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

ConditionHereditary Spastic Paraplegia Type 50
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age4-72
SponsorElpida Therapeutics SPC

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Male and females between the ages of 4 months to 72 months at the time of screening.

Molecularly-confirmed diagnosis of SPG50 (confirmed by a CLIA certified, CE-marked, or equivalent lab): Genomic DNA mutation analysis demonstrating bi-allelic pathogenic or likely pathogenic variants in the AP4M1 gene.

Subjects must have features of neurologic dysfunction by clinical history and physical examination.

Stable doses of concomitant medications such as anti-spasticity medications, anti-seizure medications, behavioral management medications, sleep medications, and special diets, supplements, or nutritional support for at least 3 months prior to Screening. If recent changes (< 3 months) in medications, the subject may be allowed per Investigator judgement.

Disqualifiers

None

Trial design

Design model

Parallel

Treatments tested in this trial

  • MELPIDA

    Genetic

    Gene Therapy agent

Treatment groups

24 Participants
are divided into 2 treatment groups
Group A: MELPIDA TreatmentExperimental treatment 1 intervention
Group B: Matched Prospective Concurrent Control ArmNo intervention 0 interventions

Trial outcomes

Primary outcomes

1

Gross Motor Function Measure (GMFM-88) Defined Major Milestones

Change in total percent score of the 8 Major Motor Milestone Scores from baseline in treated group compared to change in total percent score of the 8 Major Motor Milestone Scores from baseline in untreated controls 1. #24: Sit on mat: Maintain, arms free, 3 seconds 2. #38: Prone: Creeps forward 1.8m (6') 3. #52: On the floor: Pulls to stand at large bench 4. #67: Standing: 2 hands held: walks forward 10 steps 5. #69: Standing: Walks forward 10 steps 6. #84: Standing: Holding 1 rail: walks up 4 steps, holding 1 rail, alternating feet 7. #85: Standing: Holding 1 rail: walks down 4 steps, holding 1 rail, alternating feet 8. #88: Standing on 15cm (6") step: Jumps off, both feet simultaneously

Time frame
156 weeks

Secondary outcomes

1

Composite Endpoint Defined by the Win Ratio

Composite Endpoint Defined by the Win Ratio using matching criteria after 156 Weeks of Follow-up of the 8 selected items and the raw scores of the Cognitive domain of the Bayley Scale of Infant and Toddler Development 4th Edition (Bayley-4).

Time frame
156 weeks
2

Developmental Milestones- Bayley-4 Cognitive Domain

Developmental Milestones- Bayley-4 Cognitive Domain-Change in Total Raw Score from Baseline

Time frame
156 weeks
3

Gross and Fine Motor Function (GMFM-88 full scale)

Gross and Fine Motor Function (GMFM-88 full scale) - Change in Total Score from Baseline.

Time frame
156 weeks
4

Disease Severity (Spastic Paraplegia Rating Scale )

Disease Severity (Spastic Paraplegia Rating Scale ). Change in Total Score from Baseline.

Time frame
156 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Elpida Therapeutics SPC

Lead sponsor

University of Texas Southwestern Medical Center

Collaborator

Hospital Sant Joan de Deu

Collaborator

Source ClinicalTrials.gov
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