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New treatment option for Spasticity, Muscle
Official title Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
ClinicalTrials.gov ID: NCT05518188
What this study is testing
What is MELPIDA?
MELPIDA is an investigational medicine, given as an once-daily injectable medicine, being studied as a potential treatment for spasticity, muscle.
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
- MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss. Outcomes will evaluate the safety and tolerability of a single dose of MELPIDA, which will be measured by the treatment-associated adverse events (AEs) and serious adverse events (SAEs).
- Phase 2: a mid-size study of how well it works
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 10
You may be able to join if
- Age 4 months-10 years old
- Confirmed diagnosis of SPG50 disease by:
- Genomic DNA mutation analysis demonstrating homozygous or compound heterozygous, confirmed pathogenic variants in the AP4M1 gene
- Clinical history or examination features consistent with SPG50 and that include neurologic dysfunction
- Parent/legal guardian willing to provide written informed consent for their child prior to participation in the study
You likely can't join if
- Inability to participate in study procedures (as determined by the site investigator)
- Presence of a concomitant medical condition that precludes lumbar puncture (LP) or use of anesthetics
- History of bleeding disorder or any other medical condition or circumstance in which lumbar puncture is contraindicated according to local...
- Inability to be safely sedated in the opinion of the clinical anesthesiologist
- Active infection, at the time of dosing, based on clinical observations
- Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer
See the full eligibility criteria
- Age 4 months-10 years old
- Confirmed diagnosis of SPG50 disease by:
- Genomic DNA mutation analysis demonstrating homozygous or compound heterozygous, confirmed pathogenic variants in the AP4M1 gene
- Clinical history or examination features consistent with SPG50 and that include neurologic dysfunction
- 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
- Ability to stand for more than 5 seconds OR
- Ability to take 5 steps independently or with a walker OR
- Modified Ashworth Scale score 2 or below (Ankles).
- Inability to participate in study procedures (as determined by the site investigator)
- Presence of a concomitant medical condition that precludes lumbar puncture (LP) or use of anesthetics
- History of bleeding disorder or any other medical condition or circumstance in which lumbar puncture is contraindicated according to local institutional policy
- Inability to be safely sedated in the opinion of the clinical anesthesiologist
- Active infection, at the time of dosing, based on clinical observations
- Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer
- Inability of the patient to undergo MRI according to local institutional policy
- Inability of the patient to undergo any other procedure required in this study
- The presence of significant non-SPG50 related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study
- 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
- Contraindication to MELPIDA or any of its ingredients
- Contraindication to any of the immune suppression medications used in this study
- Clinically significant abnormal laboratory values (GGT, ALT, and AST, or total bilirubin \> 3 × ULN, creatinine ≥ 1.5 mg/dL, hemoglobin [Hgb] \ 20 g/dL; white blood cell [WBC] \> 20,000 per cmm) prior to gene...
The study team makes the final eligibility decision.
Where it's taking place
- Dallas, Texas, United States
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 10 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Dallas, Texas, United States. 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.