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Tests treatment safety and results for Angelman Syndrome
Official title A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
ClinicalTrials.gov ID: NCT07181837
What this study is testing
What is MVX-220?
MVX-220 is an investigational medicine, given as a treatment applied to the skin, being studied as a potential treatment for angelman syndrome.
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
- The purpose of this study is to evaluate the safety and efficacy of MVX-220 gene therapy in children and adults with Angelman syndrome with UBE3A gene deletion, uniparental disomy, or imprinting center defect genotypes.
- 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 50
You may be able to join if
- The participant's parent/legal guardian must provide written informed consent.
- Symptoms consistent with AS and documented genetic confirmation of one of the following genotypes resulting in a diagnosis of AS:
- Full maternal UBE3A gene deletion causing AS in the region of 15q11.2-q13
- Uniparental disomy
- Imprinting center defect
You likely can't join if
- Clinically significant medical finding other than AS, that, in the judgment of the Investigator would make the participant unsuitable for...
- Laboratory abnormalities including but not limited to:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> upper limit of normal (ULN)
- Total and/or fractionated bilirubin (direct and/or indirect) \> ULN
- Gamma-glutamyl transferase (GGT) \> ULN
- Estimated glomerular filtration rate (eGFR) below the lower limit of normal (LLN) for age
See the full eligibility criteria
- The participant's parent/legal guardian must provide written informed consent.
- Symptoms consistent with AS and documented genetic confirmation of one of the following genotypes resulting in a diagnosis of AS:
- Full maternal UBE3A gene deletion causing AS in the region of 15q11.2-q13
- Uniparental disomy
- Imprinting center defect
- The participant must be 18 to 50 years of age, inclusive (for adult participants), or 4 to 8 years of age, inclusive (for pediatric participants), at Screening.
- The participant must have the ability to ambulate independently.
- The participant must be on stable antiepileptic medications (with no changes within 1 month prior to the Screening visit, except for weight associated dose adjustments). Key
- Clinically significant medical finding other than AS, that, in the judgment of the Investigator would make the participant unsuitable for participation.
- Laboratory abnormalities including but not limited to:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> upper limit of normal (ULN)
- Total and/or fractionated bilirubin (direct and/or indirect) \> ULN
- Gamma-glutamyl transferase (GGT) \> ULN
- Estimated glomerular filtration rate (eGFR) below the lower limit of normal (LLN) for age
- Hemoglobin \< 8 g/dL
- White blood cell (WBC) count outside the normal range for age
- Platelet count \< LLN
- Partial thromboplastin time (PTT) outside the reference range
- PT/International normalized ratio (INR) outside the reference range
- Any known history and/or family history of hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) or multisystem inflammatory syndrome (MIS).
- Any known history and/or family history of disordered complement function and/or complement gene mutation(s).
- History of systemic lupus erythematous, Still's disease, rheumatoid arthritis, and/or other severe autoimmune conditions per judgment of the Investigator.
- Any known history of thrombotic microangiopathy (TMA)/microangiopathic hemolytic anemia, or hypercoagulable conditions including, but not limited to, disseminated intravascular coagulation (DIC), deep venous thrombosis...
- Current therapy with high dose immunosuppressants.
- Prior or current treatment with an investigational drug within 6 months or 5-half-lives of the hospital admission whichever is longer.
- Prior treatment with an antisense oligonucleotide within 1 year of hospital admission.
- A history of gene therapy administration.
- Any contraindication to ICM administration procedure, including contraindications to imaging, contrast use, anesthesia, or any condition that would increase the risk of adverse outcomes from the ICM procedure.
- Any contraindication to glucocorticoid use
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Chicago, Illinois, United States
- Boston, Massachusetts, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
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 years to 50 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Chicago, Illinois, United States; Boston, Massachusetts, 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.