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

Part of paid clinical trials in Dallas, Texas.

Sponsor
Elpida Therapeutics SPC
Study ID
NCT06692712
Phase
PHASE3
Status
Recruiting
Apply to this trial

Conditions

  • Hereditary Spastic Paraplegia Type 50

Eligibility Criteria

Sex
ALL
Age
4 Months - 72 Months
Healthy Volunteers
Not accepted

Interventions

  • MELPIDA — GENETIC
    Gene Therapy agent

Study Details

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).

Key Dates

Start date
Apr 1, 2026
Status verified
Apr 2026
Primary completion
Feb 28, 2032
Completion
Jun 1, 2032

Study Design

Enrollment
24 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: MELPIDA Treatment
    Eligible subjects (N=8) will receive a single open-label intrathecal administration of MELPIDA and follow up to week 260.
  • No Intervention: Matched Prospective Concurrent Control Arm
    Approximately 16 untreated age- and disease- matched controls with confirmed AP-4-related disease (SPG47, SPG50, or SPG52) will be enrolled and attend study visits concurrent with the MELPIDA treatment arm.

Primary Outcome Measure

Gross Motor Function Measure (GMFM-88) Defined Major Milestones [ Time Frame: 156 weeks ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of Texas Southwestern Medical CenterDallasTexas75025-

Find similar trials in Dallas, TX

By research site
University of Texas Southwestern Medical Center· Dallas, TX

Related Studies