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Tests treatment safety and results for Hereditary Spastic Paraplegia Type 50
Official title Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.
ClinicalTrials.gov ID: NCT06692712
What this study is testing
What is MELPIDA?
MELPIDA is an investigational medicine, being studied as a potential treatment for hereditary spastic paraplegia type 50.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 72
You may be able to join if
- Inclusion: For the treatment group
- 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...
- people 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...
See the full eligibility criteria
Who can join
- Inclusion: For the treatment group
- 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.
- people 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...
- Parent/legal guardian willing to provide written informed consent for their child prior to participation in the study,
- people and caregivers must demonstrate the ability to travel to the study center. For the 30 days post treatment people must reside within 100 miles (approximately 160 km) of the clinical site. For the control group
- Male and females between the ages of 4 to 72 months at the time of screening.
- A molecularly confirmed diagnosis of SPG47, SPG50 or SPG52 (confirmed by a CLIA certified, CE-marked, or equivalent lab). Genomic DNA mutation analysis demonstrating bi-allelic pathogenic variants in the AP4B1, AP4M1...
- people must have features of neurologic dysfunction by clinical history and physical examination.
- Parent/legal guardian willing to provide written informed consent for their child prior to participation in the study.
- Subject able to comply with all protocol requirements and procedures.
- people and caregivers must demonstrate the ability to travel to the study center. Exclusion For the treatment group
- Loss of one of the 8 major motor milestones within the last 12 months. Milestones defined as:
- #24: Sit on mat: Maintain, arms free, 3 seconds
- #44: 4 Point: Crawls or hitches forward 1.8m (6')
- #53: Standing: Maintains, arms free, 3 seconds
- #67: Standing: 2 hands held: walks forward 10 steps
- #69: Standing: Walks forward 10 steps
- #84: Standing: Holding 1 rail: walks up 4 steps, holding 1 rail, alternating feet
- #85: Standing: Holding 1 rail: walks down 4 steps, holding 1 rail, alternating feet
- #88: Standing on 15cm (6") step: Jumps off, both feet simultaneously
- Inability to participate in the clinical evaluation as determined by the principal investigators.
- Clinically significant abnormal laboratory values (hemoglobin \ 20 g/dL; white blood cell \> 20,000 per cmm, platelets count \ ULN; GGT, ALT, and AST or total bilirubin \> 1.5 × ULN, creatinine ≥ 1.5 mg/dL) prior to...
- Presence of a concomitant medical condition (eg, scoliosis or bleeding disorder) that precludes a lumbar puncture or use of anesthetics for sedated procedures.
- Documented cardiomyopathy or significant congenital heart abnormalities.
- History of severe/life-threatening allergic reaction to sirolimus, tacrolimus, corticosteroids, or gadolinium.
- Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer, or interactions with the immunosuppressive agents.
- Any item which would exclude the subject from being able to undergo MRI according to local institutional policy, or any other procedure.
- The presence of significant AP-4 related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study.
- Recent or planned elective surgical procedures (within 6 months) that would confound the scientific rigor or interpretation of results of the study.
- Failure to obtain appropriate informed consent.
- Reason to believe that the subject or parents of the subject will not comply with the study procedures outlined in the study protocol.
- Have received an investigational drug within 30 days prior to screening or plan to receive an investigational drug (other than gene therapy) during the study.
- Enrollment and participation in another treatment clinical trial 90 days before first visit (screening). For the control group
- Loss of one of the 8 major motor milestones within the last 12 months. Milestones defined as:
- #24: Sit on mat: Maintain, arms free, 3 seconds
- #44: 4 Point: Crawls or hitches forward 1.8m (6')
- #53: Standing: Maintains, arms free, 3 seconds
- #67: Standing: 2 hands held: walks forward 10 steps
- #69: Standing: Walks forward 10 steps
- #84: Standing: Holding 1 rail: walks up 4 steps, holding 1 rail, alternating feet
- #85: Standing: Holding 1 rail: walks down 4 steps, holding 1 rail, alternating feet
- #88: Standing on 15cm (6") step: Jumps off, both feet simultaneously
- Inability to participate in the clinical evaluation as determined by the principal investigators.
- Any other situation that would exclude the subject from undergoing any other procedure required in this study.
- The presence of significant AP-4 related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study.
- Recent or planned elective surgical procedures that would confound the scientific rigor or interpretation of results of the study.
- Failure to obtain appropriate informed consent.
- Reason to believe that the subject or parents of the subject will not comply with the study procedures outlined in the study protocol.
- Have received an investigational drug within 30 days prior to screening or plans to receive an investigational drug (other than gene therapy) during the study.
- Enrollment and participation in another treatment clinical trial 90 days before first visit (screening).
The study team makes the final eligibility decision.
Where it's taking place
- Dallas, Texas, United States
- Barcelona, Spain
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 4 months to 72 months. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Dallas, Texas, United States; Barcelona, Spain. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.