

Status
Not yet recruiting
Phases
Phase 2
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2025-524481-15-00
Enrollment
2
Registered
2026-06-23
Start date
Unknown
Completion date
Unknown
Last updated
2026-06-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Heriditary spastic paraplegia (SPG50)

Brief summary

• Change in total percent score of the eight Major Motor Mile-stones derived from the Gross Motor Function Measure (GMFM)-88 from baseline at W156.

Interventions

DRUGMelpida

Sponsors

Rigshospitalet, Elpida Therapeutics SPC
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
0 Years to 17 Years

Design outcomes

Primary

MeasureTime frame
• Change in total percent score of the eight Major Motor Mile-stones derived from the Gross Motor Function Measure (GMFM)-88 from baseline at W156.—

Outcome results

None listed

Source: EU CTIS · Data processed: Jun 24, 2026