A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
ConditionAngelman Syndrome
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age4-50
SponsorMavriX Bio, LLC
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
The participant's parent/legal guardian must provide written informed consent.
Full maternal UBE3A gene deletion causing AS in the region of 15q11.2-q13
Uniparental disomy
Imprinting center defect
Disqualifiers
Clinically significant medical finding other than AS, that, in the judgment of the Investigator would make the participant unsuitable for participation.
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
Trial design
Design model
Single group
Treatments tested in this trial
MVX-220
GeneticAAVhu68 viral vector
Treatment groups
12 Participants
are divided into 3 treatment groupsGroup A: Cohort 1: Adults ages 18-50Experimental treatment 1 intervention
Group B: Cohort 2: Children ages 4-8Experimental treatment 1 intervention
Group C: Cohort 3: Optional cohort, adults and children ages 4-50Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Incidence of Adverse Events, Serious Adverse Events, and Adverse Events of Special Interest as assessed through clinical safety, laboratory tests, ECG, vital sign measurements, and physical examinations
Time frame
Up to Week 104Secondary outcomes
1
Change in communication ability as assessed by the Observer Reported Communication Ability (ORCA) measure
The ORCA measure is a caregiver reporter assessment of communication ability that was developed specifically for Angelman syndrome. The ORCA measure produces a single score that is an estimate of an individual's overall level of communication ability, with higher T-scores reflecting greater communication ability.
Time frame
From Baseline to Week 1042
Change in developmental milestones as assessed by the Bayley Scale of Infant and Toddler Development, Fourth Edition (Bayley-4)
The Bayley-4 is a performance-based assessment of developmental functioning across communication, cognition, and motor skills. The total raw score reflects the sum of all the item scores within a subdomain, with higher scores reflecting greater ability.
Time frame
From Baseline to Week 1043
Change in adaptive behaviors as assessed by Vineland Adaptive Behavior Scale (VABS-3)
The VABS-3 assesses adaptive behaviors across multiple domains through a clinician-directed interview of a caregiver of an individual with AS. The total raw score reflects the sum of all the item scores within a subdomain, with higher scores reflecting greater ability.
Time frame
From Baseline to Week 1044
Change in Symptoms by the Angelman Severity Assessment (ASA)
The ASA is a clinician-reported outcome measure for Angelman syndrome. The clinicians rate their overall impression of the improvement in disease-related symptoms utilizing a 7-point scale, ranging from "very much improved" to "very much worse".
Time frame
From Baseline to Week 104Sponsors and contacts
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Source ClinicalTrials.gov